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FDA Moves Forward With Its Guidance Framework for Rare Disease Trials: 2026

August 3, 2026 by sjwilliamspa

FDA Moves Forward With Its Guidance Framework for Rare Disease Trials: 2026

Reporter: Stephen J. Williams, Ph,D.

In 2025, the Trump Administration had determined it wants to streamline the FDA and clinical trials in order to expedite much needed drugs for various terminal diseases such as cancer and rare childhood diseases.  In 2026, under the guidance of the new FDA commissioner, Dr. Makary, the FDA initiated guidance for the proposed changes in February and available for comments at that time.  This has been a growing discussion aver the years and the FDA wanted, for many years, to figure out they could speed bringing new therapies to market, especially for terminally  ill patients. Given the progress of biomarkers development early in the drug discovery process and new computational technologies, the time seems to be ready for such changes in trial design and even for the long drug development process.  In addition the FDA up to this point was focused on other matters so it wasn’t really at the forefront of their to do list.

From: https://www.fda.gov/news-events/press-announcements/fda-launches-framework-accelerating-development-individualized-therapies-ultra-rare-diseases

FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases

For Immediate Release:

The U.S. Food and Drug Administration today issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations.

The draft guidance, issued by the Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research, specifically discusses genome editing and RNA-based therapies such as antisense oligonucleotides but leaves open the potential that this framework may apply to additional tailored therapeutics provided they directly address the underlying specific cause of the disease.

“President Trump promised to accelerate cures for American families — and we are delivering, especially for children with ultra-rare diseases who cannot afford to wait,” said Health and Human Services Secretary Robert F. Kennedy, Jr. “We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most.”

“This guidance is a critical step the FDA is taking to tailor our regulatory approach to patients with ultra-rare conditions,” said FDA Commissioner Marty Makary, MD, MPH. “It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases.”

The draft guidance focuses on therapies that target a specific genetic, cellular or molecular abnormality and are designed to correct or modify the underlying cause of disease. Key criteria include:

  • Identifying the disease-causing abnormality.
  • Demonstrating the therapy targets the root cause or proximate biological pathway.
  • Relying on well-characterized natural history data in untreated patients.
  • Confirming successful target drugging or editing.
  • For traditional approval, therapies should demonstrate improvement in clinical outcomes, disease course, or biomarkers if they are established to predict clinical benefit.

“Designing treatments unique to individual patients has always been the promised goal of personalized medicine,” said Chief Medical and Scientific Officer and Center for Biologics Evaluation and Research Director Vinay Prasad, MD, MPH. “After 25 years the FDA has, for the first time, outlined a framework to facilitate these approvals. The Plausible Mechanism Framework is a revolutionary advance in regulatory science.”

“The Plausible Mechanism draft guidance creates a novel framework through which cutting-edge treatments tailor-made for patients with ultra-rare diseases can be used as a basis for FDA approval,” said Center for Drug Evaluation and Research Acting Director Tracy Beth Høeg, MD, Ph.D. “We anticipate our Plausible Mechanism draft guidance will inspire industry to place increased focus on individualized therapies, thereby driving innovation, improving safety, lowering costs and offering more patients with ultra-rare diseases a unique shot at a life-saving treatment.”

Because genome editing technologies are designed to be highly specific to unique DNA sequences, a product targeting different mutations in a single gene could be included in a single product application and potentially evaluated through the use of master protocols that evaluate these product variations in a single trial. A highly supported “plausible” mechanism of action may then be used to support the addition of other such genome editing product variants, intended to treat patients with mutations that were not included in the clinical trial used to support the original approval.

The FDA recognizes that an adequate and well-controlled clinical investigation in this context will include a small sample size, therefore, investigation results should be sufficiently robust to exclude chance findings. When determining effectiveness, the FDA considers the specific disease, the strength of the evidence and the challenges of conducting clinical investigations for individualized therapies.

 

In June of 2026, the FDA formalized their discussion into guidelines for discussion so it appears the following are not set into register of regulations as of yet.

From https://www.fda.gov/drugs/guidances-drugs/guidance-documents-rare-disease-drug-development

Guidance Documents for Rare Disease Drug Development

In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in agency guidances means that something is suggested or recommended, but not required.

Below are selected guidances that are relevant to rare disease drug development, organized by topic. This list does not include all FDA guidances on or relevant to rare disease drug development but represents our most commonly used guidances. This list may be updated periodically.

I have kept the original text in order for reference and to provide the background for these changes “in their words”.

However there are a few themes in these guideline changes and discussions including:

  • definitions of rare diseases
  • reduction of complexities and simplification of trial design and requirements (the FDA wants to go to a more ONE trial with very well designed controls than the two trial design for INDs (I will discuss this further in a near future post)
  • accelerated approval which will entail streamlining both the submission and approval process for rare conditions
  • heavy reliance on biomarkers during drug development and clinical trials (which is already in frequent use in pharma)
  • early communication with the FDA
  • more reliance on plausible mechanism of action to reduce number of studies needed

Rare Disease

Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause
The purpose of this guidance is to describe considerations for generating substantial evidence of effectiveness and evidence of safety for individualized therapies based on a plausible mechanism framework.

Rare Diseases: Considerations for the Development of Drugs and Biological Products
This guidance clarifies FDA’s thinking on important considerations in rare disease drug development to ultimately assist rare disease drug and biologic product developers in conducting successful drug development programs.

Rare Diseases: Natural History Studies for Drug Development: Draft Guidance for Industry
FDA is publishing this draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products for rare diseases. A natural history study collects information about the natural history of a disease in the absence of an intervention, from the disease’s onset until either its resolution or the individual’s death. Although knowledge of a disease’s natural history can benefit drug development for many disorders and conditions, natural history information is usually not available or is incomplete for most rare diseases; therefore, natural history information is particularly needed for these diseases.

Rare Pediatric Disease Priority Review Vouchers
This guidance provides information on the implementation of section 908 of the Food and Drug Administration Safety and Innovation Act (FDASIA), which added section 529 to the Federal Food, Drug, and Cosmetic Act (the FD&C Act). Under section 529, FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified in that section.

Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings : Draft Guidance for Industry
The purpose of this draft guidance is to assist sponsors of drug and biological products for the treatment of rare diseases in planning and conducting more efficient and productive pre-investigational new drug application (pre-IND) meetings. Drug development for rare diseases has many challenges related to the nature of these diseases. This draft guidance is intended to advance and facilitate the development of drugs and biological products for the treatment of rare diseases.

Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition That Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies : Guidance for Industry
This document provides guidance to sponsors on the evidence necessary to demonstrate the effectiveness of investigational new drugs or new drug uses intended for slowly progressive, low-prevalence rare diseases that are associated with substrate deposition and are caused by single enzyme defects. This guidance applies only to those low-prevalence rare diseases with well-characterized pathophysiology, and in which changes in substrate deposition can be readily measured in relevant tissue or tissues.

Pediatric Rare Diseases–A Collaborative Approach for Drug Development Using Gaucher Disease as a Model : Draft Guidance for Industry
The purpose of this guidance is to facilitate drug development in pediatric rare diseases. In particular, it discusses a new possible approach to enhance the efficiency of drug development in pediatric rare diseases using Gaucher disease as an example.

Inborn Errors of Metabolism That Use Dietary Management: Considerations for Optimizing and Standardizing Diet in Clinical Trials for Drug Product Development: Guidance for Industry
This guidance describes the Food and Drug Administration’s (FDA’s) current recommendations regarding how to optimize and standardize dietary management in clinical trials for the development of drugs that treat inborn errors of metabolism (IEM) for which dietary management is a key component of patients’ metabolic control. Optimizing dietary management in these patients before entry into and during clinical trials is essential to providing an accurate evaluation of the efficacy of new drug products.

Accelerated Approval

Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway
For drugs granted accelerated approval, sponsors have been required to conduct confirmatory studies postapproval to verify and describe the anticipated effect on irreversible morbidity or mortality or other clinical benefit. In the Consolidated Appropriations Act, 2023 (CAA), Congress amended section 506(c) of the FD&C Act (21 U.S.C. 356(c)), to provide additional authorities to help ensure timely completion of such trials, including that FDA “may require, as appropriate, a study or studies to be underway prior to approval, or within a specified time period after the date of approval, of the applicable product.” This draft guidance, when finalized, will describe FDA’s interpretation of the term “underway” and policies for implementing this requirement, including factors FDA intends to consider when determining whether a confirmatory trial is underway prior to an accelerated approval action.

Accelerated Approval – Expedited Program for Serious Conditions
Accelerated approval is one of FDA’s expedited programs intended to facilitate and expedite development and review of new drugs to address an unmet medical need in the treatment of a serious or life-threatening condition. The purpose of this guidance is to provide information on FDA’s policies and procedures for accelerated approval as well as threshold criteria generally applicable to concluding that a drug is a candidate for accelerated approval. This guidance also describes the procedures for expedited withdrawal of approval of a product approved under accelerated approval and the revisions Congress made through the Consolidated Appropriations Act, 2023 (Public Law 117-328). Additional programs to expedite product development and review are covered in other guidances.

Benefit-Risk

Benefit-Risk Assessment for New Drug and Biological Products
The intent of this guidance is to clarify for drug sponsors and other stakeholders how considerations about a drug’s benefits, risks, and risk management options factor into certain premarket and postmarket regulatory decisions that the Food and Drug Administration (FDA or Agency) makes about new drug applications (NDAs) submitted under section 505(c) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) as well as biologics license applications (BLAs) submitted under section 351(a) of the Public Health Service Act (PHS Act).

Biomarkers

For general information on Biomarkers, please see About Biomarkers and Qualification

Biomarker Qualification: Evidentiary Framework
This draft guidance provides recommendations on general considerations to address when developing a biomarker for qualification under the 21st Century Cures Act (Cures Act), enacted on December 13, 2016, that added a new section to the Federal Food, Drug, and Cosmetic Act (FD&C Act). Qualification of a biomarker is a determination that within the stated context of use, the biomarker can be relied on to have a specific interpretation and application in drug development and regulatory review.

Qualification Process for Drug Development Tools
This guidance describes the qualification process for drug development tools (DDTs) intended for potential use, over time, in multiple drug development programs.

Clinical Outcome Assessments (COAs) and Endpoints

For information on the COA Qualification Program, please see Clinical Outcome Assessment (COA) Qualification Program

Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments
This guidance (Guidance 3) is the third in a series of four methodological patient-focused drug development (PFDD) guidance documents that describe how stakeholders (patients, caregivers, researchers, medical product developers, and others) can collect and submit patient experience data and other relevant information from patients and caregivers to be used for medical product development and regulatory decision-making.

Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making
This guidance (Guidance 4) is the fourth in a series of four methodological patient-focused drug development (PFDD) guidance documents that describe how stakeholders (patients, caregivers, researchers, medical product developers, and others) can collect and submit patient experience data and other relevant information from patients and caregivers to be used for medical product development and regulatory decision-making.

Multiple Endpoints in Clinical Trials Guidance for Industry
This guidance provides sponsors and review staff with the Agency’s thinking about the problems posed by multiple endpoints in the analysis and interpretation of study results and how these problems can be managed in clinical trials for human drugs, including drugs subject to licensing as biological products.

Clinical Pharmacology

Exposure-Response Relationships — Study Design, Data Analysis, and Regulatory Applications 
This document provides recommendations for sponsors of investigational new drugs (INDs) and applicants submitting new drug applications (NDAs) or biologics license applications (BLAs) on the use of exposure-response information in the development of drugs, including therapeutic biologics. It can be considered along with the International Conference on Harmonisation (ICH) E4 guidance on Dose-Response Information to Support Drug Registration and other pertinent guidances (see Appendix A).

Bioavailability Studies Submitted in NDAs or INDs – General Considerations
This guidance provides recommendations to sponsors and applicants submitting bioavailability (BA) information for drug products in investigational new drug applications (INDs), new drug applications (NDAs), and NDA supplements. This guidance contains recommendations on how to meet the BA requirements set forth in 21 CFR part 320 as they apply to dosage forms intended for oral administration.

General Clinical Pharmacology Considerations for Pediatric Studies of Drugs, Including Biological Products
This guidance assists sponsors of investigational new drug applications (INDs) and applicants of new drug applications (NDAs) under section 505 of the Federal Food, Drug, and Cosmetic Act (the FD&C Act), biologics license applications (BLAs) under section 351(a) of the Public Health Service Act (PHS Act), and supplements to such applications who are planning to conduct clinical studies in pediatric populations.

General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products Guidance for Industry
This guidance is intended to assist sponsors of investigational new drug applications (INDs) and applicants of new drug applications (NDAs), biologics license applications (BLAs), and supplements to such applications who are planning to conduct clinical studies in neonatal populations. This guidance provides recommendations for neonatal clinical pharmacology studies, whether the studies are conducted pursuant to section 505A of the Federal Food, Drug, and Cosmetic Act (FD&C Act), section 505B of the FD&C Act, or neither.

Assessing the Effects of Food on Drugs in INDs and NDAs – Clinical Pharmacology Considerations
This guidance provides recommendations to sponsors planning to conduct food-effect (FE) studies for orally administered drug products under investigational new drug applications (INDs) to support new drug applications (NDAs) and supplements to these applications for drugs being developed under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355).

Population Pharmacokinetics
This guidance is intended to assist sponsors and applicants of new drug applications (NDAs), biologics license applications (BLAs), abbreviated new drug applications (ANDAs), and investigational new drugs (IND) applications in the application of population pharmacokinetic (PK) analysis.

Clinical Pharmacology Considerations for Antibody-Drug Conjugates Guidance for Industry
This guidance provides recommendations to assist industry and other parties involved in the development of antibody-drug conjugates (ADCs) with a cytotoxic small molecule drug or payload. Specifically, this guidance addresses the FDA’s current thinking regarding clinical pharmacology considerations and recommendations for ADC development programs, including bioanalytical methods, dosing strategies, dose- and exposure-response analysis, intrinsic factors, QTc assessments, immunogenicity, and drug-drug interactions (DDIs).

Drug-Drug Interaction Assessment for Therapeutic Proteins Guidance for Industry
The purpose of this guidance is to help sponsors of investigational new drug applications (INDs) and applicants of biologic license applications (BLAs) determine the need for drug-drug interaction (DDI) studies for a therapeutic protein (TP) by providing a systematic, risk-based approach.

Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease
The pharmacological effect of a targeted therapy is often related to a particular molecular alteration, and many diseases are caused by a range of different molecular alterations (some of which may be rare). Therefore, a targeted therapy may have differential effects among patients with the same disease who have different molecular alterations. The purpose of this guidance is to describe general approaches to evaluating the benefits and risks of targeted therapeutics within a clinically defined disease where some molecular alterations may occur at low frequencies.

Clinical Pharmacogenomics: Premarket Evaluation in Early-Phase Clinical Studies and Recommendations for Labeling
This guidance is intended to assist the pharmaceutical industry and other investigators engaged in new drug development in evaluating how variations in the human genome, specifically DNA sequence variants, could affect a drug’s pharmacokinetics (PK), pharmacodynamics (PD), efficacy, or safety. The guidance provides recommendations on when and how genomic information should be considered to address questions arising during drug development and regulatory review.

Clinical Trials

All clinical trials guidances are listed here.

Enhancing Participation in Clinical Trials — Eligibility Criteria, Enrollment Practices, and Trial Designs
This guidance recommends approaches that sponsors of clinical trials intended to support a new drug application or a biologics license application can take to increase enrollment of a representative population in their clinical trials. This guidance considers both demographic characteristics of study populations (e.g., sex, race, ethnicity, age, location of residency) and non-demographic characteristics of populations (e.g., patients with organ dysfunction, comorbid conditions, disabilities, those at the extremes of the weight range, and populations with diseases or conditions with low prevalence). Enrolling participants with a wide range of baseline characteristics may create a study population that more accurately reflects the patients likely to take the drug if it is approved and allow assessment of the impact of those characteristics on the safety and effectiveness of the study drug.

E8(R1) General Considerations for Clinical Studies
This guidance describes internationally accepted principles and practices in the design and conduct of clinical studies of drug and biological products. The guidance is intended to assist sponsors and other parties that design clinical studies, and to promote the quality of the studies submitted to regulatory authorities, while allowing for flexibility.

Multiple Endpoints in Clinical Trials Guidance for Industry
The purpose of this guidance is to describe various strategies for grouping and ordering endpoints for analysis and applying some well-recognized statistical methods for managing multiplicity within a study in order to control the chance of making erroneous conclusions about a drug’s effects. Basing a conclusion on an analysis where the risk of false conclusions has not been appropriately controlled can lead to false or misleading representations regarding a drug’s effects.

E17 General Principles for Planning and Design of Multi-Regional Clinical Trial
With the increasing globalization of drug development, it has become important that data from multiregional clinical trials (MRCTs) can be accepted by regulatory authorities across regions and countries as the primary source of evidence to support marketing approval of drugs (medicinal products). The purpose of this guidance is to describe general principles for the planning and design of MRCTs with the aim of increasing the acceptability of MRCTs in global regulatory submissions.

Decentralized Clinical Trials for Drugs, Biological Products, and Devices
This draft guidance provides recommendations for sponsors, investigators, and other stakeholders regarding the implementation of decentralized clinical trials (DCTs) for drugs, biological products, and devices. In this guidance, a DCT refers to a clinical trial where some or all of the trial-related activities occur at locations other than traditional clinical trial sites.

Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products: Guidance for Industry
The purpose of this guidance is to assist industry in developing enrichment strategies that can be used in clinical investigations intended to demonstrate effectiveness (and in some cases safety) of human drugs and biological products. This guidance defines several types of enrichment strategies, provides examples of potential clinical trial designs, and discusses potential regulatory considerations when using enrichment strategies in clinical trials.

Ethical Considerations for Clinical Investigations of Medical Products Involving Children
Clinical investigations in children are essential for obtaining data on the safety and effectiveness of drugs, biological products, and medical devices in children and to protect children from the risks associated with exposure to medical products that may be unsafe or ineffective. Children are a vulnerable population who cannot consent for themselves and who therefore are afforded additional safeguards when participating in a clinical investigation. Such safeguards are an essential requirement for the initiation and conduct of pediatric investigations as part of a medical product development program.

Master Protocols for Drug and Biological Product Development
This guidance document provides recommendations on the design and analysis of trials conducted under a master protocol as well as guidance on the submission of documentation to support regulatory review.

There are some other Guidances which were issued and I will discuss them in another post.  These include Guidances on minimizing use of animals for preclinical toxicology, Innovative clinical trial design especially for gene and cell therapies, and use of AI in designing of clinical trials.

For more information go to the FDA website at : https://www.fda.gov/drugs/guidances-drugs/guidance-documents-rare-disease-drug-development

Other Articles on FDA Guidances on this Open Access Scientific Journal Include:

FDA Guidance on Use of Xenotransplanted Products in Human: Implications in 3D Printing

FDA Guidance Documents Update Nov. 2015 on Devices, Animal Studies, Gene Therapy, Liposomes

FDA Cellular & Gene Therapy Guidances: Implications for CRSPR/Cas9 Trials 

New FDA Draft Guidance On Homologous Use of Human Cells, Tissues, and Cellular and Tissue-Based Products – Implications for 3D BioPrinting of Regenerative Tissue

FDA Guidelines For Developmental and Reproductive Toxicology (DART) Studies for Small Molecules

 

Posted in Artificial Intelligence in Medicine - Applications in Therapeutics, children, Clinical & Translational, Drug Development Process, FDA, FDA Regulatory Affairs, New Drug Approval | Tagged , , , , , , , , | Leave a Comment »

Artificial Intelligence TRANSFORMS Medicine, Biotech and Healthcare, How?

August 3, 2026 by 2012pharmaceutical

Artificial Intelligence TRANSFORMS Medicine, Biotech and Healthcare, How?

Curator: Aviva Lev-Ari, PhD, RN

WORK-IN-PROGRESS

Explosion of Podcasts on the topic include the following Selected list:

How AI Is Transforming Healthcare, Biotech and the Future of Medicine

Forbes

Jun 7, 2026

AI-driven innovation is rapidly reshaping the scientific ecosystem and redefining long-term value creation across healthcare, biotech, and life sciences. This session will explore the key forces accelerating progress in each of these sectors and how investors are making concentrated bets to fund startups with deep domain expertise and a clear ability to leverage AI in pursuit of breakthrough outcomes. Forbes Assistant Managing Editor Steve Bertoni sits down with Morgan Cheatham, M.D., Partner, Head of Healthcare and Life Sciences, Breyer Capital, Shalabh Gupta, M.D., Founder, CEO, President and Chairman, Unicycive, and Bradley Tusk, Founder & CEO, Tusk Ventures, at the 2026 Forbes Iconoclast Summit in New York City.

Transcript

Script SOURCE

https://youtu.be/nTpnCZvBmFc?si=dl5iWs75GZ4AFLFP

Posted in Artificial Intelligence - Breakthroughs in Theories and Technologies, Artificial intelligence applications for cardiology, Artificial Intelligence Applications in Health Care, Artificial Intelligence in CANCER, Artificial Intelligence in Health Care - Tools & Innovations, Artificial Intelligence in Medicine - Application for Diagnosis, Artificial Intelligence in Medicine - Applications in Therapeutics, Artificial Intelligence in Psychiatry, BioTechnology - Venture Creation, BioTechnology - Venture Creation, Venture Capital, Deep Learning in Pathology, Machine learning in predicting type 2 diabetes | Leave a Comment »

In Memoriam: Nobel Laureate J. Michael Bishop for discovery of Retroviral Oncogenes

July 31, 2026 by sjwilliamspa

In Memoriam: Nobel Laureate J. Michael Bishop for discovery of Retroviral Oncogenes

Reporter: Stephen J. Williams, Ph.D.

Among the many cancer researchers who have passed this year (listed in a post here), Dr. Bishop, who won the Nobel Prize with Harold Varmus for their discovery of retroviral oncogenes deserves special mention.  Since Peyton Raus noticed that an infective agent could transfer oncogenic stimulus for formation of new sarcomas in chickens injected with a fractioned tumor lysate, the search for the molecular basis a viral elements could induce tumorigenesis had remained elusive for most of the 20th century.  From medieval times cancer had been thought to have some infective component (and people used to think that tumors themselves were able to infect others).  The identification that a virus could induce tumors in animals was a revolutionary discovery and many investigators had tried to uncover human viruses which could induce tumors as well for a large part of the 20th century.

 

Drs. Bishop and Varmuses work to determine the human homologue to the chicken virus led to a revolutionary discovery of how growth factor signaling and their proliferative effects could be explained on the molecular level and how such proteins, when mutated, could lead to abnormal proliferation and growth.

From the AACR

Source: https://www.aacr.org/professionals/membership/in-memoriam/j-michael-bishop/

J. Michael Bishop

In Memoriam: J. Michael Bishop

(02/22/1936 – 03/20/2026) Member since 1998

J. Michael Bishop, MD, a Fellow of the AACR Academy who shared the Nobel Prize for Physiology or Medicine in 1989 with Harold E. Varmus, MD, for their discovery of the cellular origin of retroviral oncogenes, died March 20, 2026, at the age of 90.

Bishop served as chancellor of the University of California, San Francisco, from 1998 until he retired in 2009. He presided over a major expansion of UCSF, a university that is devoted entirely to biomedical sciences.

Born in York, Pennsylvania, in 1936, Bishop grew up in a rural area and attended elementary school in a two-room schoolhouse. He received a bachelor’s degree in chemistry from Gettysburg College in 1958 and a medical degree from Harvard Medical School in 1962.

After a residency at Massachusetts General Hospital and research at the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, and the Heinrich Pette Institute in Hamburg, Germany, he joined the faculty of UCSF as assistant professor of microbiology and immunology in 1968. He became a full professor in 1972, and in 1981 he was named director of the university’s George F. Hooper Research Foundation.

In 1970, Varmus joined Bishop’s lab as a postdoctoral fellow. They began studying the Rous sarcoma virus to test the theory that healthy body cells contain dormant viral oncogenes that cause cancer when triggered. Bishop and Varmus discovered that the viral genes that cause cancer in humans and animals do not originate within viruses, as previously thought, but instead begin as normal genes within healthy cells that act to control cellular growth and division. This finding indicated that these benign genes (called proto-oncogenes) can be picked up by certain viruses and then transformed into oncogenes.

This discovery that cancer could be caused by the malfunction of normal genes revolutionized the understanding of tumorigenesis and greatly expanded the scope of associated scientific areas such as cancer detection as well as drug discovery and development.

In addition to receiving the Nobel Prize, Bishop’s honors included election to the National Academy of Sciences in 1980 and as an Honorary Fellow of the American Association for the Advancement of Science in 1987, and as a Foreign Member of the Royal Society. He received the Albert Lasker Award for Basic Medical Research in 1982, the Gairdner Foundation International Award and the Alfred P. Sloan, Jr., Prize from the General Motors Cancer Research Foundation in 1984, and the Dickson Prize in Medicine in 1986. He was presented with the 2003 National Medal of Science by President George W. Bush. He was a member of the inaugural class of Fellows of the AACR Academy in 2013.

 

Posted in CANCER BIOLOGY & Innovations in Cancer Therapy, cancer-general, Mourning the Loss of a Scientific Leader, Nobel Prize Winners, Oncolytic virus & OncoViro-Therapy | Tagged , , , , , , | Leave a Comment »

Companies Actively Engaging Artificial Intellegence for Drug Design: 2026

July 31, 2026 by sjwilliamspa

Companies Actively Engaging Artificial Intellegence for Drug Design: 2026

Curator: Stephen J. Williams, Ph.D.

Work-in-Progress

This is not an exhaustive list of all AI companies involved in the drug discovery process.  Some like Chai Discovery will be featured in separate posts and this will be constantly updated as companies are acquired and formed. 

I want to start with a Great interview and white paper by McKinsey where they interview key opinion leaders on the business intelligence on AI firms in the drug discovery world and what to look for in companies that are in this space.

Source: https://www.mckinsey.com/industries/life-sciences/our-insights/how-ai-could-revolutionize-drug-discovery

How AI could revolutionize drug discovery
 | Video
By Alex Devereson, Christoph Sandler, and Lydia The
Watch VIDEO
Artificial intelligence could help scientists develop better medicines faster—and thus improve millions of people’s lives. But for that to happen, companies will need to change the way they work.

Below is a Mckinsey report on How AI will Change the Future of Biotech

https://www.mckinsey.com/featured-insights/the-next-normal/biotech

CHARTING THE FUTURE

AI will be embedded into everyday research

The AI-driven drug discovery industry continues to grow, fueled by new entrants in the market, significant capital investment, and technology maturation. We’ve identified more than 250 companies working in the industry. More than half of them are based in the United States, but key hubs are emerging in Western Europe and Southeast Asia as well. The best of these companies will fully integrate AI into research workflows, as the exhibit shows. By putting AI at the center of the research engine, companies can transform research at scale—and bring about dramatic improvements in patient outcomes. For the full article, see:

Parts of a high-throughput screening (HTS) process embedded with AI technology

1. High-throughput screen commenced with diverse compound sets Scientist selects diverse compound sets (a set of chemical compounds with a wide range of chemical structures) as first high-throughput screen

  • In silico/ on-the-chip simulations
  • In-vitro/ ‘wet lab’ experiments

2. Automated compound selection and transfer Using HTS machinery, individual compounds are transferred to individual wells of cells under experimental conditions

3. Computer-vision-based hit selection Cell response to each compound is measured using microscope analysis (eg, through computer vision techniques); promising compounds are labeled “hits”

4. Automated machine learning (ML) model training from screen outcomes Information from HTS for first few plates is automatically transferred into an ML pipeline, which “learns” how cells respond to each kind of chemical structure

5. Compound library inferencing and prioritization ML algorithm then scans the remainder of the library compounds and predicts which plates should be prioritized to identify the highest number of hits in the next screen

6. Automated compound selection based on ML recommendations ML recommendations are automatically queued and used in the next round of HTS. The cycle continues, with the algorithm continuously learningfrom “real world” outputs. Recommendations trigger scientists to explore new chemical space and begin downstream screening processes more quickly. These recommendations feed into the selection of chemical compounds in step 1

DOWNLOADS

Human bodies are incredibly complex. It takes many years to discover even just one new medicine to successfully treat a disease. Could artificial intelligence help speed up that process? McKinsey experts believe so. (The following transcript has been edited for clarity.)

Video

How AI could revolutionize drug discovery

Faster and better

 

How relevant and useful is this article for you?

Lydia The: What excites me about AI and drug discovery is the convergence between technology, drug development, and biology, which is going to lead to better drugs being developed faster—using all of the capabilities that Silicon Valley and the tech ecosystem have developed—to help us have even greater impact on patients.

Christoph Sandler: Today, to discover and develop a drug takes more than ten years.

Alex Devereson: We might be able to have drugs in one-tenth of the time, from being discovered to being able to treat patients. Today, many diseases simply have no treatments whatsoever. I think, and I hope, we’re going to see a world where we can generate therapies that can treat those patients very effectively. Fundamentally, we will have life-changing, game-changing drugs—on a scale and at a pace that we’ve never seen before—getting to the right patient at the right time.

The promise of personalized medicine

Christoph Sandler: In the not-so-distant future, we might collect health data across different inputs: from wearables, from our electronic medical records, or from clinical or academic research. And we will have the opportunity, on a voluntary basis, to upload these data into a central, secure, trusted data storage system.

Lydia The: You could imagine using the data to figure out not just what drug might work for you but exactly what drug would work for you at what time, in what sequence, in what dose—really personalized to you.

Will AI replace scientists?

Lydia The: What we’ve found is that technology doesn’t supplant the people. Rather, it will enable scientists to do things faster and better—and potentially develop insights that humans would not be able to develop at all.

Alex Devereson: Scientists will be able to discover things with machine learning that they could never have thought of by themselves, generate entirely new ideas, and move at a pace at which one person can do what it would have taken 100 to do before.

Lydia The: While in previous generations, scientists would spend a lot of their time—maybe even the majority of their time—on manual efforts such as pipetting from one tray to another tray or manually curating and cleaning data, I think AI will help us do all of those things in a more automated and quick way, and develop hypotheses that can then lead a scientist to think through, “What would the next experiment be? What are the implications of the data?”

What companies should do today

Alex Devereson: I think the challenge a lot of companies have is that they want to explore new ideas but, for good reason, they are reluctant to commit until they’ve seen some results and some tangible impact. So they explore a lot of pilots.

Lydia The: We have a term for that. We call it “pilot purgatory”: companies focus on one pilot, and they see the returns in a single pilot, but they don’t establish that approach and way of operating across their organization.

Define a ‘North Star’

Lydia The: One of the most important things for companies to escape pilot purgatory is a real mindset shift, from the top end all the way through the rest of the organization.

Christoph Sandler: It is important for the organization to define what a North Star for them should be, so that the data and analytics transformation of the R&D function can be targeted toward that North Star.

Identify—and solve—the biggest pain points

Alex Devereson: Truly understand what your biggest scientific and operational pain points are. For the lab scientists, for the patients who are in your clinical trials, for the patients who might get the drug: What is the biggest unsolved problem today?

Embed analytics into decision making

Christoph Sandler: Bring the organization on board and help them understand the potential of data and analytics.

Alex Devereson: You need to really, truly redesign a process that embeds analytics, where it’s not something on the side; it’s truly a part of the decision making.

Christoph Sandler: And you need to establish trust in the data and in these models. Equally important as the technical part is the human part.

Deliver value quickly

Alex Devereson: Don’t set up a project that delivers only after five years. Have a view on how you can deliver value in three months—and on what it takes in terms of analytics, data, and technology—with a relentless laser focus on value for the patient and for the scientific process.

 

 

Source: https://www.statnews.com/2026/06/03/alnylam-partner-with-inceptive-nucleics-ai-foundation-models/?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-_DOWjLTkMjWQ-ewilam07aNlz79w35ktjIKopXav_x3PpkOIfQIqnnMJlxdznIvjv-lGeo3tI4BUwIuxqEclEu4fHA9Q&_hsmi=422199873&utm_content=422199873&utm_source=hs_email
Biotech Correspondent

The inventor of some of AI’s technical underpinnings is shifting focus to an RNA startup. Also, Rick Pazdur has some ideas on how to determine whether Revolution Medicines’ pancreatic cancer drug can work in a first-line setting. And an oncologist explains why she believes the failed Grail trial matters.

artificial intelligence

The AI architect taking aim at RNAi

Jakob Uszkoreit helped create the transformer architecture — the “T” in ChatGPT — that sparked the generative AI boom. Now he’s trying to do something just as ambitious in drug development, STAT’s Brittany Trang writes. His startup, Inceptive Nucleics, is building biological foundation models that can be applied across a wide range of sequence-based medicines, from RNA interference therapies to mRNA and antisense drugs.

That vision caught the attention of Alnylam, which yesterday announced that it had struck a three-year partnership worth up to $2 billion in potential milestone payments and royalties, along with a $30 million upfront investment. The idea is that AI could perhaps do more than analyze biological data — and instead design the molecules themselves.

 

Top 10 Leading AI Drug Discovery Companies Transforming the Market: Trends, Technologies, Growth Outlook & Competitive Landscape (2026-2034)

IntelMarketResearch

MARKET OVERVIEW:

Artificial Intelligence (AI) is redefining the future of pharmaceutical research, making drug development faster, smarter, and more cost-effective than ever before. What once took more than a decade and billions of dollars can now be significantly accelerated through advanced machine learning algorithms, generative AI, predictive analytics, and computational biology.

AI Drug Discovery Market is projected to grow from USD 3.41 billion in 2026 to USD 10.45 billion by 2034, registering a robust 15.7% CAGR. This remarkable expansion reflects the pharmaceutical industry’s increasing reliance on AI-powered platforms to improve research efficiency, reduce clinical failures, and identify breakthrough therapies for complex diseases.

As healthcare systems worldwide demand faster innovation and precision medicine continues to evolve, AI is becoming an essential component of modern drug discovery rather than an experimental technology.

Key Technologies Powering AI Drug Discovery

The industry’s rapid growth is supported by several advanced technologies that work together throughout the drug development pipeline.

Machine Learning helps identify disease targets, recognize biological patterns, and predict therapeutic outcomes using historical datasets.

Deep Learning improves molecular analysis by recognizing highly complex biological relationships that conventional computational methods may overlook.

Generative AI creates entirely new molecular structures designed for specific therapeutic applications, enabling scientists to explore millions of virtual compounds before physical synthesis.

Predictive Modeling estimates toxicity, efficacy, pharmacokinetics, and safety profiles early in development, reducing unnecessary experimentation.

Molecular Docking Simulations evaluate how drug molecules interact with proteins, helping researchers prioritize candidates with the highest therapeutic potential.

AI-Driven Clinical Trial Optimization assists pharmaceutical companies in selecting suitable patient populations, improving trial design, and increasing the probability of successful outcomes.

Top 10 Leading AI Drug Discovery Companies Driving Innovation

The competitive landscape is rapidly evolving, with established technology providers and specialized biotechnology companies competing to develop next-generation AI platforms.

1. Insilico Medicine

Insilico Medicine has become one of the industry’s most recognized innovators through its end-to-end AI drug discovery platform. The company combines generative AI with biological research to accelerate target identification and therapeutic development.

2. Exscientia

Exscientia focuses on AI-designed precision medicines and has established multiple collaborations with leading pharmaceutical organizations to improve drug candidate selection and optimization. Exscientia is a pioneering artificial intelligence-driven pharmatech company that was acquired by Recursion Pharmaceuticals in November 2024

3. Atomwise

Known for its deep learning platform, Atomwise uses artificial intelligence to identify promising small-molecule therapies across oncology, infectious diseases, and rare disorders.

4. Recursion Pharmaceuticals

Recursion Pharmaceuticals integrates high-content imaging, automation, and AI to analyze millions of biological experiments, enabling large-scale phenotypic drug discovery.

5. BERG

BERG (now operating or known as BPGbio) specializes in biology-driven AI by combining multi-omics datasets with artificial intelligence to identify biomarkers and novel therapeutic opportunities.

6. Cyclica

Cyclica’s AI platform focuses on polypharmacology, helping researchers understand complex drug-protein interactions while improving drug design efficiency. , focusing on MatchMaker and POEM technology platforms before being acquired by Recursion for $40 million in May 2023.

7. GNS Healthcare

GNS Healthcare applies causal AI and patient-level analytics to support personalized medicine and identify drug repurposing opportunities.

8. DeepCure

DeepCure develops generative chemistry platforms capable of designing optimized molecules while reducing early-stage research complexity.

9. Schrödinger

Schrödinger combines computational chemistry, molecular simulation, and AI technologies to improve molecular modeling and accelerate pharmaceutical innovation.

10. IBM Watson Health & DeepMind Health

These technology pioneers continue expanding AI capabilities across healthcare by leveraging advanced analytics, large-scale biological data processing, and intelligent research platforms that support drug development initiatives. https://www.ibm.com/products/watsonx

There are many other companies that are using AI in Innovative ways to support drug discovery.  Here is another list from

 

11 Innovative Companies Using AI for Drug Discovery

Contributing Writer

By Sherri Gordon

Published December 15, 2025

Overview

A look at 11 companies using AI-driven platforms to reshape drug discovery, including generative models, computational chemistry, and data-centric biology.

 

For more articles on AI and healthcare on this Open Access Scientific Journals please see our Portals at

Medicine with GPT-4 & Chat GPT

AGI, generativeAI, Grok, DeepSeek & Expert Models in Healthcare

Artificial Intelligence: Genomics & Cancer

Posted in Artificial Intelligence Applications in Health Care, Artificial Intelligence in CANCER, Artificial Intelligence in Medicine - Applications in Therapeutics, Deep Learning, Drug Development Process, Machine Learning, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence | Tagged , , , , , , | Leave a Comment »

AI-Native Drug Discovery Landscape 2026 – How LPBI Group Differentiates in the New Era of Foundation Models

July 19, 2026 by 2012pharmaceutical

AI-Native Drug Discovery Landscape 2026 – How LPBI Group Differentiates in the New Era of Foundation Models

Curators: Aviva Lev-Ari, PhD, RN with Grok Assistence

As the race to build powerful biology foundation models intensifies, several well-funded AI-native companies have emerged with ambitious platforms for protein design, small-molecule generation, and multimodal drug discovery. While these companies bring strong technical capabilities, LPBI Group occupies a distinct and complementary position in the ecosystem.

Key AI-Native Players (2026)

 

Competitive Landscape Table for AI-Native in Drug Discovery

Company Focus Stage LPBI Differentiation
Isomorphic Labs (DeepMind) Protein/small molecule design using AI Advanced (AlphaFold3 based) LPBI offers curated multimodal training data + COM methodology (they need high-quality data to train/validate)
EvolutionaryScale Protein design (ESM models) Early commercial LPBI’s strength is in clinical/therapeutic context + mechanism-of-action curation across full disease spectrum
Chai Discovery Multimodal AI for drug discovery Early LPBI provides the upstream high-provenance corpus + ontology they would need for better results
Recursion Pharma Phenotypic screening + AI Clinical stage LPBI’s expert-curated literature + images + COM complements their wet-lab focus
Insilico Medicine Generative AI for drug design Clinical stage LPBI’s causal reasoning framework + AJAUS offers continuous refresh they lack
AlignedHQ.ai Generative AI for protein design & therapeutic optimization Early-stage LPBI supplies the high-provenance, expert-curated multimodal corpus and COM Tool Factory that significantly enhances model accuracy and reduces failure rates in downstream development

How LPBI Group Differentiates

LPBI Group does not compete directly in building foundation models. Instead, we provide the critical upstream layer these companies and hyperscalers urgently need:

  • A 9 GB private multimodal corpus of expert-curated scientific content (6,290+ articles, 48 e-Books, 7,500+ images, 300+ podcasts)
  • The 17-part Composition of Methods (COM) Tool Factory, including AJAUS (autonomous 24/7 refresh) and Rosetta Stone Ontology (causal mapping)
  • 15 Subject Matter Small Language Models (SLMs) ready for concatenation into proprietary LLMs and MFMH
  • Proven track record of 4–5×+ uplift in novel causal relationship extraction when combined with frontier models

Strategic Positioning

While AI-native startups excel at model architecture and computation, they still face the persistent bottleneck of high-quality, causally structured, provenance-rich training data. LPBI Group’s vertically integrated assets and methodology offer a defensible moat and a true “own-both” advantage when partnered with hyperscalers or pharma companies.

This complementary role positions LPBI Group as the ideal upstream partner for the next generation of domain-aware AI in Health.

AlignedHQ.ai as Partner vs. Insilico Medicine

AlignedHQ.ai is a strong potential partner.

Why?

  • They are AI-native focused on protein design and generative models — directly complementary to LPBI’s strengths in curated biomedical literature, mechanism-of-action, and multimodal data.
  • They would benefit enormously from access to LPBI’s high-quality training data and COM methodology.
  • Partnership model: They use LPBI data (licensed) + Grok 5 / SpaceXAI compute and frontier models → Co-develop specific therapeutic pipelines.

Insilico Medicine is also a good candidate but slightly less ideal than AlignedHQ for early partnership because:

  • Insilico is more advanced clinically (has candidates in trials) and may want more control.
  • AlignedHQ appears earlier-stage and more open to collaboration.

Recommendation: Start with AlignedHQ.ai as a proof-of-concept partner (easier entry, high complementarity). Use success there to approach Insilico and others from a position of strength.

Where does AlignedHQ.ai’s Domain Knowledge in Medicine Come From?

From public information:

  • Primarily from public + licensed datasets (PDB, UniProt, scientific literature, clinical trial data, etc.).
  • They rely heavily on large-scale public biomedical databases and pre-trained models (e.g., AlphaFold derivatives).
  • Like most AI-native drug discovery companies, they have limited proprietary clinical/therapeutic context compared to LPBI’s expert-curated, mechanism-rich corpus.
  • Their domain knowledge is model-derived rather than expert-curated at source.

This is LPBI’s Core Delta: AlignedHQ (and similar companies) excel at model architecture and generation but lack the deep, traceable, expert-validated biomedical knowledge that LPBI has built over 14+ years. This is why access to LPBI’s portfolio would be highly valuable to them.

Grok, 7/19/2026

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In Memoriam: In Remembrance of Cancer Researchers who passed in 2026

July 13, 2026 by sjwilliamspa

In Memoriam: In Remembrance of Cancer Researchers who passed in 2026

Reporter: Stephen J. Williams, Ph.D.

Source: https://www.aacr.org/professionals/membership/in-memoriam/

The following remembrances of American Association of Cancer Research (AACR) prominent member who have recently passed in 2026 is given below.  Each have contributed seminal research and discovery in the field of cancer biology and cancer risk.  In many cases, their discoveries transformed the way  we understand and treat cancer.  A separate In Memoriam for Nobel Leaureatte Dr. J. Michael Bishop will be given in a separate post.

Joseph F. Fraumeni, Jr., MD, FAACR (04/01/1933 – 06/22/2026)

Headshot of Joseph Fraumeni

Joseph F. Fraumeni, Jr., MD, FAACR, a renowned cancer epidemiologist, a Fellow of the AACR Academy, and a former member of the AACR Board of Directors, died June 22, 2026, at the age of 93. A career researcher and leader at the National Cancer Institute, Fraumeni was a co-discoverer of the genetic condition now known as the Li-Fraumeni syndrome and launched the U.S. Atlas of Cancer Mortality, which mapped geographic variations in cancer.Born April 1, 1933, in Boston, Fraumeni earned a bachelor’s degree from Harvard College, a medical degree from Duke University School of Medicine, and a master of science in epidemiology from the Harvard University School of Public Health. He completed medical residencies at Johns Hopkins Hospital and the Memorial Sloan-Kettering Cancer Center. A member of the AACR since 1968, Fraumeni served on the AACR’s Board of Directors from 1983 to 1986. He also served the AACR as an assistant editor, senior editor, and editorial board member for Cancer Epidemiology, Biomarkers & Prevention and an assistant editor for Cancer Research. The AACR recognized him with the AACR-American Cancer Society Award for Research Excellence in Epidemiology and Prevention in 1993 and the AACR Award for Lifetime Achievement in Cancer Research in 2009. He was inducted as a member of the inaugural class of Fellows of the AACR Academy in 2013. Fraumeni was a fellow of the American College of Physicians, the American Association for the Advancement of Science, and the American Academy of Arts and Sciences, and a member of the Institute of Medicine, the Association of American Physicians, and the National Academy of Sciences.

In 1962, Fraumeni joined the Epidemiology Branch of the National Cancer Institute (NCI) as a commissioned officer in the U.S. Public Health Service (USPHS). He went on to hold several leadership positions at the NCI, including posts as head of the Ecology Studies Section, chief of the Environmental Epidemiology Branch, director of the Epidemiology and Biostatistics Program, and founding director of the Division of Cancer Epidemiology and GeneticsHe retired from the USPHS in 1999 with the rank of rear admiral and assistant surgeon general. When he retired from NCI in 2017, he was named Scientist Emeritus. He authored or co-authored more than 900 scientific publications.

His research focused the epidemiology of high cancer risk populations and, in 1969, led him to discover a familial syndrome of early-onset cancers of the breast, brain, and other malignancies known as Li-Fraumeni Syndrome.  Li-Fraumeni Sydrome is characterized by inherited mutations in the p53 tumor suppressor gene.

Li-Fraumeni Syndrome

from Cleveland Clinic: Li-Fraumeni syndrome is a rare genetic disorder that increases the risk you and your family members will develop cancer. Everyone with this condition has a 90% chance of developing one or more types of cancer by age 60. About half develop cancer before they turn 40. Females with Li-Fraumeni syndrome almost always develop breast cancer.

Below is the original reference published with his colleague the late Dr, Federick Li.  Thier work together over the years helped develop the discovery of cancer susceptiblitiy genes and the importance of mutations of these genes linked to increased risk of developing cancer.

Li FP, Fraumeni JF Jr. 1969. Soft-tissue sarcomas, breast cancer, and other neoplasms. A familial syndrome? Ann Intern Med 71: 747–752.

Four families were identified in which a pair of children had soft-tissue sarcomas: three sets of sibs and one set of cousins. One parent of each affected child developed cancer; carcinoma of the breast occurred in three mothers under 30 years of age. Other young adults in these families had a high frequency of cancer, with no evidence of underlying genetic disorders known to carry a high risk of neoplasia. The increased familial susceptibility to cancer was manifested not only by the large number of members affected but by a seeming excess of multiple primary neoplasms.

It wasn’t until the 1990’s that Malkin et al. that germline mutations in TP53 were associated with this disease

Malkin D, Li FP, Strong LC, Fraumeni JF Jr, Nelson CE, Kim DH, Kassel J, Gryka MA, Bischoff FZ, Tainsky MA, et al. 1990. Germ line p53 mutations in a familial syndrome of breast cancer, sarcomas, and other neoplasms. Science 250: 1233–1238.

A similar syndrome named Lynch syndrome also  gave rise to early increased risk of multiple cancers but due to germline mutations in mismatch repair genes like MLH1, MSH2, MSH6, or PMS2.

Lynch HT, Mulcahy GM, Harris RE, Guirgis HA, Lynch JF. 1978. Genetic and pathologic findings in a kindred with hereditary sarcoma, breast cancer, brain tumors, leukemia, lung, laryngeal, and adrenal cortical carcinoma. Cancer 41: 2055–2064.

 

Pierre Chambon, MD, FAACR, (02/07/1931 – 05/05/2026)
Pierre Chambon

Pierre Chambon, MD, FAACR, a Fellow of the AACR Academy who was a pioneer in the structure and expression of genes, died May 5, 2026, at the age of 95. Chambon’s early work contributed to the discovery of PolyADPribose, the discovery of multiple RNA polymerases, major contributions to the elucidation of chromatin structure, and the discovery of animal split genes. Later work included the discovery of multiple promoter elements and their cognate factors. His research on nuclear receptors has had a marked influence on the understanding of signal transduction and endocrinology in vertebrates.

Born February 7, 1931, in Mulhouse, France, Chambon received his medical degree from the University of Strasbourg in 1958. He joined the university as a research associate, becoming an associate professor in 1962 and professor of biochemistry in 1968. He founded the Institute for Genetics and Cellular and Molecular Biology in 1994 and served as its director until 2002. He then founded the Mouse Clinical Institute and served as director until 2006. He held the chair of molecular genetics at the Collège de France from 1993 to 2003 and served as chair of molecular genetics and biology at the University of Strasbourg Institute for Advanced Study from 2012 to 2021. Chambon was elected to the French Academy of Sciences in1985, the same year in which he was elected a foreign member of both the U.S. National Academy of Sciences and the American Academy of Arts and Sciences.

Juliet M. Daniel, PhD

Juliet M. Daniel, PhD, a cell biologist who was a distinguished university professor at McMaster University in Hamilton, Ontario, and member of AACR since 2002, died April 28, 2026. She was 61 years of age. Noted for her work on genetic risk factors for breast cancer, Daniel discovered and gave the name “Kaiso” to a gene associated with triple negative breast cancer in women of African descent. Born in Barbados in 1964, Daniel obtained a bachelor’s degree in life sciences from Queen’s University in Kingston, Ontario, in 1987 and a doctorate in microbiology from University of British Columbia in Vancouver in 1993. She conducted postdoctoral research at St. Jude Children’s Research Hospital in Memphis and Vanderbilt University in Nashville. She joined McMaster as an assistant professor in 1999, the first black woman to become a member of the Faculty of Science. She was promoted to associate professor in 2005 and professor in 2012. Daniel was appointed associate dean of research and external relations for the Faculty of Science on an acting basis in 2020 and permanently in 2021. She was named strategic advisor to the university president for the Canada-Caribbean Institute (CCI) at McMaster in 2024. She was named a distinguished university professor, the highest faculty honor, in 2025. Among many other honors, she was elected a fellow of the Canadian Academy of Health Sciences in 2025, received the inaugural Canadian Cancer Society Inclusive Excellence Prize in Cancer Research in 2020, and was awarded an honorary doctorate in science by the University of the West Indies in 2021.

Philip S. Low, PhD

Philip S. Low, PhD, the Ralph C. Corley distinguished professor of chemistry at Purdue University, an inventor and entrepreneur with more than 100 patents to his credit, and an emeritus member of AACR, died March 4, 2026, at the age of 78. He also served as Purdue’s Presidential Scholar for Drug Discovery and was for a time as director of the university’s Center for Drug Discovery. Low held more than 100 U.S.-issued patents through Purdue Innovates and is listed on 600 U.S. and international patents and 145 invention disclosures. He founded seven companies based on based on work conducted at Purdue, one of which, Endocyte Inc., was sold to Novartis in 2018. Born in Ames, Iowa, in 1947, Low earned a bachelor’s degree in chemistry from Brigham Young University in 1971 and a doctorate in biochemistry from the University of California, San Diego, in 1975. He joined the Purdue University faculty in 1976. An AACR member since 2005, Low received the AACR Award for Outstanding Achievement in Chemistry in Cancer Research in 2015 in recognition of his research on low molecular weight ligand-targeted therapeutic and imaging agents. In the same year, he also received the American Chemical Society (ACS) George & Christine Sosnovsky Award for Cancer Research and was elected to the National Academy of Inventors. In August 2025, Low was named the recipient of the ACS Alfred Burger Award in Medicinal Chemistry for 2026. He also received the Order of the Griffin and the Morrill Award from Purdue.

For more remebrances of past AACR members please visit: https://www.aacr.org/professionals/membership/in-memoriam/

Other recent In Memoriam on this Open Access Scientific Journal Include:

News from AACR; In Memoriam: Nobel Leaureate David Baltimore, Ph.D

In Memoriam: Professor Yitzhak Apeloig, President and Distinguised Professor of the Technion

 

 

 

Posted in Cancer Prevention: Research & Programs, Drug Discovery Chemistry, Gene Regulation, Li-fraumeni syndrome., Mourning the Loss of a Scientific Leader, Patents, TP53 - Germline mutations | Tagged , , , , , , , , , | Leave a Comment »

Urgent Request for Action on Proposed Rule Affecting Federal Funding Grant Review

July 7, 2026 by sjwilliamspa

Urgent Request for Action on Proposed Rule Affecting Federal Funding Grant Review

Reporter: Stephen J. Williams, Ph.D.

Yesterday I received an email from the Mesothelioma Applied Research Foundation (MARFA), as well as Life Sciences PA and the American Association for Cancer Research (AACR).  This was on a little publicized proposed change to the NIH scientific review process allowing the Office of Management and Budget (OMB) to have direct authority over the funding of grants which had passed the rigorous scientific review process.  This proposed change by the current administration could negate decision made by NIH grant review panels as well as NIH program project directors and NIH administration as well as other funding agencies.

OMB has put a deadling of July 13, 2026 for comments by the public on this proposed change so it is of urgent matter that US scientists be informed of these potential changes.

I have included the three letters below: from MARFA, Life Sciences PA, and AACR.

From the Mesothelioma Applied Research Foundation

For decades, scientific breakthroughs in mesothelioma have depended on one essential principle: research should be guided by science.

A proposed rule from the Office of Management and Budget (OMB) could weaken that principle by allowing political appointees to override scientific peer review when making federal research funding decisions. It could also allow active federally funded research projects to be terminated if deemed inconsistent with changing agency priorities.

For the mesothelioma community, the stakes are especially high.

As a rare cancer, mesothelioma research already faces significant funding challenges. Every federal grant helps advance promising discoveries, supports clinical trials, and gives patients access to new treatment options. The proposal could also restrict international scientific collaboration—partnerships that are often essential for rare disease research, where researchers must work across borders to enroll enough patients and share critical scientific knowledge.

Learn more about the Proposed Rule and how it affects mesothelioma patients and research.

We need your voice.

Please take just a few minutes to submit a public comment asking OMB to protect independent scientific peer review, preserve ongoing federally funded research, and support international collaboration for rare diseases.

Submit your public comment here: https://www.federalregister.gov/documents/2026/05/29/2026-10817/regulation-for-federal-financial-assistance#open-comment

The comment period is open until Monday, July 13th. Don’t delay submitting your comments.

Every comment demonstrates that patients, families, researchers, clinicians, and advocates are paying attention and understand what is at stake.

Thank you for standing up for the future of mesothelioma research.

Together, we can help ensure that scientific discovery continues to be driven by evidence, collaboration, and the needs of patients.

 

From Life Sciences PA
To the Membership of Life Sciences PA:

Today, we are urging you to contact your United States members of Congress on Office of Management and Budget (OMB) proposed revisions to “Guidance for Federal Financial Assistance.” You can do so through the Life Sciences PA Advocacy Action Center.

The OMB proposal, which is currently accepting public comments, would make significant changes to how research funding decisions are administered across federal agencies, including the NIH and NSF. The public comment period closes in just one week, Monday, July 13, 2026, and public comments can be submitted HERE. However, it is equally important for U.S. Senators and Representatives to hear directly from their constituents on matters like this. We encourage your outreach beyond the public comment period.

For decades, independent, merit-based scientific peer review has been the foundation of federal research funding. Currently, the NIH is investing over $2.5 billion in more than 4,300 research projects across the Commonwealth. These investments have helped establish Pennsylvania as a global leader in research and development for innovative therapies and technologies. The revisions proposed by OMB put in jeopardy the transparency, predictability, and scientific integrity of that process.

Federal research investments are critical to the success of Pennsylvania’s leading life sciences ecosystem. They support scientific discovery at our academic research institutions, foster crucial partnerships with industry, and help advance the therapies and technologies that improve the lives of patients in the Commonwealth, across the country, and around the world. Maintaining certainty, consistency, and confidence in this established research funding process is essential to sustaining scientific progress and continued economic growth.

Life Sciences Pennsylvania is sharing our concerns with members of the Pennsylvania Congressional delegation, as well as submitting public comment reaffirming the importance of preserving an independent, science-driven research funding system. We urge you to share your perspectives by submitting concerns to your U.S. Senators and Representatives HERE.

 

From AACR

AACR Statement and Call to Action Concerning OMB Proposal to Rewrite the Rules for Scientists and Physicians Who Are Improving Public Health and Saving Lives

PHILADELPHIA – The American Association for Cancer Research (AACR) is deeply concerned that the recent action of the White House Office of Management and Budget (OMB) to propose a new set of regulations on how federal grants are awarded and managed is a major threat to the National Institutes of Health (NIH) and its lifesaving mission to accelerate progress for patients with cancer and the hundreds of other diseases that afflict millions of Americans.

AACR strongly opposes many of the changes that OMB has proposed in its recently issued rule, titled “Regulation for Federal Financial Assistance.” A considerable number of the regulations included in the rule, if finalized, will be extremely disruptive to the lifesaving research that the federal government funds and oversees, mainly through NIH.

Therefore, AACR calls on the Administration to abandon this harmful proposal, as proceeding along these lines will irreparably damage NIH, an agency that is widely revered as the world’s leading medical research enterprise for its support of the innovative discoveries and groundbreaking treatments that are increasing survival rates and enhancing the quality of life for patients.

According to OMB, the intention of these proposed revisions is to improve transparency, accountability, and oversight of federal awards while reducing administrative burden and ensuring responsible stewardship of taxpayer resources. While AACR shares these goals and supports efforts to strengthen the effectiveness, integrity, and accountability of federal grant programs, a significant number of the provisions in this proposal would in fact increase administrative complexity, create uncertainty for grant recipients, reduce transparency in funding decisions, and undermine the merit-based processes that have effectively guided federal research investments. In short, this OMB proposal is reckless and does not meet the high U.S. standards required for a meritorious, impactful research grant program.

AACR has identified several especially concerning provisions in this OMB proposal that will severely set back our nation’s medical science enterprise and delay the groundbreaking treatments that are pivotal to improving patient outcomes and saving lives:

  • OMB proposes to markedly expand the power of political appointees to override expert assessment of scientific merit by subjecting every federal research funding decision to political review. These politically appointed officials are unlikely to have subject-matter expertise and would also be instructed to determine whether grant proposals are aligned with the Administration’s political priorities, regardless of their scientific merit. If political appointees are required to provide oversight of federal grant decisions and are allowed or encouraged to ignore the advice of highly qualified scientists, it will threaten the significant progress in patient outcomes that has been achieved over many decades.The merit review process, in which scientific experts in a particular field are brought together to review scientific proposals and assign scores that are based on the quality of the application and its potential to advance new knowledge, has underpinned many discoveries that have led to major improvements in public health. This objective approach to scientific review establishes a foundation of trust within the broader scientific community and throughout the U.S. population. Revising the rules to establish an environment that sidesteps traditional and transparent scientific metrics will weaken overall confidence in the U.S. research enterprise. [This area corresponds to provision 200.205 in the OMB proposal.]
  • OMB proposes to expand agency authority to suspend or terminate awards based on changing political priorities at the agency or program goals that no longer align with the Administration’s concept of “Gold Standard Science,” which is a term the Administration uses to terminate research not because it is unsound, but because it does not fit a preferred political or methodological agenda. These suspensions and terminations could take place regardless of whether the recipient is performing the project based on the previously approved scope of work. Therefore, an active grant, including a multi-year award that is already underway, could be terminated because of a political or ideological agenda.This OMB-proposed provision would create uncertainty for researchers, institutions, and patients. As one example, this proposed change may have an adverse effect on patient accrual to cancer clinical trials, as patients with cancer may be less likely to enroll if ongoing support for these clinical trials is uncertain. Cancer research often requires years of sustained investment, and permitting political appointees to discontinue support for a grant or clinical trial after the grant or clinical trial has already been awarded or begun threatens scientific progress, wastes taxpayer resources, and destabilizes research programs and studies on which patients are relying for their survival. [This area corresponds to provisions 200.340; 200.341; and 200.342 in the OMB proposal.]
  • OMB proposes to create additional barriers to scientific collaboration by imposing undue restrictions on international partnerships that are often essential for making advances against cancer and other human diseases. Modern cancer science relies on global networks of researchers, clinical trial participants, data resources, and specialized expertise. Limiting these collaborations will slow the pace of discovery and innovation. As one example among many, international collaborations are vitally important for pediatric cancer research. Because childhood cancers are rare, pooling global patient data, resources, and expertise accelerates clinical trials, drives breakthroughs in drug-resistant subtypes, and bridges survival disparities across high- and low-income countries. [This area corresponds to provisions 200.202 and 200.220 in the OMB proposal.]
  • OMB proposes to prohibit all federal funding related to diversity, equity, and inclusion. OMB’s justification includes vague language that could be interpreted in ways that restrict research on cancer disparities, access to care, and differences in outcomes among all patient populations. Cancer touches every community, yet, sadly, its burden is not shared equally. A person’s race, ethnicity, income, ZIP code, insurance status, access to screening, ability to enroll in a clinical trial, and proximity to overall high-quality cancer care shape whether cancer is found early, treated effectively, and ultimately cured. [This area corresponds to provisions 200.218 and 200.300 in the OMB proposal.]
  • OMB proposes to restrict activities that are fundamental components of innovative scientific research and that are essential to how research findings are shared and translated into patient benefit. Provisions in the OMB proposal to limit funding support for scientific publications, journal subscriptions, and attendance at scientific conferences would hinder the ability of researchers to disseminate discoveries, learn about emerging advances, and establish highly productive collaborations. [This area corresponds to provisions 200.432; 200.454; and 200.461 in the OMB proposal.]

For decades, the framework that supports America’s scientific research enterprise through agencies such as NIH and the National Cancer Institute has fueled transformative discoveries in cancer prevention, detection, treatment, and survivorship. This established system has reduced the U.S. cancer death rate by 35% since 1991, resulting in more than 4.8 million U.S. lives saved. This progress has been made possible because of research funding decisions guided by the rigorous review of grant applications by scientific experts and their assessment of these grants to improve public health—NOT guided by political and ideological considerations.

If this OMB-proposed regulation is ultimately finalized, it will severely weaken the U.S. federal research grant program that has supported American innovation and medical breakthroughs for decades. It will also upend the collaborative and evidence-based model that has resulted in U.S. leadership in cancer research and medical science.

 

Click here for Instructions on how to Submit a Comment to OMB

Click here to Contact Your Members of Congress about the proposal

It is imperative that all US scientists respond to this potential disasterous change.  NIH grant review panels take their time out of their schedule to volunteer to review grants and committ great effort and time to this labor of love.  In addition, those who write the grants have spent countless hours, days and months meticulous preparing exellent grants for review.  These scientists show an utmost committment to the conduct of great science and a dedication to their field of expertise.  Many volunteer countless hours and resources for their research, the scientific community and for patients. And most importantly, scientists include verbage and material in grants BASED ON SOUND SCIENCE, not their opinions.

Please take some time to respond to your lawmakers before July 13, 2026

Thank you

 

Posted in Bio-Ethics, Federal Budget Appropriations, Funding Opportunities for Cancer Research, Law and Medicine Conflicts, Population Health Management, Scientist: Career considerations | Tagged , , , , , , , | Leave a Comment »

2026 Tang Prize in Biopharmaceutical Science Awarded to Three Pioneers of Cellular Immunotherapy Revolutionizing Treatments for Blood Cancers and Solid Tumors

July 5, 2026 by sjwilliamspa

2026 Tang Prize in Biopharmaceutical Science Awarded to Three Pioneers of Cellular Immunotherapy Revolutionizing Treatments for Blood Cancers and Solid Tumors

Reporter: Stephen J. Williams, Ph.D.

From the Tang Prize Committee Anouncement

Source: https://www.tang-prize.org/en/media_detail.php?id=2097

On June 16, the Tang Prize in Biopharmaceutical Science announced its 2026 laureates. The prize, whose laureates from previous cycles have subsequently received Nobel Prize honors, has drawn wide international attention. Three leading scientists in the field of cellular immunotherapy, Drs. Steven A. Rosenberg, Michel Sadelain, and Carl H. June, have been named joint laureates “for the discovery and development of tumor-infiltrating lymphocyte (TIL) and chimeric antigen receptor T-cell (CAR-T) therapies, which have revolutionized treatment for blood cancers and solid tumors.”

Immunosuppression in the Tumor Microenvironment (TME) can lead to exhaustion of T cells, which are responsible for identifying and attacking cancer cells. Cellular immunotherapy uses a patient’s own immune cells, including genetically engineered CAR-T, to recognize and destroy cancer cells, and has emerged as one of the most transformative advances in cancer treatment in recent years. The contributions of the three laureates have laid the foundation for a new era of “living drugs,” turning the patient’s immune system into a powerful medicine with far-reaching impact.

Since the first FDA approval in 2017, CAR-T therapy has already benefited over 30,000 patients with blood cancers worldwide. These therapies provide life-saving options for patients with recurrent and/or refractory blood cancers. Furthermore, TIL therapy has established a new option for treating advanced solid tumors, especially metastatic melanoma. Recent advances in CAR-T therapy in 2026 have also expanded into areas such as CRISPR-Cas9-based cell engineering, treatment of autoimmune diseases such as systemic lupus erythematosus, cardiac injury repair, and research targeting senescence.

Academician Wen-Chang Chang, Chair of the Tang Prize Selection Committee in Biopharmaceutical Science, noted that the Tang Prize in Biopharmaceutical Science recognizes successful drug development, as well as medical and technological research that leads to clinical treatment breakthroughs. In the field of tumor immunology, the inaugural Tang Prize laureates, Drs. James P. Allison and Tasuku Honjo, respectively identified CTLA-4 and PD-1 as key inhibitory immune checkpoints, paving the way for the development of antibody drugs known as immune checkpoint inhibitors and bringing major advances to the treatment of many cancers, particularly certain solid tumors. This year’s three laureates specialize in cellular immunotherapy. From Dr. Rosenberg’s pioneering clinical work with TILs, to the foundational advances by Dr. Sadelain and Dr. June that brought CAR-T therapy toward maturity and clinical application, the three scientists helped turn the human immune system into a powerful anti-cancer medicine, creating breakthrough treatments for malignant blood diseases such as leukemia, lymphoma, and multiple myeloma.

 

The pioneering work in this field was fundamentally established by Dr. Steven A. Rosenberg, widely known as the “Father of Cancer Immunotherapy.” As Chief of the Surgery Branch at the National Cancer Institute (NCI) since 1974, he has built the foundational clinical framework for adoptive cell therapy (ACT) 1. In the 1980s, Dr. Rosenberg showed that high-dose interleukin-2 (IL-2) could stimulate T cell proliferation and enhance their ability to kill cancer cells, leading to regression of metastatic tumors — the first clinical proof of T cells’ anti-cancer potential, leading to the FDA approval of IL-2 as the first cancer immunotherapy agent. He also demonstrated that TILs could induce regression of metastatic melanoma. In the 1990s, he achieved another milestone by receiving the first regulatory approval to introduce foreign genes into humans3.

From The National Cancer Institute

Source: https://ccr.cancer.gov/staff-directory/steven-a-rosenberg 

Dr. Rosenberg has pioneered the development of effective immunotherapies for patients with advanced cancer. His studies of cell transfer immunotherapy using tumor infiltrating lymphocytes (TIL) have resulted in durable complete remissions in patients with advanced solid cancers. He has also pioneered the development of gene therapy and was the first to successfully insert foreign genes into humans. His recent studies of the adoptive transfer of genetically modified lymphocytes using chimeric antigen receptors (CAR) or conventional T-cell receptors (TCR) have resulted in the regression of metastatic cancer in patients with lymphomas and solid cancers. His current research is aimed at defining the host immune response of patients to their cancers. These studies emphasize the ability of human lymphocytes to recognize unique cancer antigens and the identification of anti-tumor T-cell receptors that can be exploited to develop new cell transfer immunotherapies. As Chief, Dr. Rosenberg also oversees the Branch’s extensive clinical program aimed at translating scientific advances into effective immunotherapies for patients with advanced cancers arising from the breast, gastrointestinal tract, and pancreas.

Please Watch VIDEO

Dr. Michel Sadelain and Dr. Carl H. June are two leading pioneers in the development of CAR-T cell therapy. In the early development of antigen receptor engineering, research teams incorporated the intracellular CD3ζ chain4— responsible for transmitting activation signals — which Dr Sadelain found to be ineffective on its own.  He discovered that integrating an additional CD28 co-stimulatory domain2 directly into the receptor yielded T cells with therapeutic potential, thereby establishing the core architecture that has become standard framework for all subsequently FDA-approved CAR-T therapies. In addition, Dr. Sadelain identified a molecule named CD19 as a potential target for treating B cell malignancies, which include leukemias and lymphomas, and provided the first demonstration that human CD19 CAR T cells could treat cancer in mice. In 2013, his team at Memorial Sloan Kettering Cancer Center (MSKCC) reported the first significant therapeutic responses to CD19 CAR-T cells in adults with relapsed and refractory acute lymphoblastic leukemia (ALL).

Dr. June made the key breakthroughs that carried CAR-T therapy toward clinical success. He helped demonstrate that CD28 co-stimulation2 as essential for T-cell activation and applied the anti-CD3 and anti-CD28 bead expansion protocol, which has become the global manufacturing standard for CAR-T cells. He also engineered CAR constructs incorporating the 4-1BB (CD137) co-stimulatory domain and the T-cell receptor-zeta chain (TCR-ζ) to enhance the proliferation and long-term survival of transferred T cells, while also addressing the challenges of large-scale manufacturing. Dr. June then led the first successful clinical trials of CD19-targeted CAR-T cells, achieving durable remissions in patients with chronic lymphocytic leukemia (CLL) and acute lymphoblastic leukemia (ALL). His partnership with Novartis culminated in Kymriah becoming the first FDA-approved CAR-T therapy in 2017, marking a major step in bringing CAR-T therapy from research into clinical medicine.

Among the many patients who have benefited from CAR-T therapy, the story of Emily Whitehead is especially compelling. She was the first pediatric patient to receive CAR-T therapy. Diagnosed with ALL at just 5 years old in 2010, she underwent more than 16 months of chemotherapy without success before receiving CAR-T therapy in 2012. After treatment, her cancer went into complete remission, and she has remained healthy for 14 years. Over the years, Emily has continued to share her story publicly, advocating and raising funds for leukemia patients every year. Her recovery has moved and inspired countless patients and families.

Please Watch VIDEO on Dr. June

Footnotes:

  1. Adoptive cell transfer (ACT) is a form of immunotherapy in which a patient’s own immune cells  are collected, expanded or modified outside the body, and then reinfused into the patient to attack cancer.
  2. CD28 is a co-stimulatory receptor on T cells that provides a crucial “second signal” for T-cell activation, proliferation, and survival.
  3. Because gene-transfer research raises ethical and safety concerns, it had long been subject to strict regulation. The 1990 approval laid an important foundation for later genetically modified immune cell therapies.
  4. The CD3ζ chain is an intracellular signaling chain in T cells that helps initiate T-cell activation and immune-killing responses.

 

About the Tang Prize

Since the advent of globalization, humanity has enjoyed unprecedented benefits from advances in civilization and science. Yet a multitude of challenges, such as climate change, the emergence of new infectious diseases, the widening wealth gap, and moral degradation, have surfaced along the way. Against this backdrop, Dr. Samuel Yin established the Tang Prize in December 2012. It consists of four award categories: Sustainable Development, Biopharmaceutical Science, Sinology, and Rule of Law. Every two years, four independent and professional selection committees, comprising many internationally renowned experts, scholars, and Nobel laureates, choose Tang Prize laureates who have made substantive contributions and generated a far-reaching impact on the world, regardless of race, nationality, gender, or religion. A cash prize of NT$50 million (approximately US$1.6 million) is allocated to each category, with NT$10 million (approximately US$320,000) of it being a grant intended for research or educational outreach programs to encourage professionals in every field to examine mankind’s most urgent needs in the 21st century, and become leading forces in the sustainable development of human society through their outstanding research outcomes and active civic engagement.

 

For more articles on Tumor Infiltrating Lymphocytes and CAR-T Therapy on this Open Access Scientific Journal please see:

Tumor Infiltrating Lymphocytes (TIL) as a first of kind FDA approved immunotherapy for cancer
2018 Albany Medical Center Prize in Medicine and Biomedical Research goes to NIH’s Dr. Rosenberg and fellow immunotherapy researchers James P. Allison, Ph.D., and Carl H. June, M.D.
2018 Nobel Prize in Physiology or Medicine for contributions to Cancer Immunotherapy to James P. Allison, Ph.D., of the University of Texas, M.D. Anderson Cancer Center, Houston, Texas. Dr. Allison shares the prize with Tasuku Honjo, M.D., Ph.D., of Kyoto University Institute, Japan
Live Notes from @AACR’s #cbi16 Meeting on Precision Medicine: Success Story and Dr. Stephan Grupp

 

Posted in Albany Medical Center Prize in Medicine and Biomedical Research, CANCER BIOLOGY & Innovations in Cancer Therapy, Cancer-Immune Interactions, CAR-T, Engineering Better T Cells, Immune Engineering, Immunotherapy, Interviews with Key Opinion Leaders (KOLs), Life Sciences Breakthrough Prize | Tagged , , , , , , , | Leave a Comment »

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Tumor Infiltrating Lymphocytes (TIL) as a first of kind FDA approved immunotherapy for cancer

June 12, 2026 by sjwilliamspa

Tumor Infiltrating Lymphocytes (TIL) as a first of kind FDA approved immunotherapy for cancer

Reporter: Stephen J. Williams, Ph.D.

 

Source AACR; https://www.aacr.org/about-the-aacr/newsroom/pillars-cancer-care/boosting-the-immune-response-to-cancer/?utm_source=twitter&utm_medium=social 

Decades of pioneering research led to a first-of-its-kind FDA approval for a new type of immunotherapy—tumor-infiltrating lymphocyte (TIL) therapy.

Tumor infiltrating lymphocytes (TILs) have been thought for years to be a key immune regulator of the growth of tumor cells and these specialized T-cells have been found in many tumor microenvironments, especially in solid malignancies.  It was felt, if one could purify these immune cells and genetically alter them to induce a killer T-cell response,  these modified TILs would be a great therapeutic.  However it has been a challenge to purify, modify, and induce these cells to be able to infiltrate the tumor microenvironment.  These issues restricted their therapeutic utility towards solid tumors and posed this challenge for decades.  However, just recently the FDA has approved a TIL therapy for metastatic melanoma, especially for those melanomas that failed PD-L1 immunotherapies or B-Raf inhibitors (if expressing the corresponding B-Raf mutation.

Jennifer Ficko has been cancer-free for more than seven years, thanks to a clinical trial and an innovative form of immunotherapy. Diagnosed with stage 4 melanoma in 2010, she enrolled in several clinical trials to little avail—the tumor either didn’t respond, or the treatment led to debilitating side effects. That was until 2017, when Jennifer enrolled in a clinical trial evaluating lifileucel, a novel type of immunotherapy called tumor-infiltrating lymphocyte (TIL) therapy. The treatment left her weak for months afterward—but it worked. Her tumors disappeared, and she has not had any recurrences since. The success of lifileucel for Jennifer and many other patients enrolled in the clinical trial led to its approval in 2024 (under the brand name Amtagvi), making it the first treatment of its kind to be greenlit by the U.S. Food and Drug Administration (FDA).

“Today I’m doing fabulously, and I am just thankful that I was given this opportunity,” said Jennifer, who was featured in the AACR Cancer Progress Report 2024.

The premise of TIL therapy was pioneered by Steven A. Rosenberg, MD, PhD, chief of surgery at the National Cancer Institute (NCI) and a Fellow of the AACR Academy, who long hypothesized that the patient’s immune system could be a powerful ally in the fight against cancer.    “The accumulation of associative evidence led me to spend my entire career trying to find immunotherapies for the treatment of patients with cancer,” he said.Dr. Rosenberg remained committed to developing effective TIL therapy for more than three decades, leading the field in developing, testing, and enhancing this novel form of cancer treatment—research that was made possible by federal investments in the NCI.

What is TIL Therapy?

Lifileucel and other TIL therapies under investigation work through the same basic principle: collect the patient’s tumor tissue through biopsy or surgery, isolate from the tissue the T cells that have infiltrated the tumor (called TILs), promote proliferation of the isolated TILs to increase their number, and deliver the expanded TILs back into the patient along with an infusion of the protein IL-2 to stimulate TIL proliferation and activation within the patient’s body.

The 30-year Journey From Discovery to FDA Approval

The story of TIL therapy can be traced back to 1986, when Dr. Rosenberg and colleagues reported the discovery of TILs in human tumors and a method to expand them in the lab. When the human TILs were expanded and injected into mice, they led to regression of metastatic tumors in the liver and lungs. During the 1980s and ’90s, Dr. Rosenberg spearheaded clinical trials at the NCI testing TILs in patients. The trials illustrated the promise of TILs for cancer therapy, but they also revealed the shortcomings of this approach—namely the short-lived nature of treatment responses. Dr. Rosenberg and others continued to explore ways to overcome the challenges facing TIL therapy. In the early 2000s, they found that using chemotherapy to deplete the patient’s own immune cells prior to TIL infusion (called lymphodepleting conditioning) increased response rates and made responses more durable. Over the following decade, it became increasingly clear that TILs could be effective for patients whose melanomas did not respond to established treatments, and, in late 2023, researchers reported that almost 50% of patients who were treated with lifileucel after prior therapy were alive four years later—data that led to the historic FDA approval of lifileucel in January 2024.

The Importance of Cancer Research

“We have had a lot of progress in [treating] melanoma in the last 20 years,” said Harriet Kluger, MD, Jennifer’s oncologist and a professor of medicine at Yale University who was involved in the clinical testing of lifileucel. “We are able to control metastatic melanoma, and possibly even cure, in at least half of our patients now, but half isn’t enough. “That’s why these new therapies are important. Particularly, lifileucel is approved for patients in whom the other approved drugs don’t work,” she added. “Any time we can get results in that setting, we are getting closer and closer to our overall goal, which is curing 100% of our patients 100% of the time.”

Advances like these rely on investments to fund the basic, translational, and clinical research that pave the way for life-saving therapeutics for patients. “Cancer research is expensive, scientific research is expensive. And the more people we have that are smart, that have been educated appropriately, that are creative and innovative, the more of those people we can bring into research against deadly diseases such as cancer, the more rapidly progress will be made,” said Dr. Rosenberg.“The resources to do that, provided by the government as well as private institutions, I think [are] going to play a very important role. It has played an important role and will continue to play an important role.”

Other Articles of Note on Cancer Immunotherapy and Tumor Infiltrating Lymphocytes on this Open Access Online Scientific Journal Include:

Cancer-free after immunotherapy treatment: Treating advanced colon cancer – targeting KRAS gene mutation by tumor-infiltrating lymphocytes (TILs) and Killer T-cells (NK)
LIVE – 8/29 – CHI’s Oncolytic Virus Immunotherapy and ADOPTIVE CELL THERAPY, August 28-29, 2017 Sheraton Boston Hotel | Boston, MA
Another Promise for Immune Oncology
Issues Need to be Resolved With ImmunoModulatory Therapies: NK cells, mAbs, and adoptive T cells
Sleeping Threats: Immune System’s Watch on Dormant Cancer

 

 

Posted in CANCER BIOLOGY & Innovations in Cancer Therapy, Cancer-Immune Interactions, Cytokines, Efficacy of Anti-Tumor T Cells, Engineering Better T Cells, Immunotherapy | Tagged , , , , , , , , , | Leave a Comment »

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