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Alliance for Cancer Gene Therapy to honor Dr. Crystal Mackall with Edward Netter Leadership Award

Posted in Biological Engineering, Biological Networks, Gene Regulation and Evolution, Cancer - General, Cancer and Current Therapeutics, Cell Level, Childhood cancer, Drug Delivery Platform Technology, Gene Therapy & Gene Editing Development, Genetics & Innovations in Treatment, Interviews with Key Opinion Leaders (KOLs), Interviews with Scientific Leaders, Life Sciences Breakthrough Prize, Metabolic Immuno-Oncology, Metastasis Process, Oligonucleotide Therapeutics & Delivery, tagged Alliance for Cancer Gene Therapy, Cancer immunotherapy, CART therapy, Edward Netter Leadership Award, gene therapy, modulation of cell and gene therapies, Stanford, University of Pennsylvania on April 8, 2023| Leave a Comment »

Alliance for Cancer Gene Therapy to honor Dr. Crystal Mackall with Edward Netter Leadership Award

Reporter: Stephen J. Williams, PhD

Past recipient and cancer research pioneer Carl June, MD, to present award to Dr. Mackall

Alliance for Cancer Gene Therapy (ACGT) will award the Edward Netter Leadership Award to Crystal Mackall, MD, of Stanford University, at the ACGT Awards Luncheon on March 30 at Riverpark restaurant at the Alexandria Center for Life Science, located at 450 E. 29th St., New York City.

Named for ACGT co-founder, Edward Netter, the award recognizes a researcher who has made unparalleled and groundbreaking contributions to the field of cell and gene therapy for cancer. Dr. Mackall is a leader in advancing cell and gene therapies for the treatment of solid tumors, with a major focus on children’s cancers.

In addition to being an ACGT research fellow and a member of ACGT’s Scientific Advisory Council, Dr. Mackall is the Ernest and Amelia Gallo Family professor of Pediatrics and Medicine at Stanford University, the founding director of the Stanford Center for Cancer Cell Therapy, associate director of the Stanford Cancer Institute, leader of the Cancer Immunotherapy Program and director of the Parker Institute for Cancer Immunotherapy. She has led numerous groundbreaking clinical trials to treat children with sarcomas and brain cancers.

“There is exciting progress happening in the field of cancer cell and gene therapy,” said Kevin Honeycutt, CEO and president of ACGT. “We continue to see the FDA approve cell and gene therapy treatments for blood cancers, while research for solid tumors is now progressing to clinical trials. These successes are linked to the funding of ACGT, and Dr. Crystal Mackall is one of the best examples of a researcher who refused to accept the status-quo of standard cancer treatment and committed to developing novel cell and gene therapies for children with difficult-to-treat tumors. ACGT is proud that Dr. Mackall is an ACGT Research Fellow, a member of ACGT’s Scientific Advisory Council, and the newest recipient of the Edward Netter Leadership Award.”

The ACGT Awards Luncheon will celebrate the non-profit organization’s 20th anniversary and usher in a new decade as the only nonprofit dedicated exclusively to funding cancer cell and gene therapy research. ACGT funds innovative scientists and biotechnology companies working to harness the power of cell and gene therapy to transform how cancer is treated and to drive momentum toward a cure.

The Edward Netter Leadership Award will be presented to Dr. Mackall by Carl June, MD, of the University of Pennsylvania, who received the honor at ACGT’s 2019 Awards Gala. ACGT grant funding enabled Dr. June to research and develop cell and gene therapies that led to the first FDA approvals of CAR T-cell therapies for cancer.

For information about purchasing a ticket to the ACGT Awards Luncheon, visit the ACGT Awards Luncheon website (https://acgtfoundation.org/awards/), call Keri Eisenberg at (475) 400-4373, or email keisenberg@acgtfoundation.org.

Alliance for Cancer Gene Therapy (ACGT)

For more than 20 years, Alliance for Cancer Gene Therapy has funded research that is bringing innovative treatment options to people living with deadly cancers – treatments that save lives and offer new hope to all cancer patients. Alliance for Cancer Gene Therapy funds researchers who are pioneering the potential of cancer cell and gene therapy – talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. One hundred percent of all public funds raised by Alliance for Cancer Gene Therapy directly support research and programs. For more information, visit acgtfoundation.org, call (203) 358-5055, or join the Alliance for Cancer Gene Therapy community on Facebook, Twitter, LinkedIn, Instagram and YouTube @acgtfoundation.

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Real Time Coverage @BIOConvention #BIO2019: Issues of Risk and Reproduceability in Translational and Academic Collaboration; 2:30-4:00 June 3 Philadelphia PA

Posted in Computational Biology/Systems and Bioinformatics, Conference Coverage with Social Media, Global Partnering & Biotech Investment, Investment in Technological Breakthrough, Life Sciences Breakthrough Prize, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, tagged academic advancement, Academic publishing, basic research, Big data, collaboration, data validity, Innovation, Pharmaceutical industry, Public–private partnership, research funding, tech transfer on June 3, 2019| Leave a Comment »

Real Time Coverage @BIOConvention #BIO2019: Issues of Risk and Reproduceability in Translational and Academic Collaboration; 2:30-4:00 June 3 Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

Derisking Academic Science: The Unmet Need

Translating academic research into products and new therapies is a very risky venture as only 1% of academic research has been successfully translated into successful products.

Speakers

Richard SollSenior Advisor, Strategic Initiatives at WuXi AppTec

Jim AudiaExecutive Director atChicago Biomedical Consortium

Collaboration from Chicago area universities like U of Chicago, Northwestern, etc. First phase was enhance collaboration between universities by funding faculty recruitment and basic research. Access to core facilities across universities. Have expanded to give alternatives to company formation.

Curtis KeithCSO atBlavatnik Biomedical Accelerator, Harvard University

Half of the partnerships from Harvard and companies have been able to spin out viable startups.

Nadim ShohdyAssistant Dean, Therapeutics Alliances atNYU Langone Health

Most academic PI are not as savvy to start a biotech so they bring in biotechs and build project teams as well as developing a team of ex pharma and biotech experts. Derisk as running as one asset project. Partner as early as possible. A third of their pipeline have been successfully partnered. Work with investors and patent attorneys.

Dominique VerhelleSr Director, Strategic Academic Alliances, Center for External Innovation atTakeda

Focused on getting PIs to get to startup. Focused on oncology and vaccines and I/O. The result can be liscensing or partnership. Running around 50 to 60 projects. Creating a new company from these US PI partnerships.

Most projects from Harvard have been therapeutics-based. At Harvard they have a network of investors ($50 million). They screen PI proposals based on translateability and what investors are interested in.

In Chicago they solicit multiple projects but are agnostic on area but as they are limited they are focused on projects that will assist in developing a stronger proposal to investor/funding mechanism.

NYU goes around university doing due diligence reaching out to investigators. They shop around their projects to wet their investors, pharma appetite future funding. At Takeda they have five centers around US. They want to have more input so go into the university with their scientists and discuss ideas.

Challenges:

Takeda: Data Validation very important. Second there may be disconnect with the amount of equity the PI wants in the new company as well as management. Third PIs not aware of all steps in drug development.

Harvard: Pharma and biotech have robust research and academic does not have the size or scope of pharma. PIs must be more diligent on e.g. the compounds they get from a screen… they only focus narrowly

NYU: bring in consultants as PIs don’t understand all the management issues. Need to understand development so they bring in the experts to help them. Pharma he feels have to much risk aversion and none of their PIs want 100% equity.

Chicago: they like to publish at early stage so publication freedom is a challenge

It Starts with One Health: A Guide to Translational Science from Research to Investment

115B, Level 100

Speakers

William KareshExecutive Vice President for Health and Policy atEcoHealth Alliance

Marianne de backerVP, Global M&A Operations and Divestitures (Pharmaceuticals Group), Global Infectious Diseases & Vaccines Business Development atJohnson & Johnson Innovation

Randal J. KirkChief Executive Officer atIntrexon

Dr. Hilary MarstonMedical Officer/Policy Advisor, Office of the Director, NIAID atNational Institute of Health

Tina ParkerProgram Officer – Translational Centers of Excellence and Research Coordination Section Office of Biodefense, Research Resources and Translational Research, DMID/NIAID atNIH

Novel Approaches to Improve Reproducibility in Academia-Industry Collaborations

112AB, Level 100

Speakers

Florence BietrixOperations Manager at EATRIS

Dr. Nathan CoussensSenior Research Scientist at National Center for Advancing Translational Sciences, National Institutes of Health

Leonard FreedmanCSO atFrederick National Laboratory for Cancer Research

Marian Hajduch Scientific Director at IMTM

Weida TongDirector Division of Bioinformatics and Biostatistics at NCTR/FDA

Dr. Freedman: Most scientists responding to Nature survey said yes a reproduceability crisis. The reasons: experimental bias, lack of validation techniques, reagents, and protocols etc.

And as he says there is a great ECONOMIC IMPACT of preclinical reproducability issues: to the tune of $56 billion of irreproducable results (paper published in PLOS Biology). If can find the core drivers of this issue they can solve the problem. STANDARDS are constantly used in various industries however academic research are lagging in developing such standards. Just the problem of cell line authentication is costing $4 billion.

Dr. Cousins: There are multiple high throughput screening (HTS) academic centers around the world (150 in US). So where does the industry go for best practices in assays? Eli Lilly had developed a manual for HTS best practices and in 1984 made publicly available (Assay Guidance Manual). To date there have been constant updates to this manual to incorporate new assays. Workshops have been developed to train scientists in these best practices.

NIH has been developing new programs to address these reproducability issues. Developed a method called

“Ring Testing Initiative” where multiple centers involved in sharing reagents as well as assays and allowing scientists to test at multiple facilities.

Dr.Tong: Reproduceability of Microarrays: As microarrays were the only methodology to do high through put genomics in the early 2000s, and although much research had been performed to standardize and achieve best reproduceability of the microarray technology (determining best practices in spotting RNA on glass slides, hybridization protocols, image analysis) little had been done on evaluating the reproducibility of results obtained from microarray experiments involving biological samples. The advent of Artificial Intelligence and Machine Learning though can be used to help validate microarray results. This was done in a Nature Biotechnology paper (Nature Biotechnology volume28, 827–838 (2010)) by an international consortium, the International MAQC (Microarray Quality Control) Society and can be found here

The MicroArray Quality Control (MAQC)-II study of common practices for the development and validation of microarray-based predictive models

However Dr. Tong feels there is much confusion in how we define reproduceability. Dr. Tong identified a few key points of data reproduceability:

Traceability: what are the practices and procedures from going from point A to point B (steps in a protocol or experimental design)
Repeatability: ability to repeat results within the same laboratory
Replicatablilty: ability to repeat results cross laboratory
Transferability: are the results validated across multiple platforms?

The panel then discussed the role of journals and funders to drive reproduceability in research. They felt that editors have been doing as much as they can do as they receive an end product (the paper) but all agreed funders need to do more to promote data validity, especially in requiring that systematic evaluation and validation of each step in protocols are performed.. There could be more training of PIs with respect to protocol and data validation.