Healthcare analytics, AI solutions for biological big data, providing an AI platform for the biotech, life sciences, medical and pharmaceutical industries, as well as for related technological approaches, i.e., curation and text analysis with machine learning and other activities related to AI applications to these industries.
LIVE Day 1 – 2017 MassBio Annual Meeting March 30, 2017 8:00 AM, Royal Sonesta Boston
Reporter: Aviva Lev-Ari, PhD, RN
Real Time Coverage: Aviva Lev-Ari, PhD, RN
#MassBioAM17 #PatientDriven @pharma_BI @AVIVA1950
Day 1
Breakfast and Registration
8:00 am–9:00 am
Welcome Remarks & MassBio Board Elections
Plenary
9:00 am–9:30 am
Robert Coughlin, CEO, MassBio
63,000 emp
1600 drugs in the pipeline
3 million cures in the near future
Life Sciences Challenges
Negative impact on Biotech Industry: Policies from the New Administration: Repeal & Replace ACA, Travel Ban, Health coverage, cost and access
proposed cuts to NIH, devestation to Institutions in MA
Drug costs and Price Transparencies
MassBio — opposed these policies from Washington
MassBio will continue to lead like with ACA
MassBio will continue to embrace Value-based Contracting and will continue to suppost generics which will lower drug costs
MA is HQS of Biotech, partnered with NEHA, biopharma and BlueCross BlueShield
ENsuring Patient access and drug affordability
MA Health Insurance (BlueCross BlueShield) partners with Biotech and Pharma to identify strategy to reduce cost and Monitor Value-based Contracting
Vibrant Healthcare Sector and Innovative Biotech
Saving Lives is the goal
List of Sponsors is Long: Catalent, Corealis, fisherscientific, Amgen, Biogen, Nutter, SHire, Radius, Snow, Uniqure, MA Life Sciences Center
Charlie Brown in the afternoon, committed to the goals of MassBio
Abbie Celniker, Chair, MassBio Board of Directors
MassBio works as a collective advocacy agency of multiple stakeholders
Work together as Partners
Engagement, Involvement
Community experiencing growth and maturation, average income $140,000 commercial Lab work spaces
Diversity workforce, immigrants, spectrum of cultures, gender and training
Survey: Female Mangers represent commitment, credentialing, 50% of companies surveyed SVP less hopeful than above and below
Culture and Values alignment for employ retention, primarily Women
Opening Keynote
Plenary
9:30 am –10:15 am
Dan Schorr, Founder, Chief Icecream Officer, VICE CREAM
Coffee Break
10:15 am–10:30 am
Challenges the Future Generation of the Industry Faces
Better Business
10:30 am –11:30 am
MODERATOR:
Johannes Fruehauf, Co-founder, LabCentral
Postdoc at Harvard, transition from Academic environment to commercial, not awareness of Biotech industry, in Germany went through Medical School and was aware of Big 200 years old Pharma companies
CROs in Boston, Automated Labs, workflow outsourced if alck of funding
PANELISTS:
Will Adams, CSO, President, Founder, Riparian Pharmaceuticals – CVD drug development
human genetics, differential biology, therapeutics for CVD
Lab at HMS research on Vasculture, one important pathway
NIH – $500,000
Using CROs
Chris Colbert, Director of Programming, Harvard Innovation Lab – seminars open to the Public
Harvard Entrepreneur activity for students and Alunmi, started EdTech in 2014, defining problems and solutions
Education vs outcomes – capacity to start a business
complementary to Education, resources to become entrepreneur
individuals are driving the process
AI
Tim Wang, Co-founder, KSQ Therapeutics
at MIT, now with VC at KSQ, 30-40 emp, hires are computentional biology, cancer biology from pulls of Boston-based Postdocs,
involvement in wriitng a business plan and funding
being active and interdisciplinary
Aging
Trends in Science
10:30 am –11:30 am
Evolution of Analytics in Life Sciences: The Past, the Present and Future of Analytics
Convergence
10:30 am –11:30 am
The MassBio Annual Awards Luncheon
11:45 am –1:15 pm
11:55 – 12:00 Patient Story
12:00 – 12:30PM 2016 Joshua Boger Innovative School of the Year Award – Presented to Hingham High School
12:30 – 1PM 2016 Henri A. Termeer Innovation Leadership Award
Presented to David Meeker, Executive VP and Head of Sanofi Genzyme
Sources of inspirations Henri A. Termeer the founder of Genzyme
Consumer of HealthCare for 62 years is critical: Trust and Access for vulnerable patient when they are ill
Return on Investment in Drugs, incentives to develop vs generics, BioSimilars, Price will drop
Societal contract: Recognizing the value and the Pharma Industry needs to increase self regulating
If Gov’t will regulate PRICE of drug, innovations will decrease
Thank you for the award
1:00 – 1:15 2017 MassBio Leading Impact Award introduced by EDAC Chair Mark Bamforth – Lab Space
Presented to Pfizer, Accepted by Morrie Birnbaum, SVP & CSO, Internal Medicine
2100 emp in MA
single building in Kendall Square
Andover facility
three R&D units: Drug Discovery
Pfizer, Accepted by Morrie Birnbaum, SVP & CSO, Internal Medicine
2014 – Kendall Square site: Becoming Premier Pfizer’s Research Center, close to Science, Scientists and Patients
Recognition that working with Foundations and Academic centers Not able to do it alone
Close relations with MassBio
Thanks to MassBio
Remarks from Governor Charlie Baker
Plenary
1:15 pm –1:30 pm
Patient Driven – Governor Charlie Baker
MA #1 in STEM occupations
Knowledge ecosystem at Kendall Square
Smart Fibers HQS: MIT and MA players joint investment
Robotics Competition in MA
BioPharmaceutical Competision: WPI, MIT State of MA, ULowell – joint investment
IBM Watson in MA, MA Life Sciences Center
Discovery and inquiry – MA is a place people look from where solutions will come
Four big element: Grant from MA Life Sciences Center gave Lab equipment to Brimfield High School
Collaborate with Academia for apprenticeship
Partnership: Draper Lab and Mass Eye and Ear: develop drug eluting device with drug for Macular Degeneration to replace the needle in an injection into the eye
Invest in workforce – human training skills for the technology sector
Public Transportation Investment: Replace from mechanical to digital in 2022 all switches of the T system – Double capacity on Red Line, manufactured in Springfield, MA
Affordable Housing Units: MA State Housing Dept largest owner of Housing in MA in area near Public Transportation
MassBio – Part of the MA economy with less problems that other industries
Cancer Moonshot and other Cancer related issues
Plenary
1:30 pm–2:30 pm
MODERATOR: John Hallinan, CBO, MassBio
PANELISTS:
Laurie Glimcher, President & CEO, Dana-Farber Cancer Institute
Was part of Cancer Moonshot – 10 key directions
Fund $1.8 Billion, fund needs be approved each year
Funds for 12 implimentation Teams – Applications for 10 key directions: How a Tumor evolve and resist treatment
Create an initiative of Data Sharing acquired from Patients
understanding mutation unique
cancer immunotherapy: fight tumor, stimulate immune system CHeck-Point blockers – only 20% of patients are responsive
Big data to identify new drug targets so more patients will respond
Tsunami in BioMedical Research regarding NIH to face $1.2 Billion cut (equal indirect cost) – Patients are best advocates –
MA figured out how to do collaboration among stackholders
Several Cancers will become Chronic diseases not quite Cure in 5-10 years
Prevention and early detection: Vaccination, HPV (only 14% girls get vaccine for Cervical, anal and throat) in Japan 0% of HPV.
environment that surrounding the tumor, immune cells suppressing the immune system
Mathew Meyerson, Prof. of Pathology, Dana-Farber Cancer Institute, Broad Institute
3-D cancer genomics sequencing for every cell, new therapies interaction of Cancer and the environment, computing and Internet use for Patient direct involvement in Research – enrollment
Child Cancers
Prevention: Vaccination, HPV, HEP B
HIV a cocktail of drugs, kill a cancer from many angles
access to clinical trial: Connect Clinical Trials information with Doctors
Electronic cigarets and Lung cancer – connection needs to be researched
Chandra Ramanathan, Head East Coast Innovation Center, Bayer US
Bayer Partnership with Broad Institute
Immunotherapy Combination of Biomarkers
Sharing Data from Clinical Trials
Seema Singh Bhan, Head, Public Policy, Foundation Medicine
Technology to
harness Big data
Momentum via NCI, Cancer Moonshot
Foundation Medicine: Collaboration: Pharma, Goventment, FDA, Academic Institution 0 Liquid Biopsy assays developed and go through the Regulatory system
Patient-Doctor – List of Clinical Trials available for Patients
2:30 – 3:15 Breakout Sessions
2:30 pm–3:15 pm
CRISPR and What’s Next
Trends in Science
2:30 pm–3:30 pm
MODERATOR:
Scott Gillis, CEO, Onsite Therapeutics, Inc.
implantation of gene edited cells in Human in China
Bio Ethics
Jim Burns, President & CEO, Casebia Therapeutics, ex-Sanofi-Genzym
How to deliver tissue specific in vivo
Hymophylia
Immunodefficiency
30 emp – 60 emp year end
Incubate ideas
Joint venture CRISPR Therapeutics and Casebia
guidelines from the National Academy
George M. Church, Harvard Prof. of Genetics – now speaks for Genesis
In one year Clinical trial of porcine organ lab made – organ transplant
build new tool set write not read
Mattew Stanton, VP, Head of Chemistry, Moderna Therapeutics, ex-Merck
Sperm Analysis by Smart Phone, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 1: Next Generation Sequencing (NGS)
Sperm Analysis by Smart Phone
Reporter and Curator: Dr. Sudipta Saha, Ph.D.
Low sperm count and motility are markers for male infertility, a condition that is actually a neglected health issue worldwide, according to the World Health Organization. Researchers at Harvard Medical School have developed a very low cost device that can attach to a cell phone and provides a quick and easy semen analysis. The device is still under development, but a study of the machine’s capabilities concludes that it is just as accurate as the elaborate high cost computer-assisted semen analysis machines costing tens of thousands of dollars in measuring sperm concentration, sperm motility, total sperm count and total motile cells.
The Harvard team isn’t the first to develop an at-home fertility test for men, but they are the first to be able to determine sperm concentration as well as motility. The scientists compared the smart phone sperm tracker to current lab equipment by analyzing the same semen samples side by side. They analyzed over 350 semen samples of both infertile and fertile men. The smart phone system was able to identify abnormal sperm samples with 98 percent accuracy. The results of the study were published in the journal named Science Translational Medicine.
The device uses an optical attachment for magnification and a disposable microchip for handling the semen sample. With two lenses that require no manual focusing and an inexpensive battery, it slides onto the smart phone’s camera. Total cost for manufacturing the equipment: $4.45, including $3.59 for the optical attachment and 86 cents for the disposable micro-fluidic chip that contains the semen sample.
The software of the app is designed with a simple interface that guides the user through the test with onscreen prompts. After the sample is inserted, the app can photograph it, create a video and report the results in less than five seconds. The test results are stored on the phone so that semen quality can be monitored over time. The device is under consideration for approval from the Food and Drug Administration within the next two years.
With this device at home, a man can avoid the embarrassment and stress of providing a sample in a doctor’s clinic. The device could also be useful for men who get vasectomies, who are supposed to return to the urologist for semen analysis twice in the six months after the procedure. Compliance is typically poor, but with this device, a man could perform his own semen analysis at home and email the result to the urologist. This will make sperm analysis available in the privacy of our home and as easy as a home pregnancy test or blood sugar test.
The device costs about $5 to make in the lab and can be made available in the market at lower than $50 initially. This low cost could help provide much-needed infertility care in developing or underdeveloped nations, which often lack the resources for currently available diagnostics.
Swiss Paraplegic Centre, Nottwil, Switzerland – A World-Class Clinic for Spinal Cord Injuries
Author: Gail S. Thornton, M.A.
Co-Editor: The VOICES of Patients, Hospital CEOs, HealthCare Providers, Caregivers and Families: Personal Experience with Critical Care and Invasive Medical Procedures
Article ID #232: Swiss Paraplegic Centre, Nottwil, Switzerland – A World-Class Clinic for Spinal Cord Injuries. Published on 3/23/17
WordCloud Image Produced by Adam Tubman
The Swiss Paraplegic Centre (SPC, www.paraplegie.ch) in Nottwil, Switzerland, is a privately owned, leading acute care and specialist hospital employing more than 1,500 health professionals in 80 different occupations that focuses on world-class primary care and comprehensive rehabilitation of patients with spinal cord injuries. In addition to the SPC’s extensive range of medical and therapeutic care, treatment and services, the hospital offers advisory services, as well as research in the areas of paraplegia [paralysis of the legs and lower body, typically caused by spinal injury or disease], tetraplegia [also known as quadriplegia, paralysis caused by illness or injury that results in the partial or total loss of use of all four limbs and torso], prevention and related conditions. With 150 beds, the SPC provides modern facilities for rehabilitation and therapy, diagnostics, surgery, ongoing care, orthopedic technology, as well as social services and 24-hour emergency care.
In its 26-year history, the SPC has provided treatment and care to more than 20,000 in-patients. That number continues to grow exponentially due to the reputation of the SPC. In fact, the SPC’s staff performs their duties with effectiveness, expediency and cost-efficiency measures, requiring highly developed process-led medicine, centered around the needs of the patient.
The areas of medical specialty and centers of excellence include the Swiss Paraplegic Centre (SPC), the Swiss Spinal Column and Spinal Cord Centre (SWRZ), the Centre for Pain Medicine (ZSM) and the Swiss Olympic Medical Center (SOMC). These centers respectively offer patients cutting-edge medical treatment based on the most advanced research in areas covering treatment and rehabilitation cases of acute paraplegia, vertebral and spinal cord surgery, as well as services relating to pain management, sports medicine and preventive health checks.
Alongside the core focus on paraplegiology, the SPC is also equipped with the necessary medical facilities, allowing for the lifelong care of paraplegic patients. The SPC provides individually-tailored, comprehensive treatment in three phases (acute, reactivation and integration) using highly skilled staff and state-of-the-art equipment. The aim is always to re-establish a patient’s personal functionality, self-image and lifestyle to the fullest possible extent, with a holistic approach to treatment that includes mental, physical and psycho-social aspects, such as career, family and leisure activities.
Specialist services available at the SPC include amongst others orthopedics, neuro-urology, pain medicine, sports medicine, prevention, clinical research, emergency medicine, vehicle conversion and rehabilitation techniques. Medico-therapeutic treatments, such as physiotherapy, ergotherapy and training therapy are available, alongside advice and counseling services, such as professional reintegration.
The SPC is the largest of Switzerland’s four special hospitals for paraplegics and tetraplegics located in Nottwil/Lucerne, a town in central Switzerland on the shores of Lake Sempach. The other three facilities are in Basel, Sion and Zurich. Nowadays, the SPC consistently treats more than 60 percent of people with spinal cord injury in Switzerland and is fully occupied year-round.
Image SOURCE: Photographs courtesy of Swiss Paraplegic Centre, Nottwil, Switzerland. Interior and exterior photographs of the hospital.
Below is my interview with Hospital Director Dr. Med. Hans Peter Gmünder, M.D., which occurred in March, 2017.
As a privately owned clinic with a specialty in the rehabilitation of patients with spinal cord injuries, how do you keep the spirit of research and innovation alive?
Dr. Med. (medicinae) Gmünder: The goal of the Swiss Paraplegic Foundation, an umbrella organization that encompasses the Swiss Paraplegic Centre, is to create a unique network of services for people with spinal cord injury, from primary care through to the end of their lives. Its aim is to provide comprehensive rehabilitation and to reintegrate those affected into family life, society and the working environment.
We want to maintain our pioneering and leading role in the fields of acute medicine, rehabilitation and lifelong assistance to people with spinal cord injuries. By providing a comprehensive network of services featuring solidarity, medical care, integration and lifelong assistance, as well as research all in one place, we are unique in Switzerland and in other countries around the world.
People with spinal cord injury rely upon our network of services, which are at their disposal throughout their lives. The challenge facing us is to continually adapt these services to reflect current research and treatment to comply with our mission of delivering high-quality services. The trust which has been placed in us obliges us to continue our success story.
We have our own research department, closely linked to the Swiss Paraplegic Centre, and dedicated employees who draw upon their wide-ranging professional networks to stay on top of the latest international research.
We have a few examples that we’d like to share with you.
In 2013, the World Health Organization (WHO) published its first international health report on the topic of spinal cord injury, “International Perspectives on Spinal Cord Injury.” It was developed in collaboration with Swiss Paraplegic Research in Nottwil and a team of international experts.
In the summer of 2014, the Swiss Paraplegic Centre became the first rehabilitation center in Switzerland to implement exoskeletons [external covering for the body that provides both support and protection] in the rehabilitation and training of patients with spinal cord injury. Our experiences are included in an international study, and will contribute to the development of useful mobility aids for people with spinal cord injuries.
At the end of October 2016, an estimated 9,000 visitors came to Nottwil for two days of celebrations to mark five anniversaries — the Swiss Paraplegic Foundation turned 40, the Swiss Paraplegics Association was 35, the Swiss Paraplegic Centre celebrated 25 years, Swiss Paraplegic Research reached 15 years, and it was the 80th birthday of the founder and honorary president, Dr. Med. Guido A. Zäch, M.D.
What draws patients to the Swiss Paraplegic Centre?
Dr. Gmünder: We support people with spinal cord injuries throughout their lives. It is the unique, holistic approach to acute medicine, rehabilitation and lifelong medical, professional and social assistance that draws patients from Switzerland and many other countries to our clinic in Nottwil.
For example, in cases where we have individuals involved in serious accidents, the comprehensive rehabilitation of a patient with spinal cord injury begins at the scene of the accident. The aim of comprehensive assistance follows in three stages – acute, reactivation and integration phase – through the appropriate, individual deployment of specialist personnel and instruments. We rescue the individual at the scene of the accident and provide the right acute therapy. What follows is an initial rehabilitation through specialists in diagnosis, surgery, therapy and care, and then comes lifelong support and care with the aid of specialists.
Following the disproportionately high percentage of people with tetraplegia admitted to the Centre for initial rehabilitation in 2014, our specialist clinic reported a higher proportion of people with paraplegia in 2015. Spinal cord injuries resulted from an accident in around half of all initial rehabilitation cases: falls led to the spinal cord injury in the case of 43 percent of people affected, sports accidents with 35 percent and road traffic accidents in 18 percent. In fact, 52,482 nursing days were clocked for a total of 1,085 in-patients who were discharged from the clinic after initial rehabilitation or follow-up treatment in 2015.
In fact, some of our patient success stories mentioned on our web site involve these individuals:
“I was a cheesemaker for 33 years with my own dairy; gardening was my second love. That was before I had my accident helping out on my son’s farm. I need a new hobby now that I will enjoy, that will fill my time and give me something to do when I get back home. Making art out of lime wood could appeal to me. While it is difficult for me to make the small cuts in the wood as I lack strength in my hand, patience will reap rewards. My most important objective? To be able to stand on my own feet and take a few steps again. I should have achieved that by the time I am discharged from the clinic in five months.” — Josef Kobler (58), tetraplegic following an accident.
“Since being diagnosed with a spinal cord injury, I come back to Nottwil a lot. For instance, to go the Wheelchair Mechanics Department to have the settings of my new wheelchair optimized. It replaces my legs and must fit my body perfectly. However, in most cases I attend the Centre for Pain Medicine of the SPC as an outpatient in order to have the extremely severe pains and muscle cramps, which I suffer from every day, alleviated. They became so severe that I had a pain pump with medication implanted at the SPC. It is apparent now that unfortunately the effect isn’t permanent. We are now giving electrostimulation a try. This involves applying electrodes to the vertebral canal. If I could finally get my pain under control, I would be able to return to work and set up my own business. That is my biggest wish. I have had an idea about what I could do.” — Hervé Brohon (41), paraplegic following an accident.
“I have always been passionate about cooking and have enjoyed treating my family and guests to my dishes and to the aperitifs that I have created myself. I absolutely want to be able to do that again. As independently as possible, of course. That is my objective. I have availed of the opportunity on a few occasions to try out the obstacle-free practice apartment and kitchen at the SPC. If I am able to go home in four weeks, my kitchen will also be adapted to be wheelchair-friendly. Whether I am cooking for two, four or six people is a much bigger consideration as a wheelchair user. I now have to consciously allow for time and effort. However, one thing is certain: I can’t wait to welcome my first guests.” — Isa Bapst (73), paraplegic following an accident.
How is the Swiss Paraplegic Centre transforming health care?
Dr. Gmünder: The Swiss Paraplegic Centre offers an integrated healthcare structure, including a wide range of medical specialists covering every aspect of medical care for those with spinal cord injuries.
In selected core disciplines for the care of people with spinal cord injuries, we also treat a large number of patients without spinal cord injuries. This relates primarily to pain medicine, spine- and spinal cord surgery and respiratory medicine.
In fact, the Swiss Paraplegic Foundation, our umbrella organization, has been an unbelievable success story, operating a network of services to benefit people with spinal cord injury.
Our Chairman of the Board of Trustees, Dr. Sc. Techn. (scientiae technicarum) Daniel Joggi, knows what it’s like to become totally dependent as he has been in a wheelchair for the past four decades.
Dr. Joggi tells his story: “I have been a wheelchair user ever since I had a skiing accident 39 years ago. I know what it is like to become totally dependent from one second to the next. How doggedly you have to battle to recover as much of your mobility as possible and, more especially, to be able to live a self-determined life again after a long process of resilience. The inner resolve it takes to plot a new course in life, to have relationships with others from a different perspective and to acquire new job skills. Therefore, I am eternally grateful along with all the other people in Switzerland with paraplegia and tetraplegia for the help, support and great solidarity that allow the Foundation to deliver all the services which are so immensely valuable to us.”
At the Swiss Paraplegic Centre, a 24-hour emergency department is staffed to handle any emergency. Please provide your thoughts on this critical component of diagnosis and care for newly diagnosed patients.
Dr. Gmünder: Yes, our Centre is recognized by the Swiss Union of Surgical Societies as a specialist clinic for first-aid treatment of paraplegics.
Statistics and experience clearly show that in 80 out of 100 cases, the damage to the spine and the spinal cord is not definite immediately after an accident. In the first six hours, there are real chances to mitigate or even avoid an imminent cross-paralysis. After that it is usually too late.
In addition to transferring an individual directly to the SPC, appropriate acute care is another important criterion for the success of the individual affected by spinal cord issues. That means that individuals are in the right place for the subsequent, comprehensive rehabilitation.
The benefits for our patients are:
Emergency service around the clock by specialists trained to minimize damage to the spinal cord and spine;
Admission and treatment of all patients with paraplegia from all over Switzerland;
Specific knowledge and practical experience in comprehensive rehabilitation of paraplegics;
Comprehensive range of medical and therapeutic services under one roof;
Modern equipment for precise, careful diagnostics and operations;
Consultancy and network for external experts in areas not covered by the SPC;
Interdisciplinary work in well-established teams; and
Central location proximity and quick access from all parts of the country.
What is your connection to the Swiss Paraplegic Research and its mission of getting “strategy into research” and “research into practice?”
Dr. Gmünder: The Swiss Paraplegic Research (SPR), connected to the Swiss Paraplegic Centre, is part of the Swiss Paraplegic Foundation (SPF) and is an integral part of the Nottwil campus.
It is the mission of Swiss Paraplegic Research to sustainably improve the situation of people with paraplegia or tetraplegia through clinical and interdisciplinary research in the long-term. The areas that are aimed to be improved are functioning, social integration, equality of opportunity, health, self-determination and quality of life.
Our Swiss Paraplegic Research has been supported by the Federal Government of Switzerland and by the Canton of Lucerne for eight years as a non-university research institution. We are proud of this accomplishment.
Our main research domains are in the areas of aging, neuro-rehabilitation, musculo-skeletal health, preserving and improving function of upper limbs, pain, pressure sores, respiration, urology and orthopedics.
The goal of Swiss Paraplegic Research is to promote the study of health from a holistic point of view, by focusing on the ‘lived experience’ of persons with health conditions and their interaction with society. We are, therefore, establishing a research network for rehabilitation research from a comprehensive perspective on a national and international level. This network will make it possible to practically apply the latest research findings to provide the best possible care and reintegration for people with paraplegia or tetraplegia.
This year, we received the approval of 18 new research projects and we had a total of 36 studies in progress under review, undertaken by and with the involvement of the Clinical Trial Unit (CTU), the department for clinical research at the Centre. For example, the successful implementation of a multi-center study on the use of walking robots (exoskeleton) merits special mention. Research was carried out in that study into the wide range of effects of maintaining movement for people with spinal cord injury.
The CTU will continue to carry out research in Rehabilitation Engineering in a cooperation with Burgdorf University of Applied Science and the research group headed by Professor Kenneth Hunt. The “Life and Care” symposium on breathing and respiration organized by the CTU provided a platform for an international knowledge exchange with national and international experts. This is crucial for further scientific development in respiratory medicine. In 2015, the CTU also launched the CTU Central Switzerland, in association with Lucerne Cantonal Hospital and the University of Lucerne. It supports clinics which are actively engaged in research with specific services, thereby enhancing Switzerland’s standing as a center of research.
How does the Swiss Paraplegic Foundation support your vision?
Dr. Gmünder: The Swiss Paraplegic Group includes the Swiss Paraplegic Foundation, which was established in 1975, two partner organizations — the Benefactors’ Association and the Swiss Paraplegics Association, and six companies owned by the Foundation. Those six companies are the Swiss Paraplegic Centre, the Swiss Paraplegic Research, Orthotec AG, ParaHelp AG, Sirmed Swiss Institute of Emergency Medicine AG, Seminarhotel Sempachersee AG.
The Swiss Paraplegic Foundation, founded by Dr. Med. Guido A. Zäch in 1975, is a solidarity network for people with spinal cord injuries, unrivaled anywhere in the world. Its work is based on the vision of medical care and comprehensive rehabilitation for people with paraplegia and tetraplegia, with a view towards enabling them to lead their lives with self-determination and with as much independence as possible, supported by the latest advances in science and technology.
The unique network of services of the Foundation is a strategic mix of Solidarity, Research, Medicine and Integration and Lifelong Assistance. Let me elaborate on these services.
Solidarity
The Foundation provides a comprehensive range of services for every area of a person’s life who has a spinal cord injury. The Nottwil campus serves to be a center of excellence for integration, assistance and lifelong learning for our patients.
The Foundation ensures that its benefactors and donors are aware of our list of services and can support us longer term.
The Foundation establishes a national and international network that will guarantee better basic conditions for people with spinal cord injury.
The Foundation encourages training of specialized personnel in the field of spinal cord injury.
Research
The Foundation contributes to the sustainable improvement of health, social integration, equal opportunities and self-determination of people with spinal cord injury by carrying out rehabilitation research.
The Foundation works closely with the World Health Organization (WHO) and encourages exchanges with universities and institutions locally and globally for the latest scientific findings and conducts academic training at the University of Lucerne.
The Foundation develops high-quality care standards for its patients.
Medicine
The Foundation offers all medical services needed for professional acute care and rehabilitation of people with spinal cord injury and encourages patients to become involved in their therapy and to take responsibility for their lives.
The Foundation strengthens relationships with partners in specific disciplines and local institutions to benefit people with spinal cord injury.
The Foundation is a member of committees with political influence to ensure that its patients receive highly specialized medical care.
Integration and Lifelong Assistance
The Foundation establishes a network throughout Switzerland to help people with spinal cord injury.
The Foundation offers comprehensive services to meet people’s needs to improve their integration into society.
The Foundation encourages people with spinal cord injury to lead an independent life and educate family and friends so they can provide the necessary support.
Moreover, in cases of hardship, the Foundation makes contributions towards the cost of walking aids, equipment and amenities for people with paraplegia and tetraplegia. It also takes on uncovered hospital and care costs.
Current market research shows that the Swiss Paraplegic Foundation ranks among the three most highly rated aid organizations in Switzerland. Can you please elaborate on why?
Dr. Gmünder: That is true. The Foundation is highly rated in terms of goodwill, innovation, competence and effectiveness. In addition, it is regarded as undoubtedly the most competent organization representing people with disabilities in Switzerland, according to several market research surveys.
So that we can continue to meet the demand for our patients, families and other visitors, plans are under way to upgrade our clinic and hotel on our premises.
We generally have interest from visitors to visit our Centre. Our guided tours and events enabled the general public to see how the foundation concept is put into practice, day in, day out. In Nottwil, 160 guides provided more than 11,000 visitors with a glimpse into the operations at our specialist clinic.
Additionally, we organized more than 5,000 scientific meetings attended by more than 170,000 people in 2015. And our wheelchair athletes take part in two major competitions, the IPC Athletics Grand Prix and the UCI Para-cycling World Championships, at our Nottwil site. It is our hope to continue to motivate individuals with spinal cord injuries to be involved in healthy exercise.
Since you became Hospital Director, how have you changed the way that you deliver health care or interact with patients?
Dr. Gmünder: It is important to me that the patients and their needs are the focus of our efforts. As such, one of my main tasks is to align our processes with our patients.
Here are some examples:
We started construction with a newly expanded Intensive Care Medicine, Pain Medicine and Surgical Medicine department last year to provide patients with an expanded variety of cross-linked treatments.
Certified as a nationwide trauma center, our Swiss Spinal Column and Spinal Cord Centre has become increasingly recognized throughout the country with large numbers of non-paralyzed patients, who have severe spinal cord injury, being referred to our facility. It is under the medical leadership of the Head of Department Dr. Med. Martin Baur, M.D. This highly specialized acute care facility recently received certification as a specialist center for traumatology within the Central Swiss Trauma Network.
We believe in developing the next generation of professionals and our Department of Anesthesia was recognized as a center of further training; the first two junior doctors have been appointed and postgraduate courses in anesthesia nursing are already available.
Our Swiss Weaning Centre, where individuals learn to breathe without a machine, has brought specialists from Intensive Care Medicine, Speech Therapy, RespiCare and Spinal Cord Medicine even closer together in a new process structure for respiratory medicine. At the same time, the Swiss Weaning Centre reported increased referrals from university hospitals and private clinics, as well as numerous successes with patients who had proved to be difficult to wean from respiratory equipment.
Our Centre for Pain Medicine, one of the largest pain facilities in the country, reported a further increase in inpatient treatments. Epiduroscopy, which was introduced in 2014, has proved to be a success. It is a percutaneous, minimally invasive procedure which is used in the diagnosis and treatment of pain syndromes near the spinal cord.
We reached a milestone in tetra hand surgery. The team of our doctors has been consulting at two other spinal cord injury centers and have used these occasions to show doctors around the country what possibilities there are for improved hand and grip functions, leading to an enhanced quality of life.
In what ways do you rehabilitate the whole patient? Why is this important early on in treatment?
Dr. Gmünder: In accordance with our vision, we are not just focusing on physical rehabilitation but on the entire person in their social environment (leisure, work, housing, mobility). Due to our broad organizational structure, we have many resources at our disposal. The rate of reintegration for people who did their primary rehabilitation at the Swiss Paraplegic Centre is almost 65 percent – one of the highest in the world.
Because we work to address diagnosis, treatment and management of traumatic spinal cord injuries with our patients, we take great care in working with patients on their medical disabilities, physical disabilities, psychological disabilities, vocational disabilities, social aspects and any health complications. That means that we not only treat patient’s medically, but also we treat them through therapy and complementary medicine, such as art therapy, sports and water therapy and homeopathic medicine.
At the SPC, we nurture a culture which is characterized by common values and shared objectives, namely commitment, leadership, a humane approach, cooperation and openness and fairness in our dealing with one another and with our patients.
As you follow patients throughout their rehabilitation and treatment, what are you most proud of at the Centre?
Dr. Gmünder: Research has shown that early referral of a patient with a traumatic spinal injury lessens the complications, shortens the length of time in the hospital and is, therefore, more cost-effective.
We are confronted by individuals every day whose abilities have been limited by disease, trauma, congenital disorders or pain – and we are focused on enabling them to achieve their maximum functional abilities. Our patients have a better outcome and quality of life, patient-focused treatment, ongoing case management, and lifelong care.
It’s important to emphasize that our comprehensive rehabilitation of individuals with spinal cord injuries begins on the first day after the accident or trauma. On one hand, the medical treatments with paraplegia or tetraplegia are performed by a multidisciplinary medical team. And on the other hand, it is our goal to give those individuals their personality and life structure as quickly – and as best – as possible. An individual’s medical condition affects their psychological, physical and social aspects of life.
We focus on individualized treatment for the greatest possible independence for our patients. When patients are satisfied with our work and its results, they can resume a self-determined life. That is our greatest joy.
Image SOURCE: Photograph of Hospital Director Hans Peter Gmünder, M.D., courtesy of Swiss Paraplegic Centre, Nottwil, Switzerland.
Hans Peter Gmünder, M.D. Hospital Director
Hans Peter Gmünder, M.D., assumed the role of Hospital Director of the Swiss Paraplegic Centre in 2011. He is a German-Belgian double citizen.
Previously, Dr. Gmünder was Chief Physician and Medical Director of the Rehaklinik Bellikon, a rehabilitation and specialist clinic for traumatic acute rehabilitation, sports medicine, professional integration and medical expertise for 10 years in the canton of Aargau, Switzerland. He began his career at the Swiss Paraplegic Centre in the 1990s as Assistant and Senior Physician, and later as Chief Physician and Deputy Chief Physician.
He completed a B.S. degree in Business Administration at SRH FernHochschule Riedlingen in 2010 and an M.D. degree at Freie Universität Berlin in 1987.
He is married to Sabeth and is the father of two children.
Editor’s note:
We would like to thank Claudia Merkel, head of public relations, Swiss Paraplegic Centre, for the help and support she provided during this interview.
Choosing the right rehabilitation facility is one of the most important decisions a survivor of a brain or spinal cord injury will make as the type and quality of care will have a significant impact on the patient’s long-term outcome. The top 10 rehabilitation centers in the United States are (http://www.brainandspinalcord.org/2016/04/15/top-ten-rehabilitation-hospitals-usa/):
Rehabilitation Institute of Chicago
TIRR Memorial Hermann
Kessler Institute for Rehabilitation
University of Washington Medical Center
Spaulding Rehabilitation Hospital, Massachusetts General Hospital
Mayo Clinic
Craig Hospital
Shepard Center
Rusk Rehabilitation at NYU Langone Medical Center
Moss Rehab
The Rehabilitation Institute of Chicago (https://www.sralab.org/new-ric), located in Chicago, Illinois, has been ranked as the number one rehabilitation hospital in the United States for the past 24 years by U.S. News and World Report. It is a 182-bed research facility that focuses solely on rehabilitation in many areas, including spinal cord, brain, nerve, muscle and bone, cancer and pediatric. For example, the rehabilitation course for patients with spinal cord injury requires precise medical and nursing expertise, respiratory and pulmonary care and sophisticated diagnostic and therapeutic equipment. For several years, the hospital has dedicated investments in talent, space and equipment that attract a high volume of patients with challenging conditions. The high volume, diversity of condition and greater complexity enables them to expand their experience in helping patients recover from spinal cord injury. Primary goals for patients include the emergence of meaningful motor function, sensation, coordination and endurance, resolution of respiratory and vascular instability, and overall continued medical recovery from the injury or disease.
The Spaulding Rehabilitation Hospital Boston (http://spauldingrehab.org/about/facts-statistics) is ranked number five in the country by U.S. News and World Report and number one in New England. As a unique center of treatment excellence and a leading physical medicine and rehabilitation research institution, Spaulding Boston is comprised of major departments in all areas of medicine requiring rehabilitation. They are a nationally recognized leader in innovation, research and education. The facility also has been the source of significant treatment innovations with dramatic implications for a range of conditions, including amputation and limb deficiencies, brain injury, cardiac rehabilitation, pulmonary rehabilitation and spinal cord injuries, to name a few. http://spauldingrehab.org/conditions-and-treatments/list.
Whether individuals are adjusting to a life-altering illness or recovering from a back injury, they will find the care they need within the Spaulding Rehabilitation Network. Rehabilitation specialists have the training, experience, resources and dedication to help individuals:
Regain function after a devastating illness or injury,
Develop skills to be active and independent when living with chronic illness and/or disability,
Recover from surgery, work and sports injuries, and
The ACGME accredited Harvard Medical School/ Spaulding/ VA Boston Fellowship Program in Spinal Cord Injury (SCI) Medicine is a 12-month training program that offers advanced clinical training in SCI, a strong didactic component, and opportunities for research with protected elective time. The curriculum is designed to provide exposure to the full spectrum of SCI care and includes rotations at VA Boston, Spaulding Rehabilitation Hospital, and Brigham & Woman’s Hospital. Requirements include prior completion of an approved residency program in a specialty such as physical medicine and rehabilitation, neurology, internal medicine, family practice, surgery, or other specialties relevant to spinal cord injury. http://spauldingrehab.org/education-and-training/spinal-cord-fellowship.
Specifically, the Spaulding Rehabilitation Network is at the forefront of innovative treatment for major disabling conditions, including spinal cord injury (SCI), traumatic brain injury (TBI), other traumatic injuries, stroke, and neuromuscular disorders such as multiple sclerosis, cerebral palsy, and Parkinson’s disease. At Spaulding, the treatment goals go far beyond immediate rehabilitation to address long-term health and function, as well as giving patients encouragement and hope as they return to their lives in the community.
The hub of their spinal cord injury program is the Spaulding-Harvard Spinal Cord Injury Model Systems (SCIMS) Rehabilitation Program, led by experts at Spaulding Boston, a Center of Excellence in spinal cord injury rehabilitation. With the guidance of their physicians and other rehabilitation specialists and access to some of the most advanced technologies available today, their patients have the resources to strive for their highest level of neurorecovery – and to develop successful, enriching strategies for independent living.
When potentially life-altering spinal cord injury occurs, the Spaulding Rehabilitation Network clinicians are dedicated to pioneering improved therapies that can make all the difference to a patient’s immediate and long-term recovery. Their goal is to support a patient’s return to an active, productive and fulfilling life.
Whether the spinal cord injury is due to traumatic injury or illness, their team of experts will develop a treatment plan in collaboration with the patient and family. Depending on the severity of the injury, their teams work on improving function in: walking, balance and mobility; speech, swallowing and breathing; thinking (cognition), behavior and safety; dressing, bathing and other activities of daily living; incontinence, bowel and bladder function.
Their commitment is to offer a full spectrum of rehabilitation services for adults and children with spinal cord injury:
Intensive, hospital-level rehabilitation with goal-directed therapy 3 – 5 hours a day, at least 5 days a week for inpatients.
Long-term care and rehabilitation for patients with complicating conditions.
Ventilator program to wean patients off mechanical breathing support in preparation for transition to more intensive rehabilitation.
Cutting-edge spinal cord injury technologies and therapeutic techniques.
Emphasis on family participation throughout the course of care. with an inpatient comprehensive training and education series.
Seamless transition to multi-disciplinary outpatient rehabilitation.
Sports and Recreation Programs to promote fitness, develop skills, and facilitate involvement with community to “live beyond boundaries.”
Coordination of care with Spaulding’s outpatient centers.
Spaulding Rehabilitation Network is the official teaching partner of the Harvard Medical School Department of Physical Medicine and Rehabilitation (PM&R). The Spaulding network’s facilities are members of Partners HealthCare, founded by Massachusetts General Hospital and Brigham and Women’s Hospital. The knowledge and expertise of this entire healthcare system is available to patients and caregivers. Their continuum of superb healthcare ensures that patients will find the care they need throughout their journey and the strength they need to live their life to the fullest.
2.1.5.5 Promising research for a male birth control pill, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair
Scientists think excessive population growth is a cause of scarcity and environmental degradation. A male pill could reduce the number of unintended pregnancies, which accounts for 40 percent of all pregnancies worldwide.
But, big drug companies long ago dropped out of the search for a male contraceptive pill which is able to chemically intercept millions of sperm before they reach a woman’s egg. Right now the chemical burden for contraception relies solely on the female. There’s not much activity in the male contraception field because an effective solution is available on the female side.
Presently, male contraception means a condom or a vasectomy. But researchers from Center for Drug Discovery at Baylor College of Medicine, USA are renewing the search for a better option—an easy-to-take pill that’s safe, fast-acting, and reversible.
The scientists began with lists of genes active in the testes for sperm production and motility and then created knockout mice that lack those genes. Using the gene-editing technology called CRISPR, in collaboration with Japanese scientists, they have so far made more than 75 of these “knockout” mice.
They allowed these mice to mate with normal (wild type) female mice, and if their female partners don’t get pregnant after three to six months, it means the gene might be a target for a contraceptive. Out of 2300 genes that are particularly active in the testes of mice, the researchers have identified 30 genes whose deletion makes the male infertile. Next the scientists are planning a novel screening approach to test whether any of about two billion chemicals can disable these genes in a test tube. Promising chemicals could then be fed to male mice to see if they cause infertility.
Female birth control pills use hormones to inhibit a woman’s ovaries from releasing eggs. But hormones have side effects like weight gain, mood changes, and headaches. A trial of one male contraceptive hormone was stopped early in 2011 after one participant committed suicide and others reported depression. Moreover, some drug candidates have made animals permanently sterile which is not the goal of the research. The challenge is to prevent sperm being made without permanently sterilizing the individual.
As a better way to test drugs, Scientists at University of Georgia, USA are investigating yet another high-tech approach. They are turning human skin cells into stem cells that look and act like the spermatogonial cells in the testes. Testing drugs on such cells might provide more accurate leads than tests on mice.
The male pill would also have to start working quickly, a lot sooner than the female pill, which takes about a week to function. Scientists from University of Dundee, U.K. admitted that there are lots of challenges. Because, a women’s ovary usually release one mature egg each month, while a man makes millions of sperm every day. So, the male pill has to be made 100 percent effective and act instantaneously.
Article ID #231: Women in Science. Published on 3/13/17
WordCloud Image Produced by Adam Tubman
Acclaimed biologist Rosalind Franklin’s grave in Willesden United Synagogue Cemetery has been given listed status, Historic England announced in marking International Women’s Day this week. Franklin’s tomb commemorates her life and achievements – they include X-ray observations she made of DNA which contributed to the discovery of its helical structure by Crick and Watson in 1953. Meanwhile, Historic England has teamed with The Royal Society to highlight the achievements of 28 remarkable women noted for their achievements in the fields of chemistry, biology, physics and astronomy. The women’s stories have been explored and key historic locations mapped. They include the Marianne North Gallery in Kew Gardens (named for 19th century botanist Marianne North), the Elizabeth Garrett Anderson Hospital – founded in 1872 as the New Hospital for Women in London by Anderson, a suffragette and the first English woman to qualify as a doctor, and the Royal Academy of Arts where natural history illustrator and painter Sarah Stone was an honorary exhibitor in the 1780s.
Sure, most people have heard of Marie Curie and Rosalind Franklin, Jane Goodall and Sally Ride.
But for every female scientist whose work has been recognized and celebrated, there are thousands who have been accidentally or purposefully forgotten.
Florence Bascom (1862-1945) discovered her love for geology on a childhood trip with her father and a geologist friend of his.
She worked for the US Geographical Survey, particularly specializing in the Piedmont Plateau between the Appalachians and the Atlantic coastal plain. She was voted one of the top 100 geologistsin 1906 in an edition of a magazine called, ironically, American Men of Science.
In addition to her research, she also taught several important geologists of the next generation at Bryn Mawr College.
Marjory Stoneman Douglas: Championed the ecological importance of The Everglades
President Clinton talks with Marjory Stoneman Douglas after presenting her with a Medal of Freedom.Doug Mills/AP
Marjory Stoneman Douglas (1890-1998) moved to Miami to write for the Herald, where her father worked. She left to work for the Red Cross during World War I, then returned to the Herald before branching out on her own as a writer.
She was able to see the value and importance of the Everglades despite finding them“too buggy, too wet, too generally inhospitable.” She wrote a book called “The Everglades: Rivers of Grass,” which raised awareness about the threats the ecosystem faced.
Celia Payne-Gaposchkin (1900-1979) was the astronomer who discovered that the sun is made of hydrogen and helium.
She went to college in Britain for botany, then attended by chance a lecture given by a prominent physicist, which she found so intriguing she changed fields (the lecturer, Arthur Eddington, became an important mentor for her). She moved across the Atlantic to study at Harvard, where she spent the rest of her career.
Her dissertation was called “the most brilliant PhD thesis ever written in astronomy.” In addition to our sun, she also studied variable stars, taking more than a million photographs of them with her team.
Rita Levi-Montalcini: Made a breakthrough in understanding the nervous system
A leader in Convergence, MIT’s Koch Institute for Integrative Cancer Research will, on June 16, present its 16th annual Summer Symposium: the Convergence of Science and Engineering in Cancer Research. Convergence—the merging of historically distinct disciplines such as engineering, physics, computer science, chemistry, mathematics, and the life sciences—has created extraordinary opportunities in cancer research and care. Leaders in this emerging field will discuss innovative new approaches and technologies to better detect, monitor, treat, and prevent cancer. The symposium will also feature a panel of experts to discuss the impact of Convergence on the future of medical care.
INTRODUCTORY REMARKS
Tyler Jacks, PhD
Director, Koch Institute, MIT
David H. Koch Professor of Biology, MITLIVE – new Solutions for Cancer the mission og KI. Sponsors: Affiliates, Collaborators, Patrons, Friends, Vendors. Introduction to Prof. Sharp. 40th Anniversary of RNA related discovery leading to Nobel Prize of Prof. Sharp.
Concluding the Symposium
continum
June 15, 2018 – NanoMedicine
Fall Symposium in September 2017
Phillip A. Sharp, PhD
Institute Professor, MIT
Koch Institute, MIT
LIVE – Convergence of Science and Engineering. Key note by a convergent personality – Eric Lander.
Convergence is a blueprint for innovation – NOW transformation of Life Science that follows the transformation of Physical sccience. Molecular biology – Delbruck, transmission of genetic phenotypes. Revolution of Biological Sciences in 20th Century
1st Revolution – DNA Structure, 1953
2nd Revolution – genomics Human Genome 2003
3rd Revolution – Integration of Science and the Environment in 21st Century
Such Small investment, 3.4% of NI Funding went to PI in BioEngineering 2000-2016
Introduction to Eric Lander – 1986 – to Whitehead Institute and MIT, now at Broad
KEYNOTE SPEAKER
30 Years of Convergence
Eric Lander, PhD
President and Founding Director, Broad Institute of Harvard and MIT
Professor of Biology, Department of Biology, MIT
Koch Institute, MIT
Professor of Systems Biology, Harvard Medical School
LIVE – Personal Reflection on Convergence in BioMedicine
Process is important inconvergence — WHAT ARE WE CONVERGING TOWARD?
1985 — today — 2045
BioMedicine – born from 4 Intellectual Revolutions:
Biochemistry – since 1890
Genetics – since 1900
Molecular Biology – since 1945, 1953, 2003
Recombinant DNA – since 1970
GENOMICS comes along: a View of
Completeness, Nature, 1986 – before the flood of data
Regulatory elements: Conversation of species – finite and tractable
Evolutionary
signatures of cellular processes – connectivity map Dependency Map
3D
Programmable Genome Targeting –
CRISPR GPS
Genome-wide CRISPR Screen
Human cells – unified coordinated to classify Cells and Tissues
Combine single cells with CRISPR Screening to systematically discover cellularpathway
Comprehensive Tools
Programmable Therapeutics –
DNA-directed Chemical synthesis
DNA-encoded Chemical Libraries
Programmable activation in cell type
Healthcare systems –>> Learning systems
SPEAKERS
Synthetic biology and next-generation diagnostics
James Collins, PhD
Termeer Professor of Medical Engineering and Science and Professor of Biological Engineering, MIT
Broad Institute of Harvard and MIT
Wyss Institute
LIVE
Synthetic Biology – Reprogramming life
Design & model network
Encode into DNA plasmid
Transfer to cell
monitor results
Potential for Synthetic Gene Biology
Paper-based synthetic Biology
distribution without refrigeration
RNA sensors with Colorimetric output
Rapid prototyping: Ebola sensors
Key features of Fieldable paper-based system
CRISPR-Cas9 Component for Strain Discrimination – NASBA-CRISPR Cleavage
Paper-based Diagnostics for the GUT microbiome
Paper-based Diagnostics for HPV: Rapid, Inexpensive
Sherlock – Nucleic Acid Detection with CRISPR-Cas13a
Human Genotyping Using SHERLOCK
Single-based gene usong Shrlock
Cancer Genome and the Cloud
Gad A. Getz, PhD
Director, Cancer Genome Computational Analysis Group, Broad Institute of Harvard and MIT
LIVE – Cancer is a disease of the Genome and epigenome – Life history of a Tumor
Drivers: Cancer phenotype
Smoking
Defect in DNA repair
Mutation type: C->T
Part 1: Finding Drivers Score genes by number and type of mutations: mutation tally and score of repair
450 genes va 11 genes
33 NEW cancer genes: 4,729 tumors, 21 tumor type, 254 significant genes
Gene Catalogue by Tumor type – 2,000 samples needed – to detect 90%
Burden
clustering
Non-coding drivers: Promoter, Insulator
9 significant mutations in Breast cancer gene – promoter Hotspot in FOXA1 is activated through E2F – Estrogene receptor
Mutational Signatures
Non-negative Matrix Factorization (NMF)
posterior distribution of the signatures
Homologuos recombination repair pathway
BRCA1/2: Signature analyzer: Breast Cancer mutation signatures
Mono-alleleic inactivation of BRCA
Germline mutation PALB2 PAthogenic mutations in genes
Second Window NIR Imaging (NIR-II) – deep tissue of Ovarian Cancer
Nanoparticle Characterization
Ovarian Cancer imaging
Charged Assembly a Different way: Electrostatic Self-assembly of Oligopeptide Amphiphiles
Peptide Nanoparticles are suitable for Vivo – p53-Deficient Cells
Target: MK2 Mediates DNA Damage Repair
Small molecules: Not safe not specific
New chemical engieering approaches to convergence
Robert S. Langer, ScD
David H. Koch Institute Professor, MIT
Koch Institute, MIT
LIVE
Lysozyme
soybean Trypsin inhibitor
Alkaline phosphatase
Catalase
Combinatorial lipid synthesis
Next generation LNPs: with novel lipids – potency improvement
Nano formulations for Entdothelium – combinatorial generation
Nano particle library
Current techniques:
Electroporation
– insert inside the gene – CellSqueeze of the cytoplasm
Full transcriptome microarray
Applications to Personalized Medicine
Injectable chip with combination drug therapy inserted into the Tumor, MIS,
Confining region of tumor
Conversion : Clinicians and Engineers
Understanding transcription and splicing heterogeneity in cancer progression
Daniel Larson, PhD
NIH Stadtman Investigator, Center for Cancer Research
Head, Systems Biology of Gene Expression, National Cancer Institute
Daniel Larson, NCI – Transcription and Splicing in Heterogeneity in Cancer Progression
LIVE
TFF1
Transcription in living cells: Estrogene response
Transcription occures in bursts: seconds to several hours – regulated over multiple timescale
4oth Anniversary of Splicing
high throughput approach for labelling thousands of genes at their endogenous loci
SLC2A1
Splicing times andd burst size are highly similar across genes.
Strong conservation in Eucaryote ccells
Splicing factor mutations emerged in almost all tumor types
3″ SS recognition factor U2AF1 has a missense mutation in the zinc finger domain
DNA damage after X-Ray treatment: U2AF1 S34F cells show – cell survive high dose (20 Gy) irradiation, becomes senescent and live >1 month in culture abd secrete interleukin 8
Interleukin 8 Upregulated even before DNA damage – expression and secretion in rare cells
IL-8 induces Epithelial-Mesenchymal Transition i primary mamary epithelial cells
Stochastic transcription and RNA splicing – IL-8 Secretion
Computational Models of Cancer
Franziska Michor, PhD
Professor of Computational Biology, Dana-Farber Cancer Institute
Harvard T.H. Chan School of Public Health
Effect of dosing:Number of sensitive cells in Two drug concentrations: Sensitive vs Resistant cells
Combination Treatment
Better parameter estimation: using single cell lineage tracing data
microenvironment determinants of Treatment response
Determining Optimum radiation schedules in GBM
mathematical modeling of treatment response
understanding the intratumor heterogeneity based on mouse modeling
optimal radiation schedule –>>
dose constraints – Ultra fractionated dose vs.
Practicality constraints
slower proliferation
Radiation plus temozolomide: Optimized; time post treatment/percent survival
Treatment modalities: Immunotherapy, chemo drugs
Spherical Nucleic Acids as a Powerful New Platform for Cancer Therapy
Chad A. Mirkin, PhD
Director, International Institute for Nanotechnology
George B. Rathmann Professor of Chemistry, Department of Chemistry, Northwestern University
LIVE
Next wave of Pharmaceuticals
Small molecules’
biologics
Nucleic Acids – limitations: Address disease in the Liver
The Promise of Therapeutic Oligonucleotides
Antisense DNA
Spherical Nucleic Acids (SNA) – New way of thinking on DNA and RNA
Hybridization Thermodynamics of SNAs
SNAs enter cells rapidly and efficiently – over 60 different Cell Types
Cy5-labeled SNA
SNAs Come in many forms: Micellar SNA, Protein SNAs, Lipoprotein SNAs
Late endosome nOT lysosomeow
How does cell membrane recognition of SNAs and trigger exocytosis
SNAs as Therapeutics: Skin (topical for Psoriasis), Brain (GBM)
Immunostimulatory SNAs – activity of IS-SNAs in Vivo
Oligonucleitides strands mimicking bacterial DNA or RNA
SNA – Vaccines: Antigen presenting cells
3D Architecture of SNAs leads ti enhanced TLP9 Activation – B-cell NF-KB Activation
SNAs Vaccines: -In Vivo Testing: retired Tumor growth vs control – inserted inside liposome
Advantages SNAs Vaccine vs Standard Formulations vs adjuvant (antigen)
SNA TLR-9 Activator Treatmeent vs Linear Oligo (EMT-6 Breast Cancer Model] – it potentiates activity of Anti-PD-1 Antibodies in PD-1 Resistant tumor confers immunity
SNA Competitive advantage
Dissecting the tumor ecosystem with single cell genomics
Aviv Regev, PhD
Director, Klarman Cell Observatory and Cell Circuits Program, Broad Institute of Harvard and MIT
Professor of Biology, Department of Biology, MIT
Koch Institute, MIT
LIVE – Tumors: A complex cellular Ecosystem
How to use Genomic sto study tumors?
Option 1: Gene Atlas – single cell genomics can help dissect thie ecosystem – bulk cell
Option 2: Tumor Cell Atlas – single cell
Single cell RNA-Seq in Precision Medicine pipeline
Micronvironment of the cell – Diverse T cells, Naive, – Tumar infiltrating T cells: Activation-independent variation in exhaustion program across cells
cytotoxicity, exhausion, naive
RNA-Seq associates T cell clones with their states
Cell-types and states in the melanoma ecosystem:
single cell signature used to cluster bulk tumors by their microenvironment composition
TCGA – Tumors – melanoma vs Cell type specific signature genes
inferring cell-cell interactions: Cell type A and Cell type B: Correlation of gene’s expression with inferred proportion of cell type B (bulk samples)
CAF expression of chemokines and complement associated with CD8 T cell infiltration
Primary Test Stronger control
The ecosystem of Malignant Melanoma – 19 malignant cells
Treatment naive vs Immunotherapy resistant (ITR) – CD8 cells from ITR sample – intratumor variation
How malignant melanocyte affect T-cells?
Infiltrated tumor vs exclusion tumor
ITR has prognostic value – predicts response to anti-PD1 in Pationts
ITR Signatures in malignant and CD8 T cells
Illuminating biology at the nanoscale and systems scale using single-molecule and super-resolution imaging
Xiaowei Zhuang, PhD
David B. Arnold Professor of Science, Harvard University
Session I: NEW VIEW
Xiaowei Zhuang, Harvard University — Single-Cell Transcriptome and Genome Imaging,
High throughput image-based screening: Barcodes, gene activation/inhibition agents
Tracing the 3D conformation of Chromatin
Spatial organization of A-B compartments: Transient, Polarized, radial
Super resolution of chromatin imaging with Transcriptome imaging
EXPERT PANEL: CONVERGENCE IN HEALTH
LIVE
Cori Bargmann, PhD
President of Science, Chan Zuckerberg Initiative
Torsten N. Wiesel Professor, The Rockefeller University
LIVE – translation of the needs in computational, biology, IT, mathematician, medicine, physics and engineering. BRAIN Initiative at NIH. How to motivate? by a Problem in need for solution
Marc N. Casper, MBA
President and CEO, Thermo Fisher Scientific
LIVE – convergence from a tool perspectiveLast 20 years – integration of Biology into the physical sciences,
SCREENING AND DIAGNOSTICS
Victor Dzau, MD
President, National Academy of Medicine
LIVE
As cardiologist: Pace maker Convergence in Research.
Funding research does not encourage convergence
innovation is killing disease, need to introduce cost effectiveness – economics into the play. Recognition of cost in Precision Medicine: economic model as a tool to consider cost effectiveness,
Convergence to include SOcial Sciences and Economics
Diagnostics;
Tyler Jacks, PhD
Director, Koch Institute, MIT
David H. Koch Professor of Biology, MIT
LIVE
– Convergence at Koch
NIH created few CENTERS for research in Cancer, Brain, Nanotech, focus on Cancer did help the convergence
Next step of Convergence: common language, Learning by doing, mechanisms to encourage and continue the convergence
Better efficiency:
Which Drug to which Patient
Early detection and prevention
Nancy Simonian, MD
CEO, Syros Pharmaceuticals, Inc.
LIVE – convergence is required to understand the biology of a disease, genomics, computational biology and chemistry is a new approach
Cost of Medicines: reibbursement per value vs no concern to value. Innovation t carry premium allowing cost reduction while effciency is been exploled.
Elias Zerhouni, MD
President for Global Research and Development, Sanofi
Former Director, NIH
LIVE
Convergence of multidisciplines is a must for scientific solutions to emerge, horizontal integration
Patient Bill of Right
Defficiet every year in the US,
Moderated by:
Susan Hockfield, PhD
President Emerita, MIT
Professor of Neuroscience, MIT
Koch Institute, MIT
2012pharmaceutical
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