The New York Stem Cell Foundation Accelerating Cures Through Stem Cell Research New York Pharma Forum Susan Solomon December 6, 2013
Stem Cell Research in the United States • 1996 – Dickey Wicker Amendment • 1998 – James Thompson and colleagues isolate hESCs • 1999 – NIH Director Harold Varmus says NIH can fund human pluripotent stem cells per HSS General Counsel • 2000 – NIH guidelines released for hESC research • 2001 – Bush Executive Order • 2006 – Senate passes Stem Cell Research Enhancement Act; vetoed • 2006 – NYSCF’s privately funded, safe -haven laboratory opens The New York Stem Cell Foundation
Stem Cell Research in the United States (cont’d) • 2006 – Shinya Yamanaka derives first ever induced pluripotent stem (iPS) cells • 2008 – NYSCF supported scientist derives first patient iPS lines (ALS) • 2009 – Obama Executive Order and final NIH guidelines • Allows NIH to fund some research on, but not derivation of hESCs • Lines that were eligible under Bush era must be reviewed by a Working Group • NIH funds cannot be used for research on stem cell lines derived from SCNT or parthenogenesis The New York Stem Cell Foundation
Public Supports Stem Cell Research • 73% of Americans favor expanding ESC research • A vocal minority has controlled the message Source: Research!America The New York Stem Cell Foundation
Stem Cell Programs • States responded with their own programs • California – $3 billion over 10 years • New York – $600 million over 11 years • Connecticut – $100 million over 10 years • Maryland – approximately $10 million per year • NIH: FY12 - $1.5B for stem cell research • $146.5M for hESC ~ 13% • Private Funding • New York Stem Cell Foundation Research Institute - $100 million The New York Stem Cell Foundation
NYSCF Mission Accelerating cures for the BEN major diseases of our time through stem cell research
NYSCF Programs NYSCF Research Laboratory NYSCF NYSCF Innovators: Conference Fellows and and Investigator Symposia s
NYSCF Conferences and Symposia Annual Conferences Panels Seminar Series
NYSCF Innovators $53M dedicated to building the next generation of top stem cell researchers • 55 3-year NYSCF Fellowships over 12 years ($12M) at 17 institutions • 28 NYSCF Early-career Investigators over 6 years ($41M) at 10 institutions
NYSCF ’ s Proven Track Record In just eight years, NYSCF has achieved several breakthroughs in the field 2013 – New therapeutic approach for diabetes • Discovery of compound that restores beta cell function in genetic form of diabetes, may be applicable to all forms of diabetes. 2013 - Personalized bone graft advance • NYSCF Research Laboratory team engineers bone from skin cells 2013 – First monogenic diabetes model • Mutated genes genetically corrected 2012 - Stopping mitochondrial disease • NYSCF Research Lab team develops clinical cure for inherited disease that impacts children 2011 - Pioneering cell replacement therapy • NYSCF Research Lab team derives first ever human embryonic stem cell from human eggs - Time magazine #1 medical breakthrough of the year 2008 - Creating first-ever disease model (ALS) • NYSCF scientist has Time and Science magazines #1 medical breakthrough of the year
Why NYSCF Research Institute is Unique • Focus only on translational research – translating research into cures • Fund high-risk, high-reward “ tipping point ” experiments that traditional funding mechanisms won ’ t support • Independent and unencumbered by bureaucracy or federal restrictions • Leverage our proprietary research in collaboration with institutions around the world
NYSCF Research Institute Laboratory • 45 full time researchers • Raised and invested $100M for stem cell research • Leader in developing stem cell technologies and disease modeling • An international community of over 100 scientists collaborating to cure disease
NYSCF Research Institute ™ Building infrastructure to industrialize stem cell research Objectives: • Reproducible stem cell production • Parallel derivation & culture at scale • Quantitative quality control assays • Reproducible panels of differentiated cells • Diverse and disease populations Connect Genotype to phenotype: Representative Stem Cell Lines • in vitro GWAS • “ Clinical trials in a dish ”
NYSCF Disease Programs • Bone regeneration • Diabetes / auto-immune diseases • Heart disease • Neural disorders • Alzheimer ’ s disease • Autism • Parkinson ’ s disease • Multiple Sclerosis • Mental Illness
Why Do Cures Take So Long? Pharmaceutical & Academic Institutions Biotech Companies Identify Disease Causes Drug Development • 13 Years • $4 Billion • 99% Fail Publish Research Papers • Use small • Mainly work on large disease collections of cell markets (changing) lines from a narrow • Public companies - generally ? group of patients risk averse Then what? • Screen on mice and cells unrelated to the disease
NYSCF Provides a Bridge to Cures connecting research to cures and treatments Biotech & Academic Pharmaceutical Institutions Companies can scale reduces time, their cost, and risk discoveries
The stages of development of a ‘ typical ’ new drug DRUG PRECLINICAL CLINICAL DEVELOPMENT DISCOVERY DEVELOPMENT Phase I Phase II Phase III Phase IV Target selection Pharmacokinetics Pharmacokinetics, Small-scale trials in Large-scale Postmarketing Lead-finding Short-term tolerability, side- patients to assess controlled surveillance toxicology Lead optimization effects in healthy efficacy & dosage clinical trials Formulation volunteers Pharmacological profiling Synthesis scale-up Long-term toxicology studies 2-5 years 1.5 years 5-7 years � 50 projects 12 compounds 5 3 1.7 1 Drug Development Drug approval candidate compound for marketing
This process is amazingly inefficient and shockingly expensive Paul et al., Nature Rev Drug Discovery, 2010 First time a drug may see a human cell
Pluripotent Stem Cells
NYSCF Research Institute Goals
iPS Cell Disease Modeling • Model diseased tissue • Test drugs on diseased tissue 21
Functional Cells in a Dish Alzheimer ’ s Dopamine Producing Neurons Neurons Insulin Producing Cells Cardiac Cells
Existing Challenges with iPS cells • Artisanal product via various methods with variable quality • Few cell lines from relatively homogeneous populations • Lack of standardization • Limited scalability and slow production
A New Technology Platform: The NYSCF Global Stem Cell Array
NYSCF Global Stem Cell Array TM • Automated robotic systems for iPS cell generation, differentiation, and analysis • Minimize manual manipulation • Massive parallel processing (100s-1000s) • Produce reproducible panels of differentiated cell lines • Cell line collection • 95% of genetic diversity • Major and rare diseases
Automated differentiated cells Differentiated Cells: Cholinergic Beta Cells Cardiomyocytes Neurons Insulin/Glucagon/Nuclei cTnT/Nuclei Tuj1/CHAT/DNA • Other iPSC derived cell types produced at NYSCF: astrocytes, endothelial cells, osteoblasts, oligodendrocytes, and others. 26
Finding the Genetic Causes of Disease The NYSCF Global Stem Cell Array Puts the human genome ATCGTACGTTGCATGCATC into biological context GTACGTTACCGCAACCTG CATGCCACGTACGCATGC • Current genomic analysis allows ATGCATGCATGCACTGCAT scientists to see a connection GCATTGCATCTAACGTACG between DNA and disease TATACGGCGCATGTATAGT ATCGTACGTTGCATGCATC GTACGTACGTACGTACGTA GTACGTTACCGCAACCTG TGCCAGTTGCATGGCATG CATGCCACGTACGCATGC CATTGCATGCTTACGT ATGCATGCATGCACTGCAT • With the addition of GCATTGCATCTAACGTACG NYSCF ’ s Stem Cell Array , TATACGGCGCATGTATAGT scientists can see how the GTACGTACGTACGTACGTA DNA actually functions in TGCCAGTTGCATGGCATG the disease. CATTGCATGCTTACGT
“ Clinical Trials in a Dish ” The NYSCF Global Stem Cell Array • Replicate diseases in a dish using actual diseased human cells (not mouse cells) • Anticipate how people from genetically diverse backgrounds will respond to different drugs before clinical trials • Predict drug toxicity in the dish 28
Scaling the Research The NYSCF Global Stem Cell Array Allows scientists to: • Scale their discoveries • Test their findings in a large population • Translate their research into medicine and cellular treatments for disease
Extensive Institutional Collaborations & Key Relationships (50+) Other Countries Australia Israel Qatar Sweden United Kingdom 30
Select large scale collaborations: Michael J. Fox Foundation NIH Undiagnosed Disease Program Cure Alzheimer’s Fund Stem Cell Consortium Personal Genomes Project
NYSCF Global Stem Cell Array TM Freedom to Operate - Robust Management NYSCF Collection Sites (clinical data), SCRO Committee, and Tissue samples Human Subjects Team In-house US Collaborator International IRB Protocols MTA USDA / Customs Consent Forms MTA iPS production NYSCF Production Management Team SOPs – Freedom to Operate – Licensing LIMS Tracking and batch records Bank QC Banking NYSCF Array Distribution Team Full Documentation MTA [Academic / Government / Commercial] Distribution
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