NYSCF News Update | Vol 12, Issue 1

NEWSupdate VOLUME 12: ISSUE 1: 2019

N YS C F

The New York Stem Cell Foundation Research Institute

“W

B R I N G I N G N E W H O PE TO WO M E N W ITH R E PRO D U C TI V E C A N C E R S

e have an enormous opportunity to improve outcomes for women with reproductive cancers. The groundbreaking stem cell technologies we have developed at NYSCF give us the power to find better and more personalized treatments.”

–Susan L. Solomon, JD Ursula Matulonis, MD (Dana-Farber Cancer Institute), Laura Andres-Mar tin, PhD (NYSCF), Siddhar tha Mukherjee, MD, DPhil (Columbia University Ir ving Medical Center), and Carla Grandori, MD, PhD (SEngine Precision Medicine) Panel support provided by the Stavros Niarchos Foundation, TESARO, and GSK

When women with a reproductive cancer (such as ovarian, cervical, or endometrial cancer) start treatment, they all go through the same drill. Surgery. Chemotherapy. More chemotherapy. Wait. If the cancer comes back, start again.

The issue here is that due to severe underfunding of research into women’s reproductive cancers, this care plan has not changed in decades — most patients are prescribed the same therapies. But every patient’s cancer evolves in a unique manner, so we cannot expect the same drugs to work for everyone. And they don’t — survival rates have barely improved over the past 40 years.

We need a better way to study and treat these devastating cancers, and the members of the NYSCF Women’s Reproductive Cancers Initiative (launched this April with a panel discussion featuring members of the Initiative’s Scientific Advisory Board — a collection of 15 world-leading researchers and physicians who have spearheaded breakthrough treatments, such as PARP inhibitors, across several cancer types) are ready to take on this challenge.

Led by NYSCF’s Dr. Laura Andres-Martin, a pioneer in stem cell biology who has committed her career to applying these innovations to improve the way diseases are treated, the team is using samples of patient tumors to generate stem cells and organoids (3D aggregates of tumor tissue) that have an unlimited lifespan. These stem cells and organoids capture the uniqueness of each patient’s disease from their own cells, helping to illuminate how tumor cells interact, how the cancer started in the first place, and which treatments can stop it. This biobank of patient-specific tumor tissue that never “expires” will serve as a foundational resource for the research community to enable personalized cancer research and drug discovery.

explained Scientific Advisory Board member Dr. Siddhartha Mukherjee. “Creating a bank of this misinformation will help us understand the code that drives the growth of these cancers and then find a way to disrupt that code.”

The Initiative will begin by focusing on ovarian cancer. Ovarian cancer is especially tricky: it is hard to detect before advanced stages and relapse rates are high (tumors return in over 75% of patients).

Having a model in place that can help us identify targets for early detection and intervention will be a game-changer for this disease.

M E E T D R . L A U R A A N D R E S - M A RT I N SENIOR PRINCIPAL SCIENTIST, ONCOLOGY

Dr. Andres-Martin recently joined NYSCF to pioneer the Women’s Reproductive Cancers Initiative. Originally from Spain, Dr. Andres-Martin received her PhD from the University of Salamanca, followed by a postdoc at Weill Cornell Medicine, where she was a NYSCF – Druckenmiller Fellow.

“Women with reproductive cancers have a major unmet medical need for effective treatments,” she says. “I am excited to be applying the latest discoveries in stem cell research to help meet this need.”

“If all of biology is information, then cancer is misinformation,”

F E AT U R E D I N T H I S I S S U E FA M I LY DA Y

ALUMNI MEETINGS

SPR I NG E V E N TS

MU LT IPL E SCL EROSIS

Students and parents experience the wonders of stem cell research p.3

When the world’s top researchers meet, ideas blossom p.5

What’s next for Alzheimer’s, type 1 diabetes, and gene editing? p.6

Toward new treatments for the disease’s most severe form p.7

Contact us at info@nyscf.org or 212.787.4111

W W W. N Y S C F. O R G

U N I T I N G T O A C C E L E R AT E C U R E S BOARD OF DIRECTORS Roy Geronemus, MD, Chairman Susan L. Solomon, CEO Margo Alexander Marilyn G. Breslow Peggy Brim Karen E. Burke, MD, PhD Paul Goldberger George Lazarus, MD Richard J. Massey, PhD Paul M. Meister Stephen M. Ross Stephen M. Scherr Anita Volz Wien

LEADERSHIP COUNCIL Katherine Bristor & William Priest David A. Carmel Russell L. Carson Chuck Close Alan M. Cohen Shirley Cook Fiona Druckenmiller Jodie & John Eastman Frank Gehry Lawrence E. Golub & Karen Finerman April Gornik Marlene Hess Tania Higgins Dorothy Lichtenstein Sandra Lloyd Stephen Meringoff David Mitnick Nancy & Fred Poses Carol Roaman Julian Robertson Clifford Ross Andy Russell Ian Schrager Barbara Stovall Smith Martha Stewart David & Jane Walentas Dick Wolf

Dear Friends, At NYSCF, we identify and pursue areas in which stem cell research can have the greatest impact on human health. I am delighted that our latest addition is a focus on women’s reproductive cancers, a sorely neglected and underfunded group of diseases. These women need better research and better treatments.

So in April, we launched the Women’s Reproductive Cancers Initiative to leverage the power of stem cells in changing the way these devastating cancers are studied and treated.

We have assembled a team of talented researchers and a brilliant Scientific Advisory Board of pioneers and experts to help guide us, and I look forward to seeing the incredible advancements we will create together. It is through this type of collaboration that we make strides toward a better, healthier future for all.

NYSCF has worked for years to build an elite community of scientists, and at this year’s Innovators Retreat, I saw its impact firsthand. Many of our Innovators noted that their breakthroughs were made possible through the input and support of the peers they met through NYSCF. To foster these bonds after graduating from the NYSCF – Druckenmiller Fellowship and NYSCF – Robertson Investigator programs, our Innovators took the initiative to organize annual alumni meetings. This spring, we hosted the first of these meetings at the NYSCF Research Institute, where these top scientists reconnected and shared ideas for pushing their impactful research forward. I am continually inspired by their drive to improve the lives of patients through teamwork and ingenuity.

Each day, our impact expands, and we move closer to cures, all because of the steadfast commitment of our friends and supporters. We cannot do it without you, and we thank you for your generosity.

Susan L. Solomon, JD CEO and Co-Founder

Join us!

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, 2019

H O N O R I N G S T E M C E L L H E RO E S

OCTOBER

PETE R J . GO ULA NDRIS & KAREN ELI ZA BET H B URKE G OULAN DRIS, M D, PHD JAY K R I EG EL GR EG H I CK S & R ICK FOSTE R

J A Z Z AT L I N C O L N C E N T E R ’ S F R E D E R I C K P. R O S E H A L L

NYSCFNEWSupdate

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N YS C F – D RU C K E N M I L L E R F E L LOWS R EC E P TI O N

This June, NYSCF staff, supporters, and scientists gathered at the Asia Society in Manhattan to welcome the 2019 class of NYSCF – Druckenmiller Fellows to the NYSCF Innovator community.

Shelly and George Lazarus, MD, Kay Unger ; Fiona and Stan Druckenmiller ; Susan Scherr, NYSCF – Druckenmiller Fellow Alumna Elisa Cimetta, PhD, and Jeff Wallerstein

ROBOT S , M IC ROSCOPE S , AN D TH E SC I E NTI ST S OF TOMORROW: NYSC F HOST S S ECON D AN N UAL FAM I LY STE M C E LL DAY The day began with an educational presentation on stem cell research, after which attendees were invited to visit 14 different stations. Downstairs, students made slime, built neurons from pipe cleaners, smashed flowers frozen in liquid nitrogen, and donned lab coats in the photo booth. Upstairs in NYSCF’s labs, guests learned about different aspects of research — everything from robotics to growing bone to launching cells into space.

“Look — it’s an astrocyte!” exclaimed one student, triumphantly returning to her friends with a brightly colored image of star-shaped cells she had created with Valentina Fossati, PhD, using digital painting. “They live in the brain,” she explained, happily tucking the photo into her bag.

A student glimpses pancreatic cells made from stem cells by NYSCF scientists

“Events like this are a perfect way to get kids interested in science,” observed one parent. “Sometimes when you’re reading about science in a book, it’s hard to see how it will benefit society, so I think today has really helped perpetuate their interest in the field.”

“W ho thinks I can power this lightbulb with just the electricity from my finger?” Cecile Terrenoire, PhD, asks a room of eager students.

About half believe it’s possible (“Yeah! You can!”) and half are more skeptical (“With your finger? No way.”) As she touches the base of the lightbulb, it quickly brightens, as do the eyes of the students.

Dr. Terrenoire was teaching the students about how the body’s electricity plays a key role in health and disease. At NYSCF’s second annual Family Stem Cell Day, hosted with support from the Stavros Niarchos Foundation, students K-12 and their parents were invited to tour NYSCF’s state-of-the-art laboratories and learn about the wonders of human biology and stem cell research from scientists who work in this field every day.

“It’s on us to inspire the next generation of researchers,” remarked NYSCF’s Dr. Daniel Paull. “This event is a great opportunity to introduce people to science they didn’t know existed.”

NYSCF Vice President of Stem Cell Technology Platforms, Daniel Paull, PhD, and Chief of Staff David McKeon show students stem cell research in action

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TOP MINDS CONNECT AT ANNUAL NYSCF INNOVATORS RETREAT

W hether it is a conversation over lunch, a question at a poster, or a suggestion given

Graduating NYSCF – Robertson Investigators Drs. Valentina Greco, Kay M. Tye, Edward Chang, Lisa Giocomo,

after a talk, the interactions that take place at the annual NYSCF Innovators retreat often spark ideas that could help millions of patients around the world.

The Innovators pointed out that this incredible work is fueled by more than just funding — these community interactions enable team science.

“The funding is great, but the community is worth so much more,” remarked NYSCF – Robertson Neuroscience Investigator and Salk Institute Professor Kay Tye, PhD.

“Science is a community effort,” reflected NYSCF – Robertson Stem Cell Investigator, CRISPR pioneer, and MIT Professor of Biological Engineering Feng Zhang, and Dr. Feng Zhang On the final night of the retreat, the PhD. “There is the experimental aspect, graduating class shared their plans to further this sense of kinship and but the bigger and more important aspect is relational. Conversations give back to the community they value so deeply. They are starting a are how new ideas are formed and how advancements are made.” mentorship program, where graduating Investigators are paired with first-year Investigators to help guide the new class through what are Over the course of the week-long retreat, the NYSCF – Robertson often the formative years of their laboratories.

Investigators, NYSCF Research Institute scientists, and NYSCF – Druckenmiller Fellows shared their groundbreaking research with “This community always acts as champions for one another,” each other and gathered feedback from top minds in the stem cell noted NYSCF – Robertson Stem Cell Investigator and Professor of and neuroscience fields. This year’s retreat showcased trailblazing Genetics at Yale University Valentina Greco, PhD. “Once you are a discoveries in neurodegenerative disease, gene editing, heart disease, part of it, you want to make sure that everybody thrives.” mental illness, cancer, and much more.

NYSCF Innovators and staff

M E E T TH E 2 019 N YSC F – D RU C K E N M I L L E R F E L LOW S

T he NYSCF – Druckenmiller Fellowship was created to support young postdoctoral researchers in their pursuit of innovative stem cell research and is the largest dedicated stem cell fellowship program in the world. Fellows receive an annual stipend as well as support for their research, and are welcomed into NYSCF’s greater Innovator Community.

Stephanie Ellis, PhD

Jesus M. Gomez Salinero, PhD

The Rockefeller University

Weill Cornell Medical College

Studies competition among skin stem cells and how it protects the skin against stress

Develops methods enabling therapeutic liver regeneration via stem cells

Marissa A. Scavuzzo, PhD

Tianchi Xin, PhD

Case Western Reserve University

Yale University

Examines the unique functions of the enteric nervous system, known as the “brain inside your gut”

Explores the role of stem cells in regulating tissue growth and how these processes are influenced by aging

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S C I E NTI F I C FA M I LY R E U N I O N S : N YS C F I N N OVATO R S L AU N C H A LU M N I M E E TI N G S

NYSCF – Druckenmiller Fellows and Alumni Drs. Ana G. Freire, Evangelos Kiskinis, Elisa Cimetta, Jesus M. Gomez Salinero, Dafne Campigli di Giammar tino, Gabsang Lee, Dilek Colak, Valentina Fossati, Elena Ezhkova, Benjamin Lin, Ying Liu, Brett Shook, Ya-Chieh Hsu, Dominik Paquet, Sandra Pinho, Tianchi Xin, Kosuke Funato, Marissa A, Scavuzzo, Stephanie Ellis, Raffaella Di Micco, Sangbum Park, Larr y Luchsinger, Tae Wan Kim

W hen the NYSCF – Robertson Investigator Alumni graduated from their fifth and final year of the program, they were determined

Then in June, former members of the NYSCF – Druckenmiller Fellowship jumped on board and gathered at the NYSCF Research Institute for an alumni meeting featuring a keynote by biotech giant Robert Langer, ScD, of MIT. From developing personalized therapies for cancer to cracking the codes of neurodegenerative diseases, these alumni are making critical advances that will help patients live longer and happier lives, and the reunion gave them the opportunity to present this work and discuss where it should go next.

to stay in the NYSCF community. These leading scientists wanted to keep up with each other’s exciting advancements and solidify the bonds they formed in the program that introduced them to many inspiring peers.

So, these 24 researchers decided to have a ‘family reunion’ for the program alumni. This March marked the first NYSCF – Robertson Stem Cell Investigator and NYSCF – Robertson Neuroscience Investigator Alumni Meetings, held at the NYSCF Research Institute. The two-day symposia brought together alumni from around the world to share their latest, unpublished scientific research, get reactions from their peers, form collaborations, and even take on fun new challenges (axe throwing, anyone?).

NYSCF – Rober tson Stem Cell Investigator Alumnus Marius Wernig, MD, PhD

NYSCF CONFERENCE

Keynote Speakers Shinya Yamanaka, MD, PhD Nobel Laureate Kyoto University

S AV E T H E D AT E OCTOBER

“It’s so helpful to realize that there are other people who have the same challenges you do, and who you can draw on for advice,” said NYSCF Senior Scientific Advisor and Harvard University Professor Kevin Eggan, PhD. “And the fact that our Innovators have this desire to remain a part of our community even after they have graduated from the program is a sure indicator of their value.”

Hans Clevers, MD, PhD Hubrecht Institute Utrecht University

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T H E RO C K E F E L L E R U N I V E R S I T Y

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AL ZHEIMER’S, T YPE 1 DIABETES, AND GENE EDITING: WHERE ARE WE NOW, AND WHERE ARE WE GOING NEXT?

A lzheimer’s is a brutal disease that affects more than 44 million patients and their families. At NYSCF’s recent “Alzheimer’s Disease:

Institute of Bioethics, an organization committed to exploring the intersection of ethics and science/health policy. Panelists unpacked the scientific potential of CRISPR and the ethical conundrums around editing human DNA.

The Path Forward” discussion, the speakers were optimistic that using human stem cell models for research will help us find treatments that address the disease’s root cause.

“We’re at a point now where gene editing therapies are reaching the clinic,” noted Berman Institute Director Dr. Jeffrey Kahn, “We need to figure out, through discussions that involve scientists and the public, a playbook for how to move forward.”

“What makes me hopeful is in this room,” commented caregiver and patient advocate Dan Gasby. “This type of research is the final frontier.”

Dr. Betsy Myers (Program Director for Medical Research at the Doris Duke Charitable Foundation) is funding the development of gene editing therapies and is inspired by the promise they are already showing.

Like Alzheimer’s, type 1 diabetes (T1D) is lacking therapies that address its underlying mechanisms. In partnership with Lyfebulb, NYSCF hosted an event on “Uniting Patients and Researchers on the Road to Curing Type 1 Diabetes,” Dan Gasby (Alzheimer’s caregiver and patient advocate) where T1D patients, researchers, physicians, and advocates explored how different communities can work together to improve patients’ lives in the short and long term.

“It takes advocates; it takes doctors; it takes researchers; it takes funders. This beautiful mosaic of groups has to work together in order to find cures,” said NYSCF CEO Susan L. Solomon, JD.

Raeka Aiyar, PhD (NYSCF), Jeffrey Kahn, PhD, MPH (Johns Hopkins Berman Institute of Bioethics), Betsy Myers, PhD (Doris Duke Charitable Foundation), Neville Sanjana, PhD (The New York Genome Center; New York University), and Susan L. Solomon, JD (NYSCF)

Cures are on the horizon for several diseases thanks to CRISPR gene editing, but the power of this technology raises important ethical questions about when and how to apply it. To foster the necessary dialogue, NYSCF hosted a panel on “Gene Editing and the Future of Human Health” in collaboration with the Johns Hopkins Berman

“We have a national registry of clinical trials which currently includes six trials using gene therapies to treat sickle cell anemia,” she remarked.

“It’s a whole new world.”

We are grateful to the Stavros Niarchos Foundation for their support of our education and outreach programs.

NYSCF DISCUSSES HOT TOPICS IN BIOETHICS AT THE STAVROS NIARCHOS FOUNDATION CONFERENCE

B ecause of the rate at which science is accelerating, we now know more about disease than we ever have before, and we are seeing more lifesaving therapies reach

patients each year. However, as progress accelerates, we must do all we can to ensure that research and treatment practices remain safe and ethical. At the Stavros Niarchos Foundation (SNF) Conference held this June in Athens, Greece, Susan L. Solomon discussed the advancements NYSCF is making in the stem cell field as well as the conversations that must happen between scientists, the government, and the public to determine the best way to regulate the development and uses of new Panos Papoulias, PhD (SNF), Robert Barnett, PhD (Modern Tibetan Studies technologies.

Program, Columbia University), Claire Katz, PhD (Texas A&M) Susan Solomon, JD (NYSCF), Effy Vayena, PhD (Swiss Federal Inst. of Technology)

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NYSCF KEYNOTE: THE FUTURE OF BIOTECH IN NYC

N ew York isn’t just a hotspot for food and culture; it’s also becoming the city of choice for

biotech companies. NYSCF has played a leading role in building this sector by establishing stateof-the-art headquarters in midtown Manhattan and fostering a sense of community among local biotech organizations. At The Future of New York Life Sciences conference hosted by the New York City Economic Development Corporation (NYCEDC) and Xconomy, a leading digital media and events services company for the technology and life sciences communities, Susan L. Solomon gave a keynote address on the future of biotech in New York City and how NYSCF will continue driving innovations in this sector. Over 200 leaders from New York City institutions, governmental bodies, startups, venture capital, and pharmaceutical companies attended the event.

A NEW THER APEUTIC TARGET FOR MULTIPLE SCLEROSIS: BR AIN STEM CELLS THAT AGE TOO FAST

M yelin is like wire insulation for your nerves — it helps your cells send electrical signals to each other. In multiple sclerosis, the immune

with the more severe ‘progressive’ form of the disease who do not respond well to current drugs targeting the immune system.

system attacks myelin, leaving patients with signaling problems that can cause trouble speaking, moving, and thinking. Patients desperately need treatments that target myelination, especially those

Researchers at the University of Connecticut led by Stephen Crocker, PhD, in collaboration with Anna Williams, PhD, at the University of Edinburgh and NYSCF Senior Research Investigator Valentina Fossati, PhD, have found that brain stem cells they made from progressive MS patients look and act prematurely old. Targeting these features of ‘cellular senescence’ with a drug called rapamycin restored normal function, suggesting a path to regenerating myelin in patient brains. The research appears in Proceedings of the National Academy of Sciences.

“W

e are excited that the study of human stem cells in a dish led to the discovery of a new disease mechanism that could be targeted in much-needed therapeutics for progressive MS patients.”

– Dr. Fossati

Drs. Stephen Crocker and Valentina Fosssati

NYSCF SPEAKS OUT AGAINST FRAUDULENT STEM CELL TREATMENTS IN THE NEW YORK TIMES

PROTECTING EYE CELLS FROM STRESS IN AGE-RELATED MACULAR DEGENERATION

Iyoumagine sitting down to have a conversation with a friend, but cannot see their face. The world around them might be clear, but looking straight ahead of you, all you can see is a dark spot.

This is a reality for millions of Americans suffering from age-related macular degeneration (AMD). NYSCF is committed to making AMD a disease of the past by using stem cells to understand its causes and find new treatments.

A collaborative study published this May in Experimental Eye Research by scientists at the Yale School of Medicine and NYSCF used a stem cell model of dry AMD (the most common form of the disease) to identify a drug that may help protect patient eye cells from damage. The team tested over 5,000 compounds on human eye cells and found that one FDA-approved drug, ciclopirox olamine, showed promise for protecting cells from the harmful effects of stressors, suggesting a new route forward for treating AMD and giving patients their sight back.

Susan L. Solomon, JD (NYSCF) Source: Freeman, Demetrius (Photographer) for The New York Times

ISusan n a recent article from The New York Times, NYSCF CEO L. Solomon, JD, and Senior Vice President of Research

Scott Noggle, PhD, discuss the dangers of predatory stem cell clinics that offer unregulated and unproven stem cell treatments to desperate patients at a high cost, as well as how NYSCF is leading the charge to put a stop to these alarming practices.

Ms. Solomon and Dr. Noggle emphasize the need for increased regulation and clinical evaluation of emerging therapies. “We really need to have very significant clinical trials,” remarked Ms. Solomon. “That’s something we are in discussions with some large institutions about doing.”

Source: gouldvision

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NYSCFNEWSupdate

FUTURE CURES B E G I N W I T H YO U R S U P P O R T T O D AY !

Please make a gift to help NYSCF advance stem cell research toward the clinic.

You can donate online at www.nyscf.org/donate or by mail to:

619 W 54th Street New York, NY 10019

THE NEW YORK STEM CELL FOUNDATION 619 WEST 54 TH STREET, NEW YORK, NY 10019

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NYSCFNEWSupdate

NYSCFNEWSupdate

N Y S C F N E W S U P D AT E

On March 6th, NYSCF CEO Susan L. Solomon, JD, was joined by NYSCF staff, scientists, and board members to ring the Nasdaq bell and officially open stock trading for the day.