NYSCF News Update | Vol 12, Issue 2

NEWSupdate

N YS C F

VOLUME 12: ISSUE 2: 2019

The New York Stem Cell Foundation Research Institute

U N LOC K I NG TH E POW E R O F PREC I S IO N MEDICINE WITH STEM CELLS

Michael R. Bloomberg at the NYSCF Gala; Dr. Antony Rosen and Susan L. Solomon announce a new par tnership between NYSCF, Johns Hopkins University, and Bloomberg Philanthropies at the annual NYSCF Conference

Precision

medicine means finding the right therapies for the right patients at the right time.

Each patient has a unique disease experience and genetic blueprint, and precision medicine takes these into account to ensure patients receive tailored care rather than one-size-fits-all treatments.

Bloomberg Philanthropies, Johns Hopkins University School of Medicine, and The NYSCF Research Institute are embarking on a new precision medicine initiative leveraging the unique promise of stem cells. In the collaboration, biological samples collected through JHU’s Precision Medicine Centers of Excellence will be turned into high-quality stem cells using the fully automated NYSCF Global Stem Cell Array® technology. With patientspecific stem cell lines, disease-relevant cell types, and extensive clinical data, researchers will be better able to pinpoint the causes of disease and determine which types of therapies will work for which patient populations.

“B

“Diseases are heterogenous, but within that heterogeneity are homogenous subgroups. Finding these homogenous subgroups at scale is a major opportunity,” explained Antony Rosen, MBChB, MS, Johns Hopkins School of Medicine’s Vice Dean for Research.

loomberg Philanthropies’ mission is to ensure better, longer lives for the greatest number of people.

For years, Johns Hopkins University and the New York Stem Cell Foundation have shared that mission – and we’re honored to deepen our partnerships with them as they explore new, innovative ways to save lives through the application of precision medicine.”

Identifying subgroups and drawing conclusions about disease mechanisms takes highly sophisticated technological capabilities like those developed and housed at NYSCF. “The NYSCF Research Institute has invented and scaled the most advanced methods of human cell manipulation, which is critical for studying disease at the level of the individual patient,” noted NYSCF CEO Susan L. Solomon.

Thanks to this technology, we can create and study human cells from large –Michael R. Bloomberg populations of genetically diverse patients, Founder of Bloomberg LP enabling insights into what makes and Bloomberg Philanthropies individuals experience disease differently and which patient subgroups may benefit from which treatments. Precision medicine approaches like these are already transforming cancer care, and with At the 2019 NYSCF Conference (see page 4), a panel discussion partners like Johns Hopkins and Bloomberg Philanthropies on our explored promising developments and opportunities in precision team, we are confident that transformative therapies for many more medicine that are already being fueled by stem cell research. diseases are not far behind.

F E AT U R E D I N T H I S I S S U E T H E N Y SCF G A L A

CONFER ENCE

Celebrating advances in stem cell research p.3

Precision medicine, collaboration, and the quest for cures p.4

Contact us at info@nyscf.org or 212.787.4111

WOMEN’S REPRODUCTIVE CANCERS

Bringing better treatments to patients p.5

NEW INVESTIGATORS

Meet the 2019 NYSCF – Robertson Investigators p.6 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 Francesco Clark Paul Goldberger George Lazarus, MD Richard J. Massey, PhD Paul M. Meister Stephen M. Ross Stephen M. Scherr Kay Unger 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 are transforming human health to bring better solutions to patients. We are closer than ever to new treatments for so many diseases, and I am delighted to see the inspiring breakthroughs happening in our own laboratories and in our worldwide community, many of which were shared at our conference this fall (see page 4).

I am thrilled to share the details of a new partnership to advance precision medicine with the power of stem cells (see cover). By combining NYSCF’s cutting-edge technology and stem cell capabilities with the extensive clinical data and expertise at Johns Hopkins University School of Medicine’s Precision Medicine Centers of Excellence, and the vision and leadership of Bloomberg Philanthropies, we will be able to develop effective, personalized therapies for patients suffering from diseases with a high unmet need.

At NYSCF, our unique approach is the “tide that lifts all boats,” accelerating the entire field. We are honored to partner with many dedicated and talented organizations who are also pushing the field closer to new solutions and cures. For example, we recently partnered with NYSCF Innovator Alumnus Dr. Chris Gregg to personalize cancer treatment using big data (see page 5). And with INADcure, we are using stem cells to give new hope to families affected by a rare, mysterious neurological disease (see page 7).

NYSCF is all about impact – what stem cells bring to patients. Thank you so much for all you do to make this impact possible.

Warm wishes for a happy and healthy holiday season,

Susan L. Solomon, JD CEO and Founder

Whose hero will you be?

With your help, we can advance the most innovative stem cell research to benefit your children, their children, and generations of your family to come.

The cures of tomorrow begin with your commitment today.

Leave the legacy of cures.

Visit us at N Y S C F. O R G / P L A N N E D

Include in your will The New York Stem Cell Foundation NYSCFNEWSupdate

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TH E N YSC F G A L A A N D S C I E N C E FA I R

A t the 2019 NYSCF Gala and Science Fair, we proudly honored five Stem Cell Heroes – Peter J. Goulandris, Dr. Karen Elizabeth Burke Goulandris, Jay Kriegel, Greg Hicks, and Rick Foster – for their steadfast commitment to our work.

Top row from left: Dr. Giuseppe Maria de Peppo, Susan Scherr, Stephen Scherr ; Michael R. Bloomberg, Patti Harris 2 nd row from left: Stephen Meringoff; Susan L. Solomon, Michael R. Bloomberg; Anita Wien, Byron Wien 3 rd row from left: Zara Duffy, Matt Dillon; Alisa Doctoroff, Dan Doctoroff, David Rockwell; Gail Geronemus, Dr. Roy Geronemus, Lloyd Blankfein; Marlene Hess, Jim Zirin NYSCFNEWS Bottom row from left: Jeff Blau, Susan L. Solomon, Kathryn McAuliffe, Jay Kriegel; Bill Ackman, Dr. Neri Oxman

update

Highlights of the

2019 NYSCF Conference Nobel Laureate Dr. Shinya Yamanaka, Director of the Center for iPS Cell Research and Application (CiRA), Kyoto University, and Senior Investigator at the Gladstone Institutes, delivers his keynote presentation

Iearned n 2006, Shinya Yamanaka, MD, PhD, made a discovery that revolutionized biomedical research and him a Nobel Prize. His lab found that stem cells can be made from just a small sample of skin. In his opinion, the work being done by

NYSCF is critical for scaling up his discovery and making it as impactful as possible. And NYSCF’s private philanthropic support equips us to pursue bold, risky ideas and move faster than most institutions.

“NYSCF is a role model for me,” remarked Dr. Yamanaka in his keynote lecture at this year’s NYSCF Conference.

“Susan [Solomon] is my hero.”

During the 2019 NYSCF Conference held at The Rockefeller University on October 22–23, over 500 researchers, students, and thought leaders shared their latest findings in translational stem cell research, highlighting how stem cells are revolutionizing medicine.

“21st century medicine is really about studying wellness and the dynamics of disease, but most importantly, it’s about identifying the transitions from wellness to disease and reversing them at the earliest point,” explained biotech pioneer Lee Hood, MD, PhD, in his plenary lecture.

This approach is helping to demystify devastating diseases like Alzheimer’s, as Tracy Young-Pearse, PhD, is doing in Dr. Lee Hood (SVP and Chief collaboration with NYSCF. Science Officer, Providence St. Joseph Health; Chief Strategy “We have a cohort of 3,000 elderly Officer, Institute for Systems people who are followed clinically every Biology) year until death. Statistically, about half will get Alzheimer’s,” she noted. “We have made stem cell lines from some of them, and this allows us to see which genes put people at risk and which promote cognitive resilience.”

Many presentations highlighted how stem cells are helping to predict which drugs will be effective for which diseases and patients. NYSCF’s latest initiative to improve treatments for women’s reproductive

cancers was showcased in a panel discussion (see page 5).

“By definition, there are no animal models that are especially predictive. There’s a dire need for building new models like organoids,” said keynote speaker Hans Clevers, MD, PhD, of the Hubrecht Institute and Utrecht University, who has pioneered the conversion of tumors into organoids, personalized 3D models of cancer that he is now helping NYSCF scientists to create and use for drug testing.

Some of the most promising studies are already using stem cells to regenerate tissues destroyed by disease. The University of Modena and Reggio Emilia’s Graziella Pellegrini, PhD, showed a stunning case of the rare genetic disease epidermolysis bullosa (EB), in which a stem-cell-based therapy allowed near-complete reconstruction of a young boy’s skin.

All of the researchers at the conference Dr. Tracy Young-Pearse (Ann Romney Center for Neurologic Diseases at share one belief: that science will bring Brigham and Women’s Hospital and treatments and cures to those who Harvard Medical School) desperately need them.

“When I was training as a young surgeon, my father passed away. I couldn’t do anything to help him, and that had a huge impact on me,” said Dr. Yamanaka. “I truly believe that it is science that will help patients suffering from intractable diseases.”

NYSCF CONFERENCE S AV E T H E D AT E NYSCFNEWSupdate

OCTOBER 4

20-21, 2020

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

R A M PI N G U P R E S E A RC H I NTO WO M E N ’ S R E PRO D U C TI V E C A N C E R S

Our cancer initiative was spotlighted at our annual conference in a panel discussion featuring Drs. Hans Clevers, Siddhar tha Mukherjee, Ursula Matulonis, Michael Birrer, and Laura Andres-Mar tin

Our Women’s Reproductive Cancers Initiative has made significant progress since April’s launch.

Using patient tumors, we are creating organoids (personalized 3D cancer models with an infinite lifetime) to advance precision oncology research and drug testing.

This summer, our lead cancer investigator, Laura Andres-Martin, PhD, won a highly competitive grant from the National Cancer Institute to pilot this exciting research! Dr. Andres-Martin’s team has generated organoid lines from 48 ovarian cancer patients so far. Our ultimate goal is to build a biobank representing hundreds of patients as a resource to fuel discoveries across the greater cancer research community.

We have also recruited a world-class Scientific Advisory Board to guide this important work and accelerate its impact on patient outcomes.

Carol Aghajanian, MD

Memorial Sloan Kettering Cancer Center

Michael Birrer, MD, PhD

The University of Alabama at Birmingham

George Coukos, MD, PhD University of Lausanne

Susan M. Domchek, MD

Carla Grandori, MD, PhD

Siddhartha Mukherjee, MD, DPhil

Carl June, MD

Alessandro Santin, MD

SEngine Precision Medicine University of Pennsylvania

THE SCIENTIFIC A DV I S O RY B OA R D

University of Pennsylvania

Ronny Drapkin, MD, PhD

Yale University

Elizabeth M. Swisher, MD University of Washington

Victor E. Velculescu, MD, PhD Johns Hopkins University

Ursula Matulonis, MD

University of Pennsylvania

Dana-Farber Cancer Institute

Olivier Elemento, PhD

Kathleen Moore, MD

Weill Cornell Medicine

Columbia University

University of Oklahoma

Irving Weissman, MD Stanford University

Oliver Zivanovic, MD, PhD

Memorial Sloan Kettering Cancer Center

N YS C F PA RTN E R S W ITH A LU M N U S C H R I S G R EG G TO PE R SO N A LI Z E C A N C E R TR E ATM E NT W ITH B I G DATA

C ancer patients can often feel powerless. They want to do all they can, but many feel as though they don’t have much control over their treatment. A new venture called Storyline from NYSCF – Robertson Neuroscience Investigator Alumnus and University of Utah Associate Professor Chris Gregg, PhD, funded in part by NYSCF, is giving patients a new avenue to improve diagnosis, personalize treatments, and further our understanding of cancer.

“Storyline is a new artificial intelligence platform for analyzing cognitive and behavioral patterns where patients can answer questions about their disease experience over time: what treatments they are receiving, how they are feeling, etc.,” explained Dr. Gregg. “Then, with machine learning, researchers will identify subgroups of patients with similar disease progressions and treatment responses, ultimately helping determine which therapies will work for which patients.”

Cancer is a famously variable and patient-specific disease. Collecting and managing data about how different patients experience cancer has enormous potential to improve diagnoses, treatments, and patient quality of life. We are excited about this partnership with Dr. Gregg and the many lives it will improve.

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I N N O VAT O R C O M M U N I T Y U P D AT E S M E E T T H E 2 019 N YS C F – RO B E RT S O N I N V E S T I G ATO R S

A S U R PR I S I N G S O L D I E R I N T H E B AT T L E AG A I N S T S K I N C A N C E R : THE HAIR FOLLICLE

Y our hair follicles are good for more than birthing luscious locks. According to a new

study in the Journal of Cell Biology by NYSCF – Robertson Stem Cell Investigator Valentina Greco, PhD, of Yale University, they can also help fight skin cancer. Dr. Greco’s team discovered that when cells carrying a cancerassociated mutation in the gene HRAS arise in the hair follicle, they spark follicle regeneration, in turn encapsulating the mutant cells and stopping them from growing and spreading.

T his October, we welcomed six outstanding early career stem cell researchers and neuroscientists into the NYSCF – Robertson Investigator Program. These awards provide critical funding — $1.5 million over five years — to talented scientists as they establish their own, independent laboratories.

Ya-Chieh Hsu, PhD

NYSCF – Robertson Stem Cell Investigator Harvard University Dr. Hsu studies cell-to-cell and systemto-cell interactions using skin as a model, with the goal of regenerating fully functional skin to treat severe injuries.

Evangelos Kiskinis, PhD

CRISPR TO THE RESCUE

NYSCF – Robertson Stem Cell Investigator Northwestern University Dr. Kiskinis uses stem cells to identify opportunities for targeted and effective therapies for ALS and pediatric epilepsy.

Florian Merkle, PhD

The CRISPR family enzyme Cas13 (pink) is at the heart of the RESCUE platform, where it uses a special guide (red) to target RNAs in the cell (blue) Image Credit: Stephen Dixon

C RISPR gene editing just got more versatile. This summer, NYSCF – Robertson Stem Cell Investigator Feng Zhang, PhD, of the Broad Institute of MIT and Harvard unveiled RESCUE: a new CRISPR tool that makes temporary genetic edits, which can be preferable for some diseases to the permanent DNA alterations made by traditional CRISPR. RESCUE can target a new category of disease-causing mutations in short-lived RNA molecules; Dr. Zhang’s team reported in Science that they successfully used RESCUE in human cells to change an Alzheimer’s risk gene into a benign form.

C O N G R AT U L AT I O N S TO A N E W M AC A RT H U R G E N I U S !

NYSCF – Robertson Stem Cell Investigator University of Cambridge, United Kingdom Dr. Merkle uses stem cells to reveal processes that contribute to obesity and to identify new therapeutic targets for obesity and neurodegeneration.

Nicholas Bellono, PhD

NYSCF – Robertson Neuroscience Investigator Harvard University Dr. Bellono is interested in how organisms adapt to detect salient environmental signals based on their specific ecological or behavioral context.

Lauren O’Connell, PhD

NYSCF – Robertson Neuroscience Investigator Stanford University Dr. O’Connell studies the neural circuits behind social decision-making and how nutrition during development influences brain organization and function early in life.

Yuki Oka, PhD

N YSCF – Robertson Neuroscience Investigator Alumna Vanessa Ruta, PhD, of The Rockefeller University has been

NYSCF – Robertson Neuroscience Investigator California Institute of Technology Dr. Oka aims to understand how the brain and the body cooperate to regulate the internal body environment.

named a 2019 MacArthur Fellow for her work investigating the neural circuits that underlie innate and learned behaviors.

NYSCF CELLS GO TO SPACE!

T his December, brain cells derived from patient stem cells by NYSCF scientists are making the 250-mile journey up to the International

Space Station for a study of multiple sclerosis and Parkinson’s disease in microgravity. This study is being carried out in collaboration with scientists at Aspen Neuroscience, the National Stem Cell Foundation, and Space Tango. A test launch was completed from NASA Kennedy Space Center in July.

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NYSCF’s Dr. Valentina Fossati prepares cells for the flight at NASA Kennedy Space Center. Photo cour tesy of SpaceTango.

The Grades Are In: WO M E N A R E STI L L U N D E R R E PR E S E NTE D I N STE M , AND HERE’S HOW WE CAN FIX IT Kristin Smith-Doody (NYSCF Associate Vice President, External Programs)

When it comes to science, technology, engineering, and medicine (STEM), the victims of gender imbalance – of using less than 100% of the available brainpower – are patients.

Frustrated by the “all talk, no action” approach to gender disparities in STEM, NYSCF CEO Susan L. Solomon, JD, established NYSCF’s Initiative on Women in Science and Engineering (IWISE). One of IWISE’s first strategies was the implementation of an Institutional Report Card for Gender Equality. For the last 4 years, we have used the Report Card in our extramural grants program to collect quantitative and qualitative data about gender representation across more than 500 institutions in 38 countries. The results — published in Cell Stem Cell and covered by STAT, BBC, Reuters, Bustle, and more — demonstrate the widespread persistence of gender inequality in decision-making STEM roles, and that the imbalance lies not in recruitment, but in retention and promotion.

“Our results across hundreds of institutions show that women continue to be systematically underrepresented in STEM leadership, making up under one quarter of full professors,” explained Kristin Smith-Doody at the 2019 NYSCF Conference. “We still have a lot of work to do, and it will take a concerted effort across many groups to reach equality.”

NYSCF remains both optimistic and committed to ensuring that we achieve gender balance in STEM. We will continue to implement actionable strategies, and use the Report Card to collect data, highlight best practices, and assess interventions that improve gender representation over time. We hope that everyone will join us on this journey, because when the best minds have opportunities to lead scientific inquiries, breakthroughs will reach patients that much faster.

F O R A FA M I LY A F F EC TE D BY R A R E D I S E A S E , STE M C E L L S A R E PROV I D I N G N E W H O PE

W hen Ariya Panwala was born, she was a happy, squirmy baby — but after a few months, her parents noticed

life — she wanted to do whatever was necessary to help her daughter and other INAD-affected families. That’s what led her to NYSCF.

she wasn’t advancing as expected. Her diagnosis with infantile neuroaxonal dystrophy (INAD), a rare and debilitating disorder that attacks the nervous system, upended her parents’ lives. They learned that Ariya would lose her cognitive and motor skills, first finding it hard to walk or control her neck, then that her legs and arms would become tight and weak, with a devastating life expectancy of just 5-10 years.

“Science is our best bet to help Ariya,” she says. “The more we know, the higher our chances of being able to advance treatments.”

NYSCF is now creating cell lines from INAD-affected families to enable a multitude of research projects. INAD is genetic, so NYSCF is working on gene editing patient cells to yield insights into what drives the disease and accelerate treatment development. Gene editing may even hold promise for a cure — by directly fixing the disease-causing mutation to knock out the disease once and for all.

“It felt like I was hit by a bus,” says Leena Panwala, Ariya’s mother. “I didn’t know what INAD was, and when I turned to the internet for more information, there wasn’t much. There wasn’t a voice for this disease.”

“Gene editing is a powerful tool with a lot of promise for helping patients, especially those with rare genetic diseases like Ariya,” noted NYSCF – Robertson Stem Cell Investigator and gene editing pioneer Feng Zhang, PhD, of the Broad Institute of Harvard and MIT. “The possibility for the technique to give a child their life back is very exciting.”

So Leena decided to become that voice. She and her husband Anil launched INADcure, a foundation aimed at advancing research into INAD, developing new therapies, and hopefully finding a cure. Leena, who serves as INADcure’s President, couldn’t just sit back and watch the disease take a hold on her Leena Panwala and her daughter Ariya

Leena is optimistic. She’s hopeful for the future, and for her daughter.

She wants other families affected by INAD to feel the same.

“I look forward to seeing what the future brings. I try to inspire other families impacted by INAD to keep a positive mindset. Where we are with the science is very exciting, so there is a lot to be hopeful for.”

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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 54 th 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

N Y S C F N E W S U P D AT E At the 2019 NYSCF Gala and Science Fair, we proudly honored five Stem Cell Heroes – Jay Kriegel, Greg Hicks, Dr. Karen Elizabeth Burke Goulandris, Rick Foster, and Peter J. Goulandris – for their steadfast commitment to our work.