NYSCF News Update | Volume 13, Issue 2

NEWSupdate

NYSCF

VOLUME 13 ISSUE 2: 2020-2021

The New York Stem Cell Foundation Research Institute

Celebrating

15 Years of Innovation and Looking to the Future

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YSCF began with the aim of advancing the best stem cell research to accelerate better treatments for patients, and we have never wavered from that mission,” said NYSCF CEO Susan L. Solomon, JD. “When we

started our own laboratory 15 years ago, we hoped to make an impact. It has been much more than that — we have changed the field of stem cell research and pushed it forward. And this is only the beginning.”

Today, the NYSCF Research Institute fills 65,000 square feet, has a staff of 100 including 80 full-time scientists and engineers, and is among the most advanced stem cell laboratories in the world.

The NYSCF lab is where researchers use stem cells to accelerate cures for the major diseases of our time, including Alzheimer’s, Parkinson’s, cancer, diabetes, and multiple sclerosis. And it is where a personalized cell replacement therapy for age-related macular degeneration is soon embarking on a clinical trial (see page 5).

“NYSCF is my role model,” said Nobel Laureate Shinya Yamanaka, MD, PhD, Director of the Center for iPS Cell Research and Application (CiRA), Kyoto University, and a Senior Investigator at the Gladstone Institutes. “They have a beautiful system of automation and stem cell generation from thousands of patients and healthy volunteers. There is a gap between basic research in academia and translational work — we call it the valley of death. NYSCF is a very good way to bridge this gap.”

“NYSCF has played a role in over 20 clinical trials, and we’ve seen several NYSCF-powered therapies enter the clinic, including mitochondrial replacement therapies that prevent inheritance of deadly diseases, which are currently being used in Europe,” remarked NYSCF Senior Vice President of Research Scott Noggle, PhD.

Dr. Yamanaka delivers his keynote presentation at the 2019 Family Stem Cell Day: Students look at 2019 NYSCF Conference cells under a microscope.

NYSCF Global Stem Cell Array® & Mitochondrial Replacement Therapy

NYSCF has designed and built a world-class robotic system, the NYSCF Global Stem Cell Array , that can create high-quality stem cells from hundreds of people at a time – integrating state-ofthe-art automation engineering with artificial intelligence to give unprecedented windows into disease.

®

Contact us at info@nyscf.org or 212.787.4111

Since NYSCF’s founding, we have built and fostered an exceptional group of world-leading researchers. The NYSCF Innovator community, which includes the NYSCF – Robertson Investigators and NYSCF – Druckenmiller Fellows, now includes nearly 200 innovative early-career scientists at 50 institutions worldwide. Since 2006, NYSCF has also convened over 500 top minds annually at our translational stem cell research conference, where leading scientists have presented monumental discoveries and started countless collaborations. Through our other public education programs, NYSCF has reached thousands of people, including over 4,000 K-12 students.

LE ARN MORE AT 15YE A RS . NYSC F.O RG

Uniting to Accelerate Cures 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 Siddhartha Mukherjee, MD, DPhil Stephen M. Ross Derrick Rossi, PhD Stephen M. Scherr Kay Unger Clyde Williams

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 Kara & Stephen Ross Andy Russell Susan & Stephen Scherr Ian Schrager Barbara Stovall Smith Martha Stewart David & Jane* Walentas John Whitehead* Dick Wolf *In Memoriam

Dear Friends, The arrival of 2021 fills me with hope: a new administration committed to science-driven public policy is here, and vaccines are gradually bringing the COVID pandemic under control. But before we close the book fully on 2020, let me say how inspiring it has been for me to watch our remarkable community at NYSCF rise to the challenges of this difficult year, keeping our critical work going in the lab and expanding the scope of our mission to include special, COVID-related research projects, including the creation of lung cells for scientists around the country to use for identifying new drugs against the virus. Many of our NYSCF – Robertson Investigators have also joined the fight against the virus by developing rapid tests, evaluating new therapies, and conducting research to understand immunity. NYSCF – Robertson Stem Cell Investigator Alumnus Derrick Rossi, PhD, was a founder of Moderna, a company that, thanks to its COVID-19 vaccine, is now a household name.

Speaking of Derrick Rossi, we are delighted to have him as well as Clyde Williams and Siddhartha Mukherjee, MD, DPhil, as new members of our Board of Directors. Derrick is the first Alumnus of our program to take a seat on the Board, and it is especially meaningful to have a former NYSCF – Robertson Investigator helping to guide NYSCF’s future. Clyde, a former White House domestic policy advisor under President Obama and former political director of the Democratic National Committee, is working with us to expand our partnerships with Historically Black Colleges and Universities as well as with women’s colleges. Sid is both an esteemed member of the medical faculty at Columbia University and an acclaimed author whose “The Emperor of All Maladies: A Biography of Cancer” won the Pulitzer Prize in 2011.

And there are more reasons to be optimistic about the year ahead for NYSCF. We are marking two very important signs of progress at 619 West 54th Street: our long-planned GMP facility has been completed, allowing us to manufacture clinical-grade cells that can be used in therapies for diseases like age-related macular degeneration (see page 4). And we are getting ready to begin an expansion of our labs to the second floor of our building, which will double our research space, and give NYSCF even more capacity to stay at the forefront of stem cell research.

Susan L. Solomon, JD CEO and Founder

COVID-19 Vaccines, Variants, and Immunity March 16 | Virtual

Upcoming Events

Envisioning a Cure for Blindness

Visit us at

Patients and Scientists Unite to Accelerate Multiple Sclerosis Treatments

NYSCF.ORG/EVENTS

NYSCFNEWSupdate

March 23 | Virtual

April 7 | Virtual

In collaboration with

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

Stem Cells and Celebrities Take Center Stage at the NYSC NYSCF F Gala

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osted by CNN’s Chief Medical Correspondent Sanjay Gupta, MD, this year’s New York Stem Cell Foundation Research Institute Gala & Science Fair went virtual for the very first time. Featuring the latest updates and breakthroughs in stem cell research, the Gala brought the world of the NYSCF Research Institute laboratories to life.

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It’s about the world I can help shape

for my own daughters and their children.

Stem cell research, I think, is really likely to allow future generations to look at diseases like Alzheimer’s, diabetes, and other chronic and degenerative conditions the same way that we now see polio or smallpox: as diseases of the past. NYSCF is showing up every day to make that vision a reality.

–Dr. Sanjay Gupta

The program, themed “Show up for Science,” featured NYSCF Research Institute scientists, numerous celebrity guests, and the three 2020 NYSCF Stem Cell Heroes: award-winning architect and designer David Rockwell; internationally renowned architect Frank Gehry; and scientist, professor, and patient advocate Brooke Ellison, PhD.

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For me, this work is not just about the cures that I’m going to see in my lifetime.

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When I first became involved in stem cell research, I did it because

saw it as a critical source of hope for those for whom hope might be hard to find – those like myself who were told that the disease or disability they were experiencing was incurable or unchangeable,” said Dr. Ellison, who has been paralyzed from the neck down since age 11.

“Stem cell research has been the beacon of hope that has forced us to rethink those very ideas to redefine those words,” she continued. “But this is not just an idea. It's an action and an imperative…And there's no organization that's been more on the forefront of this fight nor more emblematic of the hope to be found in it than The New York Stem Cell Foundation.

–Dr. Brooke Ellison

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Stem cells offer us hope that in our lifetime, we will have treatments for what we think of today as untreatable conditions.

We can all help to accelerate the realization of that hope.

Do whatever you can, because whatever you do to help may actually change the life of someone you love.

–Whoopi Goldberg 3

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The program also included special appearances by Seth Rogen, Lauren Miller Rogen, Yo-Yo Ma, Jane Krakowski, Santino Fontana, Jesse Tyler Ferguson, Annaleigh Ashford, Victor Garber, NASA astronauts Serena Auñón-Chancellor, MD, PhD, and Peggy Whitson, PhD, Michael Bloomberg, Whoopi Goldberg, Kelli O’Hara, Lilli Cooper, Billy Porter, and John Slattery.

The program was directed by award-winning Broadway director Scott Ellis.

Watch the full program at NYSCF.ORG/GALA NYSCFNEWSupdate

Tackling COVID, Promoting Health Equity, and Delivering on the Promise of Regenerative Medicine: Highlights of the 2020 NYSCF Conference

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his is not abstract work. There are people on the other end of everything we do in this industry,” noted Tony Coles, MD, CEO of Cerevel Therapeutics in his Fireside Chat with NYSCF CEO Susan Solomon at the 2020 NYSCF Conference. “Every time I walk into the office, I am reminded that there are families waiting for the work we’re doing today.”

“I think the key is collaboration,” noted Rachael Pearson, PhD, of University College London, who is working toward treating blindness caused by retinal degeneration. “None of us alone can achieve all of the necessary components to take something through from first concept to clinical trials, so I think it’s really important to build a good team.”

This drive to help patients is what compels scientists across the stem cell field, and highlighting promising translational research is the focus of the annual NYSCF Conference. At this year’s meeting, held virtually, participants shared the latest discoveries across a variety of diseases including COVID-19 (see page 5), strategies for combating racial health disparities, and a vision for the future of regenerative medicine.

Racial and ethnic minorities are disproportionately affected by virtually every major disease of our time, and this gap has only been exacerbated by COVID-19. This year, the Conference featured a panel discussion that explored how we can all work together to overcome racial health disparities and ensure that all communities benefit from biomedical breakthroughs.

“This has to be an all-hands effort,” remarked Monica Webb Hooper, PhD, Deputy Director of National Institute on Minority Health and Health Disparities at the National Institutes of Health. “We must have stakeholders at all levels and all disciplines across organizations contributing to get this work done... Equitable engagement with affected communities is a cornerstone of the efforts that will address disparities and move us towards justice.”

The Conference also featured a discussion on regenerative medicine where experts who have traveled this path shared sage advice on translating scientific findings to meaningful therapeutics.

Keynote speaker and cancer immunotherapy pioneer Carl June, MD, is optimistic about the future of regenerative medicine. “I think we’re at a really great time in science because of what’s now possible. There’s work coming from biotechs and universities that didn’t happen in previous drug development efforts.”

Chuck Murry, MD, PhD, Senior Vice President and Head of Cardiometabolic Cell Therapy at Sana Biotechnology and whose lab at the University of Washington works on cellular therapies for heart disease, stressed that for science to move forward, effective communication to regulators and the public is key. “I would encourage the young people out there to not hang their citizenship at the door,” he said. “Invite legislators to come to your laboratories and tell them what it is that you’re working on. When they see that there are reasonable people doing things for the public good, science becomes less scary.”

Watch select talks and panels at Panel discussion about innovation in the time of COVID-19 and celebrating the tenth anniversary of the NYSCF – Robertson Investigator Program with Drs. Kristen Brennand, Feng Zhang, Shuibing Chen, Jay Rajagopal, Deepta Bhattacharya

NYSCF CONFERENCE NYSCFNEWSupdate

YOUTUBE.COM/NYSCF

S AV E T H E D AT E

OCTOBER 19–20, 2021

A Brand New Facility for Creating Cell Therapies

We recently finished construction on a clinical Good Manufacturing Practices (GMP) facility at The NYSCF Research Institute. This specialized

Left: Dr. Cecile Terrenoire examines RPE cells under a microscope Right: Stem-cell-derived RPE cells Image credit: Cecile Terrenoire, PhD

laboratory allows our scientists to create clinical-grade cells for use as therapies, beginning with a cell replacement therapy for age-related macular degeneration (AMD) that we are actively developing. For this treatment, scientists are turning stem cells from different AMD patients into retinal pigmented epithelial (RPE) cells, the cells lost in AMD. Each patient will then receive a transplant of ‘their own’ RPE cells to restore their vision.

“Many major diseases of our time are caused by the death of our natural cells,” explained NYSCF Senior Director of Cell Therapy Programs Howard Kim, PhD. “And cures can only really be realized when those cells are replaced.”

“The first cell therapy we're working on is for age-related macular degeneration, which is a common form of blindness,” he continued.

“In this disease, the cells at the central part of your retina called the macula begin to die, and those are the cells we're trying to replace by growing them from stem cells.”

In our new GMP facility, Dr. Howard Kim stores cells away in an incubator.

Macular degeneration is just the beginning – the new facility gives NYSCF a dedicated, unique, and safe environment in which to create cell therapies for a variety of diseases, and we look forward to bringing these therapies to patients!

COVID-19 Vaccines Are Here:

How Do We Distribute Them Ethically and End the Pandemic?

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here is finally a light at the end of the COVID-19 pandemic tunnel, thanks to a historic vaccine development effort. NYSCF gathered experts for a series of three programs, presented on Zoom, to answer key questions about the science behind COVID-19 vaccines, how vaccine allocation works, and the road back to normalcy.

“What COVID-19 vaccines do is expose healthy individuals to a [harmless] part of the virus, with the purpose of eliciting the immune system to recognize that protein as foreign and mount an immune response to it,” explained Moderna founder Derrick Rossi, PhD, who helped pioneer the mRNA technology that Derrick Rossi, PhD fuels COVID-19 vaccines. “So now when that person goes out and encounters the virus for real, their immune system is ready to mount an efficient response.”

“We have created multiple forums where people can ask any question they want to ask [about the vaccine], and we have not been dismissive,” said Valerie Montgomery Rice, MD, FACOG, of Morehouse School of Medicine’s public outreach efforts. “We have to move from vaccine hesitancy to vaccine acceptance.”

Valerie Montgomery Rice, MD, FACOG | Morehouse School

“To achieve the desired impact of vaccines, you of Medicine, Raeka Aiyar, PhD | The NYSCF Research Institute, Daniel Polsky, PhD | Johns Hopkins University, and Jeffrey Kahn, have to optimize two things,” noted Wafaa ElPhD, MPH | Berman Institute for Bioethics, Johns Hopkins University Sadr, MD, MPH, MPA. “You have to optimize the uptake of the vaccine by the population, and you need to optimize the efficacy of the vaccine itself. We have covered one of those factors: the efficacy piece. So now the challenge is: how do we achieve high uptake in our communities?”

Susan L. Solomon, JD | The NYSCF Research Institute, Wafaa El-Sadr, MD, MPH, MPA | Columbia University, Gareth Rhodes, JD | Governor Cuomo’s COVID-19 Response Task Force, and Siddhartha Mukherjee, MD, DPhil | Columbia University

Learn more at N YSCF.O RG /C OVI D -VAC C I N E S

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NYSCFNEWSupdate

Innovator Community Updates Meet the 2020 NYSCF – Robertson Investigators

Gene Editing in Human Embryos Could Lead to an Unexpected Hazard

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C RISPR gene editing holds great promise for correcting disease-causing

Dr. Dieter Egli

his October, we announced our 2020 class of NYSCF – Robertson Investigators, welcoming six talented stem cell researchers and neuroscientists into the NYSCF Innovator community. The NYSCF – Robertson Investigator Awards provide critical seed funding – $1.5 million over five years – to scientists who have established their own, independent laboratories within the last five years.

mutations, potentially as early as during embryonic development. However, before this intervention can reach the clinic, it must be proven safe and effective. NYSCF – Robertson Stem Cell Investigator Alumnus and former NYSCF Research Institute scientist Dieter Egli, PhD, of Columbia University recently reported in Cell that CRISPR gene editing in human embryos may result in an unexpected hazard: deletion of entire chromosomes.

Raffaella Di Micco, PhD NYSCF – Robertson Stem Cell Investigator

San Raffaele Telethon Institute for Gene Therapy, Italy

Dr. Di Micco is working to optimize gene therapies that use blood stem cells.

“Our study shows that CRISPR/Cas9 is not yet ready for clinical use to correct mutations at this stage of human development,” remarked Dr. Egli. “Our hope is that these cautionary findings should discourage premature clinical application of this important technology, but can also guide responsible research to achieve its ultimate safe and effective use.”

Samantha Morris, PhD NYSCF – Robertson Stem Cell Investigator Washington University in St. Louis

Dr. Morris is interested in developing regenerative therapies for gastrointestinal disease.

Organoids Just Gained Their Missing Ingredient: Functional Blood Vessels

José Ordovás-Montañés, PhD NYSCF – Robertson Stem Cell Investigator Boston Children’s Hospital

Dr. Ordovás-Montañés studies how stem cells become dysregulated in chronic inflammatory diseases and can ‘remember’ inflammation.

Jiami Guo, PhD NYSCF – Robertson Neuroscience Investigator University of Calgary, Canada

Dr. Guo studies primary cilia – hair-like, signaling antennas on virtually all cells – to understand their role in intellectual disabilities and autism spectrum disorder.

Ovarian cancer tumor organoids | Image credit: Dr. Laura Andres-Martin

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David Schneider, PhD

rganoids – 3D structures of human tissue derived from stem cells – are a critical tool for helping researchers study diseases like cancer and develop and test drugs. However, current organoid models do not contain functional blood vessels, limiting their ability to accurately model disease.

Scientists can now create organoids containing functional human blood vessels thanks to a new method published in Nature by a team of scientists led by founding member of NYSCF’s Medical Advisory Board Shahin Rafii, MD, of Weill Cornell Medicine and including NYSCF – Druckenmiller Fellows Ying Liu, PhD, and Jesus Gomez Salinero, PhD. This will allow scientists to better model many different diseases and identify effective therapies. Dr. Rafii is also collaborating with Laura Andres-Martin, PhD, and colleagues at NYSCF to create vascularized ovarian cancer organoids as part of the NYSCF Women’s Reproductive Cancers Initiative.

NYSCFNEWSupdate

NYSCF – Robertson Neuroscience Investigator New York University

Dr. Schneider studies how the brain stores and recalls memories to make and use predictions about the future.

John Tuthill, PhD NYSCF – Robertson Neuroscience Investigator University of Washington

Dr. Tuthill is researching the “sixth sense” known as proprioception — the sense of where the body is in space — aiming to answer how and where motor circuits integrate this information.

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Leveraging Artificial Intelligence for Studying and Treating Parkinson’s Disease

D rugs that aim to treat Parkinson’s disease (PD) have not fared well in clinical trials, suggesting that there is more to PD than we currently understand. The availability of patient cells offers a great opportunity to test potential drugs, but first, it is essential to define which ‘diseased’ features of the cell we want the drugs to reverse.

In a new bioRxiv preprint, NYSCF scientists, in a years-long collaboration with Google Research, present an artificial intelligence–driven platform that successfully identifies new cellular features of PD in skin cells known as fibroblasts. This powerful technology opens an avenue to discovering effective drugs for PD and other diseases.

Fluorescent images highlighting different features of fibroblasts for analysis via artificial intelligence | Image credits: Deidre Carter

Predicting How Prosthetic Implants Will Behave in the Human Body

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illions of Americans have received prosthetic implants, and as new types of implants are developed, researchers need more effective methods for predicting whether they will function properly in the human body.

A team led by NYSCF – Ralph Lauren Senior Research Investigator Giuseppe Maria de Peppo, PhD, has created a new method for testing prosthetic implants using bone tissue grown from stem cells. His team, which operates out of the William A. Marquard Family Foundation Tissue Engineering Suite at the NYSCF Research Institute, engineered a system in which a prosthetic implant is anchored to a scaffold and bone-forming stem cells grow around it, mimicking the environment of the human body and allowing scientists to test how well an implant will integrate into a patient. The study appears in Scientific Reports.

“We are excited that the system shows a promising ability to predict how an implant will behave in a patient, which could help inform the development of new implants and evaluate the efficacy of drug-delivering implants,” said Dr. de Peppo.

Figure 1 from study: Engineering the bone-implant platform. Model prosthetic implants are anchored to biomimetic scaffolds seeded with bone-forming cells.

“As we enter a new age of regenerative medicine, models like–Giuseppe this will be critical.”

Maria de Peppo, PhD 7

NYSCFNEWSupdate

Future cures begin with your support today!

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: The New York Stem Cell Foundation 619 West 54th Street, 3rd Fl, New York, NY 10019

619 W 54 th Street New York , NY 10019

Legacy of Cures Join our Legacy of Cures Society by including The New York Stem Cell Foundation in your will.

Together, we can accelerate cures for the major diseases of our time. Your support will benefit generations to come.

Please contact the Development team at (212) 365-7434 or legacyofcures@nyscf.org.

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15 YEARS of INNOVATION LE ARN M O RE AT 15 YE A RS . N YSCF.ORG

CLINICAL IMPACT COMMUNITY BUILDING TECHNOLOGY DISEASE RESEARCH EDUCATION WWW. N YS C F.ORG

Oligodendrocytes from a multiple sclerosis patient | Image by NYSCF scientist Maria Sapar, PhD