NEWSupdate VOLUME 13: ISSUE 1: 2020
N YS C F
The New York Stem Cell Foundation Research Institute
NYSCF Springs Into Action Against COVID-19 The COVID-19 pandemic is the most significant health issue facing the world today. With millions of cases worldwide, overcrowded hospitals, and no cure, there is a dire need to understand and treat this devastating disease.
At the NYSCF Research Institute, our scientists are using stem cells to create
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the specific types of lung cells affected by COVID-19 as a community resource. This will accelerate worldwide research into pressing questions like how the virus
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scientists at wor k in
the lab
infects the lungs, how genetics may make certain individuals more susceptible to severe cases of the disease, and which therapies have the potential to stop or prevent infection. Our vast biobank of stem cell lines allowed us to begin this urgent research immediately, and our labs have remained open throughout the pandemic to address this crisis. “When these lung cells get infected, they make more of the virus, but they also die – eliminating the source of mucus that cleans your lungs,” explained NYSCF Senior Vice President of Research Scott Noggle, PhD, in a research update on stem cell approaches to COVID-19. “We use those cells, created from stem cells, to test large amounts of existing drugs or find new drugs that would prevent the infection in the first place, sparing these cells and stopping the virus from replicating.” Dr. Scott Noggle
The NYSCF Research Institute
Dr. Shuibing Chen
Weill Cornell Medical College
Dr. Feng Zhang
Broad Institute of MIT and Harvard
Dr. Deepta Bhattacharya University of Arizona
Conversation with Experts: Updates on the COVID-19 Fight
Around the world, our Innovators are taking aim at the virus. Shuibing Chen, PhD
Feng Zhang, PhD
Derrick Rossi, PhD
NYSCF – Robertson Investigator Alumna
NYSCF – Robertson Investigator Alumnus
NYSCF – Robertson Investigator Alumnus
Dr. Chen is using organoids (3D clusters of tissue made from stem cells) to study the human cells that are susceptible to SARS-CoV-2 infection and has identified drugs that block the virus’s entry into cells.
A rapid paper strip test developed by Dr. Zhang, (based on the revolutionary CRISPR gene editing technology he helped pioneer) could allow point-of-care diagnosis of COVID-19 using materials that only cost about $6 per test.
A SARS-CoV-2 vaccine created by Moderna Therapeutics, founded by Dr. Rossi, has proven generally safe and able to stimulate antibody formation in early clinical trials. It has just begun a large-scale trial in 30,000 participants.
Ascommunity part of NYSCF’s new Virtual Learning and Events Program (see page 5), several members of our Innovator shared promising strides toward faster diagnosis, new treatments, hotspot surveillance, and vaccine research. The group underscored how quickly the scientific community has come together to tackle COVID-19.
“T
his pandemic is devastating, but it has also brought about the most collaborative period of science that I’ve ever experienced... I’ve probably met more people in the last four weeks than I typically would in a year, and everyone who I’ve reached out to about collaborating has been receptive. I think that speaks to how united we are in this fight.”
-Dr. Feng Zhang
F E AT U R E D I N T H I S I S S U E DI V ER SI T Y
ISSCR AWA R D
VIRTUAL EVENTS
N EU RODEGE N E R AT ION
Ensuring that medical breakthroughs reach everyone p.3
Susan Solomon receives Public Service Award p.3
Continuing science education during the pandemic p.5
How brain cells ‘go rogue’ in Alzheimer’s, multiple sclerosis, and more p.6
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 Francesco Clark Paul Goldberger George Lazarus, MD Richard J. Massey, PhD Paul M. Meister Stephen M. Ross Stephen M. Scherr Francesco Clark Clyde E. Williams, Jr.
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 and Jane* Walentas John Whitehead* Dick Wolf *In memoriam
Dear Friends, 2020 has been a year like no other. Our society was upended by the COVID-19 pandemic, which, as I write this, has claimed over 900,000 lives and changed nearly every aspect of how we live. COVID-19 is now a major disease of our time, and NYSCF’s mission and unique capabilities compel us to take it on. I am so proud of our scientists at the NYSCF Research Institute who have remained hard at work in our laboratories throughout the pandemic creating the lung cells targeted by COVID-19, offering them as a resource for leading virologists and other researchers worldwide, and partnering with experts to drive drug discovery and find new therapies. Throughout the broader NYSCF community, so many have stepped up to accelerate testing, produce PPE, compile data and information resources, and work tirelessly toward solutions for patients and society. And while we cannot hold our regular in-person events at this time, we have continued our commitment to public outreach by launching a new Virtual Learning and Events Program of webinars, live discussions, and online educational resources to help the public stay informed about breakthroughs in medical research and what they mean for all of us (see page 5). After seeing the disproportionate impact wrought by COVID-19 on the health and welfare of ethnic minorities, we witnessed numerous acts of violence against these communities that underscored the disparities that continue to exist in our nation. I am so angered by these injustices, and it is deeply painful to know that members of our staff and community regularly deal with various forms of discrimination. At NYSCF, we are committed to the values of inclusion and equality in everything we do. We want to make sure we support efforts to celebrate diversity and that we continue to strive for more inclusion, including in our research studies, as doing so is an important aspect of our ultimate goal: bettering human life. While we could never have imagined where we would find ourselves in 2020, I am inspired by how quickly we have come together to take on these unimaginable challenges. Our important work on diabetes, age-related macular degeneration, women’s reproductive cancers, and other diseases has moved forward and remains on track. I am more confident than ever that together, we will build a healthier, more robust society for the future. Thank you for your ongoing support. Warm wishes,
Susan L. Solomon, JD CEO and Co-Founder
LEGACY of CURES Join our Legacy of Cures Society by including The New York Stem Cell Foundation in your will. NYSCFNEWSupdate
Together, we can accelerate cures for the major diseases of our time. Your support will benefit generations to come.
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Please contact the Development team at (212) 365-7434 or legacyofcures@nyscf.org.
To Make Precision Medicine Possible, We Need More Diversity in Biomedical Research
D isease impacts everyone, but not in the same way. Disease risk and drug efficacy can vary a great deal across individuals, and unfortunately, much of what we know about diseases and drugs does not apply to ethnic minorities, because most studies have not adequately represented them as research subjects.
For example, 78% of people in genetics studies of disease (leading up to 2018) were of European descent. When biological data is generated solely from Caucasian research subjects, treatments often end up being ineffective or dangerous for ethnic minorities. 67% of Puerto Rican children do not respond to the drugs used in asthma inhalers. Up to 75% of Pacific Islanders are unable to convert the antiplatelet drug clopidogrel into its active form and are at higher risk for adverse outcomes following angioplasty, a common therapy for cardiovascular disorders. patients about the value of participating in our research,” she explained.
“This is simply unacceptable, and all of us in the research community must do better,” remarked NYSCF Senior Vice President of Research Scott Noggle, PhD. “We’ve had a longstanding interest in this area. The original aspiration for the NYSCF Global Stem Cell Array® was to build a system that could accelerate studies of disease across populations.”
Collaborations are a large part of NYSCF’s efforts to increase diversity in our research. Our Women’s Reproductive Cancers Initiative is creating a biobank of tumor ‘avatars’ that can be used to understand and treat these highly individualized cancers, and we collaborate with physicians at Weill Cornell Medicine and Memorial Sloan Kettering Cancer Center who provide our scientists with tumor samples from New York City’s ethnically diverse population. We also partner with Johns Hopkins University on a precision medicine initiative to use stem cells as a window into individual disease biology and tailored treatments.
The Array, NYSCF’s automated technology for producing stem cells, can make stem cells from hundreds of samples at a time, and NYSCF has built a biobank of stem cell lines – which we are continually expanding – aimed at capturing enough genetic diversity to allow assessments of personalized disease traits and drug efficacy. Creating these stem cell lines relies on skin or blood samples collected through our clinical research program, managed by Lillian Mehran, MPH, CHES.
“Our mission is to accelerate better treatments and cures for the major diseases of our time through stem cell research, and we cannot achieve this goal without ensuring our research includes diverse participants,” said Dr. Noggle. “There is still much more that we can do, and that we plan to do to make sure precision medicine reaches everyone.”
“We are continually identifying new strategies and avenues through which to recruit, whether that involves connecting with people online or forming partnerships with groups who can communicate with
Susan L. Solomon Receives ISSCR Public Service Award
N YSCF CEO Susan L. Solomon, JD, recently received the 2020 Public Service Award from the International Society for Stem Cell Research (ISSCR) for her outstanding contributions to the field. The ISSCR awards celebrate innovative work to harness the potential of stem cells, recognize exceptional research, support young researchers and the future of stem cell research, and acknowledge extraordinary public service.
“S usan Solomon has been an early and enthusiastic supporter of stem cell science,” said Doug Melton, PhD, former ISSCR president and Xander University Professor at Harvard University in a press release from ISSCR. “She
successfully campaigned for the state of New York to support the field and, more significantly, established a leading foundation dedicated to advancing stem cell science. Her commitment to supporting the entire field, particularly through the awards to young investigators has been inspirational and productive.” Dr. Doug Melton | Xander University Professor at Harvard University Leading scientists pay tribute to Susan Solomon’s transformational leadership
Dr. Paul Tesar
Case Western Reserve University
Dr. Takanori Takebe
Dr. Hans Clevers
Cincinnati Children’s Hospital
Hubrecht Institute, Utrecht University
Dr. Christine Mummery
Leiden University
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Dr. Kevin Egganupdate NYSCFNEWS Harvard University
Community Continues from Afar: Highlights of the First Virtual NYSCF Innovators Retreat
Each year in May, all NYSCF – Robertson Investigators, NYSCF – Druckenmiller Fellows, and NYSCF Research Institute scientists gather for the NYSCF Innovators Retreat: one week of discussion and camaraderie highlighted by powerful research. This year, the retreat — now in its eleventh year — was held virtually, but the science and spirit were just as strong. Each investigator confidentially presented their latest research to get cross-disciplinary feedback from their peers, and for the first time ever, the retreat included interactive panel discussions, which focused on big data and clinical translation.
Paul Tesar, PhD, a NYSCF – Robertson Stem Cell Investigator Alumnus from Case Western Reserve University, reflected that translating his drug screening platform for central nervous system diseases into a spinout company was kickstarted at the retreat. “Bringing this platform to the clinic really began with what I learned here at the NYSCF retreat,” said Dr. Tesar. “This is where I realized that what I was doing could have an impact.”
“We used to think there would be one cure for each disease, but we increasingly realized there could be as many cures as there are patients,” noted NYSCF – Robertson Stem Cell Investigator Alumna Kristen Brennand, PhD, of the Icahn School of Medicine at Mount Sinai, of her lab’s work to integrate big data with stem cell approaches to examine the genetics of patients with neuropsychiatric diseases. “We need to be better at grouping patients so that if we have a new drug that will only work on 2-3% of patients, we know who those patients are.”
The retreat ended with a toast from the graduating class of Investigators. “NYSCF means an incredible amount to me,” remarked NYSCF – Robertson Neuroscience Investigator Justin Ichida, PhD, of the University of California, San Francisco, who was also one of the first NYSCF – Druckenmiller Fellows in 2007. “I became a part of this community back when the retreat was just six scientists meeting up for a few hours. It has grown so much since then and truly feels like a family.”
Meet the 2020 NYSCF – Druckenmiller Fellows
T he NYSCF – Druckenmiller Fellowship was created to support 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.
Katie Cockburn, PhD
Katherine Stewart, PhD
Dr. Cockburn studies the process by which the human body replaces skin cells, aiming to shed light on the causes of skin diseases and cancer and develop regenerative therapies.
Dr. Stewart aims to understand how the body clears dying cells to make way for fresh, healthy cells, advancing insight into skin regeneration.
Yale University
The Rockefeller University
Hiroko Nobuta, PhD
Yongjin Yoo, PhD
Albert Einstein College of Medicine
Stanford University
Dr. Nobuta is using gene engineering approaches to create oligodendrocytes – brain cells important for typical brain function – from stem cells and use them to develop a drug screening platform for diseases such as multiple sclerosis.
NYSCFNEWSupdate
Dr. Yoo is researching cell therapies for brain disorders– specifically, a non-invasive and safe method for replacing microglia, the brain’s immune cells.
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On the Road to Personalized Therapies for Women with Reproductive Cancers
New Hope for Alzheimer’s: Scientists and Clinicians Present Emerging Therapies for a Notoriously Challenging Disease
Women’s reproductiveendometrial cancers (such as ovarian, cervical, or cancer) can be difficult
T here are more than 44 million people with Alzheimer’s worldwide, but no effective therapies. In February, NYSCF
convened top scientists and clinicians to share exciting breakthroughs towards treatments, including gene therapies that reduce Alzheimer’s risk by introducing the protective APOE2 gene into the brain being led by Weill Cornell’s Ron Crystal, MD. “I’ve been doing Alzheimer’s panels for a number of years, and this is one of the first times I’m going to come away with some hope,” emphasized moderator Max Gomez, PhD. “And please remember that we have to be the squeaky wheel for research funding. If you’re interested in curing Alzheimer’s, make a lot of noise about it.”
NYSCF’s Laura Andres-Martin, PhD
“[Organoids] are incredibly useful for understanding the variable nature of the disease and testing drugs, especially for tumors that have become resistant to therapies,” noted Susan Domchek, MD, of the University of Pennsylvania. “We’re always looking for the next great cancer models, and I’m very excited about this one.”
Drs. Max Gomez (CBS New York), Scott Noggle (The NYSCF Research Institute), Michelle Ehrlich (Icahn School of Medicine at Mount Sinai), Ron Crystal (Weill Cornell Medical College), and Sam Gandy (Icahn School of Medicine at Mount Sinai)
Dr. Susan Domchek
Progress, Challenges, and Promise: The Fight for a Type 1 Diabetes Cure
Cooped Up in Quarantine?
F or nearly a century, we have used insulin therapy to treat the symptoms of type 1 diabetes (T1D), but there is still no cure for the disease. At a panel discussion on March 2, scientists, clinicians, and patient advocates delved into promising strategies to cure T1D, including NYSCF’s work to develop cell replacement therapies by converting stem cells into pancreatic beta cells and modifying them to become invisible to the immune system.
“The story here [at NYSCF] of the pioneering work that’s happening — that this team has catalyzed — shows that there’s no singular approach to curing T1D, but that the promise of the work is very, very real,” highlighted JDRF’s Aaron Kowalski, PhD. Ben Goldberger (TIME), Dr. Bjarki Johannesson (The NYSCF Research Institute), Dr. Carol Levy (Mount Sinai Diabetes Center), Dr. Aaron Kowalski (JDRF), and Dr. Jason Baker (Weill Cornell Medical Center)
diseases to tackle, largely because of their highly individualized nature. In a recent virtual panel discussion, members of our Women’s Reproductive Cancers Initiative discussed the complex genetics of these diseases, emerging cancer therapies and strategies, and how NYSCF is creating a biobank of tumor organoids (3D ‘avatars’ of patient tumors) that recapitulate how cancer behaves in a patient’s body.
Dive Into the Exciting World of Stem Cells with Our Virtual Learning and Events Can stem cells create personalized bone grafts? How does your body use electricity? How is NYSCF conducting disease research all the way up on the International Space Station? Find out the answers to all these questions — and much more — with NYSCF’s new comprehensive virtual program of events and learning opportunities for students, parents, teachers, scientists, and the public. These resources allow students of all ages and adults to explore exciting advancements in stem cell research and learn all about what is on the horizon for groundbreaking science. Especially for those with kids in quarantine, NYSCF – Robertson Stem Cell Investigator these resources can help Alumnus Dr. Paul Tesar’s daughter joins for keep antsy minds engaged. a Stem Cells 101 webinar
Learn more at nyscf.org/virtuallearning
NYSCF CONFERENCE
Keynote Speaker
Fireside Chat with
Carl H. June, MD
Tony Coles, MD, MPH
Plenar y Lecture
O C T O B E R 20 – 21, 2020 V I RT U A L C O N F E R E N C E Register now at nyscf.org/conference
University of Pennsylvania 5
Cerevel Therapeutics
Christine Mummery, PhD
NYSCFNEWS update Leiden University
Could Inflammation be Killing Neurons? NYSCF Scientists Create New Model of Neurodegeneration
W hile astrocytes – star-shaped support cells in the brain – are typically helpful for normal brain function, a study featured on the August cover of Neuron led by NYSCF Senior Research
Investigator Valentina Fossati, PhD, suggests that in neurodegenerative diseases like Alzheimer’s, Parkinson’s, or multiple sclerosis, these cells can ‘go rogue,’ attacking the neurons they usually keep healthy. Dr. Fossati’s team established a method for creating and identifying astrocytes from stem cells, based on a protein identifier called CD49f. They observed that in inflammatory, disease-like environments, these astrocytes can turn into neuron killers. This is the first demonstration in human cells that inflammation can lead to neurodegeneration. “This is a completely new mechanism that current treatments for neurodegenerative diseases haven’t targeted before,” she remarked. “I’m very optimistic that this, along with our new system for studying human astrocyte function, will create unique opportunities to bring effective therapies to patients.” NYSCF research on August cover of Neuron
Dr. Valentina Fossati | The NYSCF Research Institute
Why is Sleep Deprivation so Bad for You? The Answer May Lie in Your Gut
P rolonged sleep deprivation can lead to far worse than fatigue and irritability — in some cases observed in animals and reported anecdotally in humans, it’s fatal.
According to a study in Cell led by NYSCF – Robertson Neuroscience Investigator Dragana Rogulja, PhD, of Harvard University, sleep deprivation likely causes premature death due to a buildup of toxic molecules, known as reactive oxygen species, in the gut. Dr. Rogulja’s team found that eliminating this buildup in sleep-deprived fruit flies allowed them to live normal lifespans.
Thoughts to Text: Edward Chang’s Implant Turns Brain Signals to Written Words with Up to 97% Accuracy
W ith the power of artificial intelligence, a new brain implant developed by NYSCF – Robertson Neuroscience
Investigator Alumnus Edward Chang, MD, and colleagues at the University of California, San Francisco could help patients seamlessly translate brain activity into written text. In initial testing of the technology in stroke patients (outlined in Nature Neuroscience), the device could convert brain signals to text with up to 97% accuracy. Dr. Rogulja Image credit: Anna Olivella
“So many of us are chronically sleep deprived. Even if we know staying up late every night is bad, we still do it,” remarked Dr. Rogulja. “We believe we’ve identified a central issue that, when eliminated, allows for survival without sleep, at least in fruit flies.”
This new method could allow patients who have lost their ability to speak — due to paralysis, Parkinson’s, or other conditions — to communicate quickly and effectively. Right: Dr. Chang Image credit: Noah Berger, University of California San Francisco
Going Gray? Stress — and Depleted Stem Cells — Are the Likely Culprits
Isystem t turns out that stress can, in fact, turn hair gray. Stress activates your sympathetic nervous – the system that controls your ‘fight or flight’ response – depleting a group of stem cells responsible for giving hair its pigment and resulting in gray hair, finds a study in Nature led by NYSCF – Robertson Stem Cell Investigator Ya-Chieh Hsu, PhD, of Harvard University. “By understanding precisely how stress affects stem cells that regenerate pigment, we’ve laid the groundwork for understanding how stress affects other tissues and organs in the body,” said Dr. Hsu. “Understanding how our tissues change under stress is the first critical step toward eventual treatment that can halt or revert the detrimental impact of stress.” NYSCFNEWSupdate
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Dr. Hsu Image credit: Jon Chase, Har vard Staff Photographer
Remembering Jane Walentas, Christo, Jay Kriegel
T he New York Stem Cell Foundation lost three cherished friends in recent months: artist and philanthropist Jane Walentas (1944-2020), who with her husband David was a NYSCF Leadership Council member; the artist Christo (1935-2020), who with his wife Jeanne-Claude had been
a loyal supporter of NYSCF almost since its founding, and who was honored with the NYSCF Humanitarian Award in 2011; and civic leader Jay Kriegel (1940-2019), who received the NYSCF Leadership Award at the 2019 NYSCF Gala and Science Fair last October. Jane Walentas was an important champion of NYSCF’s work whose “grace, talents, and love inspired everyone privileged to know her,” reflected NYSCF CEO Susan L. Solomon. Jane was instrumental in revitalizing the neighborhood of DUMBO, including the iconic ‘Jane’s Carousel,’ and NYSCF is deeply grateful that she brought her enthusiasm for brightening lives to our mission for cures.
NYSCF mattered to Jay Kriegel, board member Paul Goldberger said when he presented the award, because NYSCF “ties together two of Jay’s great passions: New York City, where Michael Bloomberg, Jay Kriegel, Susan L. Solomon NYSCF has become a key part of the growing biotech sector, and moving medical research forward.” Kriegel’s career, he said, “was always in the service of a better, more just life and a better, more just city.”
Ja
ne a a When NYSCF honored Christo, his award was presented by Mayor Michael n d D a v i d Wa l e n t Bloomberg, who recalled the artist’s celebrated project, “The Gates,” more than seven thousand gates hung with saffron colored fabric that were arrayed along the walkways of Central Park for what Bloomberg called “sixteen magical days” in February 2005, when “Central Park, usually cold and gray in the middle of February, was really alive with energy and excitement, and it really made a difference to America and to New York City.” Christo and Jeanne-Claude, Bloomberg said, “were not unlike the scientists and researchers supported by The New York Stem Cell Foundation, who strive to make the world a better and brighter place than they found it… They are full of ideas, and they envision a society transformed by their work.”
Jeanne-Claude, Paul Goldberger, Christo
NYSCF mourns the loss of three of New York’s greatest citizens, and their passing affirms our commitment to bettering human health.
Inspiring the Next Generation of Scientists
Ipossible nspiring young researchers has always been mission critical for NYSCF. Through the NYSCF Academy for Science and Society, made with support from the Stavros Niarchos Foundation, the Laurie M. Tisch Illumination Fund, and others, we conduct lab
tours, career panels, and an annual Family Stem Cell Day for students of all ages across New York City, including many from underserved communities. This year, we hosted our first-ever summer immersion program for high school students to learn about stem cells and career paths in science. We also host more than 20 interns each summer who work with our laboratory, engineering, and programs teams to enable pioneering research. We were delighted to continue our intern program this summer with virtual components for offsite interns. NYSCF interns at work in the lab and at home | High school students join for the NYSCF Stem Cell Research Immersive Experience
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NYSCFNEWSupdate
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Future cures begin with your support today! 619 W 54th Street New York, NY 10019
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 54 TH STREET, NEW YORK, NY 10019
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NYSCFNEWSupdate The NYSCF
SH OW U P for SC I ENCE O C T O B E R 27, 2020
GALA Science Fair &
A virtual celebration | Tuesday | 7:00 - 8:00 PM
Honoring 2020 Stem Cell Heroes
BROOKE ELLISON, PHD | FRANK GEHRY | DAVID ROCKWELL Hosted by SANJAY GUPTA, MD Directed by SCOT T ELLIS W W W. N Y S C F. O R G / G A L A NYSCFNEWSupdate For more information, please contact: NYSCF Gala Office at (212) 249-6188 or gala@nyscf.org