NYSCF News Update: Vol 16, Issue 1

PURSUING THE NEXT FRONTIER

OF MEDICINE

Pages 2 & 3

WHEN SCIENCE IS PERSONAL TOWARD A CANCER VACCINE

CAN STEM CELLS CURE BLINDNESS? CLIMB FOR CURES

The New York Stem Cell Foundation Research Institute

Experts discuss NYSCF’s AMD therapy

Five patient advocates summit Denali for NYSCF

A patient-scientist tackles diabetes

Turning cancer cells against each other

Pursuing the Next Frontier of Medicine

The story of NYSCF is often told as beginning at our Founding CEO Susan L. Solomon’s kitchen table, but as NYSCF Board Member and Susan’s husband Paul Goldberger shared at the March dedication of NYSCF’s laboratories to Susan’s legacy, that is not entirely correct.

“The real beginning wasn’t at the kitchen table – it was within Susan herself: in the passion and determination that she brought to everything,” he remarked. “And nowhere more than in her idea that there had to be a way to bring energy and money and young talent together to enable the most advanced science – political and bureaucratic obstacles be damned.”

Shortly before Susan passed away last fall, the NYSCF Board of Directors committed to naming the NYSCF laboratories for Susan, in honor of her transformational impact. To celebrate the formal dedication of ‘The Susan L. Solomon Center for Precision Medicine,’ NYSCF hosted a discussion between NYSCF Interim CEO Derrick Rossi, PhD, and former NIH Director and Scientific Advisor to the President Francis Collins, MD, PhD, on the exciting future of medicine, and where NYSCF’s pioneering stem cell research will play a critical role.

Dr. Rossi highlighted emerging efforts leveraging the mRNA technology he helped pioneer for COVID-19 vaccines to tackle other challenging diseases.

“Cancer is driven by mutation,” added Dr. Rossi. “Knowing that, and now being able to come in with this beautiful little transient genetic molecule called mRNA, we can perhaps impact the biology of cancer. So there are many, many clinical trials going on for mRNA in oncology, and I’m very excited by that.”

Dr. Collins shared that much of what he’s looking forward to will come from NYSCF itself. For example, NYSCF’s automated systems for making high-quality cells also open the door for more effective drug testing, since scientists can see how drugs affect the exact cells impacted by disease.

“No place else in the world [besides NYSCF] can do that,” noted Dr. Collins. “That investment, a testament to Susan’s vision, has made this a place that labs like mine want to come to.”

“Science right now is on this exponential curve of discovery. And don’t think that there’s ever been anything like this before. In biomedicine, there has not. We’re able to figure out how things work, how disease happens, and what to do about it at a scale like never before.”

“That is unprecedented. And NYSCF is a place that’s well positioned, really uniquely positioned, to be part of that. And I think if Susan were here today, she’d agree, and she’d want to push this to the next level.”

Learn more about the Center and ways to support NYSCF at nyscf.org/solomon-center

BOARD OF DIRECTORS

Roy Geronemus, MD, Chairman

Derrick Rossi, PhD, Interim CEO

Susan L. Solomon, JD*, Founding 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

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

Stephen Meringo

David Mitnick

Nancy & Fred Poses

Carol Roaman

Julian Robertson*

David Rockwell

Cli ord Ross

Kara Ross

Andy Russell

Susan & Stephen Scherr

Ian Schrager

Barbara Stovall Smith

Martha Stewart

David & Jane* Walentas

John Whitehead*

Dick Wolf

*In Memoriam

Dear friends,

I write to you with renewed excitement for the future of biomedical research and treatments, and for NYSCF’s integral role in bringing them to patients. For those who joined my conversation in January with Francis Collins – one of the most accomplished scientists of our time – I hope the evening filled you with as much hope and inspiration as it did me. The future of medicine is bright, and stem cells will be an integral part of the new wave of precision therapies. As Francis noted (see previous page), as NYSCF has invested so much in critical groundwork, we are poised to lead the way in the next frontier of medicine.

At NYSCF, we have always focused on building foundational technologies and investing in big ideas that will scale up research and accelerate the path to the clinic. With our facility for creating clinical-grade cells now in operation, we are closer than ever to bringing life-changing therapies to patients. We are so proud of our community’s recent breakthroughs towards cancer vaccines (p. 9), fighting rare disease (p. 5), and effective ALS therapies (p. 8).

None of what we do is possible without our dedicated community of supporters. At our annual Gala in October, we will honor four remarkable individuals as our Stem Cell Heroes: Kay Unger, Dr. Feng Zhang, and Janet and Jerry Zucker. I hope you will join us as we celebrate impactful progress, honor those in our lives affected by disease, and look ahead to a bright future of cures.

For a striking example of how far our community goes to support our work, look no further than the five outstanding advocates who reached the summit of Denali – the highest peak in North America – this spring. We are so proud of this outstanding accomplishment and for their ongoing commitment to lifesaving research (p. 11).

Thank you for continuing this journey with us, and wishing you all a safe and happy summer.

Derrick Rossi, PhD Interim Chief Executive Officer

From Patient to Scientist: Josi Wesely Takes on Type 1 Diabetes

NYSCF Principal Scientist in Functional Genomics

Josephine (Josi) Wesely, PhD, was diagnosed with type 1 diabetes when she was 10, and she was probably the only kid who showed up to her doctor’s appointments with scientific papers in hand.

“I looked into publications and went to my doctor’s office with a bunch of questions. I would say, ‘I read in this paper that they treated this animal with XYZ, and it seemed to work.’ I think he was overwhelmed,” she laughed.

She became a scientist herself, fueled with determination to find better solutions for diabetes patients.

“At NYSCF, we are using stem cells to make pancreatic organoids that can produce insulin, meaning they can react to the presence of glucose just like a healthy pancreas,” Josi explained.

Dr. Wesely uses gene editing techniques to modify these pancreatic cells in a way that could help them evade the immune attack in type 1 diabetes, all with the goal of creating a cell replacement therapy.

“It would be a dream not to have to worry about insulin costs or management. It could be revolutionary.”

Learn more about Josi’s journey and research at nyscf.org/josi

Uncovering New Treatment Options for a Rare Disease

At NYSCF, we are working to find the first therapies for INAD, a rare neurological disease affecting children that is often referred to as “pediatric Parkinson’s.” As part of a recent international collaborative study published in eLife with the patient-driven INADcure foundation and Hugo Bellen, DVM, PhD, of Baylor College of Medicine, NYSCF scientists made stem cell models from INAD patients that were used to identify new hallmarks of the disease, as well as four potential therapies that could reverse these hallmarks. The team also tested a potential gene therapy in mice that delayed neurodegeneration and prolonged lifespan.

“This study is an excellent demonstration of the power of stem cell models to help us understand and treat rare diseases. NYSCF is proud to bring our technology to bear on this important, unmet need, and we are hopeful that the therapeutic leads from this study will pay off in our continued work with INADCure and Dr. Bellen’s lab.” - Rick

Monsma,

PhD | NYSCF SVP of Scientific Operations

Leena Panwala (INADCure founder) and her daughter, Ariya (INAD patient)

Music, Mentorship, and Medicine

This May, we gathered once again for the NYSCF Innovator Retreat, in the 13th installment of an annual meeting where the NYSCF – Robertson Investigators, NYSCF – Druckenmiller Fellows, and NYSCF Research Institute scientists come together to share four days of pioneering research, camaraderie, and collaboration.

This year’s retreat featured a special song from NYSCF – Robertson Investigators Lindy McBride, PhD, and Vik Khurana, MD, PhD, about their time in the program, set to the tune of ‘Yesterday’ by The Beatles. Graduating NYSCF – Robertson Stem Cell Investigator Kevin Wang, MD, PhD, also shared a poem about his time in the program which perhaps best sums up the spirit of this community:

NYSCF, you are my friend;

I thank you for the journey’s end;

And now I look ahead with hope;

To use the knowledge that you gave;

You’ve also been a source of joy;

You’ve shared with me your wisdom’s voice;

You’ve taught me how to face the unknown;

You’ve shown me how to grow and learn;

You’ve made me who I am

And for the journey’s span

To see what lies in store

To learn and to explore

A partner and a guide

And stood by me with pride

With courage and with grace

And nd my rightful place.

NYSCF Innovators in Montauk, New York

Meet the 2023 Class of NYSCF

– Druckenmiller Fellows

Meet our 2023 Summer Interns!

We have 19 incredible interns this summer working across our laboratory, engineering, and programs teams. Hear from a few of them about why they are excited to be part of the NYSCF family and what they’re looking forward to in the future.

The NYSCF Fellowship Program supports postdoctoral researchers in their pursuit of innovative and groundbreaking stem cell science and is the largest dedicated stem cell fellowship in the world.

Kentaro Iwasawa, MD, PhD

Cincinnati Children's Hospital Medical Center

Advancing technology to grow liver tissue in a dish for patients who need transplants

Kaylee Wells, PhD

Harvard University

Stimulating production of myelin to treat diseases like Alzheimer’s and multiple sclerosis

Anupama Hemalatha, PhD

Yale University

Harnessing metabolism to develop better skin cancer therapies

Jiajie Xu, PhD

Massachusetts General Hospital

Pinpointing specialized airway cells to develop better treatments for lung disease

Brian Joseph, PhD

Columbia University Irving Medical Center

Maturing brain cells made from stem cells as a window into neurodegenerative diseases and aging

“This is my first time working in a lab, and I feel like I’m getting used to the greatest lab ever!”

“At NYSCF, I saw engineers working alongside scientists, and I don’t think I’ve seen that anywhere else.”

“Working on my project has really made me realize how much is possible within stem cell research and that what I’m working on is part of something far bigger.”

Students Go Behind the Scenes of Science

We were thrilled to host students from high schools around NYC this spring to get the inside scoop on how research is done as well as career paths in STEM!

The Holy Grail of ALS Treatment

ALS is a disease that manifests differently in each patient, with some cases resulting from genetic predispositions, and others sporadically. Because the causes vary so widely, it is difficult to find medications that will work for most or all patients. In two new Cell and Cell Stem Cell publications, scientists led by NYSCF – Robertson Stem Cell Investigator Alumnus Justin Ichida, PhD, of the University of Southern California have discovered new opportunities for treating ALS, by calming overexcited neurons and helping them clear toxic waste that could provide improvement for large groups of patients.

“Our discoveries bring us closer to achieving our big picture goal: finding treatments that can be broadly effective for all patients who suffer from ALS.” - Justin

Getting Under the Skin with Valentina Greco

NYSCF – Robertson Stem Cell Investigator

Alumna Valentina Greco, PhD, is on a mission to understand the skin. This spring, her lab at Yale University published four new studies shedding light into how the skin develops during early life, how it shields our bodies from external threats, and even how it neutralizes early stages of cancer. Dr. Greco is also a staunch advocate for equity in STEM and collaborative science.

Ichida, PhD

“Every day I commit to nourishing my excitement for science, and my own desire to do impactful science, but it all starts with scientists. We live in a world where we still claim objectivity is what makes science reliable, but in my opinion it is reliable because it’s a team approach with multiple lenses that together capture the complexity of nature.” - Valentina

Greco, PhD

What Doesn’t Kill You Makes You Stronger (And How To Make Sure It Does)

Some people are better at bouncing back from a stressful situation than others. Why? And can those of us who more easily succumb to stress teach ourselves to be more resilient? Resilience can be learned, and even reinforced, finds a new study in Nature by NYSCF – Robertson Neuroscience Investigator Annegret Falkner, PhD, of Princeton University in collaboration with NYSCF –Robertson Neuroscience Investigator Alumna Ilana Witten, PhD, also of Princeton.

“I’m very interested in the question of whether we can teach resilience. We need to think about ways to help the people who seem to be more susceptible to cope with the stresses of the world.”

- Annegret Falkner, PhD

Can Tumor Cells Be Used To Make A Cancer Vaccine?

Immunotherapies have revolutionized cancer therapies by harnessing the immune system to recognize and attack cancer cells. Immune cells called T cells can be trained to recognize cancer antigens (proteins specific to cancer cells), but figuring out which antigens to target can require considerable guesswork.

Scientists have now figured out how to turn cancer cells into ‘antigen presenting cells’ – or APCs, which can then train the T cells to recognize cancer antigens and kill cancer cells more effectively. The study, published in Cancer Discovery, was led by NYSCF – Robertson Stem Cell Investigator Alumnus Ravi Majeti, MD, PhD, of Stanford Medicine.

“In the future we might be able to take out tumor cells, transform them…and give them back to patients as a therapeutic cancer vaccine.”

- Ravi Majeti, MD, PhD

Education and Outreach

Read highlights and watch the full discussions at nyscf.org/events

How Could Stem Cells Cure Blindness?

People over the age of 75 have nearly a 30% risk of developing age-related macular degeneration (AMD), which now affects 11 million in the United States.

NYSCF is developing a cell therapy to restore vision in AMD patients using their own stem cells by leveraging technology pioneered by scientists at the National Eye Institute (NEI) in partnership with surgeons at Columbia University. The experts behind this investigational therapy recently gathered to give an update on its path to the clinic.

Lessons for Advancing Equity in STEM

In honor of Women’s History Month, NYSCF hosted a fireside conversation between equity advocates Maike Sander, MD (Max-Delbrück Center, Berlin, Germany) and Raeka Aiyar, PhD (NYSCF) who discussed the considerable progress towards gender equity in science in recent years, as well as ways to tackle the challenges ahead in realizing the dream of a truly equitable field, where impact is not limited by identity. Dr. Sander shared her international experience in scientific leadership and intentions to advance culture change.

“We have an opportunity to save a lot of people from being severely blind and keep them functioning, which is our goal. We want to keep them working, reading, driving, seeing their loved ones.” - Stanley

Chang, MD

“I want people to remember that culture change is up to each of us: it has to be lived at every level of an organization. Maybe that means joining a DEI committee to advocate for policies that can really make a change, or advocating for reward structures that perhaps redefine what achievement means.” - Maike

Sander, MD

Climbing for Cures: Five Patient Advocates Summit Tallest Mountain in North America

Climbing a mountain is more similar to scientific research than you might think. You invest in a major challenge and there are a lot of unknowns and uncertainties. High highs and low lows. You could make it most of the way to the top but hit a storm and run out of time (food) before reaching the summit. If you don’t summit, you still learn a lot along the way. If you do, it can change everything.

This spring, five patient advocates (Mark McCauley, Dieter Egli, PhD, James Teague, Blake Yaralian, and Knud Nairz, PhD) scaled Denali (the highest mountain peak in North America at 20,308 ft) to raise awareness and funding for stem cell research. They were inspired to take on this challenge in part as a way to honor the memory of NYSCF Founding CEO Susan L. Solomon, along with others close to them who have struggled with, or were lost to, disease. Their team name? ‘Mitosis are Cold’ (that’s a biology pun for you enthusiasts!)

The climb took just over three weeks, and the mountaineers were thrown many curveballs from Mother Nature – think: grueling winds, snowstorms, poor visibility. But the team persevered, bound together by a passion for their cause, and a belief in one another. Their first attempt at a summit had to be abandoned due to weather. They set out on a second attempt. It was their last chance. This time, everything came together.

The Climb for Cures raised $150,000, far exceeding the team's original goal of $100,000. NYSCF is extremely grateful for the support of these outstanding community members, and for their commitment to carrying on Susan’s vision of cures for the major diseases of our time.

Learn more about the cause at nyscf.org/climb

“There were definitely tears shed. I was very excited for that moment – to finally reach our goal. Denali is the most difficult of the seven continental peaks, and summiting it is my way of honoring the legacy of Susan and contributing to NYSCF.”

- Mark McCauley

Climbers at the summit

Mark preparing for the ascent

Ice walls keep out the wind at high camp

Dieter on top of the world

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 nyscf.org/donate or by mail to:

The New York Stem Cell Foundation

619 West 54th Street, 3rd Fl, 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 generations to come.

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

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