NYSCF News Update | Vol 11, Issue 2 by The New York Stem Cell Foundation

NEWSupdate

N YS C F

VOLUME 11: ISSUE 2: 2018

The New York Stem Cell Foundation Research Institute

DE MOC R ATIZI NG DI S E A S E RE S E ARC H

“The route to precision medicine will be through advanced technology that is unaffordable for me at my institute.

The only way for me to gain access to such a critical resource is through collaboration with the NYSCF team and the NYSCF Gobal Stem Cell Array,” says NYSCF – Robertson Stem Cell Investigator and Associate Professor of Neuroscience, Genetics,

and Psychiatry at the Icahn School of Medicine at Mount Sinai Kristen Brennand, PhD. Bright ideas aren’t always enough: access to resources can make or break a scientific project. Not every lab has the same capabilities, because not every lab has access to the same assets. To accelerate the pace of research, we need to make scientific resources – including technology, biospecimens, and know-how – widely available. During this year’s NYSCF Conference keynote address (more on page 4), National Institutes of Health (NIH) Director Francis Collins, MD, PhD, underscored the power of community resources in science and spoke about his lab’s partnership with NYSCF to explore the genetic basis of diabetes using patient stem cells.

“ It has been wonderful

having a chance to work with a facility that has the skills and determination to be a strong partner.

We could not do this kind of thing in our own lab.” – NIH Director Dr. Francis Collins

“It has been wonderful having a chance to work with a facility that has the skills and determination to be a strong partner,” Dr. Collins remarked. “We could not do this kind of thing in our own lab.”

”

Dr. Collins – and numerous other disease researchers – were drawn to collaborate with NYSCF because of our Global Stem Cell ArrayTM. The Array’s unique ability to create large quantities of high-quality stem cells and make them into the cell types affected in disease has transformed the scale and nature of the questions researchers can explore. While there may only be one NYSCF Global Stem Cell Array, we are working to make it widely available to other labs, helping them run studies that yield more meaningful conclusions for translational disease research. Providing community access to enabling technology like the Array has always been NYSCF’s mandate.

“I think it is really important for all the small labs to be able to think big — which NYSCF encourages,” says Dr. Brennand. “We are the ones willing to do it, but we need the resources to go after it.” NYSCF is also making the Array and other technologies available to help labs study rare neurological diseases. Rare disease communities are often small, resource-poor, and lack effective disease models, but the Array makes stem cell models accessible and affordable for these researchers.

NYSCF Senior Principal Investigator Dr. Valentina Fossati (center) and NYSCF researchers

In science, if we want to go far, NYSCF Senior Principal Scientist we have to lift each other up. Dr. Laura Andres-Mar tin Understanding and treating disease is not always easy, but by combining forces and democratizing access to scientific resources, we will be better positioned to make lifesaving cures a reality for patients everywhere.

F E AT U R E D I N T H I S I S S U E CONFER ENCE

Translational research, precision medicine, and collaboration p.4

C O L L A B O R AT I O N S

N E W I N V E S T IG ATOR S

BONE ENGI NEER I NG

Teaming up with top scientists and institutions to accelerate research p.5

Meet the Class of 2018 NYSCF – Robertson Investigators p.6

NYSCF scientists develop method for creating personalized bone grafts p.7

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 Peggy Brim Bewkes Marilyn G. Breslow Karen E. Burke, MD, PhD Paul Goldberger George Lazarus, MD Richard J. Massey, PhD Stephen M. Ross Stephen M. Scherr Stuart Smith, DPhil 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 Paul M. Meister 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 Noelle & Dick Wolf

Dear Friends, NYSCF is now realizing the promise of translating research discoveries into therapies. At this year’s NYSCF Conference (more on page 4), we heard inspiring stories of how stem cell therapies for macular degeneration and Parkinson’s disease are entering clinical trials, as well as countless presentations on the groundbreaking strides stem cell research has enabled in drug discovery and precision medicine. Precision medicine has always been central to NYSCF’s mission, and human stem cells are what make it possible. We understand that people are different, and that stem cells will help us find treatments that address each patient’s unique needs. In his keynote address at our conference, National Institutes of Health Director Dr. Francis Collins described his lab’s collaboration with NYSCF (more on page 5), in which we are using the power of stem cells to investigate the genetic underpinnings of diabetes, helping inform the development of precision therapies. Thank you to all of our new supporters and to those who have stood with us from the early days. In the coming years, we will make lifesaving treatments and cures a reality for patients everywhere. We cannot do it without your help, and we are grateful for your ongoing generosity. Warmest wishes for a happy and healthy New Year,

Susan L. Solomon CEO and Co-Founder

2019 NYSCF Events

Mark your calendars!

March 5, 2019

April 3, 2019

April 11, 2019

May 8, 2019

May 11, 2019

Unraveling the Genetics of Alzheimer’s Disease in Patient Stem Cells

Improving Outcomes of Women’s Cancers

Uniting Patients and Researchers on the Road to Curing Type 1 Diabetes

Gene Editing and the Future of Human Health

2nd Annual NYSCF Family Stem Cell Day

Panel discussion in partnership with

K-12 scientific exploration and interaction with researchers in the lab

Panel discussion

Panel discussion in collaboration with

Support for events provided by

For more information, please visit nyscf.org/events

NYSCFNEWSupdate

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

At the 2018 NYSCF Gala and Science Fair, hosted by Bridget Moynahan, NYSCF proudly honored three Stem Cell Heroes – Karin Denoyer, MD, PhD, Carol Roaman, and Ian Schrager – for their steadfast commitment to NYSCF’s work. Top row, left to right: Tom Tofield, Charlotte Meringoff, and Stephen Meringoff; Susan L. Solomon, Dr. Karin Denoyer, Carol Roaman, and Ian Schrager; Stephen Scherr, Susan Scherr, and Evan Newmark; Bottom row: Yigal Ozeri and Dr. Richard Massey; Byron Wien and Anita Wien; Matt Dillon and Roberta Mastromichele; Paul Goldberger, Jane Walentas, and David Walentas

RUNNING with H E A R T

or Geoff McGrane, NYSCF’s new Director of Business Development, running marathons is more than a personal challenge. It’s a chance to enrich someone else’s life.

Geoff has completed ten marathons: five by himself, and five with his friend Gary. Gary Genovas has cerebral palsy, a disorder that affects movement, muscle tone, and posture, that has confined him to a wheelchair. For the past few years, Geoff has run his annual marathon while pushing Gary along with him.

“People often comment on how much these races must mean to Gary, but really I think he helps me more than I help him,” says Geoff. “I’ve never met someone so consistently positive, easygoing, and grateful as Gary. Even in the face of his struggles, he is always focused on the bright side of life.”

Running has always meant a lot to Geoff — he’s been an avid runner since age 9 and was inspired to pursue it by his father.

This year, Geoff and Gary completed the Philadelphia marathon with their best time yet: 2 hours and 52 minutes. Crossing the finish line this year was especially significant for Geoff, as the race landed on his late father’s birthday.

“My dad taught me to run with heart. After he passed away in 2014, I started looking for ways that I could run while dedicating myself to something meaningful.” Geoff met Gary through the New Jersey Special Olympics, where Gary was already involved in activities like bowling and was interested in joining Geoff for the 26-mile trek.

Geoff McGrane and Gar y Genovas run the Philadelphia Marathon

“Reaching the end of a race is always an emotional experience. Not only is the race physically taxing, but crossing the finish line always reminds me of just how blessed I am to have what I have. And this year, with it falling on my dad’s birthday, I was reminded of how much this experience means to me.” NYSCFNEWSupdate

T R A N S L AT I O N A L R E S E A RC H TA K E S C E N T E R S TAG E AT N Y S C F C O N F E R E N C E

“

We’ve already seen the incredible feats we can accomplish with stem cells, and now treatments are

starting to reach patients...

We’ll have an even better understanding of what stem cells truly can do and the mysteries we can solve with them. –Dr. Lorenz Studer

” NYSCF CEO Susan L. Solomon and NIH Director Dr. Francis Collins

“M

edicine shouldn’t be one size fits all,” National Institutes of Health (NIH) Director Francis Collins, MD, PhD, told the audience of the NYSCF Conference in October. “Think about it this way: we don’t give everyone with poor eyesight the same glasses prescription. Patients heal best when we consider their individual differences.” With stem cells, we can determine the genetic and molecular factors that influence a patient’s specific disease experience and use this knowledge to create targeted treatments (a concept called precision medicine). At the thirteenth annual NYSCF Conference — a two-day meeting on translational stem cell research featuring presentations and discussions from world-leading scientists held at The Rockefeller University — the strides being made in precision medicine, disease modeling, drug testing, and clinical intervention were striking. For example, Jeffrey Bluestone, PhD, Director of the Parker Institute for Cancer Immunotherapy and a professor at the University of California, San Francisco, described his use of immunotherapies to reverse autoimmune and organ transplant rejection, paving the way for safer transplantation surgeries. Yale’s Stephen Waxman, PhD, is using stem cells to model “pain in a dish” by focusing on a rare, debilitating disorder called erythromelalgia, or “Man Dr. Jeffrey Bluestone on Fire Syndrome,” in which even the slightest warmth causes an extreme burning sensation in a patient’s hands and feet.

studying their activity.” With “pain in a dish,” Dr. Waxman was able to identify a drug that corrected the neurons’ dysfunction and reduced pain in patients. And beyond treating Man on Fire Syndrome, Dr. Waxman’s study has broader implications for alleviation of chronic pain, serving as a model for how to develop and test potentially non-addictive opioids. Weill Cornell Medical College’s Shuibing Chen, PhD, a NYSCF – Robertson Stem Cell Investigator Alumna, is studying diabetes by turning patient stem cells into pancreatic beta cells. Her team looked at one of the two genes that are known to play a role in type 1, type 2, and neonatal diabetes, finding that beta cells carrying a mutation in that gene had trouble surviving. The team then tested a drug on the developing cells and found that it was able to rescue the beta cells from dying both in the dish and in an animal Dr. Shuibing Chen model — an exciting result that suggests it could one day be used as a therapy for genetic forms of diabetes. Lorenz Studer, MD, Director of Memorial Sloan Kettering’s Center for Stem Cell Biology and a founding member of NYSCF’s Medical Advisory Board, is beginning a clinical trial in Parkinson’s disease to create and transplant new dopamine neurons (the cells lost in the disease) made from stem cells, so as to replace the damaged cells and restore normal dopamine signaling in the brain.

“One day it would have sounded like science fiction to model pain using stem cells,” said Dr. Waxman. “But we have managed to do it by taking stem cells from patients with the disorder, turning them into sensory neurons — the cells that help us feel pain — and then

“We’ve already seen the incredible feats we can accomplish with stem cells, and now treatments are starting to reach patients,” said Dr. Studer. “I’m excited for the coming years because I think we’ll have an even better understanding of what stem cells truly can do and the mysteries we can solve with them.”

NYSCFNEWSupdate

N Y S C F C O L L A B O R AT I O N S

Teaming Up to Take On Disease

STE M C E L L S TA K E TO S PAC E

U N R AV E L I N G TH E G E N E TI C S O F D I A B E TE S

ells act differently when gravity isn’t around. In space, where gravity is removed as a variable, scientists can gain a clearer understanding of how cells migrate, behave, and contribute to different diseases. In a new partnership with the National Stem Cell Foundation (NSCF), Summit for Stem Cell Foundation, and Space Tango, NYSCF will be sending 3D mixtures of different brain cells made from stem cells (called “organoids”) to the International Space Station to study the underlying mechanisms of Parkinson’s disease and multiple sclerosis, in search of new therapeutic insights.

he genetic factors that drive type 2 diabetes are tricky to analyze, as they are complex and vary across individuals. In a collaboration with the lab of NIH Director Dr. Francis Collins, NYSCF scientists are using the Global Stem Cell Array to produce stem cells from patients with type 2 diabetes and then turn these cells into pancreatic beta cells (the cells affected in diabetes), allowing the team to study the role that various genetic mutations play in beta cell malfunction.

PATIENTS AND SCIENTISTS WORK TOGETHER TO FIGHT DIABETES

THE PATH TO RESTORING SIGHT

acular degeneration is a devastating disease that robs people of their sight due to cell death in the retina. In a collaboration with the National Eye Institute’s Ocular and Stem Cell Translational Research Unit at the NIH, NYSCF will create stem cells from patients with macular degeneration and transform these cells into retinal pigmented epithelial cells (the cells that die in the disease), which will be made available to the wider research community for further study and development of new treatments.

his October, NYSCF partnered with Lyfebulb, a patientempowerment platform that connects chronic disease patients, industry, and investors, to address challenges facing the chronic disease community. This collaboration kicks off with an event on type 1 diabetes hosted at the NYSCF Research Institute on April 11, 2019, in which NYSCF scientists will share their recent advancements and discuss how patients and researchers can work together on the road to personalized, effective therapies.

NYSCF CONFERENCE

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

S AV E T H E D AT E OCTOBER

Hans Clevers, MD, PhD Hubrecht Institute Utrecht University

22 -23, 2 0 1 9

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

NYSCFNEWSupdate

I N N O V AT O R C O M M U N I T Y U P D AT E S 2018 NYSCF – ROBERTSON INVESTIGATORS

CO L L A BO R ATI N G TO CO N S TRU C T A B E T TE R B R A I N

his October, NYSCF welcomed six of the most talented early career stem cell researchers and neuroscientists into the NYSCF Investigator Program. These awards provide critical seed funding – $1.5 million over five years – to outstanding scientists as they move beyond their postdoctoral training to establish their own, independent laboratories.

Dmitriy Aronov, PhD NYSCF – Robertson Neuroscience Investigator Columbia University – Dr. Aronov combines virtual reality and machine learning to study how chickadees remember where they have hidden food, giving insights into human learning and memory.

Elaine Y. Hsiao, PhD

Drs. Paul Tesar and Valentina Fossati

IRetreat n a collaboration started at the annual NYSCF Innovators between the NYSCF Research Institute’s Valentina

NYSCF – Robertson Neuroscience Investigator University of California, Los Angeles – Dr. Hsiao is studying how changes in the gut can be harnessed to prevent seizures and is working to apply this emerging concept to other areas of neurology.

Fossati, PhD, and NYSCF – Robertson Stem Cell Investigator Alumnus and Case Western Reserve University’s Paul Tesar, DPhil, scientists have developed a new procedure for generating miniature 3D human “brain organoids” from stem cells. The new technique, published in Nature Methods, creates the first organoids containing oligodendrocytes (brain cells that produce myelin, a substance that helps neurons send signals), offering a new way to study disorders like multiple sclerosis or spinal cord injury.

Vikram Khurana, MD, PhD NYSCF – Robertson Stem Cell Investigator Brigham and Women’s Hospital – Dr. Khurana is using patient stem cells to model and observe a protein that becomes toxic in Parkinson’s disease, with the goal of developing personalized therapies.

A N E W LU N G C E L L W ITH A K E Y RO L E I N C YS TI C F I B RO S I S

Carolyn (Lindy) McBride, PhD

NYSCF – Robertson Neuroscience Investigator Princeton University – Dr. McBride studies the recognition of human odor by mosquitos. This work will inform efforts to curb the spread of mosquito-borne disease, such as dengue, Zika, chikungunya, and yellow fever.

Vijay G. Sankaran, MD, PhD NYSCF – Robertson Stem Cell Investigator Boston Children’s Hospital – Dr. Sankaran uses human genetics to better understand blood production and how it goes awry in disease, informing the development of improved therapies for blood disorders.

Image Credit: Montoro et al./Nature 2018

C ystic fibrosis is a life-threatening genetic disease that causes harmful mucus buildup in the lungs. A new study in Nature

Kevin C. Wang, MD, PhD NYSCF – Robertson Stem Cell Investigator Stanford University School of Medicine – Dr. Wang studies the genetic and molecular basis of stem cell biology with the long-term goal of translating the understanding of these complex mechanisms to treat human diseases.

led by NYSCF – Robertson Stem Cell Investigator, Associate Professor at Harvard Medical School, and Massachusetts General Hospital physician Jay Rajagopal, MD, identified a previously unknown cell type that plays an important role in cystic fibrosis. Now that scientists know exactly which cells suffer the worst consequences of the disease, we can develop targeted treatments.

NYSCFNEWSupdate

N Y S C F TA L K S

NYSCF RESEARCHERS DEVELOP NEW WAY TO BUILD PERSONALIZED BONE

THE PROMISE AND PERILS OF GENE EDITING

O ver a million individuals per year will suffer from fractures due to bone diseases such as cancer, osteoporosis, or osteosarcoma. Standard grafts for such defects can often spark immune rejection, do not form connective tissue or vasculature needed for functional bone, and can be quickly outgrown by pediatric patients, resulting in frequent trips to the hospital to get the grafts replaced.

Segmental Additive Tissue Engineering (SATE) of bone grafts

One way to create personalized treatments for bone injuries is by engineering bone grafts using a patient’s own stem cells, but it is difficult to apply this to large-scale bone defects. New research published in Scientific Reports from scientists at the NYSCF Research Institute led by NYSCF – Ralph Lauren Senior Investigator Giuseppe Maria de Peppo, PhD, describes a new bioengineering technique called Segmental Additive Tissue Engineering (SATE) which allows researchers to combine segments of bone engineered from stem cells to create large-scale, personalized grafts that can seamlessly integrate into the body. “Having effective treatment options in place for personalized relief, no matter the severity of a patient’s condition, is of critical importance,” says Dr. de Peppo. “We are confident that our new technique will one day provide such relief to patients suffering from bone diseases such as osteoporosis or osteosarcoma and injury.”

Dr. Giuseppe Maria de Peppo

Dr. Jeffrey Kahn, Dr. Barr y Coller, and Susan L. Solomon

enetics have a major influence on how patients develop disease and respond to treatments. In a talk at the Stavros Niarchos Foundation Conference last June, NYSCF CEO Susan L. Solomon, JD, discussed how the revolutionary new technology of gene editing, when combined with stem cells, is helping us understand the genetic basis behind disease. Ms. Solomon also participated in a panel discussion with Jeffrey Kahn, PhD, MPH, the Andreas C. Dracopoulos Director of the Johns Hopkins University Berman Institute of Bioethics and Barry Coller, PhD, Vice President for Medical Affairs at The Rockefeller University, to confront ethical issues that are emerging as gene editing becomes more advanced.

SHEDDING LIGHT ON A MYSTERIOUS DISEASE AFFLICTING MILLIONS

Dr. Raeka Aiyar chairs a panel discussion at Stanford University

yalgic encephalomyelitis, also known as chronic fatigue syndrome (ME/CFS), is probably the most common disease you’ve never heard of. Estimated to affect as many as 2.5 million Americans, its main symptoms are exertion intolerance, cognitive and functional impairments, and consistently unrefreshing sleep. Yet no approved treatments exist: ME/ CFS is badly underfunded and often dismissed as psychosomatic. This fall, NYSCF’s Director of Scientific Outreach Raeka Aiyar, PhD moderated the annual Community Symposium on the Molecular Basis of ME/CFS at Stanford University. Hosted by renowned Stanford geneticist Ron Davis, PhD, whose son is afflicted by the disease, the symposium featured many remarkable findings about the immune and metabolic features of ME/CFS. Dr. Aiyar spoke about the promise of stem cell research for unraveling complex diseases and NYSCF’s relevant advances in similar neurological conditions. 7

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 ! 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

FACEBOOK.COM/NYSCF TWITTER.COM/NYSCF INSTAGRAM.COM/NYSCF VIMEO.COM/NYSCF

NYSCFNEWSupdate

N Y S C F N EWS U PDAT E NYSCFNEWSupdate NYSCF Scientists at 2018 NYSCF Gala and Science Fair