NYSCF News Update | Vol 11, Issue 1

NEWSupdate

N YS C F

VOLUME 11: ISSUE 1: 2018

The New York Stem Cell Foundation Research Institute

NYSC F HOSTS I NAUGU R AL FAM I LY STE M C E LL DAY

Thanks to the support of the Stavros Niarchos Foundation, NYSCF opened its doors to students in the New York City area for its first-ever Family Stem Cell Day this spring.

Family Stem Cell Day is an event that allows students in grades K-12 to learn about research, tour the NYSCF laboratories, and participate in science-themed activities at each of the day’s 13 stations.

In the NYSCF laboratories, students were excited to have an upclose look at the research process, observing firsthand how NYSCF robots are making stem cells, exploring the role of electricity in the human body (did you know you can power a light bulb with just static electricity from your own body?) and learning about NYSCF’s plans to send cells into outer space.

Many of these stations allowed students to peer into microscopes and observe bioengineered human bone, neurons, and beating heart cells.

“It looks like a starry night,” remarked one student gazing upon a dish of cells.

“ It looks

Children build bristlebots from toothbrushes, pipe cleaners, batteries, and miniature motors

like a starry night... ”

NYSCF Researcher Juliana Hsu, PhD, shows a student how to use a microscope

Downstairs in the NYSCF Commons, which houses our lecture hall and café, students exercised their creativity by assembling biological structures out of pipe cleaners and coloring real cell images captured by NYSCF researchers. Students also made slime, dressed up like scientists for a photo booth, and watched a demonstration on the magic of liquid nitrogen.

NYSCF Family Stem Cell Day provided students with the unique opportunity to speak one-on-one with researchers and ask questions about stem cells and what it’s like to be a scientist.

NYSCF believes that the future of science depends on participation from younger generations. We are dedicated to getting students excited about STEM fields. Our staff had a great time meeting so many enthusiastic future scientists, and we look forward to the next Family Stem Cell Day!

A group of students obser ve bone grown in NYSCF’s laboratories

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

N E W C L A S S OF FE L L OWS

I N NOVATOR S R ET R E AT

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

Dedication of the Stavros Niarchos Foundation Gallery p.3

Meet the Class of 2018 NYSCF – Druckenmiller Fellows p.4

NYSCF hosts annual Innovators Retreat p.5

Partnering to produce a therapy for Parkinson’s disease p.5

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 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 Andy Russell Barbara Stovall Smith Noelle & Dick Wolf

JUNIOR LEADERSHIP COUNCIL (EXECUTIVE BOARD) Sabrina Bertucci Daniel Brancusi Francesco Clark Maggie Close Alex Goldberger, Co-Chair Daniyal Hussain Brandon Law Mark McCauley, Co-Chair Charlotte Meringoff Craig J. Moskowitz, MD Max Mullen Thomas J. Pacchia Melissa Rothberg Richard W. Rundle Ben Sherman

NYSCFNEWSupdate

Dear Friends, Thanks to your support, we are closer than ever to providing personalized therapies for patients everywhere. NYSCF Innovators at the NYSCF Research Institute in Manhattan and at 70 additional laboratories around the world are working to develop new treatments, many of which are now in or approaching clinical trials.

At our annual NYSCF Innovators Retreat (which you can read more about in this newsletter), I was thrilled to see the extraordinary progress that these scientists are making to better understand and treat the major diseases of our time. They are taking the bold steps needed to bring therapies out of the lab and into the clinic.

It was clear to me from watching this group interact and learn from each other that research works best when it is collaborative—when people from different backgrounds with different ideas join forces to approach a problem from a new angle. The global NYSCF Innovator community fosters a unique and dynamic platform for scientists to engage with each other and to build lasting research collaborations.

Our Innovators are paving the way for a brighter future by pursuing big ideas and creating groundbreaking technologies. One NYSCF Innovator who exemplifies this pioneering mindset is Dr. Feng Zhang, who continues to revolutionize the field of medical research through his work with CRISPR gene editing. CRISPR has completely changed the way we study disease, and we are excited to see what it will bring for the future and how it will change the world.

Together, we will improve the lives of people around the globe through stem cell research.

Thank you for your continued support of NYSCF.

Susan L. Solomon CEO and Co-Founder

S AV E T H E DAT E

T U E S D AY

O C TO B E R

16 , 2018

J A Z Z AT L I N C O L N C E N T E R ’ S F R E D E R I C K P. R O S E H A L L

H O N O R I N G S T E M C E L L H E RO E S

Karin Hehenberger, MD, PhD Founder and CEO of Lyfebulb Carol Roaman Philanthropist Ian Schrager Chairman and CEO of Ian Schrager Company 2

BU I LDI NG A B R AI N : U S I NG STE M C E LL S TO U N D E R S TA N D N E U R O D E G E N E R AT I O N

N eurodegenerative diseases affect millions of people worldwide, yet much remains unknown about what triggers their onset, how

they progress, and how to effectively treat them. To address these issues, NYSCF and Tufts University are embarking on a unique and exciting collaboration that will help find cures for neurological disease.

Tufts’ expertise in biomedical engineering and NYSCF’s expertise in stem cell biology and neurodegeneration provides an ideal partnership to tackle the diseases of the brain. The collaboration will initially focus on Parkinson’s disease (PD) with the intent to later expand into Alzheimer’s disease, autism spectrum disorder, and schizophrenia.

The researchers are interested in exploring two factors that may contribute to PD: genetic mutations and the relationship between the brain, intestinal, and immune systems. To investigate these factors, the researchers will be using human cells to create 3D models of the brain.

First, NYSCF scientists will generate brain cells from stem cells.

Then, the cells will be placed onto a scaffold engineered by Tufts.

The cells will integrate with the scaffold, creating a structure that mimics the architecture, function, and composition of the human brain. With this in place, the team can manipulate the model to observe how genetic mutations affect brain function and how cells from different systems affect each other’s behavior. This will help researchers identify opportunities for early diagnosis and develop new therapies.

Parkinson’s disease neurons imaged by NYSCF Researcher Ana Sevilla, PhD

NYSC F HOST S RI B BON CUT TI NG E VE NT TO OPE N T H E S TAV R O S N I A R C H O S F O U N D AT I O N G A L L E R Y

O n April 4th, NYSCF dedicated the Stavros Niarchos Foundation Gallery, which allows visitors to see NYSCF’s research in action in our laboratories, helping to bridge the gap between scientists and the public.

Leadership from both organizations came together for the ribbon cutting ceremony to officially open the gallery. The event featured remarks from NYSCF CEO Susan L. Solomon and Stavros Niarchos Foundation Co-President Andreas Dracopoulos.

Susan L. Solomon, NYSCF CEO; Andreas Dracopoulos, Stavros Niarchos Foundation Co-President; Dr. Roy Geronemous, NYSCF Board Chairman; and Roula Siklas, Stavros Niarchos Foundation Program Officer prepare to officially open the Stavros Niarchos Foundation Gallery with a ribbon cutting

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The Stavros Niarchos Foundation supports organizations that aim to achieve a broad, lasting, and positive impact on society. They are a vital supporter of the NYSCF Research Institute and NYSCF Outreach and Education programs.

NYSCFNEWSupdate

NYSC F WE LCOM E S 2018 NYSC F – DRUC K E N M I LLE R FE LLOWS A N D H O N O R S D R . RUTH L E H M A N N

T his June, NYSCF hosted a reception to welcome the 2018 class of NYSCF – Druckenmiller Fellows to the NYSCF Innovator

community and to honor Ruth Lehmann, PhD, for her work as founding co-chair of NYSCF’s Fellowship review committee.

Collectively, the current NYSCF – Druckenmiller Fellows and alumni have published over 1000 papers throughout their careers.

56% of them now hold leadership positions in science, and they hail from 40 institutions in 12 countries around the world.

NYSCF honored Dr. Lehmann, Chair of the Department of Cell Biology and Director of the Skirball Institute of Biomolecular Medicine at NYU Langone Medical Center, for her critical role in helping NYSCF launch its Fellowship program, encouraging young scientists to pursue stem cell research, and for her impact on the research community.

Past and current NYSCF – Druckenmiller fellows unite: Drs. Panos Douvaras, Bjarki Johannesson, Samuel Taylor, Tae Wan Kim, Alexandros Strikoudis, and Benjamin Lin, Susan L. Solomon, JD, Drs. Raphael Lis, Ana G. Freire, Brett Shook, Ly-sha Ee, Hongda Li, Kosuke Funato, Sangbum Park, Ying Liu, and Maria Mar yanovich

Dr. Ruth Lehmann and Susan L. Solomon

M E E T TH E 2 018 N YSC F – D RU C K E N M I L L E R F E L LOW S

T he NYSCF – Druckenmiller Fellowship was created to support young postdoctoral researchers in their pursuit of innovative stem cell research and is the largest dedicated stem cell fellowship program in the world. Fellows receive financial support over two years and are also welcomed into NYSCF’s greater Innovator Community.

Ana G. Freire, PhD

Ly-Sha Ee, PhD NYU School of Medicine Studies the role of key genes in stem cell reprogramming

Memorial Sloan Kettering Cancer Center Models pediatric brain cancer to understand its development

Samuel Taylor, PhD

Alexandros Strikoudis, PhD

Albert Einstein College of Medicine

Columbia University Models pulmonary fibrosis to identify genetic risk factors

NYSCFNEWSupdate

Kosuke Funato, PhD

Hackensack University Medical Center Visualizes the interaction between blood stem cells and their environment

Targets blood cancer stem cells to halt relapse in patients

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“A unique opportunity to interact with researchers doing incredible work.”

NYSCF Innovators at the Annual Retreat

NYSCF HOSTS ANNUAL I N N O VAT O R S R E T R E AT

make the retreat a highly anticipated gathering, with many scientists expressing that they look forward to it all year.

C ontrary to its name, one of the central themes of this year’s NYSCF Innovators Retreat was moving forward.

The 2018 retreat featured presentations on groundbreaking research in human organoids (3D aggregates of cells that model organ development and structure), neurodegenerative disease, cancer, mental illness, biological engineering, social cognition, genome editing, and more.

Whether it is moving forward by bringing a therapy to clinical trials, developing a new research technique, or pursuing a novel and promising question—NYSCF Innovators are taking the bold steps needed to make a lasting impact on disease research and patient care.

Dr. Pete Coffey, the inaugural NYSCF – Robertson Stem Cell Prize Recipient and Professor of Neuroscience at the University of California, Santa Barbara and Professor of Visual Psychophysics at University College London, delivered a keynote address on what it takes to bring a cell therapy from the lab to clinical trials. Dr. Coffey and his team recently developed a stem cell-based therapy for macular degeneration that restored vision in patients who lost their sight to the disease. These patients went from being unable to read or see objects in front of them to, in some cases, getting their driver’s license back.

Once a year, the NYSCF Innovators depart from their respective labs and convene for a week of presentations and discussions on cutting-edge science. This event gives researchers a chance to share their work, get feedback from their peers, and form collaborations that will open the door for new projects. These unique opportunities

N Y S C F – R O B E R T S O N I N V E S T I G AT O R PA R T N E R S W ITH N OVO N O R D I S K TO PRO D U C E TH E R A PY F O R PA R K I N S O N ’ S D I S E A S E

M alin Parmar, PhD, has spent the past ten years meticulously examining brain cells. She does this to better understand the basis of Parkinson’s disease and develop methods for turning stem cells into dopamine

neurons—the cells lost in the disease. Now, she’s getting ready to take these cells from the lab to the clinic.

This spring, Dr. Parmar (Professor of Developmental and Regenerative Neurobiology at Lund University, Sweden, and a NYSCF – Robertson Investigator) kicked off a collaboration with Novo Nordisk, one of the world’s largest pharmaceutical companies, to develop a large-scale cell therapy for Parkinson’s disease.

This new partnership will allow her to scale up the research and development of her stem cell therapy, accelerating its progress to clinical trials.

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Malin Parmar, PhD

NYSCFNEWSupdate

COMMUNITY BUILDING

One of the cornerstones of advancement is the sharing of success stories and new ideas. By hosting and participating in meetings of stem cell leaders, NYSCF is helping to ensure that innovative research is well supported and broadly disseminated.

N YS C F H O S T S M E E T I N G O F H E A LT H R E S E A RC H A L L I A N C E

T his January, NYSCF hosted a meeting of the Health Research Alliance (HRA),

a group of nonprofit research funders. The organizations in the HRA all share one goal: to make sure funded research will result in tangible benefits for patients.

Attendees engage in a Q&A session following a presentation

The meeting featured a keynote address from Bruce R. Conway, PhD, Director of the Robertson Therapeutic Development Fund at The Rockefeller University as well as a panel discussion on how best business practices can be used to accelerate translational research.

NYSCF Researcher Valentina Fossati, PhD, speaks with guests

NYSCF PERSONALIZED MEDICINE SERIES

DR . ED BOYDEN EXPL AIN S HIS GROUNDBRE AKING NEUROTECHNOLOGIES

F or examples of out-of-the-box thinking, look no further than the work of NYSCF – Robertson Investigator Alumnus and Y. Eva Tan Professor in Neurotechnology at MIT, Ed Boyden, PhD. Dr. Boyden recently visited NYSCF headquarters to give an update on his research in neuroscience and biotechnology.

Dr. Boyden spoke about two advancements he helped pioneer: optogenetics and expansion microscopy. In optogenetics, the activity of brain cells is stimulated or repressed by exposing them to certain wavelengths of light. In expansion microscopy, researchers inflate samples of brain tissue to a size that is easier to study, by weaving a special polymer through the sample and adding water, expanding the polymer, and pulling apart the tissue’s molecules.

Ed Boyden, PhD

N YS C F H O S T S S T E M C E L L S I N T H E C I T Y

P recision medicine, biotechnology, organoids, drug discovery: these are all words one might hear in a conversation about stem cell research. This May, NYSCF’s Junior Leadership Council and guests gathered at NYSCF headquarters for Stem Cells in the City, an event which featured a panel discussion that unpacked what terms like these mean and why they will matter for our loved ones struggling with disease.

The panel, moderated by NYSCF Junior Leadership Council Board Member Rich Rundle, featured NYSCF Vice President of Business Development Elizabeth Schwarzbach, PhD, and Vice President of Automation Systems and Stem Cell Biology Daniel Paull, PhD. The NYSCF Junior Leadership Council and guests listen to a panel discussion moderated by JLC Board Member Rich Rundle and featuring NYSCF’s Dr. Elizabeth Schwarzbach and Dr. Daniel Paull

NYSCFNEWSupdate

“Since stem cells allow us to test drugs on the actual human cell types affected by a disease, we can use them to better understand why some drugs work well in one person but not in another,” explained Dr. Schwarzbach.

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I N T E R N AT I O N A L S T E M C E L L R E S E A R C H CON FE RE NC E S HOWCAS E S PROG RE S S

T he annual International Society for Stem Cell Research (ISSCR) conference – the world’s largest stem cell-focused meeting – took place in Melbourne, Australia this June. The meeting highlighted

recent advances that stem cells have enabled in disease research and therapeutics. The NYSCF community and staff were represented among over 3500 participants.

NYSCF – Robertson Stem Cell Investigator alumna Shuibing Chen, PhD, received the ISSCR Dr. Susan Lim Award for Outstanding Young Investigator.

“Science is never one person,” she said, thanking her collaborators, including NYSCF. NYSCF Innovators have won this award in 5 of the last 6 years.

The NYSCF-sponsored “Road to the Clinic” session featured therapies for which clinical trials are imminent or underway. NYSCF – Robertson Stem Cell Prize Recipient Pete Coffey, PhD, described his cellular therapy that restored eyesight in patients with age-related macular degeneration.

“For stem cell therapies, the question has always been: Are we there yet?” said the session chair. “I think we are.”

NYSCF’s Dr. Elizabeth Schwarzbach, ISSCR CEO Nancy Witty, and NYSCF’s Dr. Raeka Aiyar

NYSCF CONFERENCE O C TO B E R

23 - 24 2 0 1 8

The Rockefeller University, New York City K E Y N OT E S P E A K E R

Francis Collins, MD, PhD

Director, National Institutes of Health

13th A N N U A L C O N F E R E N C E Register at www.nyscf.org/conference

NYSCF AND JOHNS HOPKINS UNIVERSIT Y’S BERMAN INSTITUTE T E A M U P T O C O M B AT S T E M C E L L T O U R I S M

T his April, NYSCF hosted a panel discussion with the Johns Hopkins Berman Institute of Bioethics to focus on a growing issue: stem cell tourism.

“Stem cell tourism” refers to the rising industry of unregulated, unproven stem cell treatments at a high cost to the patient and a great profit to the clinic. These treatments often leave patients with little relief and can even be harmful.

The panelists discussed what must be done to prevent fraudulent clinics from administering dangerous therapies and how to tell whether a stem cell treatment is legitimate.

Susan L. Solomon, NYSCF CEO; David Carmel, Vice President of Strategic Alliances at Atara According to panelist David Carmel, Vice President of Biotherapeutics; Francesco Clark, Founder and CEO of Clark’s Botanicals; and Dr. Jeffrey Kahn, Strategic Alliances at Atara Biotherapeutics and a founding the Andreas C. Dracopoulos Director of the Berman Institute and a professor at Johns Hopkins’ Bloomberg School of Public Health participate in a panel discussion on stem cell tourism and emeritus board member of NYSCF, patients should ask themselves the question: What am I getting here? “Stem cells aren’t all the same, so you need to know what type of cells are involved. And it also has to be in a controlled setting that is approved by an independent review board,” he says.

There are many steps that patients can take to help them make informed decisions about stem cell treatments, and participants were encouraged to seek out resources and consult experts before signing up for potentially risky therapies. One resource from the International Society for Stem Cell Research is a website called A Closer Look At Stem Cells (www.closerlookatstemcells.org), which offers advice on how to evaluate stem cell treatments.

The panel also took questions from the audience, speaking about the process of moving treatments out of animal trials and into the clinic as well as what steps established institutions should take to address stem cell tourism.

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NYSCFNEWSupdate

FUTURE CURES B E G I N W I T H YO U R S U P P O R T T O D AY !

Please make a gift to help NYSCF advance stem cell research toward the clinic.

You can donate online at www.nyscf.org/donate or mail a check to:

619 W 54th Street New York, NY 10019

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 A student par ticipates in a demonstration of “Liquid Nitrogen Magic” at NYSCF Family Stem Cell Day