NYSCF News Update | Vol 10, Issue 1

NYSCF

The New York Stem Cell Foundation Research Institute

NEWSupdate VOLUME 10: ISSUE 1: 2017

N Y SCF OPE NS N E W ST E M C E L L HU B I N M A N H AT TA N “This facility is home to some of the most brilliant scientists and researchers in the field of stem cell research, and we are so fortunate to have it right here in New York,” -U.S. Senator Kirsten Gillibrand

NYSCF Laboratories

This April, NYSCF officially opened its new 40,000 square foot headquarters and Research Institute in midtown Manhattan. The new facility expands NYSCF’s capabilities and capacity to build resources for the entire field and allows scientists to conduct the most advanced stem cell research to develop new and better treatments and cures for patients in need.

Tony Coles, MD, Chairman and Chief Executive Officer, Yumanity Therapeutics; Howard Zemsky, President and Chief Executive Officer, Empire State Development; and James Patchett, President & CEO, New York City Economic Development Corporation.

“In just 12 years, this organization has grown from one office with a handful of employees to an enormous group of researchers around the world. The New York Stem Cell Foundation strengthens New York City as a global center of research and innovation.”

-Michael R. Bloomberg

Key NYSCF collaborators, supporters, and friends spoke at the opening ceremony and ribbon cutting, including Michael R. Bloomberg, Founder, Bloomberg LP and Bloomberg Philanthropies and three- term New York City Mayor; Stephen M. Ross, Chairman and Founder, Related Companies and NYSCF Board Member;

NYSCF Laboratories

Speakers and guests seated at the Opening Ceremony

The new NYSCF facility features a glass-enclosed laboratory that houses the NYSCF Global Stem Cell Array, the unique, NYSCFdeveloped automated robotic technology for creating stem cell lines, among other cell culture facilities. NYSCF will also have a highly specialized GMP laboratory facility for manufacturing cells for therapies. Additional laboratories include molecular biology, imaging, cell sorting, and electrophysiology suites, and extensive bench space.

Contact us at: info@nyscf.org 212.787.4111 www.nyscf.org

UNITING TO ACCELER ATE CUR ES 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

Dear Friends, This spring, we moved into our new global headquarters and laboratories, fulfilling a long-time dream. Our transformational facility is already allowing us to expand our research so that we can have a greater impact on patients everywhere. Our new state-of-the-art laboratories, including a future GMP facility to make cells for therapies, cement our role as a leader in the field, and position us to host and collaborate with other leading scientists, hospitals, and research organizations on the cutting-edge of science that will get us to cures.

LEADERSHIP COUNCIL Katherine Bristor & William Priest David A. Carmel Russell L. Carson Chuck Close Alan M. Cohen Shirley Cook Fiona & Stanley Druckenmiller Jodie & John Eastman Frank Gehry Lawrence E. Golub & Karen Finerman April Gornik Marlene Hess Tania Higgins Dorothy Lichtenstein Sandra Lloyd Paul M. Meister Stephen Meringoff David Mitnick Nancy & Fred Poses Carol Roaman Julian Robertson Eric A. Rose, MD Andy Russel Alice Shure Barbara Stovall Smith Noelle & Dick Wolf JUNIOR LEADERSHIP COUNCIL (EXECUTIVE BOARD) Sabrina Bertucci Daniel Brancusi Francesco Clark Maggie Close Carolyna De Laurentiis Briggs Fraser Greg Geronemus Alex Goldberger, Co-Chair Daniyal Hussain Brandon Law Mark McCauley Charlotte Meringoff Craig J. Moskowitz, MD Max Mullen Neil O’Donnell, Esq. Thomas J. Pacchia Katherine S. Pier, MD Melissa Rothberg Richard W. Rundle, Co-Chair Ben Sherman Lindsay & Edward Timlin

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Our new home also marks a new chapter for NYSCF, for New York as a biotechnology hub, and for stem cell research around the world. Just this May, NYSCF scientists reported on an exciting new protocol to make microglia, the immune cells from the brain, from stem cells.

Not only is this protocol transformational for research today, it is also optimized for use in high-throughput experiments, such as those enabled by the NYSCF Global Stem Cell Array, which will greatly accelerate our progress towards new treatments and cures.

We have already broken in our new meeting spaces, hosting a ribbon cutting ceremony, multiple educational tours for local students and faculty, city and biotech industry meetings, and our annual new Fellow’s reception. We are so excited to see what NYSCF’s new home will further enable our scientists and collaborators to achieve.

In addition, I am thrilled to welcome world-renowned neurologist and Parkinson’s disease research and clinician Dr. Melissa Nirenberg as NYSCF’s Chief Medical Officer. Joining NYSCF from NYU Langone Medical Center, Dr. Nirenberg will serve a key role as we continue to advance therapies to patients in need.

We wouldn’t be here today without the steadfast and generous support of our community and friends. NYSCF’s supporters have truly changed stem cell science, and we are deeply grateful for all you have done. Only with your continued help can we bring the promise of stem cell research to patients in need. Thank you for joining us on our journey.

With warm regards,

Susan L. Solomon CEO and Co-Founder

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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

N Y SCF DE V E LOPS N E W TOOL

TO ST U DY BR A I N DISE A SE

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icroglia, the immune cells of the brain, are increasingly implicated in neurological disorders, including Alzheimer’s disease, Parkinson’s disease, and multiple sclerosis. Because of their mysterious involvement in many different devastating diseases, scientists have long desired to study them in the lab, but they have proven extremely difficult to obtain – until now.

NYSCF Research Institute scientists have developed a robust method for deriving microglia cells from human induced pluripotent stem cells. This new protocol, published in Stem Cell Reports, now enables scientists around the world to generate this critical cell type from individual patients and improve our understanding of the role of microglia in neurological malfunction.

Microglia

(Photo: Panos Douvaras)

system. Now, studies on healthy and diseased microglia derived from human stem cells will complement ongoing research in mouse models and shed light on the role microglia play in both disease and in health.

In addition, this microglia protocol is optimized for use in highthroughput experiments, such as drug screening and toxicity testing, and has the benefit of allowing such experiments to be carried out on multiple patient samples. NYSCF scientist Dr. Panos Douvaras was the first author on the paper, and NYSCF scientists Dr. Valentina Fossati and Dr. Scott Noggle were co-senior authors.

NYSCF strives to not only conduct groundbreaking research, but also to develop tools, models, and methods that improve and accelerate the work of the entire stem cell field. This new microglia protocol achieves both goals, and will enable new and exciting research around the world.

Prior to this breakthrough, microglia were rarely studied due to the difficulty involved in obtaining them from the human nervous

N Y SCF HOSTS “ST E M C E L L S I N T H E CIT Y ”

T he NYSCF Junior Leadership Council hosted likeminded young professionals at their annual “Stem Cells in the City” event at the Bowery Hotel in Manhattan. JLC members, friends, and guests mingled with NYSCF researchers and learned about NYSCF’s latest breakthroughs.

Top row from left to right: Daniel Paull, PhD, addresses the crowd; Alex Goldberger, Jonathan Koenig Jr., Danielle Haber, Mark McCauley; Bottom row from left to right: Allyson Papageorge, Charlotte Meringoff; Kellindo Parker, Ane Roseborough; Sean DesMarteau

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2017 N Y SCF I N NOVATOR S R ET R E AT

Who Matter”, leaders of President Obama’s Brain Initiative, TED talk speakers, and Presidential Early Career Award winners.

The 2017 annual NYSCF Innovators Retreat convened a group of nearly seventy scientists and researchers from the NYSCF Innovator

Each NYSCF Investigator and graduating Fellow presented his or her latest, and often unpublished, research and provided the community with a special insider’s look into the future of stem cell research and neuroscience. The scientific presentations, covering a remarkably broad scientific scope, ranged from the neuroscience of decision-making to diabetes to liver regeneration and cardiovascular disease.

Community for a weeklong scientific meeting in Montauk, New York.

The retreat brings together current and future leaders in stem cell research and neuroscience who represent some of the most brilliant scientific minds in the world, including Time magazine-named “People

NYSCF Innovator Community in Montauk

T R A N SL AT ION A L A DVA NC E S

Research highlights from the NYSCF Innovator Community: breakthroughs moving stem cell and neuroscience research to the clinic.

MINI LI V ER S FROM STEM CELL S

SA FER DEEP BR A IN IM AGING

N YSCF – Robertson Stem Cell Investigator Dr. Takanori Takebe

D eep brain stimulation via implanted electrodes has proven to

This research could lead to both better drug testing for liver disorders as well as future cell and even whole organ replacement therapies using tissues derived from stem cells.

Published in Cell, the scientists created a way to send electrical signals deep within the brain via electrodes placed on the scalp, as opposed to implanting them deep within the brain. This breakthrough has broad implications, enabling easier and safer delivery of deep brain stimulation for treatment and opening up new avenues of research.

of Cincinnati Children’s Hospital Medical Center led an international team of researchers in bioengineering human liver tissues. The research, published in Nature, uncovered previously unknown networks of genetic-molecular crosstalk that control the developmental processes leading to liver tissue, greatly advancing efforts to generate healthy and usable human liver tissue from human pluripotent stem cells.

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be an effective therapy for Parkinson’s disease, epilepsy, and depression among other disorders; however, it remains invasive and risky. NYSCF – Robertson Neuroscience Investigator Alumnus Dr. Ed Boyden and his team at MIT Media Lab have developed a revolutionary new method of noninvasive deep brain stimulation.

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T H E R O C K E F E L L E R U N I V E R S I T Y, N E W YO R K C I T Y

T R A N S L AT I O N A L S T E M C E L L RESEARCH CONFERENCE

DR . M E L IS SA N IR E N BERG JOI NS N YSCF AS CHIEF MEDICAL OFFICER

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elissa Nirenberg, MD, PhD is joining NYSCF in a newly created role of Chief Medical Officer. Dr. Nirenberg comes to NYSCF from the NYU School of Medicine, where she has been an Associate Professor of Neurology and specialist in movement disorders.

Dr. Nirenberg will bring her medical background to NYSCF and work with NYSCF scientists and collaborators to translate their research into therapies and facilitate upcoming clinical trials. “After almost 20 years of patient care, I recognize the challenges faced by people with Parkinson’s disease and related disorders. I share the frustration of my patients and their loved ones that we are only able to treat the symptoms, but have been powerless to change the course of the underlying disease. At NYSCF, I will finally have the opportunity to do more than just treat symptoms - I will be helping to facilitate cures,” said Dr. Nirenberg.

Melissa Nirenberg, MD, PhD

Dr. Nirenberg pursued her undergraduate education at Yale University and subsequently received a PhD in neuroscience and an MD from Weill Cornell. She completed a neurology residency at the University of California, San Francisco, and a movement disorders fellowship at Columbia University.

BUILDING BR A IN ORG A NOIDS

PROGRESS IN PARKINSON’S

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N YSCF – Robertson Stem Cell Investigator Alumna Dr. Paola

he traditional cell replacement therapy model involves replacing diseased cells in a patient with healthy cells created in the lab. NYSCF – Robertson Stem Cell Investigator Alumnus and NYSCF – Robertson Stem Cell Prize Recipient Dr. Marius Wernig and his team at Stanford University explored a new method of potential cell replacement therapy for Parkinson’s disease in their latest Nature Biotechnology paper. The researchers directly converted astrocytes, a type of brain cell, into the dopaminergic neurons lost in Parkinson’s.

Arlotta and a team of researchers at Harvard University created complex, 3D human brain models called “organoids” using induced pluripotent stem cells. These models are capable of growing and maturing to an unprecedented level. Typical organoids are developed and tested in a matter of weeks. The organoids, reported in Nature, were cultured and matured for nine months or longer and developed key traits such as spontaneously active neural networks, dendritic spines and light-sensitive cells.

These human brain models enable a next-level platform for neuropsychiatric disease modeling and drug discovery.

This research represents a potential breakthrough in cell replacement therapies in which cell replacement may be achieved through gene therapy instead of whole cell replacement.

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EDUCATING THE NEXT GENER ATION

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he NYSCF Science and Technology Education Program, or NYSCF STEP, provides educational opportunities for young students and introduces them to the exciting world of science and stem cell research. Through seminars at local schools, internship opportunities at the NYSCF Research Institute, and special laboratory tours for students, the Program promotes scientific literacy, educates young people on the promise of stem cell research, and encourages students to study science and technology.

W ELCOMING OUR 2017 SUMMER INTER NS

PA RTNER ING W ITH PR EP FOR PR EP

N YSCF partners with the Prep for Prep organization to place promising college students as interns in the NYSCF Research Institute

laboratories during their summer breaks. The NYSCF summer internship program offers these exceptional students the opportunity of a lifetime to work in cutting-edge research facilities with some of the best stem cell scientists in the world.

NYSCF University 2017 Summer Interns

T he NYSCF Summer Internship Program offers high-achieving undergraduate students the opportunity to work in New York City at

Prep for Prep interns par ticipating in discussion held at NYSCF

A delegation of Prep for Prep summer interns from across New York visited NYSCF’s headquarters and laboratories, touring the stateof-the-art facilities and utilizing the commons meeting area for an information and career panel, which included NYSCF scientists and staff. NYSCF STEP and other outreach programs strive to share the promise of stem cell research with the scientists of tomorrow and offer a glimpse into potential future career paths in STEM fields.

the NYSCF Research Institute. For ten weeks, the students get a rare glimpse into the fast-paced world of stem cell research, insight into life at an entrepreneurial nonprofit research institute, exposure to career opportunities in science, and connections to valuable professional and peer networks. Our 2017 interns hail from top universities all around the country and entertain a wide variety of interests both extramurally and at the lab bench.

SEO SCHOL A RS EX PLOR E FUTUR E CA R EERS IN STEM CELL SCIENCE

N YSCF hosted a group of college-age students who are part of the SEO Scholars, a free eight-year academic program that gets underserved

public high school students to and through college with a 90% college graduation rate. The students met several NYSCF scientists over an informal lunch, heard a Stem Cell 101 lecture, took a tour of the new NYSCF Research Institute Laboratories, and finally, participated in a career panel where they were able to ask the scientists questions about their background and life as a scientist.

SEO College Scholars and NYSCF Scientists

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2017 N Y SCF – DRUCK E N M I L L ER POST DOC TOR A L F E L LOW S

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he New York Stem Cell Foundation Fellowship Program supports the most talented young postdoctoral scientists in the pursuit of innovative and groundbreaking stem cell research. The 2017 NYSCF – Druckenmiller Postdoctoral Fellows join the NYSCF – Robertson Investigators and NYSCF Research Institute scientists to form the global NYSCF Innovator Community of over 140 scientists who are leading their generation in cutting-edge research.

Raphael Lis, PhD Weill Cornell Medical College

Sangbum Park, PhD Yale University

Maria Maryanovich, PhD Albert Einstein College of Medicine

Julia TCW, PhD Icahn School of Medicine at Mount Sinai

Dr. Lis aims to find a cure for sickle cell anemia, a disorder that causes red blood cells to become misshapen and break down. His research could provide an approach to generate an unlimited supply of healthy red blood cells to replace the deformed cells caused by sickle cell mutation.

Dr. Park’s research focuses on wound repair with a goal to understand how stem cells and immune cells interact during the complex wound repair interaction and process. This research will help scientists better understand chronic wound repair and tissue regeneration.

Dr. TCW uses brain cells derived from induced pluripotent stem cells to study a mutant form of APOE, a major genetic risk factor for Alzheimer’s disease. Her research aims to understand the mutant APOE’s contribution to the neurodegeneration seen in the disease.

Dr. Maryanovich studies the effects of aging on the environment in which blood stem cells are produced, specifically the role nerves play in the longevity of stem cells that support blood formation.

Her approach will help advance therapies for blood diseases associated with aging.

W E LCOM I NG T H E N Y SCF – DRUCK E N M I L L ER F E L LOW S

N YSCF friends and staff gathered at NYSCF’s new headquarters in Manhattan to announce and celebrate the new 2017 class of NYSCF – Druckenmiller Postdoctoral Fellows.

Top row from left to right: Stanley Druckenmiller, Fiona Druckenmiller, Susan L. Solomon; George Lazarus, MD, Gail Geronemus, Roy Geronemus, MD, Susan L. Solomon, Shelly Lazarus Bottom row from left to right: Ken Langone, Jeff Wallerstein; Sjoukje van der Stegen, PhD; Clifford Ross, Cynthia Whitehead, Paul Goldberger

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THE NYSCF WEBSITE G E T S A N E W LO O K

619 W 54th Street, New York, NY 10019

V I S I T N Y S C F. O R G

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From left to right: Howard Zemsky, Dr. Roy Geronemus, Michael Bloomberg, Susan L. Solomon, Stephen M. Ross, Dr. Tony Coles, and James Patchett (Photo: Bloomberg Philanthropies)