Introduction
Seven years ago, I was inspired by the enormous potential of stem cells to cure diabetes and many other devastating diseases. I saw that the promise of stem cell research stood in stark contrast to the negative federal political climate and the general lack of government support for stem cell research.
The New York Stem Cell Foundation (NYSCF) was established in 2005 to provide private support for human embryonic stem cell research. Today it supports 40 full-time researchers on its own staff, as well as many more researchers worldwide, and has become a full research institute. The organization's cofounder and chief executive officer, Susan L. Solomon, shares the NYSCF's successes and plans for the future.
Several leaders in the field, including Dr. Harold Varmus, Dr. Doug Melton, and Dr. Paul Nurse, were enthusiastic about this new area. With their encouragement and that of several others, I cofounded the New York Stem Cell Foundation (NYSCF) in 2005 to use private philanthropy to stimulate stem cell research.
NYSCF opened its privately funded stem cell laboratory in New York City in March 2006 and initially facilitated a collaboration between Columbia University and the Harvard Stem Cell Institute. Researchers at Harvard had the tools to attempt somatic cell nuclear transfer, but legal restrictions in Massachusetts made it difficult to perform their research. Moreover, critical types of experiments could not be performed in federally funded laboratories at the universities, so researchers at these two institutions turned to the NYSCF laboratories to carry out this groundbreaking research.
NYSCF became known as a laboratory where scientists could conduct cutting-edge human embryonic stem cell research free of federal restrictions. The NYSCF laboratory has become a full research institute and is 12 times as large as when it opened in 2006, employing more than 40 full-time researchers.
The mission of NYSCF is to accelerate cures for major diseases through collaborative stem cell research. We think that stem cells are the most promising medical opportunity of our time and see NYSCF as a critical catalyst for the best scientists in the world to perform the cutting-edge research to find cures and treatments for those we love. The urgency of our mission is particularly personal to me, as one of my sons has type 1 diabetes and I lost my mother and father to cancer and heart disease.
Another aspect of NYSCF's core mission is to expand the field of stem cell research itself. In addition to direct support of research, our Innovators Program provides fellowships for postdoctoral researchers and for early career investigators. Each fall we convene what has become the preeminent annual translational stem cell research conference. We also conduct a wide range of public programs.
Because we are primarily privately supported, our ability to fund research and conduct our other programs is unaffected by the frustrating and debilitating cycles of off-again, on-again, off-again NIH support. However, most of our collaborators are not as lucky, and there is no satisfaction for us in continuing to work when so many of our colleagues and collaborators cannot.
Significant Impact
A particularly important breakthrough (a discovery that Time magazine named the number one medical research breakthrough of 2011) was performed by NYSCF scientist Dr. Dieter Egli and his team, all working in our laboratory. Stem cell scientists had previously discovered that making patient-specific stem cells from human cells was very different from performing somatic cell nuclear transfer with other animal cells. The NYSCF team under Dr. Egli, who focuses on diabetes, managed to create and derive the first-ever human embryonic stem cell line that contained a patient's genome. Although further work is needed to perfect the technique, this science could eventually enable researchers to make human embryonic stem cells that contain only the patient's genes. Such stem cell lines would be ideal for stem cell therapies.
In 2008, Dr. Kevin Eggan, a chief scientific advisor to NYSCF and principal faculty member of the Harvard Stem Cell Institute, produced human stem cell lines from the cells of patients afflicted with a version of amyotrophic lateral sclerosis (ALS). NYSCF supported Dr. Eggan's work, which was published in Science and marked the first time scientists had replicated in a laboratory the specific human cells affected by a disease. Dr. Eggan's work went on to be cited as the number one scientific breakthrough of 2008 by the journal Science and also by Time magazine.
Dr. Eggan's previous work deriving patient-specific stem cell lines allowed scientists to see that the death of nerve cells in ALS is a sort of “assisted suicide” in which supporting cells in the spinal cord play an active, deleterious role. He is currently building on this knowledge to identify future drugs to stop this process.
Although the derivation of patient-specific cell lines is now common, at the time Dr. Eggan's goal was thought of as impossible to achieve, and as a result, he encountered difficulties obtaining grants from traditional funding sources for his research. As I see it, his work marked a turning point for stem cell science because drugs are only as good as the material they are tested on. Work like Dr. Eggan's provides a window that allows scientists to recreate a disease in a dish and study its processes, moving us one step closer to cures.
Former NYSCF-Druckenmiller Fellow and current NYSCF-Robertson Investigator Dr. Gabsang Lee has made significant process in identifying therapeutics for a rare disease called familial dysautonomia. Dr. Lee has screened compounds on induced pluripotent stem cells derived from patients with this devastating disease. Dr. Lee was formerly a postdoctoral fellow in the laboratory of Dr. Lorenz Studer at Memorial Sloan-Kettering Cancer Center and is now an assistant professor at Johns Hopkins University.
NYSCF also supports the work of Dr. Derrick Rossi, a NYSCF-Robertson Investigator and an assistant professor at Harvard Medical School. Dr. Rossi was named on Time magazine's “People Who Mattered” list in 2010 and on its “Time 100” list of most influential people in 2011.
With the guidance of our scientific advisors, including Zach Hall—NYSCF board member, former president of the California Institute for Regenerative Medicine, and director of the National Institute of Neurological Disorders and Stroke—we have supported many of the top scientists in the New York area and around the world, researchers who we hope will one day make a significant impact on improving the lives of patients. The NYSCF also supports the work of Dr. Lee Rubin at Harvard University, who is developing therapeutics for Lou Gehrig's disease.
To date, NYSCF's focus has primarily been on preclinical work, concentrating research on translating basic science into “de-risked” trials that will be picked up by large companies and turned into treatments. However, we did award Dr. Pete Coffey the inaugural NYSCF-Robertson Stem Cell Prize in 2011. Dr. Coffey is beginning a clinical trial later this year aimed at treating age-related macular degeneration using human embryonic stem cells.
We are also performing work in diabetes and other areas that we hope will be moving toward a clinical stage shortly. In addition, we have collaborations in place with the two largest public cord blood banks in the U.S. (the National Cord Blood Program at the New York Blood Center and the Carolinas Cord Blood Bank at Duke University). With these collaborations we aim to help treat patients with many forms of cancer.
A “Bridge to Cures”
A critical gap exists between the research conducted at academic institutions and the work done by biotech and pharmaceutical companies. In the scientific community this gap is often referred to as “the valley of death.”
Although academic institutions do critical work in uncovering the cause of disease, their work often does not translate into drug therapies. This is due, in part, to the fact that academic institutions work with small collections of cell lines from a narrow group of patients—far smaller than the scale needed for effective drug screening. Conversely, biotech and pharmaceutical companies mainly work on drugs that can already be seen to have large market potential, and because they are public companies, they are risk-averse. These companies have taken a wait-and-see approach to stem cells.
To solve this problem, we need the ability to de-risk potential drugs so pharmaceutical and biotech companies will move forward with them. We have done this with our Drug Discovery Center and Personalized Medicine Bank. The Personalized Medicine Bank will allow researchers to scale their discoveries and translate their experiments into medicine, find the genetic causes of disease, replicate diseases in a dish using the human cells that are actually getting sick, and anticipate how people from genetically diverse backgrounds will respond to different drugs before clinical trials—in essence a clinical trial in a dish.
We like to think of NYSCF as being a bridge to cures between academic institutions and biotech companies. NYSCF's focus is on translational research, as this area is not traditionally well funded by conventional means such as NIH funding. Although basic research is critically important to the progression of translational research, the translation of basic biomedical research into safe and effective clinical applications remains a slow, expensive, and failure-prone endeavor. NYSCF hopes to bridge this valley of death by engaging in and funding research that will catalyze the generation of innovative methods and technologies, which will in turn enhance the development of diagnostics and therapeutics to get to cures.
All the hope and hype associated with stem cell research has generated enthusiasm among patients about stem cell treatments for a variety of diseases. However, few stem cell treatments are close to being ready for therapeutic use.
Many stem cell “clinics” have exploited this hope, and there has been a worldwide proliferation of clinics that offer regenerative medicine using stem cells. These “medical tourism” treatments—ranging from injecting a patient's own stem cells back into his or her own body to treat Parkinson's disease to “stem cell facials” to make a person look younger—are at best a waste of money and at worst extremely dangerous.
These clinics have real potential to damage the legitimacy of the entire stem cell field. Patients need to know that science moves slowly and that the only safe way to currently receive a stem cell treatment (other than a bone marrow transplant or other commonly used procedure) is to participate in one of the ongoing clinical trials.
Although clinical trials often proceed slowly, stem cell research remains the best chance we have to discover cures for several major diseases. There are very promising clinical trials currently in place and new breakthroughs every day. Stem cells are also already being used as a tool for drug discovery and yielding tangible results. This is a very exciting time to be part of the stem cell field.
Fund-Raising and Legislation
One of the obstacles that research programs often encounter is that they cannot make their work accessible to members of the nonscientific public who might be funding their projects. At NYSCF we have put an emphasis on conveying complex scientific concepts in ways that are accessible to lay people. We have made it a priority to support our scientists with training and opportunities to help them communicate their science to people who have very little understanding of their work. This not only helps fund-raising but is critical for their career development.
We also are involved in education efforts on both the federal and state levels. Studies have shown that around 72% of Americans support stem cell research, and yet a small but vocal minority is limiting research that could save millions of lives.
This is part of the reason that our public education and outreach programs are so important. By ensuring that the public has straightforward, accurate information about stem cells, we hope to allow more people to understand the need for this critical work.
Meet Susan L. Solomon
Susan L. Solomon is the cofounder and chief executive officer of the New York Stem Cell Foundation, which was established in 2005 to provide private support for human embryonic stem cell research. An active health care advocate, Ms. Solomon is on the board of directors of the Juvenile Diabetes Research Foundation and is a member of both New Yorkers for the Advancement of Medical Research and the New York Council of the Joslin Diabetes Center. An attorney by training, she practiced law with Debevoise and Plimpton before beginning a 20-year career in news media, entertainment, and investment banking.
Ms. Solomon was founding chief executive officer (CEO) of Sothebys.com and has served as chair and CEO of Lancit Media Productions and as president of Sony Worldwide Networks, a pioneering Internet broadcasting provider. A member of the board of directors and the executive committee of Regional Plan Association, Ms. Solomon has also has worked as an executive at MacAndrews and Forbes Holdings and MMG Patricof and Co., and she was the founding executive of Solomon Partners.
Ms. Solomon has three sons and is married to Paul Goldberger, the Pulitzer Prize-winning writer on architecture.