What We've Done
HSCI – A New Approach to Understanding and Treating Disease
In 2004, Harvard University announced the establishment of the Harvard Stem Cell Institute, a collaboration of world-class scientists from the university and its affiliated hospitals dedicated to advancing stem cell science and building a foundation for the future of regenerative medicine. HSCI is a networked organization that was formed to bridge the gap between academic research and real-life medical applications, to truly go from “bench to bedside.”
The Harvard community is one of the largest concentrations of biomedical researchers in the world. HSCI has united over 70 stem cell experts among its principal faculty alone, making it many times larger than any other major stem cell research institution in the world. This depth and breadth of expertise in a geographically constrained area enables people to collaborate closely. This has allowed HSCI as a group to achieve results such as creating more ESC and iPS cell lines and publishing more papers in the leading journals than any other entity. Even more unique among such institutions, HSCI includes affiliates from professional schools in public health, business, law, and public policy.
The primary goal of the HSCI is to move basic biological discoveries from the lab bench to new treatments, whether stem cell-based or stem cell-derived. To achieve these goals the HSCI bridges the University and affiliated hospitals to engage in activities that include:
- the funding of research, facilities and programs across institutional boundaries
- in-depth exploration of the multiple social, political, religious, ethical, and financial issues that surround stem cell research
- active engagement in public policy issues and public education.
Developed a portfolio approach to scientific research funding
- seed grants – about 10 early stage projects per year
- core facilities – from shared equipment centers to a drug screening and a reprogramming center
- disease programs – multi-lab programs tackling key questions in selected disease areas
- Developed courses and classes for scientists and students
- Developed a bioethics course with Case Western for science administrators
- Consulted legislators, the NIH and other deliberative bodies on stem cell related policy
- Developed an annual summer internship program for undergraduates
- Conducted a series of public forums
- Piloted a training course for high school science teachers
- Developed an online book of stem cell biology
Our seed grants – designed to take on risky and innovative projects – can have far-reaching results. One example, highlighted below, is a seed grant HSCI funded several years ago to create a disease specific cell line and cell type. Over the subsequent two years the project attracted further funding, resulted in several major publications, and was awarded TIME magazine’s top medical breakthrough award in 2008.
Selected results from our Disease Programs include:
- Blood – First creation of multiple disease-specific cell lines
- Cancer - New program analyzing fresh tissue tumors to determine cancer stem cells
- Cardiovascular - Proof of cardiac progenitor cells and integration of cardiac progenitor cells onto new thin film material to create beating heart tissue
- Diabetes – Landmark proof of the ability to “directly differentiate” adult pancreatic cells into beta cells in vivo
- Kidney – Identification of the regenerative cells in the kidney; new mouse kidney injury model
- Nervous System - Definition of “molecular recipe” for upper motor neurons; proof of impact of glial cells on motor neurons in ALS; derivation of disease specific cell lines for ALS and subsequent differentiation into motor neurons; identification of chemical compounds to address spinal muscular atrophy.
New core facilities continue to emerge from advances in the basic science. For example, several HSCI scientists published some of the leading papers in the reprogramming field over the past two years. As a result of that work, HSCI established a new iPS Core facility. Having this core facility coupled with access to unique patient populations in the hospitals will allow the entire HSCI community to develop specific disease cell lines more efficiently and effectively than they could on their own. In conjunction with our Therapeutic Screening Center, these in vitro disease models will allow us to develop better drugs more efficiently as a result of being able to study the effect of drugs in more accurate, human cell-based models.
In the space of five years, HSCI has developed a new business model of a virtual R&D network that creates a new way for the university and the hospitals to interact to accelerate research in one of the most exciting fields of science today. Our model of cross-lab teams at all levels - from junior to senior faculty - is proving itself out in the scientific results we are achieving which in turn attract further support. As one scientist said, “The work could not have progressed this far without HSCI funding and inspiration.” These teams also benefit from the guidance of the Executive Committee who can pool their skills and insights to make decisions quickly and allocate capital and resources accordingly.
Extending this work into the educational area, Harvard’s new cross-school Department of Stem Cell and Regenerative Biology is developing an undergraduate curriculum that will launch this fall as well as a new concentration. Linking faculty from the medical school, the hospitals and the university to provide students an integrated multi-disciplinary perspective is not only exciting in itself, but represents the future of how science will be taught. As such this will provide a new path for education and career development that takes students from the fundamentals of biology to its applications in curing diseases.