The Harvard Stem Cell Institute — a concept, not a place.

HSCI We are stem cell science.

We are a collaborative. We are an idea. We are a unique organization.

We are dedicated to developing treatments and cures for the diseases and conditions that are overwhelming us as a people, both medically and financially.

HSCI is a we, not an it, and we are biolo-gists, physicians, and biomedical engineers who focus on stem cell science because stem cells are the cellular foundation of the entire human system. Every cell, in every organ of the body, arises from a cluster of cells, and in order to fully understand basic human development, or what goes awry in the developmental process to trigger dis-ease, we need to understand how a stem

Harvard Stem Cell Institute

■■ 255 Faculty■■ 20 Institutions■■ 1 Promising Scientific Field

Collaborative science speeding the development of safer, more effective treatments for disease

cell gives rise to a specialized cell. For example, what signals tell a stem cell to develop into a pancreatic islet cell, which in a healthy individual produces the insulin needed to regulate metabo-lism? And what is it in an individual with diabetes that derails that normal process?

A Harvard collaborativeThe Harvard Stem Cell Institute is a collaborative of more than 225 faculty members, a community of 1,000 lead-ing scientists, post doctoral fellows, graduate students and undergraduate students all of whom have two things in common: we are part of the Harvard University family, affiliated with one or more of the University’s nine schools and 11 affiliated hospitals and medical institutions; and, most important, we are all working to advance the understand-ing of stem cells in basic research and regenerative medicine. Together we are the linchpin of Harvard’s pluripotent ef-forts to advance stem cell science. Our co-directors, Doug Melton, PhD, and David Scadden, MD, are the co-chairs

of Harvard’s new cross-school Depart-ment of Stem Cell and Regenerative Biology (SCRB), whose faculty are all key members of HSCI. Prior to the founding of HSCI in 2004, almost all of us were working independently. In fact, our co-directors, Melton, then a developmental biologist in Harvard’s Department of Molecular and Cellular Biology, and Scadden, a physician-scientist treating cancer patients and do-ing research at Massachusetts General Hospital, had never met before coming together to found HSCI.

The researchers who collectively are the Harvard Stem Cell Institute are working with all forms of stem cells. Some focus primarily on human embryonic stem (hES) cells, those cells that arise in the very initial phases of embryonic devel-opment and give rise to all the body’s cells. Some are dedicated to understand-ing the role of so-called adult stem cells, stem cells in some organs of the body—skin and blood for example—which exist throughout healthy life and develop into only specific types of cells. And other HSCI scientists are working

HSCI Programs to target & cure diseases

■ ■■ Blood Disorders■ ■■ Cancer■ ■■ Cardiovascular Disease■ ■■ Diabetes■ ■■ Kidney Disease■ ■■ Nervous System Diseases■ ■■ Regenerative Therapeutics

on induced pluripotent stem (iPS) cells, stem cells that are created in the labora-tory by manipulating adult cells, turning back their developmental clock to the point where they are the equivalent of

embryonic stem cells. The goal of all this research is three-fold:

■■ to produce disease-specific stem cells that allow scientists to study

HSCI Developing & building living tools

■ ■■ Therapeutic Screening Center■ ■■ iPS Core Facility■ ■■ Flow Cytometry Core Facilities■ ■■ Genome Modification Facility■ ■■ Center for Human Cell Therapy

HSCI is a collaborative made up of scientists and other scholars from across the schools and affiliated medical institutions of Harvard University.

■■ the development of a disease in a laboratory dish, rather than in patients

■■ to create normal and disease-specific stem cells that can be used as targets for the development of drugs

■■ ultimately, to produce specialized or differentiated cells tailored to a par-ticular patient to use as treatments for disease

Disease in a dish Being able to study a disease in a dish offers research possibilities previously beyond reach. Because so many of to-day’s greatest disease challenges—heart disease, diabetes, and Parkinson’s, to name just three—develop over years, by the time a patient develops symptoms, the biological or environmental factor(s) that triggered the disease may be long gone from the body. This means that though we can study the effect of the disease on patients, we may not be able to study the development of the disease, which we need to understand if we are going to develop more effective treat-ments and cures. But creating the very

cells affected by a disease allows us to watch the disease progression from cell birth to cell death. Having a way to see what is happening to the cells will enable us to develop treatments to block or reverse the disease process.

New targetsAnd being able to create disease-specific stem cells gives us a new set of targets for drug development. This use of stem cells as tools with which to develop treatments and cures for disease has become a major focus of research at the Harvard Stem Cell Institute. In fact, we are so convinced of the promise of this area of research that we have launched one of the very few drug development programs outside the pharmaceutical world.

New therapiesSome of our efforts focus on using cells themselves as therapies, creating patient-specific cells as replacements for organs and cells damaged by disease. For example, researchers in our

wHSCI Underwriting the future of stem cell science

■ ■■ Seed Grants■ ■■ Junior Faculty Programs■ ■■ HIP Internship Program■ ■■ Clinician-Scientist Program■ ■■ NIH Training Programs■ ■■ Graduate Fellowships

Cardiovascular Disease Program, work-ing between one of the hospitals and the Engineering School, have already produced a functioning strip of the type of muscle cells that are killed by heart attack.

Focus on resultsThe creation of HSCI, with its emphasis on collaborative science; on discoveries, rather than titles and roles; on results, rather than credit; has brought people together in ways formerly unimagi-nable in the historically siloed world of academic science, culminating with the establishment of SCRB, which is a de-partment of both Harvard’s Cambridge-based Faculty of Arts and Sciences, and Harvard Medical School in Boston. With joint projects, shared facilities, regular seminars, research presenta-tions, working group gatherings, and regular communications to our growing community, we have made scientific collaboration a way of life, rather than a catchphrase.

We create collaborations and chan-nel our support for stem cell research

through several programs using cutting-edge science and state-of-the-art technology. These projects signal our receptiveness to innovation and include:

■■ Seed Grants to provide early fund-ing for innovative projects consid-ered to be “high risk/high reward.” These are the kind of studies that would typically not be funded through the usual research grant mechanisms.

■■ Core Platforms such as our Thera-peutic Screening Center and iPS Core Facility to provide resources and services that are beyond the means of most individual labs and to make available to the HSCI community the technical advances developed in individual labs.

■■ Disease-focused Programs that depend upon collaboration across laboratories to create new insights through shared interests in specific health challenges.

Managing from bench to bedsideWe provide support for scientists and clinicians working at the intersection

HSCI Where team science gets results

■ ■■ Seed grant project led to breakthrough in cell reprogramming

■ ■■ 5 HSCI faculty from three HSCI-affiliated institutions received an NIH Grand Opportunities Grant

■ ■■ 3 HSCI research teams received a total of $27 million from NHLBI Progenitor Cell Biology Consortium Award

of basic research and its application to patients. As we underwrite the future of medicine, we know where and how to put our resources to work. Our Execu-tive Committee, comprised of a dozen stem cell experts from our multiple institutions, decides where to invest the money we raise. This committee not only decides how much money goes into our major programs—seed grants, core platforms and disease programs —but it also holds the funding re-cipients accountable on a regular basis. For example, the commit-tee can quickly decide to put more resources behind a project that is going well or, start a new project when a new technology arises or, terminate a project if progress is unsatisfactory. We have the flexibility to deploy money and people according to the judgment of some of the best scientists and clinicians in the world.

Risks for resultsWe maximize our resources by invest-ing in excellence. At HSCI we think of our expenditures in research projects as investments but unlike a financial investor, the expected return is not purely monetary. Our annual survey of our funded projects shows significant results, from key findings to generation of new intellectual property to being able to secure additional funding. Our strategy emphasizes value—economic, therapeutic and scientific—in the search for new drugs and therapies. We are generating economic value through new intellectual property and new start-up companies that have already resulted from our work. We are generating

therapeutic value through the new clini-cal solutions we are developing even if they don’t have large markets. We are generating new scientific value through funding basic research and making it widely available to our fellow scientists.

Partnering with HSCIHSCI presents an outstanding oppor-tunity for advancing the state of stem cell science and regenerative medicine,

and hastening the de-velopment of treatments for, thus far, intractable chronic diseases. Our col-laborative, interdisciplin-ary approach, our depth

of scientific talent, our ability to invest in shared technology, and our access to large, well characterized patient popula-tions and clinical expertise will enable us to translate research into therapies —both cells and drugs—that can help extend and save lives. Support for the Institute is much more than support for basic research and education—it is an investment in the future of medicine.

Harvard Stem Cell InstituteHolyoke Center, Suite 727W1350 Massachusetts AvenueCambridge, MA 02138, USA617-496-4050hsci@harvard.eduwww.hsci.harvard.edu

Photo Credits: front-left, BD Colen; front-center, John Dimos/Eggan Lab; front-right, iStockPhoto; 2, Justin Ide; 3 & 5, BD Colen.

Advancing basic science today for advanced

treatments tomorrow