Whitehead Initiative in Stem Cell and Regenerative Biology

Exploring the regenerative power of stem cells

Embryonic and adult stem cells hold great promise for harnessing the body's own ability to regenerate damaged tissues and cells. This emerging field of biomedicine offers the hope for one day treating many incurable conditions, such as neurodegenerative disease, spinal cord injuries, diabetes, and sickle cell anemia. 

But surprisingly little is known about the basic biology that affords these cells such astounding power. Scientists at Whitehead Institute for Biomedical Research have launched a broad-based research initiative to decipher the complex interplay of internal programs and external cues that enable stem cells to form many different cell types and to self-renew almost indefinitely. As this knowledge gradually translates into the clinic, physicians may one day use these cells to alleviate human suffering.

Deciphering nature’s complexity

While many research institutions are experimenting with stem cell therapies to treat specific diseases, Whitehead researchers seek to understand the underlying biochemical and genetic regulators of stem cells at work in natural processes of development and regeneration. Understanding how these events occur in nature is the first step toward designing customized cell-based therapies.

Whitehead Institute is uniquely equipped to undertake this challenge by virtue of its internationally recognized research programs in developmental biology and genomic analysis, and its history of successfully tackling the “big questions” at the frontiers of biology. 

The Institute is intimate, highly collaborative, and home to many of the world’s preeminent biologists. While it is wholly independent in its governance, finances, and research, all Members are professors in the MIT department of biology. Not only do Whitehead Members participate fully in the life of MIT, but this close relationship brings MIT doctoral candidates—some of the world’s most talented graduate students—into Whitehead labs where they help push the envelope of discovery. 

A legacy of leadership and discovery

Since 1982, Whitehead has significantly shaped the world of biological science. Seminal discoveries include identifying the genes that cause the earliest stages of cancer, developing the first transgenic mouse model of a human genetic disease, identifying molecular mechanisms of obesity, revolutionizing our understanding of how RNA influences gene regulation, and deciphering mechanisms of neurological disorders like Parkinson’s and “mad cow” disease. Whitehead was the single, largest institutional contributor to the Human Genome Project. Its scientists are also expert in engineering experimental technologies that enable research to occur on scales unimaginable just a few years ago. 

With 15 Members and five Whitehead Fellows who serve as principal investigators, and more than 380 visiting scientists, postdoctoral fellows, graduate students, and undergraduate students, there are hundreds of investigator-initiated collaborations ongoing between Whitehead researchers and colleagues in academia and industry. For example, three of the Harvard Stem Cell Institute’s principal scientists trained at Whitehead, and joint research flourishes between labs. The Institute’s intellectual property and material transfer policies are designed to maximize the broadest dissemination of Whitehead science to the world.

Whitehead advances in regenerative biology

Whitehead scientists have been pioneering the field of stem cell research for more than two decades, working with mice as an experimental organism. In 2002, they succeeded in creating custom-tailored cellular therapies to partially restore immune function in an immune-deficient mouse. This breakthrough established for the first time that a combination of nuclear transplantation, gene therapy, and embryonic stem cell differentiation can be used to cure genetic disorders. With the creation of the Whitehead Human Embryonic Stem Cell Facility, researchers have begun to apply the knowledge gained from such work into human stem cells.

Since 2002, Whitehead scientists have made many other dramatic discoveries that highlight the promise of stem cell research:

• Researchers in the Richard Young lab uncovered the process responsible for granting human embryonic stem cells the potential to become any type of cell in the body. 

• The Jaenisch lab demonstrated a technique (altered nuclear transfer) for creating embryonic stem cells without destroying a viable embryo.

• Researchers in the Young and Jaenisch labs collaborated with Harvard University’s Douglas Melton and MIT’s David Gifford to map the genomic regulatory network that controls stem cell development, in both mouse and human embryonic stem cells.

• The Harvey Lodish lab discovered how to multiply mouse adult stem cells from the blood 30-fold, a promising development for transplantation therapies.

Private help for a public challenge

Whitehead researchers hope one day to use their growing knowledge of stem cell biology to reverse the genetic and biochemical programming of mature cells back to a pluripotent, embryonic stem cell-like state—without the intermediary of an embryo—creating an inexhaustible source of customized replacement cells. 

Unfortunately, present restrictions on federal support for research on human embryonic stem cell lines pose a financial obstacle to this research. No other area of biomedical research is so promising, yet so constrained. Private support is needed to allow Whitehead science to write the next chapter in stem cell research.

Investing in stem cell science

Whitehead scientists have joined together to launch the Whitehead Initiative in Stem Cell and Regenerative Biology. The Initiative will provide the scientific foundation for stem cell therapies to treat a wide range of human disorders. Philanthropy, sponsored research, and other support in the range of $100 million over the next six years will be required to meet total funding needs.

Present restrictions on federal support for research on human embryonic stem cell lines pose a financial obstacle. No other area of biomedical research is so promising, yet so constrained. Private support is needed to allow Whitehead scientists to write the next chapter in stem cell research.

Giving opportunities

Research funds—Research support is needed to launch high risk-high potential early stage projects to establish proof of principle, and to underwrite research in human embryonic stem cell lines that are ineligible for federal research support. (Expendable funds ranging from $40,000 to $500,000 per year)

Core research facilities—Understanding complex processes of stem cell and regenerative biology requires a state-of-the-art research infrastructure, including dedicated human embryonic stem cell, RNA interference, cell-sorting, microarray, proteomics, and bioimaging core facilities. (Capital and expendable funds ranging from $100,000 to $1,500,000) 

Technology development funds—Support is needed to develop and adapt experimental platforms to yield understanding of stem cell and regenerative biology (Expendable funds ranging from $75,000 to 500,000) 

Support for postdoctoral scientists—Expendable full-year fellowship ($90,000 per year)

Support for graduate students—Expendable fellowships of five years duration ($50,000 per year)

UROPs—Expendable research support for undergraduates ($5,000 per student per student)