Many tissues and organs in our bodies have a remarkable capacity to regenerate in response to injury or disease. If we slice our finger, for instance, our blood replenishes and our skin heals; we can even donate a section of our liver and it will quickly regenerate to its former size.
Our bodies, by recruiting stem or progenitor cells when the need arises, can in some cases “treat” us. Stem cell scientists are striving to understand the common pathways by which this complex process occurs, which will enable them to identify targets for therapies that could trigger or accelerate tissue and organ regeneration or, in cases when this process is going awry, to block it. These regenerative therapies could offer relief or cures to millions suffering from conditions such as heart, lung, or kidney disease; nervous system diseases; and countless other afflictions.
This year, Harvard Stem Cell Institute funded two translational research projects—the Tissue Regeneration and Repair Project and the Regenerative Therapeutics Project—that will focus on understanding and harnessing the body’s intrinsic regenerative potential with the goal of finding therapies for patients. Both grants were awarded through HSCI’s Junior Faculty Program, which provides small interdisciplinary, cross-institutional teams of early-career scientists with three years of funding and access to HSCI’s varied resources.
Launched in 2007 by a self-assembled group of young investigators, the Junior Faculty Program has been so successful that it is now an ongoing, highly competitive HSCI grant program. According to Leonard Zon, MD, HSCI’s Executive Committee chair, the projects that receive funding are highly collaborative; cut across traditional boundaries of diseases, specialties, and institutions; and support young investigators tackling big, high-risk projects. “This program has exceeded all expectations, resulting in significant research breakthroughs, additional major funding, influential scientific papers, intellectual property, and promising new collaborations that advance stem cell research,” says Zon.
Tissue Regeneration and Repair Project
The Tissue Regeneration and Repair Project is co-led by David Breault, MD, PhD, of Children’s Hospital Boston, and Benjamin Humphreys, MD, PhD, of Brigham and Women’s Hospital. The other principal investigators are Akio Kobayashi, PhD, of Brigham and Women’s Hospital, Jay Rajagopal, MD, of Massachusetts General Hospital, and Joslin Diabetes Center’s Tom Serwold, PhD, and Yu-Hua Tseng, PhD.
The group comprises an interdisciplinary team of basic scientists and physician-scientists with expertise across diseases in the areas of developmental biology, immunology, mouse genetics, stem cell isolation and transplantation, and mouse models of injury. By developing common tools, including clinically relevant mouse models, the investigators seek to understand organ failure and repair in disease-specific states and to harness the potential of progenitor cells for regeneration and repair. “Our strategy, if successful, will identify new therapeutic targets and facilitate translation to the clinic,” says Humphreys, adding that the group intentionally includes physician-scientists so that the research will “always retain its clinical focus.”
Regenerative Therapies Project
The Regenerative Therapies Project is led by Trista North, PhD, of Beth Israel Deaconess Medical Center. The other principal investigators are Fernando Carmago, PhD, of Harvard’s Department of Stem Cell and Regenerative Biology, Wolfram Goessling, MD, PhD, of Brigham and Women’s Hospital/Dana-Farber Cancer Institute, David Langenau, PhD, of Massachusetts General Hospital, Jeffrey Karp, PhD, of Brigham and Women’s Hospital, and Qiao (Joe) Zhou, PhD, of Harvard’s Department of Stem Cell and Regenerative Biology.
Using a two-pronged approach, this group seeks to identify pathways that control principle aspects of the growth and repair of multiple organ systems, and translate these findings into small molecule-based therapies to enhance the regenerative capacity of many tissue types. This work will be enhanced by the group’s expertise in diverse areas that include drug delivery, cell reprogramming, and other relevant skills. “Our ultimate goal,” says North, “is to bring a compound of wide therapeutic potential from bench to bedside for use in regenerative medicine.”