The program’s sole mission is to explore, understand, and translate the promise of stem cells into effective clinical therapies and treatments. Its leaders have developed a three-pronged strategy to fulfill this charge:
- Develop basic methods for creating customized stem cells that can be turned into any tissue in the body.
- Apply those methods to the diseases most likely to yield the first breakthrough treatments. Blood diseases such as leukemia are likely to be first because the science is furthest along for them and the method for delivering the cells, blood stem cell transplantation, is well established. These disorders are the Program’s primary focus, but major progress in its iPS cell research has enabled expansion to other diseases more rapidly than anticipated. Intensive research now focuses on solid cancers, liver diseases, and rare conditions that shed light on more common ones.
- Make iPS cells and other technologies developed here available to colleagues worldwide and so speed progress on a vast array of disorders.
To deliver on the promise of stem cell research as swiftly as possible, the Stem Cell Program at Boston Children’s Hospital devised a meticulous business plan. Developed in consultation with business, philanthropic and biotech leaders, the plan lays out the milestones and investments necessary to bring the Program’s first stem cell therapy to clinical trials within five years and deliver renewed hope to patients.