Children's Hospital, Boston

Stem Cell Research

The history of science and research at Children's is rich with breakthroughs in stem cell understanding and use. The hospital first opened in 1869 in the South End neighborhood of Boston with only 20 beds. In 1985 Children's received a $17 million grant from the Howard Hughes Medical Institute to develop a molecular genetics research program. Out of that program came work from Drs. Louis Kunkel and Eric Hoffman that deduced the genetic mutation involved in Duchenne muscular dystrophy that leads to the absence of a critical protein.

At present, Dr. Kunkel is collaborating with scientists in an effort to restore this protein to patients' muscles, using healthy muscle stem cells. Dr. Evan Snyder of Children's obtained human neural stem cells from fetal tissue in 1998.

Embryonic stem cells are the only stem cells that are truly omnipotent, meaning that they can differentiate into any cell type in the human body. In 2003 Dr. George Daley, a scientist with both medical and doctoral degrees, developed germ cell lines (precursors to sperm and egg cells). An understanding of how germ cells develop could lead to the ability to re—create germ cells from further developed cells and, potentially, to create embryonic stem cells. Further research is needed to discover how to direct embryonic stem cells to become a desired cell type.

Stem cell research is not based solely on regeneration or guided differentiation; much research has been conducted in the field of cancer stem cells. Cancer stem cells are presumably the core of a cancer, [Page 95]reproducing and providing the impetus for the cancer to spread. One theory is that if cancer stem cells could be isolated and killed, the cancer would be killed as well. In 2006, Dr. Scott Armstrong's research team identified cancer stem cells for leukemia. By understanding the differences between leukemia stem cells and healthy blood stem cells, it may be possible to engineer drugs that specifically target cancer stem cells while leaving healthy blood stem cells intact.

That same year, Drs. Sean Wu and Stuart Orkin, together with their research teams, discovered a particular heart stem cell that is the precursor to two important cardiac cell types. Research into this stem cell is important because by harnessing the cardiac stem cell and directing its differentiation into a particular heart cell, doctors may be able to treat or cure devastating heart defects.

Also in 2006, Dr. George Daley and his team showed that a woman's unfertilized ova, or eggs, could be used to generate custom stem cells genetically tailored to the woman herself. Tissue transplantation is often difficult because of the risk of the recipient's immune system rejecting the transplant; in contrast, custom stem cells could lead to the creation of custom tissues that would not be rejected by the patient's immune system.

...