July 12, 2010
Gene therapy trial launched for "bubble boy" syndrome
David Williams MD
Gene therapy holds the promise to cure a variety of diseases caused by mutations of a single gene. But clinical trials stalled after five boys with the immune deficiency disease SCID-X1 ("bubble boy disease") developed leukemia as a result of their treatment in Europe. Now, an international trial, sponsored in the U.S. by Children's David Williams MD, Chief of Hematology/Oncology and Director of Translational Research, hopes to revive this potentially life-saving treatment.
The new trial, the first to be given the green light by the FDA, will use a completely redesigned gene-delivery vector. "We and our collaborators expect it to be much safer," says Luigi Notarangelo MD, of Children's Division of Immunology, principal investigator of the study.
Click photo to enlarge
The Children's team, which also includes Sun-Yung Pai MD of the Division of Hematology/Oncology, is one of five in the U.S. and Europe participating in this trial. Children with SCID-X1 will be infused with stem cells isolated from their own bone marrow, in which the missing gene is introduced. Six months later, their immune systems will be checked to determine whether their ability to fight infections is indeed restored. The children will then be monitored for another 14.5 years to detect any possible cancers.
Gene therapy is potentially a great option for these children, who otherwise would be at risk of developing the life-threatening graft-versus-host disease from a bone-marrow transplant, the only existing treatment. If this trial succeeds, it's expected to pave the way for treating several other single-gene diseases with gene therapy.
"It is clear that the many years of research investment and development are now paying off," says Williams. "We believe that continued investment in basic and translational research using this technology will lead to additional advances, bringing new therapies to children worldwide."
Related links
Wall Street Journal article, Study Shows Hope for Gene Therapy
Thrive post, New hope for gene therapy
News
DF/CHCC ranks best in New England, tops in country
For the third year in a row, Dana-Farber/Children's Hospital Cancer Center (DF/CHCC) was ranked the number one pediatric cancer program in New England, and one of the best in the country in U.S. News & World Report's 2010-2011 Best Children's Hospitals issue. read more
Upcoming Events
Join us at SIOP Boston 2010
Dana-Farber/Children's Hospital Cancer Center is proud to welcome the 42nd Congress of the International Society of Paediatric Oncology (SIOP) to Boston, October 21-24, 2010. SIOP features an exciting scientific and social agenda for all members of the pediatric oncology community, including oncologists, surgeons, radiotherapists, pathologists, nurses, basic scientists, epidemiologists, biostatisticians, psychologists, and parents and survivors. Don't miss this opportunity to interact with your the global colleagues. Visit www.siopboston2010.com and register today.
