Pediatrics and Gene Therapy: A Conversation with David Williams

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For David Williams, MD, the field of pediatrics offers two great attractions.

“It is wonderful because of the kinds of patients you take care of,” he says. “But also because of the personalities of pediatricians – in pediatrics you find very compassionate and caring people.” Williams embodies that compassion and combines it with a drive to solve the medical problems of young patients, often with the use of cutting-edge technology.

In some 25 years of research, a major focus, Williams says, has been on understanding how blood-forming stem cells engraft into the bone marrow. He has developed landmark techniques for introducing genes into these stem cells, which are then given to patients as a form of gene therapy. (Read more about his career.)

Perhaps his most difficult and gratifying achievement has been using these methods to cure two very young boys of the  “bubble boy disease” (SCID, or severe combined immunodeficiency disease) they had lived with since birth. The gene implants restored their immune systems so they no longer needed protection from viruses that usually cause little disease in most us, but are deadly in patients with SCID.

The goal of gene therapy – a field that has had many ups and downs – is to treat patients with inherited diseases by transferring healthy genes into their cells to correct errors in their DNA. Williams, who is chief of the Division of Hematology/Oncology and director of Translational Research at Boston Children’s Hospital, as well as associate chair of Pediatric Oncology at Dana-Farber, has been pursuing gene therapy since his days as a research fellow at the MIT Cancer Center and the Whitehead Institute. (You can read more about Williams’ research into gene therapy and Fancomi Anemia on Vector, Boston Children’s Hospital scientific and clinical innovation.)

Now, Williams and his colleagues are expanding their gene therapy efforts to other congenital disorders. Currently, he is working on an ambitious project with Stuart Orkin, MD, the chair of Pediatric Oncology at Dana-Farber, aimed at curing sickle-cell disease with gene therapy.

The American Society of Hematology (ASH), the world’s largest professional society concerned with the causes and treatment of blood disorders, has chosen Williams as its next vice president; he will become president in 2015, becoming only the second pediatrician to hold that prestigious post.

A passion and compassion for caring for children is a family affair for Williams; his wife, Cindy, a pediatric nurse practitioner, is program director of Nursing Research at Boston Children’s Hospital; their daughter is a senior pediatric resident at Children’s Hospital of Philadelphia.

As a counterpoint to the intense life in lab and clinic, Williams and his family have made farm animals a part of their lives for many years. At their home outside of Boston, they keep horses, chickens, and bees. Williams and his daughter began riding together when she was a child. The pediatrician says he supplies many people at Dana-Farber and Boston Children’s – including Dana-Farber President Edward J. Benz Jr., MD – with fresh eggs and honey.

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