The pediatric specialists at Dana-Farber/Boston Children's Cancer and
Blood Disorders Center also participate in the care of children and teens with
a variety of conditions that do not come under the umbrella of cancers or blood
disorders. These conditions include genetic immunodeficiencies, metabolic
disorders, vascular anomalies, and cardiovascular diseases such as pulmonary hypertension
and pulmonary vein stenosis.
Our doctors work closely with other specialists to develop a multidisciplinary approach tailored to the needs of each child.
This sometimes includes treatments originally designed for treating cancers and blood disorders—including chemotherapy and anti-angiogenic therapies—as appropriate.
We also offer advanced treatment strategies—including stem cell transplant and gene therapy—for select conditions. Some of these treatments
have been groundbreaking for children and teens with cancers and blood disorders, and are being shown to be effective for other diseases as well..
A stem cell transplant (also
called bone marrow transplant) is the infusion of healthy stem cells into the body to stimulate new bone marrow growth.
While often used to treat cancers and blood disorders of
children and teens, stem cell transplants are becoming an attractive treatment
option for a growing range of conditions arising from problems with cells from
the bone marrow, such as:
Our genes, which hold the code for all of our body's
functions, are made of DNA. Damage to DNA, such as a mutation, is an underlying
cause of the genetic defects that lead to cancers, blood disorders, and other
conditions. Gene therapy delivers
DNA into a patient’s cells to replace faulty or missing genes, or add new genes.
Doctors at Dana-Farber/Boston Children's offer gene therapy clinical trials to treat:
We hope to extend gene therapy to additional conditions in
Through clinical trials and research, Dana-Farber/Boston Children's is at the forefront of innovative treatments, including gene therapy.