Physicians from Dana-Farber/Boston Children's Cancer and Blood Disorders Center will showcase their advances at the Annual Meeting of the American Society of Pediatric Hematology Oncology (ASPHO).
ASPHO President: Amy Billett, MD
Trustee at-large: Akiko Shimamura, MD PhD
Learn more about our innovations and clinical trials below:
The sickle cell gene therapy clinical trial will involve a single infusion of autologous bone marrow-derived CD34+ hematopoietic stem cells (HSC) cells transduced with a lentiviral vector containing a micro RNA-embedded short-hairpin RNA targeting BCL11A. This gene therapy technology is distinct in that it both turns off the sickle hemoglobin expression and turns on fetal hemoglobin production.
One of our novel gene therapies has effectively stabilized cerebral adrenoleukodystrophy (CALD) in 15 out of 17 patients who participated in a recent clinical trial. The first results of this trial were published in October 2017 in the New England Journal of Medicine.
As one of the top pediatric cancer centers in the country, we are a certified treatment center providing the FDA-approved CAR T-cell therapy called KYMRIAH to patients who are up to 25 years old with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL).
Led by Kimberly Stegmaier, MD, researchers at Dana-Farber/Boston Children's have found that CDK12 inhibitors pack a particularly lethal punch to Ewing sarcoma, a rare cancer typically affecting children and young adults. These findings were recently published in Cancer Cell.
Dr. Mark Kieran of Dana-Farber/Boston Children's has discovered a very promising new pathway to preventing tumor recurrence — "resolvins" could be used in complement with chemotherapy, radiation and targeted therapies to stave off the tumor-promoting effects of dead cancer cell debris.
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