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 PhDConference Planning Committee Chair: Denise M. Adams, MD
Dana-Farber/Boston Children’s physicians will be presenting at ASPHO.
View the schedule of speakers
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).
Carl Novina, MD, PhD, is leading efforts to develop an enhanced CAR T-cell therapy that would leverage a novel small molecule to destroy tough-to-treat cancers, like diffuse intrinsic pontine gliomas (DIPG), with less off-target side effects.
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.
Dana-Farber/Boston Children’s researchers, led by Stuart Orkin, MD, have revealed how BCL11A controls the switch in the body’s production of fetal hemoglobin to adult hemoglobin, which could lead to another strategy to cure sickle cell disease.
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