Exciting news for the ALD Community! Our friends at Bluebird Bio Inc. have sponsored the Starbeam Study to study gene therapy for boys with ALD who don’t have a bone marrow transplant sibling match.
Autologous hematopoietic stem cell (HSC) gene therapy may offer a safe and effective alternative to allogeneic bone marrow transplant for patients with cerebral adrenoleukodystrophy (ALD), according to interim results of a phase 2/3 study. After gene therapy with Lenti-D gene therapy (Bluebird Bio Inc), most patients have had evidence of neurologic and radiographic disease stabilization and all are free of major functional disabilities (MFDs) at up to 2 years’ follow-up in some patients.
“The only downside to this treatment is that it requires early identification of the disease,” said Brendan Lanpher, MD, medical geneticist at the Mayo Clinic, Rochester, Minnesota, who wasn’t involved in the study, told Medscape Medical News. “Currently, that is only possible in families who already have had an affected child, so aggressive and early screening can find the patients before symptoms are progressed. If this treatment is confirmed to be effective and becomes available, the need for universal newborn screening for this disease will become even more imperative.”
Read the complete article here!