While the interim results from the Starbeam study are early, with only three of the 17 patients having completed the study thus far, we are pleased to see evidence of neurologic and radiographic stabilization of CALD,” said David Davidson, M.D., chief medical officer, bluebird bio. “All patients are free of MFDs and most have had no progression in NFS. Brain MRI is the primary tool to quantify disease activity, and it is encouraging that most patients in the study have had stabilization of Loes score and resolution of gadolinium enhancement. We look forward to sharing additional data from this trial as the results mature, and would like to express our gratitude to the study investigators, and especially to the patients and families who are participating in this trial.”

David Williams receives research support from bluebird bio for research related to sickle cell disease, and the company has licensed intellectual property from Boston Children’s Hospital for technology related to gene therapy for hemoglobinopathies that Williams co-invented.

About the Starbeam (ALD-102) Study

The Starbeam Study is assessing the efficacy and safety of an investigational gene therapy in boys up to 17 years of age with CALD. It involves transplantation with a patient’s own stem cells, which are modified to contain a functioning copy of the ABCD1 gene. This gene addition should result in the production of functional adrenoleukodystrophy protein (ALDP), a protein critical for the breakdown of very long chain fatty acids (VLCFAs). Buildup of VLCFAs in the central nervous system contributes to neurodegeneration in CALD.

Subjects enrolled in the study are:

  • Eligible for allogeneic hematopoietic stem cell transplant (HCT) but with no matched sibling donor
  • Confirmed early-stage, active CALD as indicated by gadolinium enhancement on MRI
  • Have a Loes score between 0.5 – 9.0
  • Have an NFS of one or less

About CALD

Also known as Lorenzo’s Oil disease, cerebral adrenoleukodystrophy (CALD) is a rare and fatal, X-linked, inherited, neurodegenerative disease that primarily affects young boys. CALD involves a progressive destruction of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms usually occur in early childhood and progress rapidly if untreated, leading to severe loss of neurological function and eventual death. In boys affected by CALD, learning and behavioral problems are often observed in mid-childhood between the ages of 3 and 15 years (median age 7). The worldwide incidence rate for ALD is approximately one in 21,000 male newborns; of those, 30-40 percent are affected by CALD, the cerebral form of the disease.

Currently, the only effective treatment option for patients with CALD is allogeneic HCT. Complications of allogeneic HCT include a significant risk of treatment-related mortality, graft failure, graft-versus-host disease (GvHD) and opportunistic infections, particularly in patients who undergo allogeneic HCT and do not have a matched sibling donor.