Product Pipeline

CCALD Product Development

bluebird bio’s CCALD product program has the potential to halt the progression of childhood cerebral adrenoleukodystrophy (CCALD) by providing a functional gene delivered to the brain by the patient’s own cells that have received a corrective gene. The results of the ongoing CCALD trial, based upon work conducted by INSERM, were named as one of the “Scientific Breakthroughs of 2009” by the journal Science.

Clinical Insights
Data from a pilot study treating X-linked childhood cerebral adrenoleukodystrophy (CCALD) were presented in May 2010 at the American Society of Gene and Cell Therapy’s (ASGCT) 13th Annual Meeting. The findings demonstrate continued stable expression of CCALD protein at 10 to 15 percent of circulating monocytes and disease stabilization at three years in two CCALD patients. Both continue to show neurological stabilization with no biological adverse effects to date. Initial data on a third patient shows stable expression of the protein at 20 months; however, current data are insufficient to determine disease stabilization. New findings demonstrate the third patient’s cognitive functioning within normal range at 16 months.

bluebird bio plans to initiate larger clinical studies in CCALD in both the United States and Europe in 2011.

About ALD
Adrenoleukodystrophy (ALD) is a rare, inherited neurological disorder that, in its most severe form, causes damage to the myelin sheath (an insulating membrane that surrounds nerve cells in the brain) and progressive dysfunction of the adrenal glands. Also known as Lorenzo's Oil disease, ALD is estimated to affect one in every 21,000 boys worldwide. In the childhood cerebral form (CCALD), symptoms usually occur between the ages of 4 and 10. Boys afflicted with this form of ALD develop normally until the onset of symptoms. The symptoms of this disorder often progress rapidly and, in a matter of years, can lead to a vegetative state and, ultimately, death. Current treatment options are limited to allogeneic stem cell transplantation when there is an appropriate donor. Allogeneic transplants carry a significant risk of serious morbidity and death.