Bluebird Bio gene therapy posts encouraging results in treatment of rare brain disease

pharmafile | April 21, 2016 | News story | Medical Communications, Research and Development Bluebird Bio, Phase II, gene therapy, lenti-d, paediatric, phase 2, phase 3, phase III, rare brain disease 

Bluebird bio (NASDAQ: BLUE) has announced positive interim data from a Phase II/III trial evaluating their Lenti-D gene therapy in paediatric patients with rare brain disorder, cerebral adrenoleukodystrophy (CALD).

In the ongoing STARBEAM study, 17 young boys were treated with Lenti-D and none of them developed the major functional disabilities which are typical of the disease. Furthermore the therapy helped to stabilise neurological function in all of the participants, with one exception.

Also known as Lorenzo’s Oil disease, CALD is a rare and fatal, neurodegenerative disease that involves a progressive destruction of myelin, which is the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control.

Currently, the only treatment option available is a risky allogenic stem cell transplantation which, although it can be efficacious, it can result in graft-versus-host complications and requires a matched donor, usually a sibling.

One caveat amid these positive results is that five of the 17 patients experienced a re-emergence a marker for CALD progression, gadolinium enhancement, after 12 months. Two of the boys involved saw that issue resolved after 18 months.

David Williams, president of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, says: “It is exciting to see a potential autologous treatment option for these patients. While these study results are early, they are encouraging, and we look forward to further understanding the impact that treatment with Lenti-D gene therapy could have on patients as these data evolve.”

David Davidson, chief medical officer at Bluebird Bio, comments: “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 stabilisation of CALD… 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.”

Sean Murray

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