Gene Therapy, Results

Bluebird’s gene therapy shows promise in keeping lethal Lorenzo’s Oil disease in check

Bluebird Bio CEO Nick Leschly Image: Getty


After an average of 29 months following a one-time gene therapy infusion, a clear majority of boys suffering from lethal cases of cerebral adrenoleukodystrophy were alive and free of major complications from the disease — the primary endpoint in the Phase II/III study sponsored by bluebird bio $BLUE.

Publishing in the New England Journal of Medicine, investigators at Dana-Farber, Boston Children’s Hospital and Mass General recruited 17 patients with this disease, making it one of the largest gene therapy studies now underway. And they concluded that at this interim stage they have good evidence of disease stability for this very vulnerable group, with 88% still alive and complication free.

The publication amplifies the initial interim results posted last summer, with bluebird noting that they’ll be adding more results at an upcoming scientific conference.

One of the patients died from the disease — popularly known as Lorenzo’s Oil disease — and another has suffered severe and rapid disease progression.

That all still meets bluebird’s predefined benchmark for success, which was pegged at 76%.

There was one severe adverse event linked to the therapy, viral cystitis, but the investigators say that case was resolved. And while adverse events were also attributed to the myeloablative chemotherapy used to prep patients, there was no evidence of graft versus host disease or infections from the therapy.

According to the researchers, bluebird execs are engaged in talks with regulators about getting the gene therapy approved for use.

Adrian Thrasher

“This data we are seeing from this study of gene therapy is a really exciting development for boys with CALD. Not only does the data show that Lenti-D has the potential to minimize further progression of this devastating disease, but the technique also avoids the need for a matched bone marrow donor, which can be difficult to find and can delay vital treatment,” said Professor Adrian Thrasher, pediatric immunologist at Great Ormond Street Hospital and co-investigator of the study. “The results from this trial thus far are very encouraging and will pave the way for further development of state-of-the-art treatments for children with life-limiting conditions.”


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