New Trial Data Bodes Well for Experimental Cerebral Adrenoleukodystrophy Gene Therapy

According to a story from, the gene therapy company Bluebird Bio’s latest recently released results from the company’s phase 2/3 clinical trial demonstrates further encouraging data for its Lenti-D gene therapy as a treatment for cerebral adrenoleukodystrophy (CALD). In fact, the findings may be good enough to warrant a fast track approval for the experimental treatment. These findings were first publicly revealed at the latest congress of the European Paediatric Neurology Society (EPNS).

About Adrenoleukodystrophy (ALD)

Adrenoleukodystrophy is a genetic disease which is linked to the X chromosome. It is most characterized by the accumulation of fatty acids because the enzymes that process them are not functioning normally. This leads to damage of the myelin sheath, an insulating, fatty layer that coats nerve cells and is essential to their function. Adrenoleukodystrophy is caused by mutations that affect the ABCD1 gene. The presentation of this disease varies significantly. This makes the process of diagnosis very difficult in many cases. Some patients have no symptoms, but when they do appear, they often include neurodegeneration, paraparesis, neuropathy, behavioral abnormalities, adrenal insufficiency, and dementia. Symptoms often progress in severity without treatment. Treatment of adrenoleukodystrophy may include gene therapy, stem cell transplant, and changes in diet. These treatments are often only effective in the early stages of the disease. To learn more about adrenoleukodystrophy, click here.

About The Clinical Trial

The trial currently includes 15 patients who are part of a longer term monitoring portion and 14 that are currently in the primary treatment portion of the study. The main endpoint of this trial is the number of patients that are free of major functional disabilities after a 24 month period. So far, 88 percent of patients are free of disabilities at the 24 month mark.

The treatment process involves the extraction of a patient’s stem cells, their modification with Lenti-D in the lab setting, and then their reintroduction into the patient’s body. These cells can transform into various cell types that can produce the ALD protein that is typically deficient in this disease.

The study is also meeting its main safety endpoint, which is how many patients are presenting with graft-versus-host disease (GvHD) at 24 months, and so far there have been no reported cases. With these results, the future of Lenti-D, and therefore cerebral adrenoleukodystrophy treatment, is looking promising.

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