The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to the experimental drug Lenti-D, which is being produced by the gene therapy company Bluebird bio to treat cerebral adrenoleukodystrophy. The full article can be read here, at the North Carolina Biotechnology Centre website.

Cerebral adrenoleukodystrophy (CALD) is a genetic condition that usually affects boys. A variation to the ABCD1 gene causes very long chain fatty acids to build up to harmful levels, resulting in the breakdown of the myelin sheath that protects nerves. For patients with CALD, this damage tends to occur in the brain, which results in a progressive loss of physical and neurological function. The condition is also frequently associated with adrenal insufficiency, which can cause weakness, nausea, tiredness, and pain. There is not currently a treatment that is able to reverse the effects of CALD, but certain medicines can often reduce the symptoms. Doctors may offer patients treatments for relaxing muscles, treating infections, and epilepsy. Some patients may also be given the option of stem cell transplants. There is also on-going research into gene therapies that could provide better treatment options in the future.

Lenti-D, the experimental drug recently awarded Breakthrough Therapy designation by the FDA, is a gene therapy. It works by replacing the altered gene linked to CALD with a functioning copy of the same gene in the patient’s immature bone marrow cells. The cells reintroduced into the patient that contain the functional copy of the gene could then become different types of cells, such as brain cells, which are hoped to then function normally. As a result, the researchers believe that the build-up of very long chain fatty acids will be broken down, reducing the de-myelination of neurones that leads to neurological and physical symptoms.

The FDA’s decision to award Breakthrough Therapy designation to the drug is based on promising clinical results from a Phase 2/3 study in which boys who are 17 or younger were given Lenti-D. Two years after taking the drug, fifteen out of the seventeen patients on the trial were alive and free from major functional disabilities.

The FDA’s designation is expected to speed up the development of the Lenti-D.

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