Participant’s Diagnosis Halts Gene Therapy Clinical Trial

The FDA pauses the research program on a lentivirus-based treatment for a rare neurological condition after a patient developed a bone marrow disorder that could presage leukemia.

amanda heidt
| 3 min read
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Aclinical trial testing a novel gene therapy for a rare neurological disease has been put on hold after one of the participants in a Phase 3 trial developed a bone marrow disorder that can lead to cancer. The pause, announced Monday by the trial’s sponsor, bluebird bio, and mandated by the US Food and Drug Administration (FDA), was taken “out of an abundance of caution,” the company’s president of rare genetic diseases, Andrew Obenshain, said in a recent quarterly call.

The therapy targets cerebral adrenoleukodystrophy, which is caused by a mutation in the gene for an enzyme called adrenoleukodystrophy protein (ALDP) that breaks down fats. The mutation causes fat to build up in the brain, where it breaks down the insulating myelin that allows neurons to communicate with one another. Because the gene is on the X chromosome, women typically have a least one good copy, ...

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Meet the Author

  • amanda heidt

    Amanda Heidt

    Amanda was an associate editor at The Scientist, where she oversaw the Scientist to Watch, Foundations, and Short Lit columns. When not editing, she produced original reporting for the magazine and website. Amanda has a master's in marine science from Moss Landing Marine Laboratories and a master's in science communication from UC Santa Cruz.
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