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Gene Therapy Delivers Promising Results

Those suffering from various brain disorders could soon get relief in the form of gene therapy, a direct-to-brain treatment method, according to the Associated Press

What’s going on: A child living in Bangkok recently received “the first brain-delivered gene therapy after its approval in Europe and the United Kingdom for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. New Jersey drugmaker PTC Therapeutics plans to seek U.S. approval this year.”

  • U.S. studies are underway to test the efficacy of the therapy method for Alzheimer’s, Parkinson’s and Huntington’s diseases, as well as other maladies.

Why it’s important: “[S]cientists say the evidence supporting this approach is mounting—opening a new frontier in the fight against disorders afflicting our most complex and mysterious organ.”

  • In the case of the AADC-afflicted child in Thailand, a functioning version of a mutated gene was inserted via a tube in the skull. Since the one-time procedure, the girl’s brain has been able to produce the neurotransmitter dopamine, which it previously had been unable to do.
  • And all 40 or so patients in a National Institutes of Health–funded study of gene therapy for AADC deficiency “saw significant improvements.”  

What’s next: Scientists are looking into ways of delivering gene therapy without brain surgery.

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