Preliminary results from a small clinical trial to treat Huntington’s disease, a devastating neurodegenerative condition, are the first to show a substantial benefit in patients.
The treatment involved a gene therapy injected directly into the brain. Over the course of three years, patients who received the treatment had 75 percent slower progression of their symptoms compared with a control group of patients who did not receive any treatment.
“There have been a few trials that have shown some small signals if you squint,” said Dr. Steven Finkbeiner, the director of the Taube/Koret Center for Neurodegenerative Disease at the Gladstone Institutes in San Francisco, who was not involved in the trial. “But there has not been anything close to this.”
The findings were announced on Wednesday in a news release from uniQure, the company that sponsored the research. The data have not been peer reviewed or published in an academic journal and should thus be considered only as a preliminary indication of what the final results may show.
Huntington’s is a rare brain disease characterized by motor, cognitive and emotional symptoms, ultimately resulting in early death. It’s caused by a mutation in a single gene, called huntingtin, and is thought to affect about 40,000 Americans. Everyone’s brain cells produce the huntingtin protein, but in people with the disease, the gene mutation prompts neurons to make a toxic version. Those abnormal proteins accumulate and, over time, they damage and destroy brain cells.
The gene therapy directed neurons to produce a strand of RNA that attaches to and silences the huntingtin RNA, preventing the cells from making the faulty protein. The therapy was delivered via a genetically modified virus surgically injected into the brain. The virus carried a special piece of DNA that instructed the neurons to produce the silencing RNA. The virus is removed by the brain’s immune system after a week or so, but the DNA will stay in the patients’ neurons for the rest of their lives.
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The post New Treatment Shows Promise for Huntington’s Disease appeared first on New York Times.
