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posted by janrinok on Friday September 23 2016, @06:44AM   Printer-friendly

Last week, after more than six months of waiting, 17-year-old Gary Ruot of Debary, Florida, was accepted into a clinical trial testing a novel treatment for a rare degenerative eye disease that strikes fast and leads to irreversible blindness.

Called gene therapy, treatments such as this one attempt to slow or in some cases reverse an inherited disease by delivering a new gene to the site of a mutated, disease-causing one. But to get into a clinical trial for such therapies, patients must meet specific criteria set by drug makers. They must fall into a certain age group, they must have had symptoms for a defined period of time, and they must harbor the precise genetic mutation the therapy is designed to correct. Matching the right people to these experimental therapies is challenging, and the stakes are high for patients with no other options.

In Gary's case, there was little time to act. His vision first started declining in December 2015. In January he stopped playing baseball, and by February he couldn't see to drive anymore. That month he was diagnosed with Leber's hereditary optic neuropathy, or LHON, which affects a[s] few as one in 50,000 people worldwide.

"This is a devastating disorder that has no proven therapy of any great efficacy," says Nancy Newman, principal investigator in the trial and a professor of ophthalmology at Emory University School of Medicine, where it is taking place. "That's why a patient would be willing to move into the gene therapy area, despite the fact that there will always be risks."

[Continues....]

Gary's mother, Jennifer Ruot, tracked down the trial, which is sponsored by GenSight Biologics. Since it's testing a drug intended to correct the exact genetic mutation responsible for Gary's vision loss, it represented his best hope to get his sight backā€”but he wasn't eligible to join, because the minimum age was 18. A friend of his created a petition to deliver to the U.S. Food and Drug Administration to lower the age limit, and Jennifer called or e-mailed GenSight regularly asking for updates about the trial.

Sometimes the FDA needs to see good safety data in adults before giving the go-ahead to researchers to enroll children in clinical trials. This was the case with GenSight, which received regulatory approval this month to enroll patients aged 15 to 18.

GenSight was able to slightly expand the patient population for its trial because LHON is biologically the same disease in a 15-year-old and a 20-year-old, Newman says. Some gene therapy trials specifically target diseases in children or even infants. But not all gene therapies are considered safe for children, because they may damage organs that are not yet fully developed.

Designing clinical trials for gene therapy is tricky. In phase I and II trials, which are trying to establish safety, researchers seek out patients with advanced cases of the disease. That minimizes risk in case an experimental therapy goes wrong. Once a therapy moves into more advanced clinical trials, they enroll patients who have developed symptoms more recently; studying these patients is a better way to measure efficacy. But seeking patients at these different disease stages shrinks the pool of those ultimately able to participate.


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  • (Score: 1) by Warm_Fusion on Friday September 23 2016, @12:16PM

    by Warm_Fusion (6138) on Friday September 23 2016, @12:16PM (#405498)

    You're allowed to drive at 17 in the UK, but if you have a disability you're allowed to start at 16.

  • (Score: 2) by Fnord666 on Friday September 23 2016, @01:26PM

    by Fnord666 (652) on Friday September 23 2016, @01:26PM (#405515) Homepage

    You're allowed to drive at 17 in the UK, but if you have a disability you're allowed to start at 16.

    Generally speaking it's 16 in the US.