US And UK Scientists Win Million-Dollar Prize For Gene Therapy To Cure Childhood Blindness

Evie Fordham | Politics and Health Care Reporter

A group of seven scientists from the U.S. and the United Kingdom won a $1.15 million prize Tuesday for their work on a gene therapy to treat childhood blindness.

The scientists, some of whom started working on the treatment in the 1990s, were honored with the Portuguese Champalimaud Foundation’s Vision Award for their work to cure a form of Leber congenital amaurosis (LCA), reported Reuters.

Babies born with LCA have poor eyesight and are extra sensitive to light. In most cases, people with the “rare genetic eye disorder” go completely blind by age 40, according to a press release from the University of Pennsylvania School of Medicine.

LCA is caused by a mutated gene. The scientists figured out a way to transfer a corrected version of the gene to patients via innocuous viruses. They received the U.S. Food and Drug Administration’s first approval of a gene therapy for a congenital disorder in December 2017, according to the press release.

“This is the first, and still only example of successful gene therapy in humans that corrects an inherited genetic defect and is therefore a milestone in medical therapeutics,” said award jury chairman Alfred Sommer, dean emeritus of the Johns Hopkins Bloomberg School of Public Health, according to Reuters.

Of the four U.S. researchers who won the prize, one works at the National Institutes of Health’s National Eye Institute, while three are ophthalmology professors at the Perelman School of Medicine at the University of Pennsylvania. Two of those ophthalmologists have more than a professional bond — Jean Bennett and Albert Maguire are married.

The Champalimaud Foundation has handed out the annual science prize since 2006, according to Reuters. (RELATED: Scientists Are Hopeful About Gene Therapy To Cure People Who Thought They’d Deal With Hemophilia Forever)

But do not expect to see the treatment hitting pharmacy shelves in the immediate future. Gene therapy treatments like this one are used at extremely low rates for a few reasons. First, they are extremely expensive. Second, since they often treat rare genetic diseases, pharmaceutical companies do not have large potential customer bases to incentivize the companies to make the treatments, reported Business Insider.

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Tags : food and drug administration national institute of health university of pennsylvania
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