Sunlight causing pain can be treated with a drug pellet implant

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Sunlight causing pain can be treated with a drug pellet implant

A few minutes of sun exposure can cause debilitating pain. Savannah Fulkerson, an 11-year-old has erythropoietic protoporphyria, a rare genetic disorder.

She experienced pain when encountered sunlight as a preschooler. "It's like she's allergic to the sun and we'd be outside about 20 minutes or so…she'd say, 'I burn!'", her mother told. The pain was so intense that she'd scream "like she got hit by a car". According to scientists, people like Savannah can be helped with a tiny pellet of new drug inserted under the skin above hip.

"This is a life-changing treatment that will allow them to be exposed to sunlight longer without the fear of painful attacks. About 500 to 600 people in U.S. suffer from erythropoietic protoporphyria”, said study co-author Dr. Robert Desnick, dean of genetic and genomic medicine at Mount Sinai’s Icahn School of Medicine in New York. These patients can't tolerate daylight and suffer a tingling, itching and burning feeling, which is the warning signal to get off sun. Too much sun exposure can cause several days of excruciating pain. This condition often develops in childhood, causing "ingrained fear" of sunlight for life.

For the new study published July 2 in New England Journal of Medicine, researchers randomly gave the drug, afamelanotide, or an inactive placebo to 74 patients in Europe and 94 patients in U.S. They received an implant every 60 days for either 9 months or 6 months. Time spent in the sun without pain was calculated. Over six months, U.S. participants who got the drug reported a median (half reported more, half reported less) of 69 hours of pain-free sun exposure compared to about 41 hours among those who got the placebo. In Europe, over nine months, those who used the drug reported a median of 6 hours of pain-free sun exposure compared to less than an hour among the others. The patients who took the drug also reported higher quality of life.

No serious side effects were reported with the medication. Gastrointestinal problems and fatigue were about as common in those who took the drug. The drug works by stimulating melanin production. According to Dr. Sylvia Bottomley, “This treatment is the first to work. So it is a fairly big deal. Though not a cure, the beneficial effect of the medication is quite significant.” He added "It uniformly allows the patients to tolerate considerably more exposure to the sun and visible light, without real side effects, and should be welcomed by all."

The study represents the last in three stages of research required for drug approval in U.S., but it's not clear when the drug will be available to U.S. patients. "The number of potential patients to undergo this treatment is relatively small, and one can predict that there likely will be no rush by other pharmaceutical companies to get a share of this market," said Dr. Claus Pierach, an erythropoietic protoporphyria specialist. He added that the drug will probably be expensive, given the cost of development and once commercially available, it will likely be covered by insurance companies.

American Porphyria Foundation
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