FDA Approves Use of Experimental Drug for Twin Girls With Rare, Deadly Disease

The Food and Drug Administration has given the green light for physicians to treat twin girls suffering from a rare disease with an experimental, unapproved drug.

The five-year-old girls, who live in Reno, Nevada, suffer from Niemann-Pick type C disease, an incurable and fatal condition that is also called childhood Alzheimer’s. The disease causes cholesterol to accumulate inside cells of the body, gradually slowing down the movement of cells and causing severe neurological and physical problems. Most children with the disease do not live to become teens.

The twin girls were diagnosed with the disease in 2007 and are being treated at Renown Regional Medical Center. In the past few months, the girls, who wear diapers, began losing their ability to speak, according to their parents.

‘Compassionate Use’ Request Granted

The FDA this week granted special permission though the “compassionate use” program for doctors treating the girls to use the drug cyclodextrin, which has show promise in testing but is not FDA approved.

In some cases, the FDA grants limited permission for the use of unapproved drugs in selected patients who might benefit from treatment with unapproved drugs or techniques when their lives are in danger and no other approved treatment or method is available.

Cyclodextrin, which is delivered via intravenous infusion, is a sugar compound that also is added to cholesterol-free foods and has been touted as a possible treatment for the HIV virus.

At the request of the FDA, the girls’ treatment is beginning with half doses of cyclodextrin to ensure that the treatment is safe before progressing to higher doses if necessary, officials said. Doctors will keep a close eye on the girls’ conditions and evaluate the use of the drug every few weeks.

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