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That’s Chris Hempel’s argument: Niemann-Pick Type C, or NPC, causes cholesterol and other fats to build up to toxic levels inside cells, harming various organs and especially the brain until patients lose the ability to talk, walk and swallow. Only 500 children worldwide are known to have it. But a drug that could flush out that build-up, Hempel contends, just might point to a new route to fighting heart disease or Alzheimer’s.

For NPC, Hempel hopes to repurpose cyclodextrin, a sugar-like compound that’s already used in numerous products. But by itself, it wasn’t deemed to have any drug effects _ until scientists at the University of Texas Southwestern Medical Center made the surprise finding that cyclodextrin helped mice with NPC.

When her daughters were diagnosed in late 2007, Hempel desperately searched scientific journals for any hint of a treatment and ran across the Texas research.

What works in mice often fails in people, and it can take years of additional research before animal experiments lead to human studies.

“They don’t have years,” says Dr. Caroline Hastings of Children’s Hospital & Research Center Oakland in California, who leads the twins’ cyclodextrin treatment. “They really had nothing to lose.”

Subsequent studies in cats at the University of Pennsylvania show promise, too. Hempel found a Florida supplier of cyclodextrin, and worked with Hastings to file FDA applications for “compassionate use” testing of cyclodextrin in the twins. She even persuaded Johnson & Johnson, which uses cyclodextrin as an inactive ingredient in an anti-fungal medicine, to share proprietary data about the compound’s human safety and other issues to address FDA questions.

Addison and Cassidy already have serious symptoms; they’d quit talking. The cyclodextrin was first infused into their bloodstreams in 2009, but Hempel says it wasn’t penetrating the brain. So late last year, FDA allowed injections into the spinal fluid, which bathes the brain. It’s too soon to know how they’ll fare, but the family thinks the girls are more alert, and Hastings says tests show their hearing has improved.

Now, the NIH is planning a formal study of cyclodextrin in a number of NPC patients, to begin within about a year.

Hempel isn’t alone in her quest to repurpose common drugs. Consider progeria, a disease that rapidly ages children until they die of a heart attack or stroke, usually before their teens.

Collins’ lab at NIH uncovered the gene defect behind progeria, research that he says he pursued only because of meeting another mom, Dr. Leslie Gordon, founder of the Progeria Research Foundation, and her son, Sam, who has the disease. Today, clinical trials are under way using a failed cancer drug named lonafarnib that promises to block some of the progeria mutation’s effect.

“We’re very excited about the opportunities in progeria,” says Dr. Gary Gilliland of Merck & Co., which donates the drug and is watching carefully to see if the studies make further pursuit worthwhile.

There are an estimated 150 progeria patients worldwide, but Gordon points to growing evidence that the culprit protein may play a role in the heart disease that comes with regular aging, too.

However, progress is slow. Just because initial research shows a drug looks promising doesn’t guarantee broader testing. For example, a National Cancer Institute-funded team is pushing to test a certain class of drugs against a childhood cancer, Ewing’s sarcoma. But none of half a dozen manufacturers has yet agreed to provide the drugs, says Dr. Peter Adamson of the Children’s Hospital of Philadelphia. The reason, he says: They’re not showing enough promise in more common adult cancers.


EDITOR’S NOTE _ Lauran Neergaard covers health and medical issues for The Associated Press in Washington.