Drugs used to treat malaria and schizophrenia may be able to defeat theinfectious proteins that cause the human equivalent of mad cow disease.University of California at San Francisco researchers found the drugs wereeffective in treating mouse cells infected with proteins known as prions,which cause Creutzfeldt-Jakob disease.
"It's a big leap from findings in cell culture to those in humans, and we donot know if we will see a favorable response in humans. But the results wesaw, in a cell model we consider valid, make this lead worth pursuingimmediately," says Dr. Carsten Korth.
The researchers are studying the drugs quinacrine, an obsolete malaria drugwidely used during World War II to treat malarial infections of the brain,and chlorpromazine, approved to treat schizophrenia and other psychoticconditions. While they were preparing for human clinical trials, twovolunteers asked to be treated.
A young British woman with the human form of mad cow disease, and an olderAmerican with the more common form of the malady, Creutzfeldt-Jakob disease,have been treated at a San Francisco hospital with quinacrine.
"Before our trial was ready, two patients arrived on our doorstep," says Dr.Bruce Miller, a neurologist at the School of Medicine at the University ofCalifornia. "It put a lot of pressure on us to do the right thing. We feltwe could not turn them away and so gave the drug in compassionate use."
Compassionate use is the practice of giving an unapproved drug or one meantfor a different disease to treat a dying patient in the hope that the drugmight have some effect.
"It is way too early to make any claims that the drug is working," Millersays. "One person has gotten worse and about the other we just don't know.We are far from a cure. We are all hoping the drug will have some efficacy,but our eyes are wide open. It could absolutely not work."
The father of one patient, 20-year-old Rachel Forber from Merseyside innorthern England, told a London newspaper that after 19 days of treatmenthis daughter was able to walk unaided, use a knife and fork and completecoordination tests that she had previously found impossible. Forber wasgiven the diagnosis in June and told she had a year to live. She had becomedisabled and needed a wheelchair. The researchers said that both patientshave returned home, where they will continue taking the drug.
Infection can occur from eating contaminated meats, contamination throughbiological and pharmaceutical products and from cannibalism. The problemconfronting scientists right now is Mad Cow Disease, which has infectedpeople in the U.K., Europe and South America. On average, patients survive14 months after the disease is diagnosed. Of the 106 people known to havecontracted variant CJD since 1995, fewer than 10 are alive. Researchers fearthat it will hit the U.S. eventually.
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