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Drug trial hope for CJD victims
LONDON, England (CNN) -- Six British victims of the human form of mad cow disease are waiting to hear if they will be offered a possible treatment for the condition. The UK National CJD Surveillance Unit (CJDSU) is to contact the individuals to inform them of an American treatment which has shown promising results in laboratory tests. The tests involve the malaria drug quinacrine and the schizophrenia drug chlorpromazine. Both drugs have shown promise in laboratory tests on mouse cells infected with prions -- abnormally shaped proteins -- that cause variants of Creutzfeldt-Jakob disease (vCJD). Two women -- one British, one American -- believed to have forms of the disease have already been given the malaria drug at the University of California, San Francisco (UCSF) under a U.S. Food and Drug Administration policy that allows for experimental therapies on terminally ill patients. The family of the British patient said she showed significant improvement. The researchers are more cautious, however, stressing that the women were not part of their planned formal study and that no conclusions could be made about their reaction. "We don't know that this compound will work in people," Dr. Fred Cohen, a prion researcher at UCSF, told The Associated Press. The UCSF researchers say they will soon start a clinical trial to see if the drugs can help fight the human form of mad cow disease.
Scientists at the UCSF have been given federal approval to enrol more than 30 severely ill patients in a study by the end of the year. Only patients given less than a year to live will be included. Officials from Britain's Department of Health (DoH) have spoken to the U.S. team investigating the drugs. A DoH official said each of the British patients would need to judge, with their clinician, whether it was a treatment that could be useful to them. She said the DoH was looking at getting involved in trials to establish the success of the treatment, and would also consider funding research. The U.S. research is being led by Dr. Stanley Prusiner, who won a Nobel Prize in 1997 for discovering prions. George Carlson, a prion expert at the McLaughlin Research Institute in Bozeman, Montana, told AP that research rarely moved so quickly from testing on animal cells to human subjects, but he said this development warranted the speed. "This is something that can be tested right away. That's the exciting part," Carlson told AP. No cases of vCJD have been discovered in the United States. But 105 people in Europe have been diagnosed in the last five years, and thousands of cows have been slaughtered. Europeans fear that the disease, which is infectious and incubates for years without symptoms, could reach epidemic proportions. In the mouse cell laboratory experiments, the two drugs were found to inhibit the conversion of normal prions into the disease-causing, abnormal form. The results were published in Tuesday's Proceedings of the National Academy of Sciences. One of the two women to receive the malaria drug so far is Jane Forber, a 20-year-old from Britain. Her family contacted the university after she became ill. Relatives told the British media that the drug treatment helped her walk and talk again, abilities she lost as her disease progressed. However, the treatment seemed to have no effect on the other patient, an American with a form of Creutzfeldt-Jakob Disease not blamed on contaminated beef. |
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