The death of a vCJD patient who had shown a 鈥渞emarkable鈥� improvement after starting a controversial drug treatment will not affect preparations for clinical trials of the treatment in the US and UK, authorities say.
Rachel Forber was diagnosed with the human form of mad cow disease in June 2001. In August, she flew to the laboratory of Stanley Prusiner at the University of California, San Francisco for treatment with chloropromazine, an anti-psychotic, and quinacrine, which is conventionally used against malaria. Laboratory studies had suggested that both drugs can block the formation of prions, which cause vCJD.
Within three weeks, Forber showed such an improvement that the UK government announced it would fast-track a trial to evaluate the efficacy and side-effects of quinacrine on patients with vCJD and related sporadic CJD. But Forber was taken off quinacrine after suffering liver damage. She died of causes unrelated to that damage on 30 November.
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Peter Smith, deputy chair of the UK government鈥檚 Spongiform Encephalopathy Advisory Committee (SEAC), says that most of the science community had been sceptical that quinacrine was a miracle cure. 鈥淚 guess most people won鈥檛 be surprised that the unfortunate woman died,鈥� he told New Scientist.
Families鈥� dismay
Smith adds: 鈥淭here had been some in-vitro evidence it would work, but that鈥檚 a long way away from patient trials.
鈥淧atients and their relatives are often prepared to take risks when faced with a fatal disease, when really we would be happier if drugs were tried out in a cellular system first.鈥�
Forber鈥檚 death has dismayed families of patients with vCJD and sporadic CJD, says Francis Hall of the UK鈥檚 Human BSE Foundation, who knew the Forber family. 鈥淚t鈥檚 terrible for hope to be given and then snatched away like that. We鈥檙e right back where we started with no cure.鈥�
Memory improvement
Three weeks after starting treatment, Forber was no longer confined to a wheelchair. She was again able to clothe and feed herself and showed improvements in memory and alertness. But some vCJD experts suggested that she might have experienced a 鈥渘atural鈥� improvement in her condition, rather than one triggered by the drugs.
Prusiner鈥檚 team is planning to start full-scale trials of both drugs on US patients with sporadic CJD within the next three months.
The UK鈥檚 Medical Research Council also plans to start full-scale clinical trials of quinacrine at St Mary鈥檚 Hospital in London in January 2002. But UK vCJD and CJD patients who request the drug are currently being given it, St Mary鈥檚 says.
鈥淐learly we already know a lot about quinacrine and its side-effects,鈥� says Janet Derbyshire, head of the MRC鈥檚 Clinical Trials Unit. 鈥淲ith malaria, the risks outweigh the benefits, so we treat patients with newer drugs, but CJD has no cure.鈥�
But the small number of vCJD cases will make it difficult to evaluate the drug, say critics. 鈥淓ven though trials will also be carried out with sporadic CJD, that still amounts to only about 40 cases a year and those patients only tend to live about five months after diagnosis,鈥� Smith says.
Of the 113 people identified with definite or probable vCJD since 1996, eight are still alive.