Two years after the government announced urgent action to investigate possible treatments for variant Creutzfeldt-Jakob disease (vCJD), trials are still being held up because experts are unable to agree which drugs should be studied.
Professor Iain Chalmers, who heads the steering committee appointed to design the clinical trials, has told the patients' families to complain to their MPs about the delay. At a meeting of the CJD Support Network two weeks ago, Professor Chalmers said: "Families of patients who have suffered or are suffering from these terrible diseases have every reason to feel very angry about this delay, particularly as the UK is the vCJD capital of the world."
The Medical Research Council, which will oversee the trials, said national guidelines for studies of the antimalarial drug quinacrine will probably be agreed next month. Quinacrine trials have been held up in Britain since 2001, when a British woman having quinacrine treatment in California died after initially showing signs of improvement.
The council said: "There are great challenges in designing scientifically and ethically sound trials or clinical studies to help those who are ill with CJD. The issues are complex, such as the rapid deterioration of patients, the small numbers, what care and research assessment patients' families would find acceptable, and the fact that no proven treatment exists."
Last week, experts gathered in Belfast to review the progress of another patient with vCJD who is receiving experimental treatment (4 October, p 765). The family of Jonathan Simms, aged 19, last December won a legal battle to try the experimental treatment pentosan polysulphate. The drug, which is licensed in the United States and Germany for inflammation of the bladder, is injected directly into the brain of the patient with vCJD.
Mr Simms, who had already passed the average life expectancy of a patient with vCJD when his family won the case, has stabilised in the past 10 months and even shown some small signs of improvement in brain function, say his doctors. Though he remains confined to bed and unable to speak, he can now lift his arm when asked and has regained the ability to swallow.
Last month, the family of a young woman with vCJD (named only as M), also won permission in the High Court to begin treatment with pentosan polysulphate (18 October, p 886).
In all, 137 people have died in Britain from vCJD, of whom 16 died this year. At the moment, diagnoses seem to have peaked in 1999, though the Health Protection Agency says future peaks are always possible.