CJD case teenagers win right to 'unsafe' treatment

A High Court judge ruled yesterday that two families with children dying from variant CJD could seek an experimental treatment that could arrest the progression of the fatal disease, despite warnings from medical specialists.

Jonathan Simms, 18, from Belfast, and a 16-year-old girl who cannot be named for legal reasons, could become the first patients in the world to receive the drug – pentosan polysulphate – injected directly into the brain, if their parents can find a hospital that is willing to administer it.

The case sets a legal precedent for the use of experimental treatments on people with a terminal illness who are incapable of giving consent and opens the way for other patients with vCJD to seek the same treatment. There is no cure for the disease and the average length of survival from diagnosis is 14 months. Mr Simms has lived 15 months and the girl has survived three years.

Dame Elizabeth Butler-Sloss dismissed the advice of two medical committees, which warned that the treatment was unproven and risky. In guidance issued earlier this month, the Committee on Safety of Medicines said there was "no rational basis" for prescribing the drug and the CJD Therapy Advisory Group said there was "insufficient data" on its effect and safety. That guidance was being rewritten, the judge said.

The case was conducted in secrecy until judgment was delivered yesterday. During the hearing, a neurosurgeon had agreed to give the drug but his hospital trust refused to allow the procedure to be performed on its premises. The families have now asked the Department of Health to help them find another hospital where they can have the treatment.

In her judgment, Dame Elizabeth, president of the Family Division, said the fact that both children were still alive was "a tribute to the outstanding care they receive at home". She said: "In each case, the children have changed from normal, energetic teenagers into helpless invalids lying in bed and with a severely limited enjoyment of life. Both families are very well informed about the disease and the proposed treatment and its risks and benefits."

In the absence of other options, giving the treatment would be neither irresponsible nor unethical, she said. "Where there is no alternative treatment available and the disease is progressive and fatal, it seems to me to be reasonable to consider experimental treatment with unknown benefits and risks, but without significant risks of increased suffering to the patient, in cases where there is some benefit to the patient."

However, the ruling does not mean the NHS is required to provide the treatment. David Body, of Irwin Mitchell solicitors, who represented Mr Simms, said: "It's been declared that it's in the best interests of the patient that they should be treated, but no one has been compelled to treat. We have a neurosurgeon who is prepared to operate but his trust has refused ... we are now looking around for another. We have indications from the Department of Health that they are prepared to assist us."

Pentosan is not a new drug – it has been used in the US to treat bladder and bowel inflammation – but it has no licence in this country. Research on animals shows it may slow the progression of vCJD but it cannot provide a cure.

Referring to the Simms family, Dame Elizabeth said: "They would rather have [Jonathan] in his present condition than not at all and believe it is a worthwhile risk."

Don Simms, Jonathan's father, said: "We have come a long way from where we started and now it is up to the Health Secretary, Alan Milburn, to get my son treated ... My son remains quite strong, although certainly there is a neurological difficulty. If we had got this treatment sooner, it would not have been so great. We regard this treatment as experimental but it is not experiment for experiment's sake."

The Department of Health said it had "every sympathy" with the families and said it was trying to find suitable facilities for the treatment.