Hope builds for family of CJD sufferer after new drug treatment

DON SIMMS does not want to be seen as a crusader. But there are those from Taiwan to the United States who may disagree.

The lives of the Belfast man, his wife Karen and their six children were turned upside down more than 18 months ago when their son Jonathan was diagnosed with the human form of BSE, vCJD.

Jonathan, then 17, had only a year to live, the family were told. Experts said there were no drugs available to arrest what would be a slow and painful journey to death.

But the Simms had, according to Don, two choices: "Lie down and let this evil consume us or get up and fight." There was only one answer.

Today, Jonathan is still alive and, uniquely for a vCJD victim, showing signs of recovery.

Don puts it down to his son's fighting spirit but more importantly the use of an experimental compound that was only dispensed after the family fought the prevailing medical opinion all the way to the High Court in London.

Fourteen months ago, they heard about a drug compound that could help Jonathan. But the experts said Polyphosphate Pentosan (PPS) a compound used for 40 years to treat inflammation of the bowel was too dangerous.

"At that time, my son could walk, talk and feed himself," said Don.

The family spent nearly a year fighting the prevailing opinion before the High Court ruled that its use was both lawful and in the best interests of Jonathan.

By that stage last December, Jonathan, was in a near vegetative state and at one point was given only days to live.

Six months after being given his first injection of the compound into his brain, there are signs his decline has slowed and, even more remarkably, that at least some of his brain stem function, attacked and destroyed by a combination of prions and proteins, is recovering.

Jonathan has spoken single words and his gag reflexes, the weakening of which led him to choke on his saliva, appear to be improving. And there have been no side effects so far.

His own doctor. GP Mark McClean, is now asking: "Is he going to be the first to beat the disease?"

Don adds: "We are optimistic but guarded. We cannot be carried away on a tide of euphoria but he's a strong young man with a fighting spirit," he says. Don and Karen are now being approached by relatives of sufferers from across the globe.

People from Taiwan and the US have contacted the family at their home in north Belfast, said Don, who has three daughters aged 17, 15 and two and twin boys aged 10.

"Because relatively little is known about this disease, that's why we know so much? But I don't want to be seen as a crusader or a media star," he says.

PPS is a blood thinning compound injected into the brain via a catheter. In the case of vCJD its role is to attack and stop the production of prions and proteins, an overload of which eats away at the brain's vital functions.

The radical treatment has never been used to treat vCJD. Don, with the support of a handful in the medical profession but against the advice of the majority, describes the decision to go with the treatment as "jumping from a high point into the unknown".

It took a year between taking that decision and being allowed to go ahead with the injections. What if Jonathan was given them when he was still walking and talking?

Don is reluctant to publicly name either individuals or agencies who blocked his son receiving this treatment.

For the same reason he is reluctant to put into words his feelings about the delay. But deep anger hardly covers it.