An experimental drug given to a single person can’t prove much about that drug’s efficacy, but the results of an experimental drug given to a young vCJD sufferer are nonetheless intriguing and will hopefully spur further study.
Jonathan Simms, 18, had to sue Great Britain’s National Health Service to win the right to take pentosan polysulphate. The drug had never been used before in human subjects, and the NHS had refused to let Simms’ doctors give him the compound. A court overruled the NHS, saying that since Simms would certainly die anyway without the drug, that he essentially had nothing to lose.
Pentosan polysulphate is usually used for treating bladder pain, but researchers in Japan and the United Kingdom demonstrated that in mice the drug extended the incubation period of vCJD-like drugs and extending the length of time the animals survived once they were infected.
Since Simms began taking the drug in January 2003, he has experienced no side effects from the drug and has shown improvement in his condition.
New Scientist quoted microbiologist Stephen Dealler as saying,
I was really taken aback by this. At this point there have been significant physiological improvements and no side effects.
Don Simms, Jonathan’s father, told a BBC documentary,
I can categorically state that Jonathan has not got any worse. He in actual fact shows signs of improvement. We are not hailing it as a total success, but from what we have seen so far we are much encouraged.
Simms told an Irish news web site that his son’s pulse rate has returned to normal and “the salivating associated with vCJD has dramatically decreased and he looks much better.”
Source:
Patient benefits from controversial vCJD drug. NewScientist.Com, May 12, 2003.