UK MRC concludes that PPS not effective in vCJD

According to reports on the UK broadcatster BBC's news web site, the drug pentosan polysulphate, "does not stop the progression of variant Creutzfeld-Jakob disease." The conclusion is based on data from the UK Medical Research Council's assessment of the drug in the treatment of seven patients with vCJD, or related degenerative prion diseases. The MRC did concede however, that the drug appeared to extend the life span of patients, citing the case of Jonathan Simms, the UK's longest surviving vCJD patient as an example.

Pentosan polysulphate, which is marketed by US pharmaceutical major OrthoMcNeil as Elmiron, has Food and Drug Administration approval for the relief of bladder pain or discomfort associated with interstitial cystitis. In 1999, the UK Department of Health said that, despite the results of preclinical animal models suggesting that the drug had efficacy against prion diseases related to vCJD, there was not enough evidence to support its prophylactic use in humans (Marketletters passim). As a result, the compound was not included in the MRC's trial of possible treatments.

In 2002 however, the Simms family of Belfast, Northern Ireland, won a decison from the High Court that allowed the use of the compound in the treatment of their son Jonathan, which began in December of that year. Subsequently, other vCJD patients have begun PPS treatment, most notably Holly Mills from North Yorkshire who has achieved 18-months disease stability since the initiation of treatment in 2003, shortly after she was first diagnosed.