Creutzfeldt-Jakob disease future or investigational therapies

As of 2007, there is no cure for CJD, a fatal disease, and the search for viable treatments continues. An experimental treatment was given to a northern irish teenager, Jonathan Simms, in January 2003.^[1] The medication, called pentosan polysulphate (PPS) which is used to treat interstitial cystitis, was infused into the patient’s lateral ventricle within the brain. PPS did not seem to stop the disease from progressing. However, the treatment is alleged to slow the progression of the otherwise untreatable disease, and may have contributed to the longer than expected survival of the seven patients that were studied.^[2] The CJD Therapy Advisory Group to the UK Health Departments advises that data are not sufficient to support claims that pentosan polysulphate is an effective treatment and suggests that further research in animal models is appropriate.^[3] A 2007 review of the treatment of 26 patients with PPS finds no proof of efficacy because of the lack of accepted objective criteria.^[4]

Scientists have investigated using RNA interference to slow the progression of scrapie in mice. The RNA blocks production of the protein that the CJD process transforms into prions. This research is unlikely to lead to a human therapy for many years.^[5] In 2013 Karapetyan et al reported that astemizole, a medication which has already been approved for human use, has been found to have anti-prion activity and may lead to a treatment for Creutzfeldt-Jakob disease.^[6]

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