Post by Eric Minikel from Prion Alliance
Link – First details of Ionis trial disclosed
Spanish version link- Primeros detalles revelados sobre el ensayo de Ionis
Please find below links to some general information on clinical trials which may be helpful in the lead-up to the commencement of clinical trials for prion disease:
U.S. Department of Health & Human Services – Participating in Human Research
You may find the videos to be very approachable, and they have a very handy PDF with questions that people should consider asking a study investigator. Many of their resources are available in English and Spanish, which is a nice bonus!
U.S. Food & Drug Administration – Clinical Trials: What Patients Need to Know
Explanations are very basic, but include special pages on Informed Consent and explain the difference between a trial and medical treatment which is very helpful, especially for communities like ours that are relatively new to trials.
U.S. National Institute of Health – Clinical Research Trials and You
This site has some great tools as well, although they may be a little bit more technical that the Health & Human Services ones.
John Collinge (MRC Prion Unit, University College London, London, UK) discusses a first-in-human treatment programme to give PRN100, an anti-prion-protein monoclonal antibody, to patients with Creutzfeldt–Jakob disease; the report is published in the April issue of The Lancet Neurology.
In Conversation with… John Collinge (link to podcast with listing options)
A comparison of Granagard and Metformin as ageing hallmarks in the Tg E200K model.
Here is your chance to help towards a treatment or cure for prion disease. Ionis Pharmaceuticals, a company based in Carlsbad, California US that develops antisense drugs targeting the RNA. In the past, Ionis has successfully developed antisense drugs that treat diseases such as Spinal Muscular Atrophy and TTR Amyloidosis. they are working on doing the same for prion disease.
They are currently undertaking a survey on genetic testing for prion diseases (PRNP testing) and we ask anyone from a genetic prion disease family or anyone who has undergone or considered genetic testing for prion disease to please complete the survey provided in the link below.
Your help would be very much appreciated.