Cure CJD Research

Creutzfeldt-Jakob disease (CJD) is a fatal brain condition - there is currently no cure. It is one of a group of degenerative brain disorders caused by misfolded prion proteins, which destroy brain cells by starting a chain reaction with other prions.

Other similar conditions include Gerstmann-Straussler- Scheinker (GSS) and the human form of Bovine Spongiform Encephalopthy (BSE). CJD is the most rapidly progressive type and usually leads to death within 1 year of onset of illness.

Normally, the immune system recognises foreign proteins and other parts of germs as being alien to the body and this leads to the body producing specific antibodies tailored to fight that infection. Since prions are formed from one of the body’s own proteins they are not recognised in the same way by the immune system and lifesaving antibodies are not produced.

The team at the the MRC Prion Unit and the National Prion Clinic (NPC) have developed a drug PRN100 which potentially could prevent prions destroying the brain cells. If treatment is started early, before symptoms have started, the infection may be cured.

Laboratory tests have provided a “proof of principle” that PRN100 works by preventing the chain reaction and the formation of new prions, but there are still many challenges for patient treatment. This includes understanding what the potential side effects could be.

Professor John Collinge who leads the NPC comments “Given the lack of any alternative treatment for a disease that is rapidly and invariably fatal, the research, patients, relatives and colleagues feel that PRN100 should be offered to patients with rapidly progressing CJD.”

Professor John Collinge FRCP - Professor of Neurology and Head of Department of Neurodegenerative Disease, UCL Institute of Neurology

“Unfortunately we don’t have government funding for PRN100, but we are committed to proving the concept and rolling it out to our patients. We launched the Cure CJD Campaign in June 2016 and have been overwhelmed by the levels of monies raised. 

We would like to start treatment for those at the beginning of sporadic CJD which we are hopeful will demonstrate the efficacy of the drug. We will not stop until this horrific illness is cured once and for all.”

Professor Simon Mead, consultant neurologist and Clinical Lead at the NPC supported Diana during her last few months. He was instrumental in advising and recommending the palliative care for Diana.

“Diana had an extremely severe strand of GSS – her decline was rapid. I first examined her in April 2016, although it was clear she understood the medical questions it was obvious to me that she was going to pass away within a few months.”

“Lets work together to ensure that Diana's legacy is making GSS a thing of the past."

Professor Simon Mead FRCP - Programme Leader, MRC Prion Unit, UCL Institute of Neurology, Clinical Lead, NHS National Prion Clinic