People living at risk of Creutzfeldt-Jakob Disease (CJD), and other prion diseases, as well as families who have lost loved ones to this brutal brain disorder, joined forces with politicians on Tuesday, July 7th, to unlock funding for life-saving treatment.
Baroness Stedman-Scott paid tribute to her friend Baroness Newlove, who served as the Victims Commissioner for England and Wales for many years. Baroness Newlove passed away in November of last year after being diagnosed with CJD.
The Rt Hon Stuart Andrew MP, Secretary of State for Health and Social Care, gave the keynote address, celebrating the world-leading British research that might finally bring about a cure to this terrible disease.
CJD currently accounts for 1 in every 5,000 deaths in the UK. Most patients die less than a year after the onset of symptoms, and some within weeks of receiving their diagnosis, while others degenerate over several years.
CJD is caused when normal prion proteins become misshapen. These prions spread and multiply throughout the brain, leading to the death of brain cells. This process causes a loss of the normal function of the brain with progressive dementia and problems with speech, movement and balance. Over time, the patient is unable to speak or move, and with no cure available, the patient will eventually die.
But researchers at the MRC Prion Unit at University College London have developed a groundbreaking antibody treatment called PRN100, which has already shown hugely promising results in early-stage testing. Not only could PRN100 provide a cure for CJD, but it could also be used in research for other diseases, including Parkinson’s and Alzheimer’s disease.
Cure CJD, a charity founded and run by people who have been personally affected by CJD and prion diseases, brought together the researchers behind PRN100 and families affected by CJD to meet with politicians at a parliamentary reception in the House of Commons’ iconic Thames Pavilion.
The reception was a crucial step in building the cross-party and cross-industry coalition needed to raise £10 million for PRN100’s clinical trials, the next step in developing a cure.
Professor John Collinge, Director of the MRC Prion Unit, who attended the reception, said:
“CJD and other prion diseases are relatively rare, but devastating, causes of dementia. They are invariably fatal, usually within a few months of diagnosis, with no treatment at present. But there is now a unique opportunity. After over two decades of world-leading scientific research, PRN100, a groundbreaking antibody treatment that has shown extremely promising early-stage results in patients with CJD, could now make a cure possible. With the scientific and clinical expertise in place and the regulatory environment clear, all that is stopping us progressing is the financial commitment to carry PRN100 through to full clinical trials”.
Alice, whose family have been affected by the disease, commented:
“I have known for 5 years that I carry the same gene as my mum and brother that will cause me to develop CJD. In this time, I have lost my mum to CJD and become the sole carer for my dying brother, but the research hasn’t moved on due to a lack of funding.
The research team needs £10 million for the stage 2 trial that could make my brother’s life a little easier, that could have saved my mum’s life, and that could still save mine.
I sometimes daydream about the day PRN100 will become available, the day that ticking time bomb will be removed from my brain. The pure, boundless joy I will feel, the relief, who will I call? What will I do? Dancing in the streets will definitely be on the cards.
Sadly, I also often think that I have run out of time and that my symptoms have started: every time I forget a word, every time I pull a door instead of pushing it, every time I lose my balance.
But there is still hope. If we can get PRN100 through clinical trials, people might never have to live in fear of CJD as I do, or live in pain as my brother does or pass away as my mother did”.
Notes to editor
About CJD
There are three main types of prion disease. The most common form is Sporadic CJD, which occurs at random in the population as an unlucky chance event. It causes 1 in 5000 deaths in the UK. Inherited prion diseases are caused by a faulty gene passed down in families. Acquired forms of prion disease (known as iatrogenic CJD) are rarer and caused by the accidental transfer of prions during medical or surgical procedures, or in the past, from eating food contaminated with prions from BSE-infected cattle. All forms are untreatable and invariably fatal.
The prion protein is a normal brain constituent that we all have. Prions are formed when misshapen prion protein molecules stick to each other, forming seeds that grow into long chains (known as amyloid), which break and multiply, spreading throughout the brain. This process leads to the progressive death of brain cells, leading to loss of the normal function of the brain with progressive dementia and problems with speech, movement and balance. Ultimately, the patient is unable to speak or move.
In the early stages, CJD causes a variety of symptoms, some of which are commonly associated with other conditions, meaning CJD is often misdiagnosed or diagnosed late. Symptoms include personality change, memory loss, impaired thinking, anxiety, insomnia, depression, trouble speaking and jerky movements. These symptoms usually get worse quickly, and the patient often dies within a few months or even weeks after diagnosis. When suspected, the disease can usually be diagnosed with a combination of a brain scan and a test on spinal fluid. A specialist clinical service, the NHS National Prion Clinic, which works closely with the MRC Prion Unit at UCL, can assist with rapid diagnosis and supportive care and is referred most patients with suspected prion disease in the UK.
About the MRC Prion Unit
The MRC Prion Unit at University College London was established in 1997 at the government’s request in the wake of the Bovine Spongiform Encephalopathy (BSE) crisis (also known as Mad-Cow Disease).
Since then, the Unit has spent decades building Britain’s world-leading expertise in prion research, and they believe that they have now finally found a cure for CJD.
Not only could PRN100 cure CJD, but it could also be used in research for other neurological diseases, such as Parkinson’s and Alzheimer’s disease, where closely similar processes leading to the formation and spread of amyloids formed from different brain proteins are central to the disease process.
The conditions to advance PRN100 are uniquely favourable. The majority of CJD patients in the UK have joined the MRC Unit’s long-term clinical research programme, providing a wealth of comparative natural history data that means effective clinical trials can be conducted relatively quickly and efficiently with as few as 20 participants, far fewer than the many hundreds typically required in dementia research. This means the total funding needed is just £10 million, a small fraction of the cost of comparable research in Alzheimer’s disease, for example.