There was an interesting development yesterday in the UK with Matt Hancock, Secretary of State for Health and Social Care announcing a mass clinical trial of Inclisiran, a cholesterol lowering drug. Initially the trial is for 40,000 patients with twice yearly injections of the gene silencing drug. The injections are believed to half levels of bad cholesterol in under a month thereby reducing the risk of heart disease.
The treatment is expected to be approved by the European Medicines Agency (EMA) later this year and to be available to approximately 300,000 patients annually from next year in the UK. Studies suggest the drug will save 30,000 lives within 10 years in the UK.
This trial is as a result of collaboration between Novartis, the UK Government and the National Health Service (NHS) in an attempt to speed up access to breakthrough treatments.
Health Secretary Matt Hancock said “I'm determined to find ways to save as many lives as possible, and to do my best to stop terrible conditions like heart disease from taking people from their family and friends far too soon. “
What could this imply for sufferers of Motor Neurone Disease (MND)?
The above establishes that the UK is willing to conduct mass trials for up to 40,000 patients without a placebo before a treatment is approved by the European Medicines Agency (EMA) or the Medicines and Healthcare products Regulatory Agency (MHRA) or the National Institute for Health and Care Excellence (NICE).
There are approximately 5,000 MND sufferers in the UK. A clinical trial without a placebo for all 5,000 MND sufferers would be welcome news and potentially lifesaving.
What can you do?
Please raise the issue with your MP and local MND Association requesting that they advocate for early access to treatments and that they work towards a similar clinical trial for MND. Basically, a clinical trial without a placebo for all sufferers of MND using drugs / treatments that are in advanced phases of clinical trials and the FDA / EMA approval process.
The treatments that have made most progress in the FDA approval process or should perhaps be reviewed for early access include:
1- NurOwn by Brainstorm Cell Therapeutics - FDA Phase 3 Clinical Trial
2- CuATSM by Collaborative Medicinal Development (CMD) – FDA Phase 2/3 Clinical Trial
3- Regulatory T cells (Tregs) is a treatment being developed at Houston Methodist Hospital - FDA Phase 3 Clinical Trial
4- NeuroNata-R by Corestem - Licensed in South Korea / Phase 3 Clinical Trial
5- Bone Marrow Mesenchymal Stem Cells (BM-MSCs)
The BM-MSCs is a procedure that many MND sufferers are having done abroad but is currently unregulated in the UK (BBC, 12 January 2020). However, it is already performed by orthopaedic surgeons and available on the NHS for conditions other than MND. It also comprises the early part of the NurOwn treatment process. Hence, as a new treatment it (BM-MSCs) appears not to currently fall under the regulation of MHRA or the Human Tissue Authority (HTA) but is permitted under existing EU Directives (BBC, 12 January 2020).
The above list of drugs / treatments that could potentially be included in such a proposed clinical trial for MND sufferers is expected to increase with time as more approach phase 3 and the approval process or are reviewed for early approval via Accelerated Assessment / Access by FDA/ EMA / MHRA / HTA / NICE, where applicable.
(1) http://www.essexctc.nhs.uk/media-centre/1222-stem-cell-op