Our Second Strategic Priority for MND Research in Focus: Accelerating New Treatments

10 July 2023

In the next article of our Strategic Priority series, we’re diving into our second priority, Accelerating New Treatments. We identified this as a key area requiring investment in our recently-launched research strategy, Catalysing a Cure; find out in this article why it is key to helping us realise our vision of a world free of MND.

People living with MND have very few options

Nearly three decades since the approval of the first treatment for MND (riluzole), it remains the only licensed treatment for people living with MND in the UK. Drug discovery has been challenging and efforts have been hampered by significant underfunding compared to other diseases.

In recent years, MND has been catapulted to the forefront of all our minds, thanks to several high profile people sharing their journeys following their own diagnosis, including our late founder, Doddie Weir OBE. In addition, the United2EndMND campaign spearheaded by a passionate group of people living with the disease successfully campaigned for a £50m investment into targeted MND research from the UK government over the next 5 years. Furthermore, our knowledge and understanding of the underlying biology of MND has improved exponentially and for the first time, we are hearing the results of drug trials that have not only slowed the impact of disease but, in some people, improved their symptoms.

MND research is continuing to gain momentum, meaning we are now in a greater position than ever to catalyse the change that will lead to more treatment options being available to people living with MND within years, not decades.

Building a diverse treatment portfolio

MND is a complex disease with more than one single cause and many different symptoms that affect individuals in different ways. There is unlikely to be one approach that will effectively treat everyone living with MND. In the same way we treat breast cancer differently to blood cancer, and different types of breast cancer are treated differently, we must consider MND as more than one disease.

The Foundation is committed to leaving no stone unturned in our search for effective treatments. We will take a ‘portfolio approach’ to investing in a diverse range of repurposed, novel and innovative drugs for MND. By supporting several different approaches, we will increase our chances of identifying those that may benefit people living with MND, and we protect the community from too many set-backs.

Investing in every stage of the drug discovery and development pipeline

Last year, we undertook a collaborative exercise to identify research areas that were important to the MND community. The priority areas that were identified could be grouped into different stages of the drug discovery and development pipeline, so we plan to invest at every stage of the pathway from identification and validation of new targets, pre-clinical safety and efficacy testing, experimental medicine and clinical trials. We’ll work with our advisors to ensure we are investing our funding where it will have the most impact for people living with MND.

We have already supported several projects throughout the drug discovery pipeline. These include:

Investing in the development and optimisation of novel gene therapies to treat MND.
Supporting the pre-clinical testing of a repurposed drug, terazosin, and providing follow-on funding for TRUST, the first clinical trial to test the drug in people living with MND.
Investing in TRICALS clinical trials at sites across the UK; our funding has enabled recruitment to key posts at leading institutions to ensure that people living with MND have access to a diverse range of trials.

Going beyond funding

We are committed to doing more than just funding research, and will also provide non-financial support to projects that show promise. We will work with our researchers to ensure they have the right tools and connections in place to progress the research further. This could involve leveraging further investment and support and/or introducing researchers to the right people who can effectively support the research going forward. We have relationships across the international MND research community and we will use these to catalyse new collaborations where they can accelerate the development of new treatments.