The best road to a cure – the importance of following the science

Following the news at the end of 2021 that the UK Government has committed an historic £50m over the next 5 years to targeted MND research, we thought it timely to reiterate the ground breaking research plans which underpinned our campaign.

Because of the ‘very targeted’ project aims we also feel it important to be very clear about what these specific funds will NOT be spent on.

Patients want a laser focus on the acceleration towards disease halting treatments and eventual cures. The UK’s world-leading research scientists understand this and believe the ‘time is right’ to set up a coordinated translational research infrastructure. Along with us, they submitted a detailed work plan to government to achieve this.

Recently, we described the principles governing the new funding and the upcoming delivery challenge. Here’s why we believe the researcher’s plan is the most efficient, timely and beneficial way forward for all of us living with MND and our families

The plan sets out that the £50m will be allocated to help establish a ground breaking institute focused solely on the discovery and delivery of new treatments by:

1. Creating better and more measurable trials making it easier for patients to participate and to prove whether treatments are working.

2. Establishing and maintaining a world leading, on-demand, re-usable trials platform that that will be sought after by the major international pharmaceutical companies because of technologies, efficiencies and cost benefits.

3. Ensuring that findings from each and every trial (even if unsuccessful) are learned from for future trials.

4. Building an integrated drug discovery process leading to an almost continuous stream of new drugs/treatment candidates.

5 Universities and 22 research centres are already planning to pool resources and work as a cohesive group.

Most importantly, a primary aim of the plan is to offer every newly diagnosed patient a place on a trial and wider availability of trials for those already diagnosed.

These trials will utilise exciting new compounds, one or more of which, we all hope, will start to bring an end to this disease. These are exciting times for all of us living with MND.

What will the funds not be used for?

This research investment will NOT be used to:

1. Fund widespread access to experimental drugs that are, as yet, unproven. We will cover this vitally important and topical subject in our next post*

2. Fund MND research, important as it is, related to quality of life and care related treatments. These will continue to be funded by existing mechanisms eg NIHR, charities and industry.

3. Fund innovative, pioneering, perhaps high-risk new avenues of research. Experience shows that these are vital areas of research which will continue to be funded by the UK government’s MRC standard grant processes and by contributions from charity and industry.

*In our next post we will discuss how viable promising experimental treatments might be funded, in the UK, to provide the earliest potential access to patients. We also consider the consequences of not following regulations and scientific rigour.