Forging ahead – developments at the UK MND Research Institute

As we enter the summer period we thought you might like to see the following updates from the UK MND Research Institute (UKMNDRI) that you might have missed.

The UK MND Research Institute is in its first full year of operation and work has been moving ahead at speed, although we fully appreciate that to patients, and our families, the pace can never appear fast enough.

First, the Institute has its own website. This explains the various work packages, and why they are important, in user-friendly language. It’s well worth reading and bookmarking, so you can come back for updates.

For the purposes of this post, we’d like to highlight the activities we hope, through coordination and collaboration, will change the face of research in the UK and globally.

A brief reminder of the institute’s key aims:

• Understanding the patient and disease process.

• Identifying new drugs with therapeutic potential.

• Testing new drugs on innovative platforms.

Here are just some snippets of the work so far, starting with Understanding the disease process.

Improvements in measuring the progression of ALS

Researchers, supported by the UKMNDRI, have improved the way in which we use the ALS function rating scale (ALSFRS-R), which is used to measure the severity and progression of ALS.

The scale includes 12 questions that have a score of zero to four, where zero indicates no function and four indicates full function. This provides a total score out of 48.

It is useful for diagnosing people with the disease, measuring how our disease is progressing, and can also be helpful for selecting participants for clinical trials.

However, the scale is known to have some issues, and researchers have been looking at ways to improve the scale so that the results are more accurate.

You can read the full post on the UKMNDRI website here.

Involving we patients in understanding the disease

An app is being developed, to be used by patients, to help researchers with the natural history of our disease. We all know how patients vary in their disease course and the app will help aid designing future trials and might reveal risk factors. It is called Telehealth in Medicine (Research) and you may hear it referred to as TiM-R. Watch out for news on this app. It will be a user-friendly way for you to help people who are trying to help us find a cure.

What else has been going on since the foundation of Institute?

As with any complex task there is a lot going on, often what we would call ‘grunt’ work. It’s not all newsworthy, but vital. Have a read of the Institute’s history to date including

• A UK Researcher buddy scheme that links patients with researchers.

• Biomarker sampling.

• Developing cell lines to aid drug discovery.

The MND Association has also published an excellent paper on the subject of translational research, which is at the very heart of the UKMNDRI. 

The paper explains the area in some detail with many reference projects, including the first showcase project of the Institute, EXPERTS-ALS, the world’s first ALS/MND drug ‘screening’ platform in humans. EXPERTS-ALS is part of the third pillar of the Institute’s research, Testing new drugs on innovative platforms.

We have previously covered some of the exciting work going for EXPERTS-ALS here and we will continue to update you on this innovative, and globally groundbreaking, project as it looks to recruit it’s first patients this year.

Have a good summer, whatever your plans and activities. Please follow the UKMNDRI web site for the very latest updates.

#united2endmnd