Our main objective at the institute is to work together to accelerate the search for a cure for MND. We urgently need new treatments for the disease, which means identifying and testing them in clinical trials.

Central to this is a concept known as “translational medicine”. This important and collaborative discipline aims to get new treatments to patients as quickly and safely as possible. But what exactly does this involve? And why is it such an important part of our research?

What is translational medicine and why is it important in MND research?

MND is a progressive and currently incurable disease, which is why a speedy translation of basic science to much-needed new treatments is essential. But research takes time, so we must make an organised effort to coordinate and speed it up.

Translational medicine can help with this. It usually starts with basic science, which is an essential foundation of research. This involves trying to understand the biological mechanisms and genetic factors that are involved in a disease like MND. These experiments usually take place at the molecular or cellular level to identify new targets for treatment.

Once a new potential treatment is identified, the next step is making sure it is safe and effective, while also better understanding how it works. This is known as preclinical research and is usually conducted in the lab or animal models. The objective of this stage is to test whether the new treatment is promising enough to go on to be tested in clinical trials.

Clinical trials are where the new treatment is tested on human participants. These trials are typically split into three phases:

  • Phase 1 – this stage involves small-scale trials that test the safety, dosage and side effects of treatment in a small group of healthy volunteers.
  • Phase 2 – these trials are larger than Phase 1 and evaluate the effectiveness of a drug and further assess safety.
  • Phase 3 – after success in phases 1 and 2, the new treatment is compared to the current standard treatment in a large group of patients.

After a new treatment is found to be safe and effective, it is then submitted to regulatory bodies to be approved. In the UK, this is the MHRA. They review the data and ensure the treatment is safe and effective for public use, and all research and trial data are rigorously assessed.

Once the treatment is approved, it can then be implemented into clinical practice and healthcare professionals will be taught how to use the new therapy. This also involves assessing how the treatment works in the real world, which is known as post-market surveillance, and involves monitoring the long-term safety and effectiveness of the drug.

This can be a long and expensive process, which is why it is so important that we ensure the search for new patients is as efficient, safe and fast as it can be. So that it can reach people with MND and make a difference.

What are we doing?

Translational medicine is the key to accelerating the search for a cure for MND, but how are we using it in our work? Our research programme takes what we learn from our patients into the laboratory, using that knowledge to find new treatments, and then test them in clinical trials. We’ve split our work into three different pillars of research, to make sure it is efficient as possible.

1.     Understanding the patient and disease process

Within this pillar, we have three different research programmes that are aimed at understanding MND in more detail.

MND register and biobank – Here, two MND registers record everyone with MND in the country (one in Scotland and the other in the rest of the UK) and clinical information and blood samples are collected. These samples are vital as they provide DNA for genetic research, proteins for biomarker studies as well as a wide range of other molecules that are useful for research.

Telehealth in MND Research (TiM-R) – TiM-R is an app that will allow us to collect clinical information and then let researchers send questionnaires to people living with MND to learn more about their experiences. This information helps us design better clinical trials by seeing how environmental and lifestyle risks affect whether someone develops MND. This could help us better test and define new treatments. TiM-R also allows people to join the MND Register and easily contribute samples.

ALS Biomarker Study – This research project is aimed at understanding more about what causes ALS (the most common form of ALS) and what causes it in the first instance. The study is specifically looking at identifying biomarkers that can speed up the diagnosis of MND, improve ways we can monitor its progression and find new and more effect therapies.

Trajectories in Outcomes and Neurological Conditions (TONiC) – This study recruits people nationally to fill in questionnaires to improve outcomes for MND. This has already had an impact on the improvement of the ALS Functional Rating Scale. You can read more about how we improve the ALSFRS-R here.

2.     Identifying possible drugs to treat MND

We also have two programmes that are aimed at developing new drugs for MND. These work with other projects both in the UK and across the world.

Cell models – Cell lines are another essential part of research. They are used to explore how particular genes or pathways are linked to MND and how they could be targeted with new treatments.  Our researchers have developed a network of cell lines across multiple labs, using the same sets of cells and using the same methods.

Developing possible drugs – These new cell lines are also fed into a project that is looking to identify new molecules that could be as treatments or genes and proteins as targets. They are narrowed down depending on the way they’re taken or their side effects. This generates a whole new range of drugs that could be tested in trials.

3.     Testing new treatments in clinical trials

And finally, we have programmes that are looking to test newly found treatments in clinical trials. We have designed these trials to be as efficient and accessible as possible.

EXPERS-ALS – Some drugs that are already used for other conditions could be used to treat MND. These are called repurposed drugs and EXPERTS-ALS is a trial that is testing these possible treatments in about 20 to 60 people over 6 to months. This provides a potentially faster way of finding new treatments for large-scale trials in MND.

MND-SMART – Many of our researchers are also helping with the MND-SMART trial, which is a national clinical trial that tests several treatments at the same time, greatly increasing the speed at which they can be tested. It also has broad inclusion criteria and many different centres, making it highly accessible to people living with MND.

TRICALS – This clinical trial system is taking place across Europe and includes both academic-led and commercial clinical trials. Being multinational means that it can recruit people quickly and has an inclusion system that means almost 80% of those who apply can take part. These trials are testing treatment at phase 3.

This is just a quick snapshot of some of the vital work we’re doing in the search for a cure for MND. Make sure to follow us on our social channels and regularly check our website to hear about all our research breakthroughs and updates.

You can read about our other blogs and news stories here or read about our research here.