Testing new treatments in clinical trials

The MND Research Institute have three programmes to test treatments in trials; EXPERTS-ALS, MND-SMART, and TRICALS.

(i) EXPERTS-ALS

Potential treatments (see Identifying possible drugs to treat MND) that are already in use for other conditions are called repurposed drugs. These and novel drugs that have been through safety testing need to be prioritized for large scale studies, but knowing which ones to test first can be tricky. We know that a biomarker, neurofilament-L (NFL), is released when nerve cells are damaged, and can be measured in the blood. We also know that a drop in NFL means an extremely effective therapy. If there is no change, that does not mean the drug does not work, just that the effect is not so dramatic as to greatly slow MND progression, but it may still have an important effect in slowing down MND. The EXPERTS-ALS trial tests possible treatments in about 20 to 60 people over 6 to 9 months open label (ie no placebo arm), measuring the NFL level a regular intervals. Any drug that lowers NFL is given maximum priority for large-scale trials.

What we gain from this:

EXPERTS-ALS is globally unique and is a very fast way to prioritize new treatments for large-scale trials.

Testing new treatments in clinical trials

(ii) MND-SMART

Based in our Edinburgh centre, the MND-SMART trial is a flagship national clinical trial designed with patients in mind. It has broad inclusion criteria, so that most people are eligible, and recruits at many centres, so that it is highly accessible to people with MND who might not live near a large research centre. All drugs are liquid based, there is twice the chance of being on active treatment as on placebo, and crucially, the trial is a platform adaptive design. That means several treatments can be tested at the same time, and can move from Phase 2 to Phase 3, greatly increasing the speed at which new drugs can be tested.

What we gain from this:

MND-SMART is a rapid, patient-friendly way to test new treatments.

(ii) TRICALS

The TRICALS clinical trial system is pan-European and uses standard training and tools, so that new trials are quick to set up. It includes both academic-led and commercial clinical trials, and being multinational, can recruit large numbers quickly. Because it does not have a fixed protocol, different trial designs can be incorporated. It also uses an inclusion system that means about 80% of people are eligible to take part.

What we gain from this:

TRICALS is a multinational clinical trials system that includes commercial and academic-led trials, testing treatments at Phase 3.