Three screened and plenty more candidates to try

Our third drug candidate ropinirole does not lower NFL levels in the EXPERTS-ALS model. We are hugely grateful to those people living with ALS who made this rapid drug screening possible through their commitment. After 30 years of drug trials in ALS, while this is another disappointment, the EXPERTS-ALS screening process is greatly speeding up the ‘train’ to inevitable success. Another drug candidate is being readied for the platform and progress continues.

The EXPERTS-ALS ‘engine’ is working even more efficiently than we hoped. In 18 months, and with about 150 participants, it has been possible to screen three drug candidates for their ability to lower NFL levels. Their failure to lower NFL tells us these drugs do not have a major effect on slowing down the core disease processes in ALS.  Before EXPERTS-ALS, the only other option to test these drugs would have been three much larger and longer studies. Such studies are still the gold standard to prove clinical benefit to patients, but they need hundreds more participants, everyone taking study drug for up to 12 months. These studies take several years from start to end, and each cost millions of pounds. EXPERTS-ALS offers enormous time efficiency and money savings, ensuring only the most promising drugs, those showing a strong human signal of likely longer-term benefit to people living with ALS, are prioritised for larger investment.

Yes. The drugs picked for EXPERTS-ALS go through a very rigorous and detailed appraisal process. A panel of scientists, including pharmaceutical industry experts, considers the totality of the published laboratory evidence and whether the drug will reach the brain in adequate concentrations. All candidates going forward must have a satisfactory safety profile from prior use for at least six months in humans. This safety requirement makes so-called ‘repurposed’ drugs (those that already have a licence for human use in another disease), the group to find candidates from more quickly and there are many candidates still to try. Meanwhile, we are talking regularly to biotech companies about bringing their novel drugs to EXPERTS-ALS too.

Failure is not an option in EXPERTS-ALS! Blood samples from participants are stored for our many science teams across the UK to develop new biomarkers and explore novel targets. The analysis model itself is breaking new ground, garnering interest from international ALS centres keen to do similar studies. Whatever the outcome of each drug testing in EXPERTS-ALS, new knowledge is acquired and the world moves closer to the goal of better therapies for ALS. Onwards and upwards.