Dr Ruben van Eijk (UMC Utrecht) has been awarded the prestigious Paulo Gontijo Award. He received the prize during the virtual 31st International Symposium on ALS/MND. The award means international recognition for his ground-breaking research into the eligibility criteria for ALS clinical trials.
The prize has been installed by the Paulo Gontijo Institute in collaboration with the MND Association and the International Alliance of ALS/MND for the best ALS researcher under the age of 35. It encourages and recognises outstanding research performed by young investigators.
Improving eligibility criteria
Ruben van Eijk, based at the UMC Utrecht (the Netherlands), is actively involved in TRICALS’ research activities. He received the Paulo Gontijo award for his innovative research that led to a computer model to improve eligibility criteria for people living with ALS. Eligibility criteria are intended to select participants that are most likely to show a response to an experimental medicine. However, in practice, this means that patients who are in an advanced disease stage, or patients with either a very fast or a very slow disease progression, are often excluded.
Ruben van Eijk: “When we compared the details of 2,904 Dutch patients with the eligibility criteria of 38 recent ALS clinical trials, we found that on average 60% of the patients are no longer eligible to participate in trials on the day they receive their diagnosis. The result of these entry criteria is that the majority of people living with ALS cannot participate in a clinical trial, and that drug candidates are tested in a patient cohort unrepresentative of the actual patient population. Importantly, we found that most eligibility criteria are ineffective to select the responsive patient group. This means that many people living with ALS are unnecessarily denied access to trials.”
The computer model uses factors such as age, disease duration, lung function and measures of daily functioning to predict a patient’s disease course. In June 2020, it was endorsed by the European Medicines Agency. By using the computer model instead of standard eligibility criteria, the percentage of people with ALS that can participate in clinical trials can be drastically increased. Ruben van Eijk: “When we apply our computer model to include patients, we can in some cases increase the number of eligible participants five-fold. It is very important that potential medicines are tested in a diverse patient population. This way we can best determine if new therapies are safe and effective, or more or less beneficial in certain subgroups. At the moment, we are preparing several trials with TRICALS that will use computer models to include patients, such as our platform trial. Our ultimate goal is to give every patient the chance to participate in clinical trials.”
van Eijk RPA, Westeneng HJ, Nikolakopoulos S, Verhagen IE, van Es MA, Eijkemans MJC, van den Berg LH. Refining eligibility criteria for amyotrophic lateral sclerosis clinical trials. Neurology. 2019 [Link]