“The annals of ALS clinical trials is strewn with failed studies. Only two out of more than 70 clinical trials have been positive, and even these showed only very modest benefit. Is this dismal record strictly due to the extraordinary complexity of neurodegenerative disease in general, and ALS in particular? Or is it due to methodological flaws that could be repaired?”
Robert G Miller, Professor of Neurology, Stanford University
Although there is not much we can do about disease complexity, improving the way treatments are trialed is something that can be achieved. Imagine a world without clinical trials, where independent companies or individuals would be allowed to sell their self-made ‘drugs’ without any evidence that they were ever used on anyone with the disease, let alone that they would improve one’s condition. No one would know what the drug is (which could simply be a water solution), how it works and whether as soon as the drug is taken, we would be poisoned.
Thankfully, this is not the case and clinical trials, although not perfect, are considered the gold standard for approving any treatment. However, there are still some improvements that can be done to make trials easier to access and provide more accurate estimates of drugs’ effectiveness much faster.
Each year, the MND Association dedicates the month of June to raising MND awareness. This year, we focus on the eyes – in most people with MND the only part of their body they can still move and the only way left for them to communicate. Alongside the Association-wide campaign, the Research Development team selected six most-enquired about topics, which we will address through six dedicated blogs.
In our previous article we introduced four MND researchers who gave us an insight what a typical day in the life of a researcher looks like and what carrying out a research study actually involves. In this continuation article, you will get the chance to look into the lives of four PhD students, who give us an overview of their projects and their usual daily duties.Read More »
To mark International Clinical Trials Day (20 May) we reflect on the ALS Clinical Trials Guidelines workshop that took place in March. The MND Association co-sponsored this successful meeting, held at Airlie House in Virginia USA. Approximately 140 delegates from across the world attended, including 11 MND researchers and doctors from the UK.
Why was this meeting held?
The meeting was a key stage in the process to update (and improve) international guidelines for clinical trials in amyotrophic lateral sclerosis (ALS, the most common form of MND).
The first international ALS clinical trials guideline workshop took place in 1998. The guidelines were designed to improve the quality of clinical trials in ALS, and provide evidence based recommendations to those designing and carrying out all stages of clinical trials.Read More »