Clinical trials: Highlights from Glasgow

This blog is part of the ‘Highlights from Glasgow’ collection of articles, where you can read about the content of some of the talks and posters presented at the 29th International Symposium on ALS/MND.

In the Clinical trials and trial design (4B) session we heard from two speakers looking at ways to improve current design of clinical trials. In his plenary talk, Mahesh Parmar (C20) provided his perspective on the necessity of changes from his experience working on cancer trials, highlighting that any efforts to improve clinical trials should be focused on Phase 3 where the most money and time is spent. One solution that stuck with a lot of clinicians attending Prof Parmar’s talk was the design used in the STAMPEDE trial, a large clinical trial assessing effectiveness of new treatments for people affected by prostate cancer, which has been running since 2005. The innovation of this approach is the ongoing protocol that allows to test multiple treatments within the same established clinical trial, allowing new drug candidates to be tested (relatively) straight away, avoiding the creation of a brand new clinical trial. This design improves efficacy of testing new treatments, systematic approach to testing, and access to a large pool of participants who could take part in multiple treatment trials over time.

Brian Dickie, the Director of Research Development at the MND Association said: “Prof Parmar’s presentation generated a lot of interest amongst clinicians who are regularly involved in MND trials and there was a strong feeling that this is the direction that we need to be taking with MND as it could increase the efficiency and reduce the cost. That said, it will take a while to put the building blocks in place and we certainly wouldn’t want to hold up trials that are already in advances stages of planning, so I would expect to see a gradual introduction of changes to trials design over the coming years.”

You can learn more about the multi-arm multi-stage trial design from Prof Parmar here.

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Lithium revisited: Is there a baby in the bathwater?

At last year’s Airlie House workshop to develop new ALS/MND Clinical Trial Guidelines the focus was, of course, on MND, but there was also important input and learning from outside the field.

One of the most fascinating presentations was from an oncologist who was explaining how detailed genetic analysis of tumours was leading to an understanding of why some experimental cancer drugs appeared to only work in a small subgroup of patients. The take home message from the cancer field was that there should be more effort made in future MND trials to identify and analyse smaller subgroups of patients, in case a potentially positive effect might be missed.

A new research paper, published in the journal Neurology, raises some intriguing findings from the trials of the drug lithium that were carried out several years ago. Lithium generated a lot of excitement when researchers in Italy reported a positive effect of the drug in the SOD1 mouse model of MND. Almost as an afterthought, their research paper mentioned that they had tested the drug in a small short-term trial in patients and it appeared to have some effect.Read More »