MND-SMART - the participant's perspective

Alan, pictured here with his wife Beverley, received his formal diagnosis of MND in January 2017. You can read more details of his MND story from an interview he gave to one of our funders, MND Scotland, in 2018. (link opens in new window)

"When the MND-SMART trial announcement was made in January it represented a major milestone for people living with MND throughout Scotland and the rest of the UK. It gave me much needed hope and belief that a cure will be found. I felt very honoured and privileged to attend the Edinburgh clinic as the first patient to start the trial.

The first two appointments I had were conducted face-to-face, and were quite intensive. The first appointment involves a number of tests (e.g. blood tests, ECG) that enabled the trial team to verify that it would be safe for me to take the trial drugs. The second appointment was used to establish a baseline of my MND progression. After that I was randomly assigned a treatment, with the possibility of receiving one of the two active drugs being tested or a placebo.

Setting realistic expectations was a key aspect of the first appointment, with the trial team stressing that:

• There was a 66% chance of being randomly assigned an active drug, or a 33% chance of being assigned a placebo
• The goal of the trial is to slow down or stop the progression of the MND. It’s highly unlikely that any of the treatments being tested could reverse existing symptoms.
• I should not expect instant results, the benefits of any treatment will be identified over the full duration of the trial
• There is the potential that some participants will experience some side effects

Despite some of these considerations, I still felt a renewed sense of hope. This was due to the potential of receiving an active drug treatment, and recognising that I’m playing a small part in the search for a cure.  Even a negative result for a specific treatment will move the scientific research forward.

The drug was provided in liquid form with my treatment dosage built up over a four week period, in order to minimise the risk of side-effects. Anytime my treatment dosage was increased it was always fully discussed and agreed with a doctor from the trial team.

Maintaining a daily diary sheet is a key task for participants, this is used to log details of the dosage taken, and any significant or noteworthy health events. Thankfully it only takes a minute to complete.

Since the initial appointments all follow up meetings have taken place from the comfort of my home via video call, and additional drug supplies have been delivered to my home via courier. These remote processes were put in place at the start of the trial, but they have become even more valuable in today’s COVID-19 environment.

Having only started the treatment in March it’s too early to talk about any benefits. I have encountered a few minor side effects, but these were manageable.

Throughout the first few months I’ve been extremely impressed by the trial team, they are highly organised and very professional."