It quickly became apparent to me at the Neuromuscular Centre that their work encompasses numerous conditions and most of them are rare. There are many gaps in the understanding of musclewasting conditions and a lack of treatments. Research is essential for understanding the causes, trialing things that can have a benefit and ultimately in finding effective treatments. Progress often happens in tiny increments and we don’t know whether or not effective treatments will come in our lifetime. Many that I have spoken to are interested in our conditions and for a range of reasons would like to be involved in research projects.
I recently met with David Tucker who is taking part in a drugs trial to look at a possible treatment for the condition he has, Sporadic Inclusion Body Myositis. Research is not always drugs trials, and there are many observational studies.
How did David get onto the research programme? His story began when David moved to Salford Hospital, as he felt that his previous clinic did not have the expertise or knowledge of his condition. On his first visit he was asked if he would participate in a new research project. He readily agreed and within a few weeks he was back to start the process. For many people it is something that they bring up with their Consultant who should be able to link them into current and active research projects.
What was the process? The initial screening determined that David was a suitable candidate for the research. There were a range of criteria and tests, such as a DEXA “dual energy X-ray absorptiometry” scan used to measure bone density, which could have ruled him out at this stage. David matched the qualifying criteria on all counts.
Next came the baseline tests to measure his muscle strength, these have been repeated periodically through the study and measure any changes in David’s strength.
In David’s trial the drug is given as an infusion. Every few weeks David has to travel to Salford for two consecutive days, a round trip of over 160 miles. The first day he arrives and is simply weighed before leaving. He explained that the reason is that the pharmacists take a day to make up the infusion specifically to his weight. While frustrating, David accepts this as essential to ensure the integrity of the study.
This is a major international study and Novartis, the drugs company, will be spending many millions on this research. They willingly pay all out-ofpocket expenses for David and a carer, including travel, food and accommodation. For the first infusion David and his wife stayed in a hotel, however he now prefers to travel daily.
Was the research a positive experience? David had just had his penultimate infusion but he hasn’t noticed any difference in his abilities throughout the study. He isn’t discouraged about this as he explained that he doesn’t know whether he is getting the drug or not. There are four groups of participants, three are receiving infusions in different doses and the last is a control group receiving just a placebo. No one knows, other than those doing the analysis, which group they are in, including the medical staff. This secrecy is so strictly controlled that two different pharmacists are involved in the preparation and the mixing of the infusion so that even they can have no knowledge of the end dose.
I asked what motivated David to be involved in the research. “ I want to help in any way I can, in the hope that it will give faith and be of help to future sufferers.”
Following the end of the trial all the participants will continue to receive infusions at the same dose until the results of the research are confirmed and if the drug is licensed . It is hoped that it will be as soon as next year. They will then receive infusions with the drug at the optimum dose, so hopefully all could benefit directly from participating in the study.
This study has required an enormous time commitment for David, but he is very positive about the experience “Although I think I am on the Placebo I am finding it a worthwhile and very interesting experience being on the Trial and obviously hope that it will benefit myself also.”
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