Photo: courtesy Medical Daily

Let’s face it, when you are feeling crummy all you want to do is be quiet, rest and not have to deal with anyone else. So, it’s not surprising that a new survey of people with primary mitochondrial disease (PMD) found that many were often less than enthusiastic about taking part in a clinical trial.

It’s not surprising because PMD, caused by problems with the mitochondria which provide energy within our cells, can lead to a wide variety of debilitating conditions including muscle weakness, visual problems, hearing problems, heart disease, liver disease, kidney disease, gastrointestinal disorders, breathing problems, neurological problems and dementia. Any one of those is bad enough, but if you combine several you can see why it would be hard for a person with PMD to get to a clinical trial site for an experimental therapy.

That’s unfortunate because right now there are no effective treatments for PMD so it’s vitally important that people take part in clinical trials that might lead to new therapies.

Obstacles and opportunities

Fortunately, this study, published in the journal PLOS One, did more than just identify the barriers to taking part in a clinical trial, it also identified some strategies to overcome those barriers.

The barriers included not just the individual’s state of health but also:

• Requiring patients to discontinue current medications
• Daily blood tests
• Requiring patients to pay for the cost of the clinical trial

Ways to encourage increased participation include:

• Direct communication with a physician involved in the trial
• Better education and outreach to people with PMD
• Working with patient advocacy groups

The study says this last point in particular is extremely important.

“We propose widespread, coordinated efforts that involve PMD patient advocacy groups to organize community education sessions that clarify the components and need for efficacious clinical trial design.”

CIRM CAP meeting

This is something that CIRM knows a lot about. Whenever we fund a clinical trial – or, in some cases a late stage pre-clinical program – we create a Clinical Advisory Panel (CAP) to support it. Each CAP consists of an independent, outside expert in whatever disease the trial is targeting, a CIRM Science Officer, and a Patient Advocate. The Patient Advocate plays a vital role in making sure this project works.

Researchers know the science, but the Patient Advocate knows what it is like to live with the disease and the limitations it may impose. They can help guide and advise the researchers on how to design a clinical trial that works for the patients and makes it as easy as possible for them to be part of the trial.

In the last few years we have created 68 CAPs, ensuring the voice of the patient, and the needs of the patient, are front and center in everything we do.

The easier it is for the patient, the easier it will be to recruit people for the trial and the more likely it is they will stay with the trial to the end. It won’t guarantee the therapy will succeed, but it gives it the best possible chance.