According to a story from nature.com, Amber Sapp’s twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne muscular dystrophy. The trial had continued for two and a half years. After all this time, Amber ultimately learned that the drug being tested had failed to have any positive impact on the disease.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, boys are mostly affected, with girls only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.
Social Media and Clinical Trials
While it was upsetting to realize that the trial had wound up being a total waste of time for Garrett, the manner in which she learned that news made the entire situation more disturbing. Amber had not been given this information by clinical trial officials; instead she learned about it via a Facebook post from another parent.
Many parents whose kids were a part of the trial regularly communicated over social media about the progress of the study, as well as how their kids were doing. Social media has begun shifting the landscape around trials, with some trials even being formed through collaboration on such sites.
Regardless, the willingness for trial participants to share info about the trials has some researchers concerned about trial integrity. Discussing info like side effects and who is in the control group could potentially alter trial results. As patients can choose to withdraw from trials at any time, someone who believes they are receiving a placebo could leave. Retention of patients in trials reflect this; retention rates are as low as they have ever been since retention was first monitored 20 years ago.
Although the trial wasn’t complete, the news that the drug wasn’t working caused Amber and many other parents to take their kids out and move on. If you knew the therapy wasn’t working, why stay? While patient advisory boards help many drug companies structure their trials, it is clear that more should be done to improve participant retention.