According to a story from SMA News Today, a recent study has demonstrated that the parents of children with spinal muscular atrophy are often skeptical about enrolling their kids in clinical trials, even though it may be the best way for them to get help. This information suggests that more should be done to inform that parents of the specifics of individual trials.
Spinal muscular atrophy (SMA) is a genetic disorder that is characterized by the death of motor neurons, which leads to the degeneration of the skeletal muscles. In its more severe forms, this can mean an early death. The condition is caused be a deficit in the protein SMN, which is expressed by all cells throughout the body and is critical for the survival of motor neurons– a lack of this protein causes these cells to die. Generally, the muscles of the torso, such as the breathing muscles are affected first, followed by the upper arms and legs. Symptoms include a loss of muscle strength and reflexes, problems swallowing, tongue twitching, and bell-shaped torso. To learn more about spinal muscular atrophy, click here.
The lack of clear information about trials can be a major barrier for participation. Parents want to be reasonably sure that there is a good chance that the trial will actually help their children, but many of them fear that their child will be given a worthless placebo, making the trial a waste of time for them. A trial can only succeed if it is able to recruit participants, which is already a challenge for a rare disease like SMA. In the past, trials have been stuck in limbo because they were not able to recruit enough patients.
The study found that the majority of parents did want their kids to be part of a trial; the problem was that parents were not given a clear understanding of what the trial would entail on a daily basis, much less what the potential risks were. Enrollment requirements could also be restrictive in many cases, preventing children who desperately needed treatment from possibly getting help.
The clinical trials process has been criticized for years, and while new approaches to conducting trials are starting to be introduced, the data highlights the need for more patient oriented protocols that will leave patients and their families more confident in the process.
