If your child has recently been diagnosed with Duchenne muscular dystrophy (DMD), you will be aware that progressive muscle wasting is inevitable. It will become more and more difficult for your child to move around independently as time progresses. While the current approved therapies can help delay disease progression, they can’t stop it altogether.
Many parents find themselves researching the latest treatments on long, sleepless nights. While experimental therapies can offer the hope of a better life for your child, parents should also understand their limitations.
What is an experimental therapy?
By definition, experimental therapies are therapies that are currently being tested by drug companies in clinical trials and have not received approval by the U.S. Food and Drug Administration (FDA). This is because the evidence proving they are safe and effective has not been firmly established. A new drug is only approved after extensive testing, usually over the course of many years.
Many of the drugs currently being tested for DMD are in a class called gene therapies.
What is gene therapy?
Unlike an infectious disease that can be treated with an antibiotic or antiviral, genetic diseases like DMD never go away, because the problem lies in the DNA of the patient themselves. To fix the problem at its foundation, doctors need to find a way to edit out or silence the problematic gene. This is known as gene therapy.
An example of a gene therapy that has received U.S. FDA approval is delandistrogene moxeparvovec-rokl, commonly known by its brand name, Elevidys. This therapy helps to slow the process of muscle degeneration. It is not, however, curative.
Accessing experimental therapies
In general, there is very limited access to experimental therapies because of the considerable risks involved. The only way you can access an experimental therapy is if you enroll in a clinical study; however, your child might receive the placebo instead of the experimental therapy that you seek.
Learn more about DMD treatment and care
In the United States, there is a “Right to Try” law that gives some patients who are terminally ill the right to access certain experimental therapies. DMD is not considered a terminal illness except at its very final stages. However, if you would like to find out more about this law and whether it may benefit your child, speak to your doctor or your lawyer for guidance.
It is important to manage your expectations about experimental therapies. While there are some that show promise (such as the experimental therapy RGX-202, which appears to stabilize muscle cells in mice studies), there is no guarantee that a new treatment will work in humans until final results have been published. There is also no timeline regarding when the U.S. FDA will consider a drug’s application to be accepted, and even more uncertainty about when it will become available for public purchase and use.
In general, experimental therapies are very expensive: costs in the millions are not atypical. This is because they require extensive testing, and may fail during the clinical trial stage due to negative effects seen in volunteer patients.
Because of all these reasons, your son’s care team might recommend focusing on the proven therapies that are currently available, such as physical therapy and approved medications. Regardless of the treatment your family and doctors decide is best, by being active in your child’s treatment plan and showing your child unconditional support, you are setting them up for the very best quality of life possible.
Sign up here to get the latest news, perspectives, and information about DMD sent directly to your inbox. Registration is free and only takes a minute.
