Cannabis can be used to treat the symptoms of a variety of rare diseases including multiple sclerosis, Dravet syndrome, Crohn disease, and amyotrophic lateral sclerosis (ALS).
So why not Duchenne muscular dystrophy (DMD) as well?
That’s the question George G. Rodney, PhD, is asking. An associate professor at Baylor College of Medicine in Houston, Texas, Dr. Rodney thinks cannabidiol (CBD)—a key component of the cannabis plant—could benefit boys and young men with the degenerative neuromuscular illness.
“If you look at the literature for CBD, this compound has been shown in a number of different diseases to modify a number of the pathways that lead to the symptoms these boys have,” he explained. “We thought that if CBD could basically have a multifactorial approach to combat many of the altered signaling pathways within Duchenne, we may have a better chance providing these patients with a better quality of life.”
Dr. Rodney works in Baylor’s Department of Integrative Physiology. In August 2022, he received a $300,000 grant from the Muscular Dystrophy Association (MDA) to study how CBD—combined with current steroid treatment—could significantly reduce inflammation while improving skeletal muscle, pulmonary, and immunological functions. His preclinical mouse study ends in mid-2025.
Patients With DMD Report Cannabis Helps With Anxiety, Coping
“Cannabinoids are known to interact with some of the same inflammatory pathways that are active in Duchenne, so there’s good reason to try it,” said Sharon Hesterlee, PhD, chief research officer at the MDA, which advocates for patients with Duchenne, among other diseases.
Natalie Truba, PhD, is a clinical psychologist at Nationwide Children’s Hospital in Columbus, Ohio. She’s seen dozens of patients with DMD for anxiety, depression, and behavioral disorders.
“Many men with Duchenne who I work with find cannabis products incredibly useful from an anxiety management and coping point of view,” she said. “I believe them when they tell me it helps them.”
Read more about DMD symptoms
Dr. Truba pointed out that young people are going to use cannabis, regardless of what their parents or doctors think. “But we want them to move away from smoking it and towards edibles,” she said, noting that teenagers and young men with Duchenne already have compromised pulmonary systems and shouldn’t be smoking anything.
Added Pat Furlong, founding president and CEO of the nonprofit group Parent Project Muscular Dystrophy: “Some clinicians are recommending cannabis for pain or anxiety. What is needed is some clarity around the various types and doses. I do think it would be useful to study cannabis and one day develop patient materials that explain in detail various types and uses. Hopefully, this mouse study will provide some insight and lead to increased interest and exploration.”
Dr. Rodney has a master’s degree from the University of Texas Health Science Center in San Antonio in muscle physiology and a PhD from Baylor in molecular physiology and biophysics. He said his lab had been working on cannabis for about a year before winning the grant to study Duchenne. And that came about only because one of his students happened to be interested in clinical translational science.
“We set her up with a colleague of ours at the neuromuscular clinic at Texas Children’s Hospital, where she shadowed some physicians in the clinic to see patients and their parents for Duchenne muscular dystrophy,” Dr. Rodney said. “Several moms asked the clinician if they should give their boys CBD. At the time, the clinician didn’t know the answer to that. So my student came back to me, asked me the question, and we started looking into it.”
Research Mouse Model Mimics Progression of DMD in Boys
The researcher said he treats mice with purified CBD in the form of drops that the animals ingest from a pipette tip, rather than injecting them intravenously. Not only is this method easier; it’s also more similar to real-life methods of administration among Duchenne patients.
“You’re not going to want to give a boy an IV injection. They probably don’t even really want to take an intramuscular injection very often,” he said. “We have done a few rounds of studies at 1 dose of CBD with minimal effects. We’re also trying to understand what dose we really need to treat the model. There is evidence in the literature that too much CBD is a bad thing as well.”
Dr. Rodney added that the most common mouse model in such studies, known as the mdx mouse, depicts the progression in boys with DMD quite well.
“As the mouse ages and becomes an adult, it stops progressing; at that stage, it doesn’t mimic the patient very well,” he said. “There’s also another mouse model in which investigators have knocked out a compensatory protein called utrophin. We have a dystrophin‑utrophin mouse that mimics a lot better the progression we see in the human population. We’re actually studying both models so that we’ll understand which model might be better suited for continued studies.”
Besides the MDA grant, Dr. Rodney is seeking $1.25 million from the Department of Defense’s Duchenne Muscular Dystrophy Research Program to study how CBD can benefit cardiac health in boys and men with DMD.