Clinical trials essential in developing new treatments for current patients and future generations

At nearly two years old, Oliver Lambright wasn’t able to walk, or even crawl. His mom, Erin Lambright, knew there was something wrong. After pursuing testing and consultations, Oliver was diagnosed with Duchenne Muscular Dystrophy (DMD), a genetic disorder that affects roughly one in 3,000 boys and is characterized by weakness in the muscles that worsens over time.

The disorder is caused by a lack of dystrophin, an important protein for protecting muscles from stress. There is no cure. People rely on medications and therapy to manage the disease.

“I didn’t sleep for weeks when his diagnosis was confirmed,” Lambright says. “I was looking on every single website looking for a tiny bit of hope that there was something out there that could help Oliver. You just feel completely helpless.”

In 2016, Oliver’s doctor, Dr. Jean Mah, associate professor in the Department of Paediatrics at the Cumming School of Medicine (CSM), told the family about a clinical trial underway at the Alberta Children’s Hospital that explores a possible new treatment for DMD. Although the Lambrights live in Grande Prairie and would have to travel to Calgary every month, it was a “no-brainer” to enter the trial, says Lambright.

“We had nothing to lose. Of course, we were nervous and weighed the pros and cons, but we were 110 per cent going to do it,” she says. “If there was any little thing that could enrich Oliver’s quality of life, we were on board.”

Clinical trials 'essential' for new treatments, learnings

Clinical trials are research studies aimed at investigating the effects of a medical intervention — such as drugs, devices or diagnostic tests — on health outcomes. At the University of Calgary, there are currently 563 active clinical trials.

As the principal investigator of Oliver’s trial, Mah says clinical trials are essential for forging the path toward new learnings and treatments.

“Every drug we use today in the clinic had to first be tested in a clinical trial,” says Mah. “Clinical trials have blazed the way for the titanium hip, for taking out your gall bladder, or for grafting around a blocked artery in the heart. If we could fix this one protein in these kids’ muscles, it would be a huge breakthrough.”

Lambright adds that their family was motivated to enter a clinical trial not only to help their son, but also other families with children who have DMD.

“There have been other parents who’ve put their children in clinical trials, and even though the trial didn’t work for them or those children have passed on, positive things have come from those trials,” she says. “It’s like everybody helping each other, even future parents that will have a little boy with this disorder.”

Approximately 100 people from around the world are participating in the same trial Oliver joined — a double-blind study where neither the participants nor the researchers know who is receiving real treatments, in order to prevent bias.

While most clinical trials recruit individuals who were diagnosed with a condition and meet certain criteria, healthy individuals can also take part in some trials as a control participant. Healthy control participants are sometimes needed to fully evaluate the benefits of a new treatment, and participating in this way can be a great opportunity to help others as well as learn about emerging technologies, therapies and ideas.

To raise awareness about the importance of clinical research, International Clinical Trials Day was celebrated throughout the world on May 17 to commemorate the first randomized clinical trial in 1747. UCalgary hosted a Clinical Trials Market Day to give researchers an opportunity to learn about the various resources and tools available that can help when planning, promoting and conducting a clinical trial. 

Dr. Jean K. Mah is an associate professor in the Department of Paediatrics, a member of the Alberta Children’s Hospital Research Institute and an associate member of the Hotchkiss Brain Institute at the Cumming School of Medicine.