The life cycle of a clinical trial
From initial idea to lifesaving innovation, find out what it takes to bring a new treatment from the lab to patients
It’s an unfortunate fact in the world of medicine: Nine out of every 10 clinical trials—those crucial research endeavors that aim to uncover new and better ways of treating disease—fail.
But even failed clinical trials provide valuable data and insights that can fuel future forays into better medicine. And when a clinical trial succeeds, it gives doctors a new or improved weapon in their fight against a specific disease—and gives patients a dose of hope.
Good luck, good science, and good results converged last year when a clinical trial involving a team of experts at M Health Fairview Masonic Children’s Hospital helped prove the effectiveness of a new gene therapy for treating cerebral adrenoleukodystrophy (ALD), a rare neurodegenerative disease that typically affects boys.
Traditionally, ALD is treated through the transplantation of blood stem cells from a healthy donor to a sick recipient. It’s effective, but for between 10 and 15% of recipients, the procedure has fatal side effects.
Troy Lund, M.D., Ph.D., Paul Orchard, M.D., and a team at Masonic Children’s Hospital played a leading role in the 10-year clinical trial that evaluated this potentially safer approach to treating the disease.
Instead of using a donor’s cells, the gene therapy protocol involves removing some of a patient’s defective stem cells, adding in a normal copy of the previously faulty gene within those cells, and putting them back in the body where they work to disrupt the disease process of ALD.
The results were more than promising; in September 2022, the U.S. Food and Drug Administration approved the gene therapy for wider use to treat ALD. Later this year, Masonic Children’s Hospital will become one of the four locations in the world to offer the treatment to patients.
“A trial like this really fulfills our promise as physician-scientists,” says Lund, whose work has been supported by the nonprofit X Out ALD. “Our goal is to take ideas and observations from the lab, develop therapies, bring them to patients, and have successful outcomes. It’s fantastic when it all works out.”
ILLUSTRATION BY KATHLEEN FU