New protocol adds a second immunosuppressant to the regimen of patients who are at higher risk for liver complications
LOS ANGELES–(BUSINESS WIRE)–Children’s Hospital Los Angeles (CHLA) is expanding its safety protocol for patients receiving Elevidys®, the FDA-approved gene therapy for Duchenne muscular dystrophy (DMD). In October, CHLA introduced a second immunosuppressant, mycophenolate mofetil (CellCept®), to the regimen of certain high-risk adolescents – boys who are older, heavier, and more advanced in their disease.
“Rather than waiting for a dangerous inflammatory reaction, we want to prevent it up front,” says Leigh Ramos-Platt, MD, Medical Director of CHLA’s Neuromuscular Disorders Center in the Neurological Institute. “We hope this approach will protect the liver while sparing patients from the extreme steroid doses we’ve had to use.”
To date, CHLA has treated 28 boys with Elevidys—more than almost any center in the country. That experience, combined with CHLA’s role as a national leader in pediatric cell and gene therapy, is critical to delivering Elevidys safely.
Focus on safety
The enhanced protocol follows reports earlier this year of two non-ambulatory teenage patients at other hospitals who died from liver failure after receiving Elevidys. In response, the therapy manufacturer, Sarepta®, temporarily halted shipments for non-ambulatory boys.
In July, following a death in a different Sarepta gene therapy trial, the FDA requested a pause for all patients. Eleven days later, after an investigation, the FDA allowed the company to resume Elevidys doses for ambulatory boys 4 or older.
After the FDA lifted the hold, Children’s Hospital Los Angeles also restarted treatment in these patients. But the team has continued to sharpen its focus on patient safety.
“From the beginning, we’ve been quite cautious in how we offer Elevidys to patients,” Dr. Ramos-Platt says. “We were very transparent with families about the lack of data and higher risks for non-ambulant boys, and only one of our non-ambulant patients chose to receive it.
“Overall, among our patients, younger boys under age 8 are doing phenomenal, and many are showing striking improvements,” she adds. “Older, heavier boys, even if they are still walking, are more complicated. That’s where we’re seeing the liver issues.”
Addressing liver risks
Liver inflammation is one of the most serious risks of Elevidys gene therapy. While the complication is rare in younger patients, many adolescents experience significant liver enzyme elevations—often requiring hospitalization and escalating doses of steroids.
“When we see elevated liver enzymes, it tells us there’s ongoing damage to the liver,” says Rohit Kohli, MBBS, MS, Chief of Gastroenterology, Hepatology, and Nutrition at CHLA. “The rapid onset of these elevations in higher-risk children is what prompted us to think about a different pre-emptive approach.”
Boys with Duchenne already take steroids to slow muscle damage, but gene therapy protocols temporarily add higher doses to prevent inflammation in the liver, heart, and muscle.
CHLA’s new regimen keeps these additional steroids in place but adds CellCept for children who are older, heavier, or more advanced in their disease. Some other centers are testing the immunosuppressant tacrolimus.
“For some children we have chosen CellCept because it’s easier to take and monitor, and it’s gentler than some other options,” Dr. Ramos-Platt says. “It’s not for everybody; it’s a case-by-case basis.”
Older boys may be more vulnerable to liver inflammation because of years of steroid use and a higher incidence of obesity-related liver disease. Dr. Ramos-Platt also theorizes that reduced muscle mass in more advanced Duchenne may lead more of the viral vector to concentrate in the liver.
The team is exploring more detailed pre-treatment liver assessments as well—including MR elastography—and has added a one-month post-infusion echocardiogram to its cardiac monitoring protocol.
“These therapies are still new, and we’re very tuned into these risks,” Dr. Kohli says. “We’re adjusting in real time.”
At CHLA, an expert multidisciplinary team—including pediatric hepatology, cardiology, nephrology, and hematology—monitors each patient closely before and after treatment. Families also have access to a 24/7 gene therapy phone line, staffed exclusively by attending neuromuscular neurologists.
“Even after gene therapy, a patient will still have Duchenne; the idea with Elevidys is to slow it down,” Dr. Ramos-Platt explains. “But while it doesn’t work for every patient, it’s the best treatment we have right now. We are continually working to make it as safe as possible for patients.”
About Children’s Hospital Los Angeles
Children’s Hospital Los Angeles is at the forefront of pediatric medicine and is the largest provider of hospital care for children in California. Since its founding in 1901, CHLA has delivered a level of pediatric care that is among the best in the world. Ranked one of the top 10 children’s hospitals in the nation by U.S. News & World Report, Children’s Hospital Los Angeles provides comprehensive and compassionate care to one of the largest and most diverse pediatric patient populations in the country. The hospital is the top-ranked children’s hospital in California and the Pacific U.S. region for 2025-26. A leader in pediatric research, CHLA is among the top 10 children’s hospitals for research funding from the National Institutes of Health. The Saban Research Institute of Children’s Hospital Los Angeles supports the full continuum of research, translating scientific discoveries into life-changing treatments for patients around the globe. As a pediatric academic medical center, CHLA is also home to one of the largest graduate education programs for pediatricians in the United States. The hospital’s commitment to building stronger, healthier communities is evident in CHLA’s efforts to enhance health education and literacy, introduce more people to careers in health care, and fight food insecurity. To learn more, follow CHLA on Facebook, Instagram, LinkedIn, YouTube, and X, and visit CHLA.org.
Contacts
Marlen Bugarin
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