Cell Therapy Developed by Smidt Heart Institute Leader Delays Disease Progression in Patients Who Have Few Treatment Options
A cell therapy developed by the executive director of the Smidt Heart Institute stabilizes weakened muscles–including the heart muscle–in Duchenne muscular dystrophy patients, a new study published in the international peer-reviewed journal The Lancet shows.

If the HOPE-2 study’s success is duplicated in the upcoming multicenter, randomized, placebo-controlled HOPE-3 clinical trial, the intravenous cell therapy could become the first Food and Drug Administration-approved treatment for Duchenne patients with advanced disease.

“This therapy is unique in that it addresses two vital needs in patients with Duchenne: physical movement and a healthy heart,” said Eduardo Marbán, MD, PhD, executive director of the Smidt Heart Institute at Cedars-Sinai, the Mark S. Siegel Family Foundation Distinguished Professor, an author on the study and the inventor of cardiosphere-derived cells (CDCs), progenitor cells derived from human heart tissue, which have been used in multiple clinical trials.

Duchenne muscular dystrophy is a rare, inherited disorder that mostly affects males. It’s caused by mutations on a gene on the X chromosome that interferes with the production of a protein called dystrophin that muscles need to function. Children born with such mutations have muscle weakness throughout their bodies. This makes it difficult for them to do normal activities like run, jump, climb stairs, stand up after sitting and pedal a bicycle. They can also become extremely sick when muscles in their hearts and respiratory organs weaken.

The prognosis for patients with Duchenne muscular dystrophy is bleak. Most use a wheelchair by the time they are teenagers and don’t typically live into their 30s. There is no cure for the disease. Currently, the only approved medical treatments are aimed at delaying loss of the ability to walk; nothing is available for patients with more advanced disease, who now outnumber those with milder symptoms.

Release date: 10 March 2022
Source: Cedars-Sinai Medical Center

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