Symptoms of Duchenne Muscular Dystrophy are improved by cell therapy

Symptoms of Duchenne Muscular Dystrophy are improved by cell therapy ...

A clinical trial at UC Davis Health and six other clinics demonstrated that a cellular therapy is beneficial to patients with late-stageDuchenne muscular dystrophy, a rare genetic disorder that causes muscle loss and physical impairments in young people.

The therapy seems to be effective in preventing upper limb and heart function damage. It is the first therapy to provide significant functional improvements in most severe cases of DMD patients.

According to Professor Rig McDonald, the national principal investigator and author of the trials, HOPE-2 is the first clinical trial to validate systemic cell therapy in DMD. McDonald is a professor and chair of physical medicine and rehabilitation at UC Davis Health. The study demonstrated a statistically significant and unprecedented stabilization of both the skeletal muscle deterioration and the heart deterioration of structure and function in non-ambulatory DMD patients.

The Lancet records from the trial have been published today.

Cellular therapy for muscular degeneration

Capricor TherapeuticsCAP-1002allogeneic cardiosphere-derived cells (CDCs) obtained from human heart muscles were used in the Phase II clinical study. These cells can improve muscle inflammation and cell regeneration.

McDonald claims that the primary purpose of the CAP-1002 therapy is to help reduce chronic inflammation problems, decrease fibrosis, increase muscle regeneration, and thus maintain or improve critical heart and skeletal function.

The study uncovered the long-term feasibility and safety of repeated intravenous infusions of CAP-1002 for the treatment of late-stage DMD. It enrolled 20 patients with DMD at seven U.S. hospitals. Participants were at least 10 years old with moderate weakness in their arms and hands. They were randomly assigned to receive either CAP-1002 or a placebo every three months for one year.

Improvements in arm, hands, and heart function are significant.

The team analyzed the upper limb function using the scale Performance of Upper Limb(PUL) motor function for DMD. They also tested heart function by using cardiac magnetic resonance imaging (MRI), spirometry measures of respiratory function and circulating biomarkers.

The researchers calculated the PUL for participants at their first infusion and after one year. They also calculated the difference between the two levels of the mid-level and upper-bow PUL scores. The study found a significant positive difference in participants who received CAP-1002, compared to those who received the placebo. There was a significantly lower deterioration of upper extremity muscle function in the cell-treated group.

According to the cardiac MRI, cardiac symmetry and function appeared to improve in participants who received CAP-1002.

Cell therapy may one day be used for more common forms of heart failure, according to the founder. The Mark Siegel Family FoundationDistinguished Professor and executive director of theSmidt Heart Institute of theCedars-Sinai Medical Centerin Los Angeles is a good example.

Moving forward

McDonald and collaborators in other hospitals in the United States are planning a phase III clinical trial, HOPE-3. This study is intended to help confirm the effectiveness of CAP-1002 in a larger cohort of patients.

According to McDonald, the FDA believes that a larger Phase III study would be the next step toward obtaining drug approval. We must ensure the therapeutic longevity and safety of CAP-1002 beyond 12 months for the treatment of muscular degeneration in the heart and skeleton.

What is Duchenne muscular dystrophy

DMD is a condition that affects around 1 in 5,000 people, mostly boys, that usually develops in early childhood, causing gradual weakness and chronic inflammation of the skeletal, heart, and respiratory tissues, causing significant difficulties such as sitting and walking. Patients with DMD often lose their ability to walk in their teenage years and develop heart and lung complications as they age.

DMD medication services are limited, but no cure has been discovered. Current therapy that targets skeletal muscles aren''t as effective in treating the heart muscle that was damaged by DMD. A therapy that stabilizes or reverses heart deterioration, while improving upper limb function, would be unique in its ability to address the immense burden of disease seen in advanced DMD patients.

This cell-based therapy is innovative in its ability to meet the critical needs of patients with the most severe disease burden and to improve both upper limb and heart function. Therapies that address the later stages of the disease may have an enormous impact on the quality of life for boys and young men with DMD and reduce the burden of care for their families, according to McDonald.

Collaborations that made HOPE possible

The HOPE-2 study was the first clinical trial by researchers and staff at the university of California Davis Alpha Stem Cell Clinic.

McDonald believes that this is a solid testimony to UC Davis Health''s leadership in stem cell therapeutic trials and clinical translational medicine.

You may also like: