The Lupus Foundation of America and the National Institute of Allergy and Infectious Diseases (NIAID) are co-funding a five-year, Phase 2 trial that will investigate mesenchymal stem cells (MSCs) as a potential treatment for moderate to severe lupus.
The lupus foundation had already announced a $3.8 million five-year grant supporting the study, and NIAID has secured an additional $720,000 for the first year.
“Robust funding is vital to moving lupus research forward. Without sufficient funding from public and private resources, research treatments will be delayed, and the search for better treatments and cures will be seriously impaired,” Sandra C. Raymond, CEO of the Lupus Foundation of America, said in a press release.
MiSLE, the Phase 2 trial (NCT02633163), will be conducted in the U.S. and is expected to begin patient enrollment soon.
It will evaluate the effectiveness and safety of mesenchymal stem cells obtained from umbilical cords for the treatment of adults with systemic lupus erythematosus (SLE).
Participants will be randomly assigned one of two MSC doses — one million cells per kg or five million cells per kg — or a placebo. All patients will continue to receive standard of care therapy.
The study’s primary goal is to determine if more patients receiving mesenchymal stem cells respond to treatment.
Responders are defined as patients who experience a four point or higher reduction in the Systemic Lupus Erythematosus Disease Activity Index score (SLEDAI), and receiving corticosteroids at a dose of 10 mg a day or less.
Researchers will also examine changes in SLEDAI scores — a measure of disease activity — along with the frequency of lupus flares, accumulation of new damage, and changes in health-related quality of life. Fatigue, pain, and depression will also be addressed.
Researchers plan to also determine if treatment with MSCs decreases the use of steroids and other therapies linked to side effects, such as damage to vital organs.
Early data from Chinese MSC trials show that patients who failed other forms of therapy benefited from mesenchymal stem cells with few adverse reactions.
“People with lupus need and deserve better treatment options now. This clinical study represents an innovative step forward in lupus research, and our hope is that we will be able to add stem cell therapy to the arsenal of treatments available for people with lupus,” Raymond said.
Mesenchymal stem cells are a type of stem cell able to differentiate into bone, cartilage, connective tissue, muscle, and fat cells. They are found mostly in the bone marrow, adipose tissue, umbilical cord, muscle, and lung.
Studies in mice suggest that stem cells may ease lupus symptoms by preventing bone loss and the decrease in mineral content. Researchers have also shown that animals treated with MSCs have fewer autoantibodies, reduced inflammation, less severe kidney disease, and improved survival.