The world’s first clinical trial of a stem cell therapy for pulmonary hypertension (PAH) has yielded positive results. PAH is a rare disease which results in high blood pressure affecting arteries in the lungs and in the heart. The disease mostly affects young women and it has a high mortality rate.

PAH is usually caused by a defective gene but in many cases its cause is unknown. Drugs can improve the symptoms experienced by the patients, but cannot repair any damage to the blood vessels in the lungs or heart. There is currently no cure for the PAH.

In the clinical trial, Canadian researchers tested a generically enhanced stem cell therapy to see if it could regenerate the blood vessels in the lungs. Seven patients with PAH had their white blood cells harvested in a process called apheresis. The white blood cells were then grown in a laboratory to produce endothelial progenitor cells. The endothelial progenitor cells were harvested and genetically modified to produce higher levels of a natural substance called nitric oxide.  Nitric oxide can widen blood vessels, making it useful for vascular repair and regeneration. The modified stem cells were then injected into the lung circulation of patients.

Researchers observed that patients had improved blood flow in their lungs following the therapy. All patients said they were able to exercise for longer and had an improved quality of life.

Lead author of the paper, Dr. Duncan Stewart says that the stem cell therapy for this deadly disease shows great promise: “We desperately need new therapies for this disease, and regenerative medicine approaches have shown great promise in laboratory models and in clinical trials for other conditions.”

Source: Stem cell therapy shows promise in small clinical trial for rare lung disease

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