Promising stem-cell clinical trial results for rare lung disease
- In a clinical trial, reserachers evaluated the effect of genetically enhanced stem cell therapy on lung blood pressure levels in pulmonary arterial hypertension (PAH).
- PAH is a rare lung disease that is characterized by very high blood pressure in the arteries that supply blood to the lungs. It generally affects young women.
- This study was not designed to be rigorous—rather it was a feasibility study; however, the researchers observed that in the days following treatment, patients had improved blood flow in their lungs, as well as enhanced ability to exercise and better quality of life for up to six months.
The results from this feasibility study are encouraging, because although there are treatments for PAH, there is a still an unmet medical need. The study, which was published in Circulation Research, focused on treating seven patients with stem cells created from white blood cells that had been harvested from their own bodies. The cells were genetically engineered to produce greater amounts of nitric oxide—the natural substance that not only widens blood vessels, but is also needed for vascular repair and regeneration. The cells were injected directly into the patients' lung circulation.
Like other stem-cell studies that are currently underway, the results are preliminary and the patient population being studied is relatively small. However, the research is viewed as promising and worth pursuing further.