Results of a preclinical study suggest that stem cell technology from Athersys (NASDAQ:ATHX) could offer a treatment for patients suffering from a rare disorder called “Hurler’s syndrome.”
Hurler’s syndrome is a metabolic disorder in which patients produce too little of an enzyme that helps the body break down a complex type of sugar molecule found in the body. Children with this disease develop nervous system problems and can die young, according to the National Institutes of Health.
In the Athersys study, an injection of the company’s MultiStem stem cell technology in neonatal mice reduced the accumulation of complex sugars in the brain and improved behavioral and motor functions in the mice, according to a statement from Athersys.
The results suggest the technology could hold promise in treating patients with Hurler’s syndrome and similar conditions in which patients’ bodies are unable to break down complex sugar molecules.
“While these conditions affect only a small number of patients, there are limited treatment options available to ameliorate or reverse disease progression, and it remains an area of unmet clinical need,” said Dr. Robert Mays, Athersys’ head of neuroscience.
Of course, it’s important to note that results seen in mice often aren’t seen in humans, and years of human testing would be required before Athersys’ treatment would be available to patients afflicted with these disorders.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Athersys’ MultiStem technology is an off-the-shelf stem cell treatment derived from the bone marrow of adults or other nonembryonic sources. The technology has shown promise in reducing inflammation, protecting damaged tissue and forming new blood vessels.
The Cleveland company is investigating the technology for the treatment of several conditions, including heart attack, stroke and blood diseases.
[Photo from flickr user Tobyotter]
