According to a story from Beyond the Dish, a new experimental treatment for spinocerebellar ataxia was given the go-ahead from the U.S. Food and Drug Administration for testing. The investigational product is in development from the Taiwanese biotechnology company Steminent Biotherapeutics, Inc. This company focuses on the development of stem cell based therapies for the treatment of neurological disorders.
About Spinocerebellar Ataxia
Spinocerebellar ataxia, which is also referred to as spinocerebellar atrophy, is a genetic, degenerative, progressive disease that has several different subtypes. It is estimated that this disease affects around 150,000 in the US per year. This disease has no current treatments and is fatal in many cases. The severity of the disease depends on the type. Symptoms of spinocerebellar ataxia include loss of coordination (affecting gait, speech, hands, and eyes), Parkinson’s like symptoms, cognitive impairment, seizures, and cerebellum atrophy. Treatment is supportive and symptomatic; certain medications can help with symptom control and physical therapy can delay somewhat the loss of function associated with the disease. Overall, this illness represents a significant unmet medical need. To learn more about spinocerebellar ataxia, click here.
Steminent’s primary product is called Stemchymal, which consists of stem cells that are derived from fat and can be harvested and standardized with a standard liposuction procedure. The product has already been cleared for three clinical trials in Taiwan. So far, Phase I trials have demonstrated that administration of Stemchymal is safe in humans. These fat-based cells contain an array of molecules and growth factors that can promote the healing process.
The drug will be tested as a treatment for a subtype of spinocerebellar ataxia called polyglutamine spinocerebellar ataxia (PolyQ SCA). Earlier tests in Taiwan suggest that Stemchymal could be a potential treatment for this disease, as they proved that the drug was safe, and patients also showed improved function with the treatment, which could be maintained for a six month period.
The drug also received Orphan Drug designation from the FDA for the treatment of the disease in late 2015. The next step for Steminent will be the preparation of the US trials and the recruitment of patients.