NU211-01/NU215-02, human umbilical cord mesenchymal stem cells for hereditary ataxia

The aim of this study is to evaluate the the safety and efficacy of transplantation of human umbilical cord-derived mesenchymal stem cells (UC-MSCs) in patients with hereditary ataxia. There were no serious transplantation-related adverse events within the 12 months follow-up of the 16 treated cases. The majority of patients showed improved Berg Balance Scale (BBS) and International Cooperative Ataxia Rating Scale (ICARS) scores continuing for at least 6 months, which indicated UC-MSC therapy could alleviate Hereditary spinocerebellar ataxia (SCA) symptoms. This study suggested that UC-MSC transplantation was safe and might delay the progression of SCA. An additional study published in The Journal of Translational Medicine, including the participation of Beike Biotechnology Company, reports statistically significant improvements in functionality and quality of life in 30 patients with hereditary ataxia treated with a combination of cord blood-derived stem cells (UC-MNCs) and physical therapy.