RNL BIO CO LTD has announced the filing of an Investigational New Drug (IND) application with the Korean Food and Drug Administration (KFDA) to initiate clinical trials phase II and III assessing the company's RNL-Astrostem(TM) stem cell drug in patients with cerebral palsy.
Cerebral Palsy is caused by non-progressive brain damage from single or multiple defect(s) on the nerve/muscular system and results in disorder in motion and sensory integration. According to Health Insurance Review and Assessment Service, the prevalence of cerebral palsy is 3.5 per 1000 male to 2.8 per 1000 female children, averaged at 3.2 per 1000 children, which is expected to increase due to reduction of premature death.
The purpose of this study is to assess conclusive efficacy of stem cells for the treatment of cerebral palsy in 45 subjects over 11 months and will be conducted through Kyung Hee University Hospital at Gangdong and THE Bethesda Hospital.
RNL-Astrostem(TM), has already completed a phase I trial to assure safety, including tests to rule out toxicity or tumorigenicity. This trial was conducted at the Seoul National University's Clinical Research Institute. In addition, the phase I study has confirmed the safety of stem cells infused by IV, as published in Stem Cells and Development ("Safety of intravenous infusion of human adipose tissue-derived mesenchymal stem cells in animals and humans," February 2011).
In the course of successful safety trials, scientists found significant evidence suggesting the potential for efficacy of stem cells in treatment of cerebral palsy. In addition, a case report with obvious result of improvement was published in another journal. RNL BIO believes that with successful trials and approval of the KFDA, it will be possible to commercialize RNL-Astrostem(TM) by 2014, revolutionizing the possibility to cure this incurable disease of children, which can only be managed with current technologies.
In the clinical study investigators employ a variety of methods to assess efficacy: K-ABC: Kaufman Assessment Battery for Children, Gross Motor Function Measure, (GMFM), box and block test, Modified Asworth Scale, MAS, finger tapping test, Brain SPECT and MRI.
Dr. JC Ra, president of RNL BIO Stem Cell Technology Institute, said, "It is our mission to find cures for incurable diseases, such as the terrible pediatric curse of cerebral palsy, through autologous stem cell technology."
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