Stemedica Cell Technologies, Inc. received the FDA's investigational new drug (IND) approval for a United States-based, Phase IIa clinical study using its allogeneic stem-cell therapy to treat subjects with mild to moderate dementia due to Alzheimer's disease (AD), the sixth leading cause of death in the United States. The study is sponsored by Stemedica International, S.A. and will start at the University of California, San Diego (UCSD) under Principle Investigator Douglas Galasko, M.D. and expand to other sites. Stemedica International will provide management and financial support for this clinical trial. The clinical trial is titled "A Phase IIa Multicenter, Randomized, Single-blind, Placebo-controlled, Crossover Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Allogeneic Human Mesenchymal Stem Cells in Subjects with Mild to Moderate Dementia Due to Alzheimer's Disease."
"This study was approved based on the excellent safety profile of Stemedica's cGMP-manufactured, hypoxically-grown stem cells and on solid pre-clinical data obtained by Stemedica International in cooperation with the École Polytechnique Fédérale de Lausanne of Switzerland and with a grant from the Swiss government," said Lev Verkh, Ph.D. Stemedica's Chief Regulatory & Clinical Development Officer. He continued, "We are very proud of Stemedica's clinical program under U.S. INDs for several indications including ischemic stroke, acute myocardial infarction, chronic heart failure, cutaneous photoaging and Alzheimer's disease. At the study's conclusion we will understand if our approach is efficacious versus placebo in subjects with Alzheimer's-related dementia, as evidenced by neurologic, functional, and psychiatric endpoints."
Stemedica's bone marrow-derived, allogeneic itMSCs are unique because they are grown under hypoxic conditions that more closely resemble the environment in which they live in the body. Compared to other MSCs, itMSCs secrete higher levels of growth factors usually associated with angiogenesis and healing. Stemedica International's AD stem cell therapies feature itMSCs, neural stem cells (NSCs) and stem cell factors, which are described in Stemedica International's U.S. Patent application #20140286910.
Promising results were achieved during a three-year, intensive, pre-clinical research project supported by a grant from the Swiss Commission for Technology and Innovation (CTI). The research was conducted at the Laboratoire d'Optique Biomedicale headed by Professor Theo Lasser at École Polytechnique Fédérale de Lausanne (EPFL) in Switzerland.
Stemedica International's pre-clinical research was led by Chief Scientist Tristan Bolmont, Ph.D. To evaluate the impact of an intravenous delivery of human mesenchymal stem cells on amyloid pathology, the well-established APPPS1 transgenic mouse model of Alzheimer's disease was used. Intravenous delivery of itMSC safely reduced cerebral Abeta pathology in APPPS1 animals analyzed one week after the last injection. Both aged and young APPPS1 mice exhibited significantly decreased Abeta amyloidosis following the itMSC treatments. Concomitantly, microglial activation was diminished in aged and young itMSC-treated APPPS1 mice. No increase of vascular amyloid or manifestation of microhemorrhages was observed following the repeated intravenous itMSC delivery. Biodistribution analysis revealed that intravenously delivered itMSC migrate to the brain and could be detected in this organ with the highest value at one hour post-delivery, decreasing after one day and subsequently dropping below detection level at one week after the injection.
According to Alzheimer's Disease International, nearly 44 million people have Alzheimer's or a related dementia. Alzheimer's and dementia are most common in Western Europe followed closely by North America. The global cost of Alzheimer's and dementia is estimated to be $605 billion USD, which is close to 1% of the world's gross domestic product.
"We are very excited to take this next step in developing a treatment for this devastating disease," says Nikolai Tankovich, M.D., Ph.D., President and Chief Medical Officer of Stemedica Cell Technologies and Executive Chairman for Stemedica International. "Our upcoming Phase IIa clinical trial will enable us to make progress towards determining if our stem cell treatment may be able to halt or slow down the progression of Alzheimer's disease and other forms of dementia."
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