From cells to the era of medicine
Aiming for early establishment of clinical use of regenerative medicine
Issues in the current regenerative medicine
Finding a proper and enough cell source
Need for the cell culture
for cell transplantation,
which accompanies with high cost and stress to the body.
Progenisyte Japan will provide the answer for these issues
We have discovered a low molecular weight compound that proliferates "neural stem cells" without a need of administered to the brain. Our medicine can increase neural stem cells by up to 600% in vivo.
Until now, only antidepressants (SSRIs) have been known as a drug that increases neural stem cells, but the increase was only up to about 20%. (Reference: Vichier-Guerre C, et al., Toxicol Lett. 2017 Nov 5;281:20-25)
This drug has 70 times higher efficacy in increasing the proliferation of neural stem cells as compared to mesenchymal stem cells. It readily crosses the blood-brain barrier and orally administrable for the therapy since we have detected the drug by HPLC in the brain tissue after a peripheral administration.
The effect is proven by a study using a Parkinson's disease animal model in which dopamine has been specifically eliminated. The peripheral administration of our drug increased endogenous neural stem cells, produced dopaminergic neuron-like cells in the striatum, and improved motor function in the rotarod test.
Current safety data
In preliminary safety studies using mice, one administration of 1000 times of the therapeutic dose did not show any acute toxicity, and daily administration of 100 times of the therapeutic dose did not show any liver or kidney toxicity up to 28 days. It also did not promote tumor cell growth and did not show any mutagenicity in the Ames test.
However, many other safety studies will be
required before we can start a clinical trial.
Toward the possibility of adaptation to various diseases,
We will continue to conduct research and development to seek many other possible applications.
We will continue to conduct research and development for seeking many other possible applications.
Cervical spine/spinal cord injury
Alzheimer's disease therapy
We have discovered that high levels of amyloid precursor protein (APP), which produces Amyloid β (Aβ), differentiate neural stem cells into glia and inhibits neuroregeneration.
Until recently, most people believed that Aβ is the cause of Alzheimer's disease, focused on its toxicity, and tried to develop therapies to reduce the Aβ deposition. We collaborated with NIH and found that a therapeutic agent for Alzheimer's disease lowered the brain concentration of APP and allowed neural stem cells to differentiate into neurons (PNAS 104(30):12506-11). Then we showed that the combination treatment of this APP lowering drug and our drug to increase endogenous neural stem cells increased neurogenesis in the brain of APP transgenic Alzheimer's disease model mice and improved their behavior in the maze test.