Cellular Pathways
Our unique approach to treating diseases harnesses the power of fundamental cellular pathways that have untapped potential to address brain diseases such as bipolar disorder and Alzheimer’s.
In the brain, GSK, CRMP2, WNT, and glutamate have been linked to cell activities including neurogenesis, synaptic plasticity, and survival. Deficiencies in how these pathways are regulated have been associated with many psychiatric and neurological conditions, including bipolar disorder and Alzheimer’s disease.
We identify and optimize candidate medicines by examining their effects on pathways on living human brain cells which have been derived from induced pluripotent stem cells.
A revolution in neuroscience
We have a novel platform driving discovery
The challenge
Research on human brain cells was historically limited due to difficulty accessing and maintaining them in culture. Differences in physiology between humans and animals limit the ability of animal models to support drug development, making human brain cells an essential component of drug discovery.
4M Therapeutics’ new technology
Stem cells can now be derived from adults with bipolar disorder, Alzheimer’s disease, and other neurologic conditions, and these cells can be induced to become neural progenitor cells and neurons whose structure and function can be studied in the laboratory.
Our models using these human cells can improve the process of drug discovery and development for the treatment of neuropsychiatric disorders and neurodegenerative diseases. We identify and optimize product candidates by examining their effects on pathways on living human brain cells which have been derived from induced pluripotent stem cells.
Innovation for patients
New research shows that neural progenitor cells derived from people with bipolar disorder have differences in the regulation and expression of several proteins. Our founders were the first to identify hyperactive GSK3β in living human brain cells derived from induced pluripotent stem cells (iPSCs) of patients with bipolar disorder and Alzheimer's disease.
While lithium has helped many people with bipolar disorder, its use is limited by the high doses required and toxicities such as kidney damage.
Using neural progenitor cell models, our founders have identified a number of candidate medicines that mimic the effects of lithium on key proteins in the brain but do so at lower doses with a lower potential for toxicity compared to lithium. Our lead compound in development, 4MT2001, is a potent and selective inhibitor of GSK3β with the potential for superior safety and efficacy.
Our goal is to demonstrate clinical success in bipolar disorder while also applying this technology and evaluating our product candidates in areas like Alzheimer’s disease, neurodegeneration, and other CNS disorders.