Evaluating the Sigma-1 Receptor as a Therapeutic Target in Human Pluripotent Stem Cell Models of Fragile X Syndrome
Principal Investigator
FRAXA Fellow
Vancouver, BC Canada
Summary
Dr. Pouladi's team is exploring a treatment of Fragile X via the sigma-1 receptor. Drugs that boost activity at sigma receptors tend to calm down overactive neurons. They are also powerful anti-inflammatory drugs.
The Science
Previous experiments with sigma-1 agonists have shown that this type of drug can reverse many symptoms in the Fragile X mouse model. Dr. Pouladi’s team will explore the potential therapeutic effects in much greater detail to fully validate this new treatment target.
“We will transform Fragile X syndrome (FXS) stem cells into human FXS neurons and will test the ability of drugs targeting the sigma-1 receptor to reverse the cellular abnormalities in these neurons,’ said Dr. Pouladi.
This class of drugs appears to be quite safe and may be useful for a wide variety of neurological conditions. This project could lead to clinical trials in the near future.
Currently there are no drugs which target sigma receptors alone, but some available drugs have “off-target” effect on this receptor. Fluvoxamine (Luvox) is the most potent sigma-1 agonist of all available drugs and this might make it a uniquely effective choice among the SSRIs for people who have Fragile X syndrome.
