- Researchers are reporting they have looked at the potential of a small molecule compound that targets the glutamate system as a treatment for multiple sclerosis (MS).
- However, they note the research was done on mice and there is a long way to go before the technique could be used on humans.
- MS is considered an autoimmune disease and treatments have traditionally targeted the immune system.
- The researchers indicated that because this drug is a compound that targets different systems in the body simultaneously, it be more successful at treating the disease.
A novel treatment approach for treating multiple sclerosis (MS) that targets the glutamate system reduced symptoms effectively in mice, according to a study published in the journal Science Advances.
Dr. Fang Liu, the lead study author and a senior scientist in the Brain Health Imaging Centre at the Centre for Addiction and Mental Health in Canada, and her research team said they created a small molecule compound that targeted the glutamate system rather than the immune system, which other MS drugs usually focus on.
Glutamate is a neurotransmitter found throughout the central nervous system, according to an article published in
The researchers in the current study note that MS has traditionally been considered an autoimmune disorder that targets myelin and causes inflammation and white matter lesions in the brain. Symptoms include vision problems, heat intolerance, paresthesia, incontinence, depression, and problems with coordination and cognition.
In this study, the researchers used two different animal models of MS.
They reported that the new drug compound reduced MS symptoms and might have the potential to repair damage to myelin. Myelin is the protective sheath around nerves that allows electrical impulses to transmit quickly and efficiently along nerve cells. Inflammation can affect myelin.
The researchers note that their medication could potentially be used in the future with other compounds to form a drug cocktail.
“There are few things in biology where there is a single definitive cause and there is evidence that the glutamate system has a role to play in MS,” Liu told Medical News Today. “There are many MS treatments already available and in the pipeline targeting the immune system, but these therapies don’t work perfectly for everyone. Having additional routes to come at the problem can only provide more options to help more people.”
“MS remains a considerable therapeutic challenge, particularly the progressive forms of the disease for which there are limited treatments,” the researchers wrote. “The solution may be to move beyond immune-modulating drugs to a different strategy and that is what our data support: a focus on protecting neurons and myelinated axons.”
Researchers are currently working to turn the molecules identified by Liu into drug-like molecules that have the potential for clinical use. She believes that the evidence of tolerability and efficacy make it an excellent candidate for human trials.
Researchers noted several limitations to the study.
The most important limitation is that the research involved mice, which may not react the same way as humans to new potential drug treatments.
Mice are often used when studying diseases and medications, according to the Jackson Laboratory.
“Humans and mice may not look alike, but almost all the genes in mice share functions with human genes. So, humans and mice get the same diseases for the same reasons,” the laboratory notes.
“The immediate next step is additional toxicity testing; we need to show our drug is safe before we can enter into clinical trials,” Liu said
After that, and assuming it goes well, the scientists will start safety testing in humans within one to two years.
“What follows are phase two and three clinical trials where our drug is made available to study participants with MS,” Liu said. “Phase three trials, in particular, can take a number of years to follow a sufficient number of participants for long enough to see how their symptoms improve (or not) over time. Approval happens once these trials are complete, only if our drug is as safe and effective as we hope it is, of course. Added together, that is seven to 10 years before our drug can be legally prescribed by doctors in the clinic.”
Dr. Saud Sadiq, the director and chief research scientist at the Tisch Multiple Sclerosis Research Center of New York, also believes this research is a long way from possibly helping people with MS.
“The researchers discovered the molecule via [artificial intelligence] and although machine learning can be helpful, I think biological data is better,” Sadiq, who was not involved in the research, told Medical News Today. “In this instance, we need additional information to show it can work in a biological setting. This is interesting research, but right now, it is mainly for researchers to build on it rather than for it to be put into practice. There is still much work to be done before it can move toward human trials.”
“In the future, I would like to see the mechanism of neurodegeneration,” Sadiq added. “This is good work, but I do question the relevance to MS.”