Healthcare (Commonwealth Union) – A group of researchers from the University of Alberta has developed a molecular compound that stimulates the immune system, offering potential as a promising treatment for cancer and persistent infections.
Khaled Barakat, a professor in the Faculty of Pharmacy and Pharmaceutical Sciences and the lead author of a recent study published in Medicine in Drug Discovery pointed out that it marks progress towards creating a small-molecule drug capable of boosting immune response, further stating that the breakthrough could pave the way for new immunotherapy approaches and improved patient outcomes.
Barakat, who is also a member of the Cancer Research Institute of Northern Alberta, pointed out that small molecules—compounds with low molecular weight that can regulate specific proteins inside cells—have the potential to revolutionize the treatment of various immunological diseases. They offer several benefits over existing methods.
At present, many therapies that target diseased cells through the immune system rely on immunostimulants. Like small molecules, these drugs work by activating the immune system, but they come with significant drawbacks. Immunostimulants are often expensive to produce and administer, requiring high doses. They also tend to have a long half-life—the time it takes for the drug’s concentration in the body to be reduced by half—which increases the risk of side effects, Barakat notes.
In contrast, small molecules are more affordable, have a shorter half-life, and are more stable at room temperature. Additionally, they can enter the bloodstream more easily and cross the blood-brain barrier—a protective system that prevents most toxins in the blood from reaching the brain—something that most immunostimulants cannot achieve.
“This is one of the big advantages,” explained Barakat. “If you have a small molecule which can cross the blood-brain barrier, you can actually start treating unreachable tumours.”
The small molecule developed by Barakat’s team, called Compound B, is a modified version of another potential compound the group was exploring. What sets Compound B apart is its higher water solubility, a crucial feature for designing a drug that can be efficiently absorbed and distributed throughout the body.
In their research, the team confirmed that Compound B activates immune cells by observing an increase in T cells (immune cells that attack specific antigens, such as cancer cells) and cytokines (proteins essential for immune system regulation).
Barakat explains that his lab begins by using machine learning and molecular modeling to filter down potential compounds to a promising few, further indicating that they simulate everything you would perform in a lab experiment, but they do it virtually on a computer.
Once their screening process highlights a few compounds of interest, like Compound B, these are tested on peripheral blood mononuclear cells (PBMCs). PBMCs contain a mix of immune cells also found in the bloodstream, enabling researchers to simulate the compound’s potential effects on the body.
Barakat pointed out that when the compound is introduced, it prompts the immune system to release specific substances, signaling that the immune response has been activated.
Research is already in progress to identify potential targets for Compound B to bind to, which will be detailed in an upcoming publication.
This interdisciplinary project includes collaborators from the University of Alberta’s Mike Petryk School of Dentistry and the departments of chemistry, oncology, biochemistry, and medical microbiology and immunology. It has been funded by the Alberta Cancer Foundation, the Canadian Institutes of Health Research, and the Li Ka Shing Applied Virology Institute.
“It is a breakthrough in our group that we’re really happy about, and we hope to follow the same procedure in developing new drugs in the future.”
