Researchers at the University of Alberta have developed Compound B, a promising small-molecule drug that efficiently activates the immune system. It offers advantages over current immunotherapies by being cost-effective, having a shorter half-life, and crossing the blood-brain barrier to treat previously unreachable tumors. Compound B shows potential as a versatile treatment for cancer and chronic diseases.
The mystery of Compound B discovery: A team of researchers at the University of Alberta (U of A) has developed a promising molecular compound called Compound B that could revolutionize the treatment of cancer and chronic infections by activating the immune system more efficiently and effectively than current drugs. This discovery, detailed in a recent study published in Medicine in Drug Discovery, opens up new avenues for immunotherapy and improved patient outcomes.
Professor Khaled Barakat, from U of A’s Faculty of Pharmacy and Pharmaceutical Studies and senior author of the study, describes the discovery of Compound B as a significant advancement in immunotherapy. “This is a step towards developing a small-molecule drug that can help activate the immune system,” says Barakat. "This could open new avenues for immunotherapy and better patient outcomes.”
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Barakat, also a member of the Cancer Research Institute of Northern Alberta, explains that small molecules like Compound B are unique because they can potentially change how immunological diseases are treated. These low molecular weight compounds can control the function of specific proteins within cells, making them more versatile and targeted compared to current treatments.
Many existing immunotherapy treatments rely on immunostimulants, which activate the immune system to target diseased cells, such as cancer cells. While these drugs have shown effectiveness in some cases, they have several drawbacks.
Immunostimulants are often very expensive to manufacture and require high doses for effectiveness. Additionally, they tend to have a long half-life—the time it takes for the drug’s concentration in the body to decrease by half—which increases the risk of adverse side effects. These treatments are also less stable at room temperature, adding complications in storage and administration.
In contrast, small molecules like Compound B offer several advantages. They are far less costly to produce and have a shorter half-life, making them safer and easier to manage. They also have the added benefit of being more stable at room temperature, allowing for more straightforward storage.
One of the most exciting aspects of small-molecule drugs is their ability to cross the blood-brain barrier, a system of specialized cells that protects the brain from harmful substances in the blood. This barrier often prevents many drugs, including most immunostimulants, from reaching the brain. However, because small molecules can cross this barrier, they offer the possibility of treating previously unreachable tumors in the brain.
“This is one of the big advantages,” says Barakat. “If you have a small molecule which can cross the blood-brain barrier, you can start treating unreachable tumors.”
Compound B is a derivative of a potential compound the research team was testing, but with a critical improvement — it is more water-soluble. This increased solubility is crucial for making a drug that is absorbed and distributed effectively throughout the body.
In their study, the team confirmed that Compound B had a powerful effect on the immune system. It increased the number of T cells—immune cells that target harmful antigens, including cancer cells—and the amount of cytokines, small proteins that play a critical role in regulating the immune response.
“This means that Compound B is effectively triggering the immune system to respond,” says Barakat. “It could be a major development in immunotherapy for cancers and chronic infections.”
Barakat’s lab discovers new compounds using machine learning and molecular modelling to screen potential candidates. These computer simulations help narrow the field to a few promising compounds, such as Compound B, which are tested in laboratory experiments.
“We do everything you would do in an experiment in the lab, but we do it on the computer,” explains Barakat.
Once a compound shows promise in these simulations, the researchers test it in peripheral blood mononuclear cells (PBMCs), which contain various immune cells in the blood. This allows them to model how the compound might affect the human body. When Compound B was added to these cells, the immune system activated, indicated by the secretion of certain materials suggesting an immune response.
The research team is now working to identify the specific targets within the body that Compound B binds to, which will be the focus of future studies.
This groundbreaking research results from collaboration across multiple disciplines at the University of Alberta, including the Mike Petryk School of Dentistry and the chemistry, oncology, biochemistry, and medical microbiology and immunology departments. The project received funding from the Alberta Cancer Foundation, the Canadian Institutes of Health Research, and the Li Ka Shing Applied Virology Institute.
Barakat and his team are optimistic about the future. “It is a breakthrough in our group that we’re pleased about, and we hope to follow the same procedure in developing new drugs.
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Compound B, showing such promising results, could represent a significant step forward in developing new, cost-effective treatments for cancer and chronic diseases, potentially saving lives and improving patient outcomes worldwide. Keep reading at Education Post News for more updates.
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