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inflammation and pain in bone joints.
Scientists have yet to identify the cause of autoimmune disorders, though they suspect genetics and/or outside environmental factors may play a role. Doctors normally prescribe anti-inflammatory drugs and steroids that reduce pain and swelling, but they have side effects, and don’t really have an impact on the underlying cause of the condition.
However, researchers in recent years have focused on ways to manipulate the immune system itself.
“A major goal of autoimmune disease research is to ‘re-educate’ the immune system by using tolerance induction strategies that selectively block or prevent deleterious immune responses while leaving protective immunity intact,” according to a report by the National Institutes of Health.
ChemoCentryx, based in Mountain View, CA, is creating small-molecule oral pills that interact with novel protein targets called chemokines and chemokine receptors. By limiting the activity of the chemokine system, the idea is that ChemoCentryx can disrupt a vital process that leads to autoimmunity, without shutting down essential immune defense functions that protect people from infections. The company has raised more than $330 million from investors.
One emerging field is called cellular bioenergetics, the science of manipulating the way cells create, store and consume energy. Until now, scientists have mostly explored cellular bioenergetics in treating cancer and obesity.
But autoimmune disorders are starting to attract more attention. The University of Colorado at Colorado Springs recently established an Institute of Bioenergetics and Immunology. The school signed a license agreement with Viral Genetics, a biotech startup based in San Marino, CA., to commercialize its discoveries.
Lycera has developed a compound called Bz-423 that interferes with the ability of diseased white blood cells known as T cells to feed itself.
In the study, the company concluded T cells primarily generate energy through oxidative phosphorylation, a metabolic process that produces adenosine triphosphate (ATP). This molecule is responsible for transporting energy between cells.
Diseased T cells have a unique feature: without ATP, the cells manufacture a superoxide that ultimately kills the cell. Bz-423, Lycera says, slows down the cell’s ability to manufacture ATP by binding itself to the enzyme underlying the molecule. Healthy T cells are not affected because they don’t behave the same way.
What excites Glick is the prospect of creating orally delivered drugs based on Bz-423. Such drugs would be less expensive than injectable drugs and lead to higher patient compliance, Glick says.
However, Glick cautions the company is still a long way off. The company is essentially creating a new class of drugs, which normally draws intense regulatory scrutiny from the Food and Drug Administration.
Glick, though, is optimistic.
“The pre-clinical info is pretty encouraging,” he says. “The company is exceptionally well capitalized. We’re ahead of schedule.”