Thanks for the Complement: A New Approach to Treating Rare Diseases


By Staff Reports

(DGIwire) – The human immune system is a fascinating and complex entity. One of its divisions—called the innate immune system—has a range of powers, including the ability to activate antibodies and tag dubious cells for destruction. According to MedCityNews, this feat is accomplished courtesy of a set of biological processes known as the complement system. In the biopharma world, the race is on to explore the complement system and base novel treatments upon it.

Already, researchers and pharmaceutical companies have developed treatments for some rare diseases—such as two blood disorders, atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH)—harnessing the power of the complement system. The most prominent commercialized treatment to date for aHUS and PNH, however, is expensive and requires infusions every other week.

“There is room for improvement in the use of complement therapeutics to address various diseases,” says Gur Roshwalb, M.D., CEO of Akari Therapeutics. “We think a fresh look at the molecular processes at work in the complement cascade can serve as a source of innovation when it comes to novel treatment.”

A protein in the saliva of a tick has been used by Akari to derive a new inhibitor of the complement protein C5, a small recombinant compact protein named Coversin. The natural Coversin molecule works by damping down the immune response of the host animal that the tick feeds off of, enabling it to repeatedly feed without damage from host inflammatory substances. Akari is currently studying Coversin as a potential treatment for a range of rare autoimmune diseases, and is intended for use once daily as a self-injected therapy.

These diseases include PNH, a rare and serious acquired disease that causes red blood cells to break apart, according to the PNH Research & Support Foundation. PNH is thought to derive from a mutation in the PIG-A gene. Its symptoms can include intermittently dark urine, fatigue, muscle weakness, abdominal pain, back pain, thrombosis (blood clots), easy bruising or bleeding, headache and shortness of breath, according to the National Institutes of Health.

Another potential use of Coversin is to treat aHUS. Affecting about one in 500,000 people per year in the U.S., according to the National Institutes of Health (NIH), aHUS causes abnormal blood clots to form in small blood vessels in the kidneys. These clots can cause serious problems if they restrict or block blood flow. There is a chance aHUS can become a chronic condition, notes the National Organization for Rare Disorders (NORD).

A third potential area is Guillain Barré syndrome (GBS), a disorder in which the body’s immune system attacks part of the peripheral nervous system. The first symptoms include weakness or tingling in the legs; these symptoms often spread to the arms and upper body, increasing until some muscles can’t be used at all and, when severe, the person is almost totally paralyzed. In these cases, GBS is a life-threatening condition.

“Giving patients with aHUS, PNH, GBS and other conditions a new treatment option reflects the importance of learning more about the complement system,” says Roshwalb.

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