Keeping Track of a Rare Blood Cell Disorder

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(DGIwire) – One of the best ways to improve understanding of a disease is to collect comprehensive data from a wide swath of the population of patients who have it. This is certainly true regarding rare diseases for which treatment options are currently limited. One of the more ambitious undertakings of this sort in recent years is aiming to help enhance our insights regarding a rare blood cell disorder: atypical hemolytic uremic syndrome (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).

Initiated in 2012, the global aHUS Registry is an observational, non-interventional, multi-center registry designed to collect demographic characteristics, medical and disease history, treatment effectiveness and safety outcomes data for aHUS patients, according to reports published in the journals BMC Nephrology and Blood. By June 2015, a total of 826 patients were enrolled in the study. Data gathered from clinicians worldwide may help to describe aHUS presentation, progression and patient outcomes, as well as defining strategies for optimization of patient care. Among the data collected as of mid-2015: the mean age of diagnosis was 23.7; 49.9 percent of patients had undergone dialysis prior to being enrolled in the registry; and 57.3 percent had been treated with the antibody eculizumab, which is approved for the treatment of aHUS.

“Important research about aHUS is key to understanding the disease,” says Gur Roshwalb, M.D., CEO of Akari Therapeutics, a clinical-stage biopharmaceutical company. “Although one product is already approved for aHUS, research is being done to find more options for patients—particularly those who are resistant to the therapy being used today.”

Akari has developed a new C5 inhibitor—a small recombinant compact protein named Coversin—derived from a protein in the saliva of the Ornithodoros moubata tick. The protein C5 ordinarily splits into two components in the complement system—a part of the immune system—to clear out foreign invaders and unwanted cells; occasionally, however, these C5 components are produced in unregulated numbers, triggering various inflammatory and autoimmune conditions, including aHUS. In July 2016, Akari announced positive interim data from the first cohort in its Phase Ib study demonstrating the maintenance of complement inhibition using once-daily dosing.

“The more we learn about those who currently suffer from aHUS, the better we will be able to determine if Coversin could serve as a viable treatment,” adds Roshwalb.

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