Antineutrophil cytoplasmic autoantibodies-associated vasculitis, or ANCA vasculitis, are autoimmune conditions that cause blood vessel inflammation, or vasculitis. The conditions stem from the immune system mistakenly attacking healthy blood vessels, causing damage and swelling that restrict blood flow.

Different types of ANCA vasculitis affect different organs. Current treatments can lead to remission — or no symptoms appearing for a time — but can not cure the condition. The treatments can also have severe side effects.

Researchers are trying to develop therapies that achieve faster remission and that keep symptoms under control longer. They are also trying to find a cure. Here are some of the therapies being developed for ANCA vasculitis:

Biological drugs

Biological therapies for ANCA vasculitis focus heavily on antibodies that prevent the immune system from generating inflammation.

Orencia (abatacept, CTLA-4-IgG1), developed by Bristol-Myers Squibb, blocks a protein called CTLA-4 that tempers immune response. The Phase 3 ABROGATE clinical trial (NCT02108860) is investigating Orecia as a treatment for a type of ANCA vasculitis known as granulomatosis with polyangiitis. The condition is also called GPA or Wegener’s.

GlaxoSmithKline developed Nucala (mepolizumab) as a treatment for an ANCA vasculitis known as eosinophilic granulomatosis with polyangiitis, also known as EGPA or Churg-Strauss syndrome.

Nucala is an antibody that targets interleukin-5, a cytokine or immune system protein that can cause inflammation.

A Phase 3 clinical trial (NCT02020889) showed that a significantly higher number of patients who took mepolizumab achieved remission, compared with those taking a placebo. Another finding was that Nucala-treated patients’ remission lasted far longer than what the placebo group achieved.

The results allowed patients to reduce the amount of glucocorticoids they were taking. Glucocorticoids are hormones with potent anti-inflammatory and immune suppression properties but that have harmful effects as well.

GlaxoSmithKline published the study’s findings in the New England Journal of Medicine. The results prompted the company to submit a new drug application for Nucala to the U.S. Food and Drug Administration in June 2017. It covers Nucala as an add-on therapy to corticosteroids.

Benlysta (belimumab), also developed by GlaxoSmithKline, is an antibody that blocks the chemical BAFF, or BLyS. BAFF stimulates the production of immune system B-cells that can cause inflammation in ANCA vasculitis. By reducing B-cell levels, GlaxoSmithKline hopes Benlysta can help ANCA vasculitis patients achieve remission. The company has completed a Phase 3 clinical trial (NCT01663623) of Benlysta as a treatment for ANCA vasculitis.

Other biological drugs targeting the BAFF pathway that have the potential to treat ANCA vasculitis include blisibimod (AMG623) from Anthera Pharmaceuticals and atacicept (TACI-Ig) from Merck Serono. They have yet to be tested in clinical trials.

Small molecule drugs

Avacopan (CCX168), developed by ChemoCentryx, is a small molecule that inhibits a protein called the C5a receptor (C5aR) that can temper inflammatory responses.

Two Phase 2 clinical trials (NCT02222155 and NCT01363388) have shown that ANCA vasculitis patients can achieve remission with it. The results were published in the Journal of the American Society of Nephrology.

A Phase 3 clinical trial (NCT02994927) of the compound is recruiting participants.

Gusperimus (15-deoxyspergualin, spanidin), developed by Nippon Kayaku, suppresses the immune system. A Phase 2 clinical trial (NCT00530075) tested it in patients with GPA. It was able to achieve remission in many of those who took it, and patients tolerated it well. The results were published in the Journal of the American Society of Nephrology.

Nippon Kayaku is conducting additional clinical trials to compare Gusperimus’ effectiveness with current therapies.

Note: ANCA Vasculitis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.