What happens in SCD?
In SCD, mutations in the gene that contains the information necessary for the production of the hemoglobin protein cause the protein to be made incorrectly. Hemoglobin is the protein that carries oxygen in red blood cells.
As a result of these mutations, the red blood cells “sickle” or twist into an unnatural shape that has difficulty traveling through small blood vessels. Moreover, the sickled cells tend to stick to the walls of the blood vessels. The combination of these two factors means that small blood vessels may become blocked, causing inflammation as the immune system tries to clear the blockages. This inflammation can also stimulate nerve cells, causing pain that can range from mild to severe.
Anti-inflammatory medications reduce inflammation through different mechanisms depending on the type. One anti-inflammatory medication is currently in development for the treatment of SCD. Another anti-inflammatory has been tested, but no updated information has been published since 2017.
ACZ885 is an anti-inflammatory treatment being developed by Novartis. The medicine has been approved by the FDA to treat systemic juvenile idiopathic arthritis and several periodic fever syndromes under the brand name Ilaris. Canakinumab, the active ingredient in Ilaris, is an antibody that reduces inflammation by binding to a pro-inflammatory signaling molecule called interleukin-1 beta (IL-1β).
A Phase 2 clinical trial (NCT02961218) is currently testing the safety and efficacy of ACZ885 in children and young adults with SCD. The study is expected to conclude in June 2020.
NKTT120 was an investigational therapy being developed by NKT Therapeutics to treat pain and inflammation in SCD. It is an antibody that triggers the death of a particular type of immune cell called invariant natural killer T- (iNKT) cells. These cells produce large quantities of inflammatory signals. By targeting them for destruction, NKTT120 may be able to reduce inflammation and reduce associated symptoms.
The safety and activity of NKTT120 was investigated in a Phase 1 clinical trial (NCT01783691) with promising results. However, the company has not published any updated data on the therapy since 2017.
Last updated: Feb. 17, 2020
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