Sickle cell disease affects approximately 100,000 Americans. Currently, the only available cure for the disease is a costly bone marrow transplant, putting it out of reach of most, with other ways of managing the disease limited to preventative care. Now however, how these symptoms are managed may change with the FDA approval of the first ever targeted therapy for Sickle Cell Disease, Adakveo, developed by pharmaceutical company, Novartis.

Normally, red blood cells are disk-like in shape. However, due to a genetic mutation for those with Sickle Cell Disease, their red blood cells resemble C-shaped crescents of “sickles”. Due to their shape, they often get stuck to the walls of blood vessels where they cause blockages. This may then result in vaso-occlusive crises in which blood flow to certain parts of the body is blocked (Lee: 2019).

Leading to severe pain and potential organ damage due to deprivation of oxygen, until now, the only way to manage such crises has been to avoid certain triggers, such as high elevation, extreme cold, physical exhaustion and certain drugs such as acetazolamide.

Now however, with Adakveo, also known as crizanlizumab-tmca, people with the disease may have another option. For those affected, a protein known as P-selectin found on the cellular lining inside blood vessel walls and platelets enables crescent-shaped red blood cells to stick to blood vessel walls, thus causing blockages. Adakveo works against ths by binding to and blocking P-selectin, thus preventing blockages and preventing vaso-occlusive crises (ibid.).

The drug’s approval comes following results from a randomized clinical trial of 198 participants. There, researchers showed those who received Adakveo required an average of 1.63 hospital visits per year, whereas those on a placebo required an average of 2.98. The trial also found that those taking the drug had a longer time until experiencing their first vaso-occlusive crisis, and that 36% of those on the drug didn’t have a vaso-occlusive crisis at all throughout the study’s duration (FDA: 2019).

According to Kenneth I. Attaga MD, director of the Center for Sickle Cell Disease at the University of Tennessee Health Science Center at Memphis, “We know this drug can decrease the frequency of sickle cell pain crisis in a significant and clinically meaningful way...The approval of crizanlizumab is an important advancement for people living with this very difficult condition (Kunzmann: 2019).”

Sources

Lee, Bruce: Forbes

FDA News

Kunzmann, Kevin: MDmag