Dr. Thompson: The recent approval of two new medications with a sickle cell disease-specific indications gives patients and their providers options that prior to this were fairly limited. I hope that one of these additional treatments will help people with sickle cell disease live with less severe pain and with improvements in the quality of their life and hopefully the length of their lives. These new drugs may not eliminate all manifestations of sickle cell disease, but could make vast improvements.
It is important to recognize that the two new medications fundamentally work through different mechanisms. Both mechanisms are powerful, both are important for controlling sickle cell disease. We know that both of these drugs can be used safely with hydroxyurea and that’s also an important finding.
Hydroxyurea is a much more well-established treatment for sickle cell disease. We’ve been using this drug for over 20 years. And we have confidence that for many individuals, and particularly adults with sickle cell disease, that there may be some real advantages to being on hydroxyurea, which should not preclude someone from considering these new therapies, especially if with hydroxyurea their sickle cell disease is still active. Having said that, one could also be treated with these new medications without taking hydroxyurea. We’re not recommending that anyone stop their hydroxyurea, but certainly for those for whom hydroxyurea in the past has either been not successful or has had side effects, they now have some new treatment options.
What are some of those challenges that sickle cell patients faced before that these could solve specifically?
Dr. Thompson: I think that the complications that these two new drugs address are anemia, the red cell breakdown, or what we call hemolysis, and pain episodes. All of these are signatures of sickle cell disease. And all of these are debilitating to patients. We know that with voxelotor, which is also called Oxbryta, patients taking it will have an increase in their hemoglobin and that their hemoglobin will be less prone to sickling. So since sickling is the fundamental abnormality in sickle cell disease, this drug has great promise in addressing sickle cell disease at its core, at the root cause of sickling.