What Are the Novel Treatments for Sickle Cell Disease?
More Programs and Publications Featuring Dr. Seethal Jacob
In this program:
For sickle cell disease, what's the latest about current and emerging treatments? Watch as expert Dr. Seethal Jacob shares advice to help improve research funding for sickle cell disease and how patients can help improve SCD care.
Transcript
Interviewer:
Can you give us an overview of the treatment landscape in sickle cell, and what are the treatment...what are the current treatments and the novel treatments that are out there or actually in the works for patients?
Dr. Seethal Jacob:
Absolutely. Hydroxyurea (Hydrea) is probably the treatment that we know the most about in regards to sickle cell disease. We've been using it for 20 to 30 years plus for sickle cell disease specifically. It's really the only disease-modifying medication that we have for sickle cell. It's shown benefits of decreasing pain crises, decreasing episodes of acute chest syndrome, decreasing hospitalizations, decreasing the need for unexpected blood transfusions, and also improving organ function and prolonging life span as well, but since that time, we've had three new medications pop up in 2017, we received approval for l-glutamine or Endari. L-glutamine has also been improved to help decrease the number of vaso-occlusive pain crises that patients have and is approved for those that are five years old and up, so does allow the treatment to be used for a wide range of ages. More recently, we had two new medications that were approved, one is called voxelotor (Oxbryta), and another is called crizanlizumab.
Voxelotor is approved for the treatment of anemia in patients with sickle cell disease, and I mentioned earlier that anemia is one of the most common complications that we see in sickle cell and by improving the anemia, we are doing so by decreasing the hemolysis or the breakdown of the red blood cells and potentially decreasing the complications that we see in sickle cell as a result of that, those studies are ongoing for the treatment of voxelotor, but we do know that it does increase hemoglobin. That's approved for individuals that are 12 years old and up, but current studies that are happening right now is looking to see if it's safe in children that are younger than 12 as well.
And then at the last medication is crizanlizumab, an IV medication that's been approved for those who are 16 years old and up, and its focus is really on those who... Whether an individual is on hydroxyurea or not, they may be having breakthrough pain crisis and frequent pain crises, and this medication is focused on decreasing the number of pain episodes in an individual with sickle cell disease has.
We'll notice that I just mentioned four medications for a disease that affects a large number of people globally, and while three of those came out within the last four years, again, which is great, we have a long way to go. Our patients deserve many more than that, and our patients deserve to know how combinations of treatments could improve their health in the long term as well.
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