What Novel Tools Are Being Used to Treat Sickle Cell Disease?

Dr. Ify Osunkwo of Levine Cancer Institute/Atrium Health discusses new therapeutic tools used to treat Sickle Cell Disease (SCD) over the last few years and how they will impact patient quality of life. Can SCD be cured early before long-term organ damage occurs? Dr. Osunkwo spotlights the need for better drug therapy, and also shares in the buzz at ASH 2019 around gene therapy options for SCD.

Ify Osunkwo, SCD QOL, Director of the Sickle Cell Disease Enterprise, Levine Cancer Institute, Atrium Health, ASH 2019, Sickle Cell, SWAY Ghana, SCD pain, SCD physical emotional, Acute pain episode, Malaria, Hemolytic, Oxbryta, Hemoglobin level, Endari, L-glutamine oral powder, Voxelotor, Crizanlizumab, ADAKVEO, SCT, Gene therapy, Curing SCD, long term organ damage

About this expert

Ifeyinwa (Ify) Osunkwo, MD, MPH

Director of the Sickle Cell Disease Enterprise

Levine Cancer Institute, Atrium Health


Q: What are some of the novel tools being used to manage sickle cell disease and improve quality of life for patients?


Dr. Ify Osunkwo: So, we have a had a bumper couple of years and in the past three years we’ve had three drugs for sickle cell disease approved by the FDA. We usually -- we previously only had Hydroxyurea, now we have Endari (L-glutamine oral powder), now we have Voxelotor (Oxbryta), now we have Crizanlizumab (ADAKVEO). And these are novel drugs that will definitely impact how well they do in terms of their physical clinical complications and symptoms, and ultimately, we believe it will impact their quality of life. And so, if you have treatment options then the goal is that they’re going to have better outcomes in terms of their quality of life and their physical functioning.


Q: What does the approval of Oxbryta mean for the sickle cell community?


Dr. Ify Osunkwo: So, the approval of Oxbryta is pretty important because globally a lot of people suffer from Sickle Cell Disease and probably live in a country where malaria is prevalent, and they have a lot of hemolysis and anemia. But even if you don’t live in a malaria prominent zone, sickle cell is a hemolytic disease, so it makes your red cells break down and you have a lower hemoglobin. Oxbryta works by increasing your hemoglobin level and in that way reducing your hemolysis to help reduce the long-term impacts of Sickle Cell Disease. And so we believe that when it’s an oral pill, that will make it more accessible in terms of taking it in compliance, but it also helps to mitigate the anemia that people of Sickle Cell Disease have.


When you’re anemic, you’re fatigued and you’re tired, and you can’t function. And the hemolysis also causes downstream damage to your organs – so we’re really excited that not just with Oxbryta, but with the ADAKVEO that was approved a couple weeks before the Oxbryta that we have more options to treat Sickle Cell Disease – reduce their pain with ADAKVEO and improve their hemoglobin level with Oxbryta.


Q: Are there any side effects to these novel agents?


Dr. Ify Osunkwo: Well right now there is a very favorable profile of side effects for Oxbryta. I think that when they did the study they reported some nausea, some abdominal discomfort, but really nothing profound that we can see in the phase three study, but you know as you take the medication you’re going to learn more about the side effects as you do your post approval analysis.


Q: What is the next chapter for sickle disease, as you see it?


Dr. Ify Osunkwo: So I think the first exciting thing we are seeing right now even at ASH [American Society of Hematology] is that there is a lot more focus on gene therapy, bone marrow transplantation, stem cell transplantation (SCT) and ways to cure Sickle Cell Disease. So there’s a number of abstracts and presentations around curative therapies as there’s a lot of focus on: "Can we cure the disease early before people end up with long term organ damage?" So I think that’s the really big focus in the next couple of years.


I think the other things we want – we want more drugs. We need more options that target different pathways to treat a very complicated single gene defect disorder. I don’t think that one drug alone is going to fix the entire pathophysiology, so we’re probably going to use most of these drugs in some combination or the other. Similar to diabetes and arthritis, we are going to need to target this pathway and that pathway to help improve the outcomes for our sickle cell patients.


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