Will New Sickle Cell Disease Drugs Transform Quality of Care for Patients?

Dr. Andrew Campbell of Children's National shares his excitement around the increasing treatment arsenal in sickle cell disease and how this shift might allow for more personalized approaches in care. Campbell speaks to this new era of multi-drug approaches, gene therapy, and the hope that these tools provide global options to treat sickle cell disease.

Sickle Cell Disease, FDA approval, sickle cell anemia, nigeria, congo, india, multi-drug treatment, hemoglobin, Oxbryta, Hydroxyurea, Voxelotor, Erythropoietin, ADVAKEO, Children’s National Hospital

About this expert

Andrew Campbell, MD

Director, Comprehensive Sickle Cell Program

Children’s National Health System

Director of the Consortium for the Advancement of Sickle Cell Disease Research (CASiRe)


Q: Is the approval of Voxelotor (Oxbryta) an exciting development for the global sickle cell population?

Dr. Andrew Campbell:
Oxbryta is -- was -- very exciting news to the sickle cell community because -- you know, first of all, we’ve only had two sickle cell disease-approved drugs for patients with sickle cell disease in the United States. The reason why this drug is important is because many patients with sickle cell disease even despite the use of the most common disease modifier or FDA-approved drug called Hydroxyurea, they still remain very anemic. What we mean by that is their hemoglobin level which measures anemia is on a low side. A lot of patients experience fatigue, they’re very tired, and also the low hemoglobin in certain patients -- in certain sickle cell disease patients -- have been linked to complications such as silent infarcts, strokes, also have been linked to kidney disease – we are not sure if it’s the cause, but it’s been linked to it. But this drug which showed to increase the hemoglobin by one gram in about two thirds of patients with sickle cell disease is a potential game-changer because we are seeking medications -- drugs -- in sickle cell disease to improve anemia in sickle cell disease. So, this drug allows us to expand the use of drugs that increase hemoglobin. 

Dr. Andrew Campbell:

Right now, there’s one called Erythropoietin [EPO], so this drug is a different one and it improves the oxygenated state of the sickle cell hemoglobin. And it allows the hemoglobin to increase, and it decreases what we call the breakdown of red blood cells. And we are very excited about it, and we hope in the future in the general population of sickle cell disease that  Oxbryta [Voxelotor] can improve those anemia symptoms. Right now, there’s no data right now on the improvement of pain, but right now we -- I guess time will tell if it can improve pain, but we are excited that the symptoms of anemia and the hemoglobin level will be higher -- based on a study -- in about two thirds of patients.

Q: Are you encouraged about the future of sickle cell?

Dr. Andrew Campbell:
Yes, I am very encouraged about the future of sickle cell disease. Now that we’ve had two new drugs with FDA approval, another one is called ADAKVEO [Crizanlizumab] which is an anti-adhesion molecule from Novartis. So, Novartis and Global Blood Therapeutics which is Oxbryta has now opened the door for newly approved trials for sickle cell disease. Many sickle cell disease trials unfortunately have failed to meet their what we call clinical goal or endpoint, but these last two drugs did. So, what this is going to do is we’re going to start thinking about multi-drug treatments for sickle cell disease, so we’re very excited for that. In the future, some patients might respond to Hydroxyurea which is already FDA approved, some patients might respond to ADAKVEO which is anti-P-selectin with Hydroxyurea. Some might respond to ADVAKEO and Oxbryta, so we now are thinking about allowing us to do more personalized medicine, right? Allow us to do a multi-drug treatment for sickle cell disease, tailored treatment, so the more drugs we have available – we’re excited! 

Dr. Andrew Campbell:

And the other is gene therapy. We are also very excited even though it’s in its infancy, this can lead to curative light treatment of sickle cell disease that can be disseminated to many patients. Right now, in a developed world -- because that’s where we are -- we hope that these new treatments, FDA approved drugs and gene therapy can lead to a more global approach to the cure of sickle cell disease because the global burden of sickle cell disease is not in Europe and the United States, it’s in countries like Ghana, Nigeria, Congo, Sub-Saharan Africa, and also India.


Related Content

The information on Diverse Health Hub is provided for educational purposes only, and is in no way intended to diagnose, cure, or treat any medical or other condition. Always seek the expert advice of your healthcare team.

© 2020 Diverse Health Hub