Until very recently, sickle cell disease was a condition that left those diagnosed very few treatment options. What was available still left patients with moderate-to-severe pain episodes and the anemia commonly attributed to the chronic condition.
However, in recent months, new developments have led to the approval of two new drugs, Adakveo and Oxbryta, that could solve those problems and drastically improve the quality and length of life for patients diagnosed with sickle cell disease.
To gain more insights into the latest developments in sickle cell treatment and research, BlackDoctor.org spoke with Dr. Alexis Thompson, currently the Hematology Section Head at Ann and Robert H. Lurie Children’s Hospital of Chicago and one of the world’s leading experts in hematology and oncology.
Could you speak to any recent developments that you've seen in sickle cell research that people should be made aware of? If so, how will they impact patients’ day-to-day lives?
Dr. Alexis Thompson: There are two principal categories of recent advances in sickle cell disease. One is the approval of new disease-modifying therapies. And the second is the early data on an additional option for a curative approach. I think these are both particularly exciting developments. These advances represent opportunities that really could change the lives of individuals with sickle cell disease.
You May Also Like
Speaking of the medications, what could the introduction of new drugs like Adakveo and Oxbryta mean for patients going forward?
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.
You May Also Like
Adakveo, which is also called crizanlizumab, works in a very different way. It actually inhibits adhesion or the stickiness of red cells to other surfaces. This is stickiness to other red cells, to white cells, or to the surfaces of the internal surfaces of blood vessels, all of which lead to obstruction or decrease in the flow in those blood vessels, decreased oxygen delivery to tissues, and pain. And the studies done so far with crizanlizumab have shown a reduction in the frequency of pain episodes and the length of hospital stay. Both of which are direct benefits in individuals who have sickle cell disease.
What's something that you wish the general public was more aware of in regards to sickle cell disease and the treatment of it, because I know there tends to be stigma with that in general?
Dr. Thompson: I want the general public to know that we are all impacted by the burden of sickle cell disease, and until fairly recently, there have been very few treatment options for individuals with sickle cell disease. Sickle cell disease is not going away. It is the most common inherited blood disorder in the world. It is a relatively rare disease in the United States, but not at all globally. It is a condition that is associated with tremendous amounts of suffering that we hope can be mitigated by better medical care.
I think that gaining more understanding of any disease, especially if that leads to new treatments, is what I believe all of us would want to see happen in terms of advances in science and medicine. Sadly sickle cell disease has not been the beneficiary of sustained research investments. I acknowledge that a lot of important research has been done over time, and perhaps finally we are seeing this investment leading to new therapies in sickle cell disease, which is extraordinarily exciting, and really all of us should celebrate this. I'm well aware that there are concerns about the costs of medications and the cost of medical care. I think these are important issues. I don't think that they mitigate the need for looking for new therapies and advances in order to treat this condition.
Right now during the times of COVID-19, everyone's being impacted in some capacity. I know with the sickle cell population, there are particular challenges, one being that the Red Cross recently announced that they have seen a significant drop in blood donations. And what are some other challenges that the population is facing and how can everyone else pitch in to help?
Dr. Thompson: I think the challenge with social distancing for COVID19 is having individuals with chronic diseases becoming isolated. Isolation in and of itself causes stress. And we know that stress with chronic conditions, like sickle cell disease, makes those diseases worse. And so to the extent that we can socially responsible ways to support our neighbors, our colleagues, our family members through what is undoubtedly the most stressful experience that many of us will ever have in our lifetimes, it is so important for individuals with chronic diseases to understand that others around them are looking out for them and that they will get the support that they need.
I think it's also important though, for individuals who have chronic illnesses to have access to high-quality medical care. And that while understandably many people have been very concerned about going to medical facilities and in particular emergency rooms during the current pandemic, we know that there are aspects of sickle cell disease, like many other chronic illnesses that still need to be managed. Having a good relationship with a provider who may be able to take care of some things with telemedicine, or can help provide guidance for families about when they do and don't necessarily need to seek care is so important. What we don't want is for people with sickle cell disease to be afraid and isolated.
