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.