Overview of Sickle Cell Treatment Landscape

Sickle Cell

Jan 6

In this program:

What sickle cell treatments are available for patients? Dr. Marwan Shaikh and hematology nurse practitioner Maya Bloomberg provide an update on current treatments, research study results, and treatment improvements currently in development.

Transcript

Ariqa Everett:

So we learned there are a lot of factors that can affect the patient's outcome of sickle cell disease. Dr. Shaikh, were there any words you wanted to share about the overview of the treatment landscape for sickle cell disease?

Dr. Marwan Shaikh:

Treatment in general. We have the four drugs that we mentioned. Hydroxyurea (Hydrea), like Maya mentioned, has prolonged survival, decreased risk of hospitalizations and everything, and then in 2017 and L-glutamine (Endari) was approved as well, and so that study also, their studies also showed very similar results. Decreased hospitalization, decreased rates of pain crises, the newer agents voxelotor (Oxbryta) and crizanlizumab (Adakveo). Crizanlizumab again, you saw a decrease in number of hospitalizations and decreased pain crises, and the interesting theory there that we had people going from multiple crisis a year down to zero, so they were not being hospitalized anymore, they were a subset of patients that we thesis one of the unique things that really stuck out to me about that. Voxelotor, the goal of voxelotor is to increase the hemoglobin and it increases it by about one gram from a seven or eight up to a nine or 10.

Okay, and so the studies weren't designed to say, “Okay, this actually reduces pain crises, this actually reduces hospitalization,” so that part we don't have...which is what we still want to know, and so again, like you mentioned, people are being put on combination therapy for the first time, and we're seeing people who are remarkably, getting better, and so I think...yes, we're right at the forefront. We need still that long-term data. The other thing that comes up with these medicines is the cost, and so these medicines aren't cheap, especially the new ones, and that's just…that's how medicine is in the healthcare system is a society and you know the insurance and sometimes get the issues with improving them or 60 percent of patients are Medicare, Medicaid, so that can impact what medicines we can treat them with. So when you come down to what's your best bang for your buck, what's going to give you the best benefit for cost, and it's going to come down to hydroxyurea, and it's unfortunate that most patients aren't on hydroxyurea either their physicians for some reason, don't...aren't prescribing it. And I... and I don't have a good reason why for that, but if patients start with that medicine, at least that's a starting point.

So when you start having those discussions with your doctor, like you mentioned just getting that first drug on there, and then educating yourself and getting interested in some of the other agents to help as well.

Maya Bloomberg, APRN:

Just looking at the landscape in general, we have so many medications there in the pipeline, we keep getting approached by so many different pharma companies to open their trial letter sensors, so where you have Agios, you have Fulcrum, you have Forma, you have so many different companies that are looking at different treatment options in pill form, so at least that'll be easier for people who are poor venous access and can't do the IV infusions. But the goal ultimately is, again, to find a medication that stabilizes their red cells to help improve the anemia and improve pain as well, so you mentioned that the voxelotor...the study really was focused more on raising hemoglobin, not necessarily pain crisis, you only had to have at least one pain crisis in 12 months, so this definitely wasn't the richest study in pain episodes, that's why you kind of have to see is your patient's main issue pain, is it more the anemia and kind of come up with a strategy that way, but we have so many exciting products, I feel like everybody is so focused on the universal cure, and gene editing definitely is something that shows some promise, but it's important to realize that gene editing is not going to be a universal cure, there's going to still be a quarter of patients around who aren't going to qualify for it if they have any antibodies.

So I think the goal should be is to be able to continue to find different treatment options to ultimately improve the quality of life, because along the way, gene therapy definitely is promising...do not get me wrong, I'm super excited about that. I think the preliminary and the interim data thus far has been really great, however, along the road, and in a much sooner time frame are going to continue to see different treatment options to address different parts of that sickle cell pathophysiology on that cascade to ultimately stabilize red cells and improve pain in the process.

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