Point-Counterpoint: Curative Therapies for SCD - Does it Make More Sense to Target the Root Cause Than All the Downstream Events

Since the initial discovery of sickle cell disease more than 100 years ago, investigators have been working to unravel both the cause of the disease and the pathophysiologic mechanisms whereby it engenders the panoply of effects it has on essentially all organ systems. However, to date, only two therapies are FDA-approved, and at best only a handful are likely to be approved in the near future. Moreover, all the current and near-term future therapies are palliative rather than curative. 

Meanwhile, hematopoietic stem cell transplantation as a curative therapy has progressed from a high-risk procedure to one that is routinely successful, although not without morbidity and still an appreciable mortality. And recently, gene therapy has been in active phase 2 clinical trials, with at least some successes reported, although gene therapy still also requires bone marrow ablative therapy and entails both known and unknown risks.

Dr. Saunthararajah will discuss the various approaches being designed and tested to achieve cure in sickle cell disease, the progress already made, and the challenges for the future.

Dr. Marilyn Telen will discuss how development of an in-depth understanding of the pathophysiology of vaso-occlusion and end-organ damage in sickle cell disease has led to the development of targeted therapies designed to reduce disease symptoms as well as prevent or ameliorate end-organ damage.