Can Microfluidic Flow-Based Diagnostics Personalize Therapy in Sickle Cell Disease? A Vision for the Future
Dr. Patrick Hines, MD/PhD
Pediatric Critical Care Medicine Physician
Pulmonary Hypertension Specialist,
and Vascular Biology Research Scientist
James Herrick’s first described the “Peculiar Elongated and Sickle-Shaped Red Blood Corpuscles in a Case of Severe Anemia” in 1910. Linus Pauling discovered the molecular basis for sickle cell disease (SCD) in 1949. Since these landmark discoveries, there has only been one FDA-approved therapy for the prevention of SCD-related complications. Fortunately, this is an historic moment in sickle cell discovery with the largest pipeline of promising SCD-specific drugs in preclinical and clinical trials in the history of the disease. In addition to targeting hemoglobin polymerization, many of these drugs target the unique erythrocyte adhesive properties that contribute to microvascular obstruction. Despite this, there is no clinical standard for assessing erythrocyte adhesion. SCD providers will soon have drugs available to prevent and relieve adhesion-mediated complications, therefore there is an urgent need diagnostic tools to guide drug selection for individual patients and monitor therapeutic response. Dr. Hines has used flow adhesion models to understand the adhesive properties of sickle erythrocytes in a simulated blood flow environment, and has worked to standardize this process for clinical testing. He recently founded Functional Fluidics, a startup company who has deployed a standardized microfluidic flow-based test into the preclinical and clinical research market. Dr. Hines will discuss advances in microfluidic flow-based blood function testing, the role these tests are play in preclinical drug validation, and the potential for applications in sickle cell clinical trials and ultimately clinical therapy.