|At the Forefront of Policy and Public Health
CHORI Scientist Receives 1.2 Million FDA Grant
Pediatriac Emergency Medicine Physician and CHORI clinical scientist Claudia R. Morris, MD, of CHORI’s Center for Sickle Cell Disease and Thalassemia, has received a highly competitive grant through the federal Food and Drug Administration (FDA) orphan drug program to study the nutritional supplement glutamine for the treatment of pulmonary hypertension (PH) in sickle cell disease and thalassemia over a three-year period.
“Our hope is to use glutamine supplementation to increase nitric oxide production and thus decrease the oxidative stress and hemolysis that can lead to pulmonary hypertension in this particularly vulnerable group of patients,” says Dr. Morris.
Within the last 5 years, PH - high blood pressure in the blood vessels leading from the heart to the lungs - has emerged as one of the major causes of death in patients with sickle cell disease, with a tenfold increased risk of mortality. Currently there are no validated standard therapies for treating PH, although studies are ongoing. Glutamine, however, could hold the key to preventing PH from developing in the first place.
"Glutamine is an amino acid, found naturally in our diet, that is metabolized by the body into many different things," explains Dr. Morris. "It's of particular interest for sickle cell disease and thalassemia because we've determined that in this patient population, the severity of a patient's PH is associated with glutamine depletion. We thought it made perfect sense to try to treat the PH by replacing the glutamine that's missing."
The new $1.2 million dollar FDA grant Dr. Morris received will allow Dr. Morris to explore three different aspects of glutamine supplementation. The first is to assess the ways in which supplementation impacts what Dr. Morris calls arginine bioavailability.
"Arginine is one of the many different metabolites that can be produced from glutamine, and is the body's only substrate for nitric oxide production, which is compromised in patients with sickle cell disease and thalassemia," says Dr. Morris. Like glutamine, it too, is found in extremely low levels in this patient population, and should be increased with the addition of its precursor, glutamine.Dr. Morris will also be conducting pharmacokinetic studies and collaborating with Jung Suh, PhD and Frans Kuypers, PhD, at CHORI to look at different biomarkers associated with oxidative stress and hemolysis - the abnormal break down of red blood cells - both of which are strongly associated with PH.
Tuesday, May 17, 2011 8:19 AM