In a move that will increase access and efficiency of clinical trials for patients with sickle cell disease, University of Texas Southwestern (UTSW) Medical Center has become a founding member of the new Sickle Cell Disease Clinical Trials Network to bring treatments and curative options to people suffering from this potentially life-shortening red blood-cell disorder.
The American Society of Hematology, which created the network, is the world’s largest professional society of clinicians and researchers who focus on blood diseases. Under the leadership of Patrick Leavey, MD, professor of pediatrics at UTSW, a collaborative North Texas clinical trial unit was developed and invited to be a participating site. The North Texas unit includes Children’s Medical Center Dallas, William P. Clements Jr. University Hospital, Parkland Health & Hospital System, Medical City Dallas, and Cook Children’s Health Care System in Fort Worth.
Sickle cell disease hits Black Americans the hardest, with the genetic disease occurring in one out of every 365 African American births. Currently, there are only four drugs used to treat sickle cell disease, even though the disease has been described in medical journals for more than 100 years. Three of the drugs have been available only since 2017.
The 10-site Sickle Cell Disease Clinical Trials Network will coordinate patient enrollment in clinical trials at several academic medical centers nationwide, attracting enough patients to make the trials feasible, expanding treatment options for these patients, and growing medical science knowledge of what works in fighting the disease.
“Sickle cell disease, being a relatively rare disease, struggles with keeping significant trials open because it takes so long to get patients. By creating these networks, you can then enroll the patients to get the answer that you’re after,” said Alecia Nero, MD, associate professor of internal medicine and pediatrics at Children’s Medical Center Dallas, adding that she’s seen clinical trials fizzle for lack of patient participation.
Nero and Leavey said they hope the network will bring significant advances against the disease by opening new collaborations with pharmaceutical companies. Nero said there are 30 to 40 new drugs that pharmaceutical companies would like to bring to clinical trials for sickle cell disease. Her highest hopes are for new treatments involving gene therapies.
Other founding members of the Sickle Cell Disease Clinical Trials Network include The Johns Hopkins University School of Medicine, the University of Chicago, and Weill Cornell Medicine.
Edited by Gary Cramer