image: Patrick Leavey, M.D. view more
Credit: UT Southwestern Medical Center
DALLAS – Aug. 24, 2021 – In a move that will increase access and efficiency of clinical trials for patients with sickle cell disease, UT Southwestern has become a founding member of the new Sickle Cell Disease Clinical Trials Network (SCD CTN) to bring treatments and curative options to people suffering from this potentially life-shortening red blood-cell disorder.
The American Society of Hematology (ASH), 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, M.D., 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.
UT Southwestern’s inclusion in the national network underscores its commitment as an academic medical center to bring the latest treatments to underserved populations. Sickle cell disease hits Black Americans the hardest, with the genetic disease occurring in 1 out of every 365 African American births.
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.
Alecia Nero, M.D., Associate Professor of Internal Medicine and Pediatrics, said the push to bring clinical trials to new heights is badly needed. 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.
“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,” Dr. Nero said, adding that she’s seen clinical trials fizzle for lack of patient participation.
Dr. Nero, who treats pediatric sickle cell patients at Children’s, is Director of the Transition Sickle Cell Program, which helps patients successfully transfer from pediatric care to adult care. Young adult patients are at increased risk of death during this critical period, making transition of care a high priority. She is also the adult sickle cell program medical director and cares for patients at Clements University Hospital and Parkland.
A description of the program was part of UT Southwestern’s application to the Washington D.C.-based ASH to become a founding member of the network.
“This was very competitive. It speaks to our institution and the leadership and ASH seeing all the things we can do at UT Southwestern.” Dr. Nero said.
Dr. Leavey said UT Southwestern’s leadership in sickle cell clinical research and its data resources also helped, along with an advisory board with community members.
“That’s really important,” he said. “We want to be conscious of what the community sees to be important in the management of their lifelong disease.”
Drs. Nero and Leavey said they hope the network will bring significant advances against the disease by opening new collaborations with pharmaceutical companies.
Dr. 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.
Dr. Leavey is a member of the Harold C. Simmons Comprehensive Cancer Center.
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About UT Southwestern Medical Center
UT Southwestern, one of the nation’s premier academic medical centers, integrates pioneering biomedical research with exceptional clinical care and education. The institution’s faculty has received six Nobel Prizes, and includes 25 members of the National Academy of Sciences, 16 members of the National Academy of Medicine, and 13 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,800 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in about 80 specialties to more than 117,000 hospitalized patients, more than 360,000 emergency room cases, and oversee nearly 3 million outpatient visits a year.