For Jason Farrar, M.D., his hometown of Little Rock is the perfect place to raise a family and establish his research career.

After graduating with a biology degree from Rhodes College in Memphis, Farrar found his calling as a physician-scientist while serving as a research technician at St. Jude Children’s Research Hospital in Memphis.

"During my year at St. Jude, I worked under a physician-scientist who studied rare immune deficiencies in children. That's where I got the bug to combine a research and clinical career," he said.

He enrolled at the University of Arkansas for Medical Sciences (UAMS) College of Medicine and found his niche in cancer through a summer research program studying a common high-risk chromosomal abnormality in infants with acute lymphoblastic leukemia (ALL). He continued his research of ALL throughout medical school, making it the subject of his honors research thesis.

From there, Farrar completed his pediatrics residency at the Children’s Hospital of Wisconsin followed by pediatric hematology/oncology fellowship in a joint program at Johns Hopkins University and the Pediatric Oncology Branch of the National Cancer Institute (NCI).

"We didn't really intend to stay on the East coast for 9 or 10 years, so when we were ready to move, I immediately thought of UAMS," he said. Inquiries were made and Farrar found a perfect fit in a position splitting his time between research at the UAMS Winthrop P. Rockefeller Cancer Institute and patient care at UAMS' affiliate, Arkansas Children's Hospital (ACH). He serves as an assistant professor in the UAMS Department of Pediatrics and sees patients in ACH's Hematology-Oncology Clinic and inpatient unit.

“I spend about 80 percent of my time at the Cancer Institute and about 20 percent in the clinic, where I treat kids with cancer and hematological conditions such as sickle cell anemia,” he said.

Farrar's main research focus is on bone marrow failure syndromes, specifically the rare blood disorder known as Diamond Blackfan anemia (DBA). Only about 40 new cases of DBA are diagnosed in the United States each year.

First recognized in the 1930s, DBA is a genetic condition that most often appears in infancy and causes profound anemia. Long-term steroid therapy and chronic blood transfusions are the most common treatments for DBA, but these are highly toxic and can lead to other challenges such as iron overload. The only curative treatment for DBA is a stem cell transplant, which is a complex medical procedure requiring a lengthy recovery.

In addition to the anemia itself, DBA also predisposes to the development of cancer. Children diagnosed with DBA develop acute myeloid leukemia (AML) and some solid tumors at a much higher rate than the general public.

"I got interested in DBA during my fellowship when I picked up a side project about a patient who had an unusual missing part of a chromosome and appeared to have DBA. I spent a couple years defining that deletion and demonstrating the gene that caused it. That led to my DBA research career," he said.

Today, Farrar continues to search for clues about the genetics of DBA. The ultimate goal, he said, is to connect the genetics with more effective and less toxic treatments for children with the condition. But his research of this extremely rare disease could have farther reaching potential as well.

"Sometimes it's hard to explain to people why you study rare diseases," he said. "From the child's perspective, DBA both alters their quality of life and can be life threatening. But from a societal standpoint, what we learn about conditions like DBA can be fundamentally important in understanding the basic processes of other diseases such as AML and myelodysplastic syndromes that affect the bone marrow and blood."

His work with the NCI's TARGET (Therapeutically Applicable Research to Generate Effective Treatments) initiative is using the most technologically advanced genomic techniques to develop new therapies for childhood diseases such as AML. As part of the cooperative group, Farrar helps to leverage clinical data and biological specimens from large-scale research studies in an effort to understand the pathways of various childhood cancers.

“This is another major research interest of mine — using modern, data-intensive techniques to explore the biological features of cancer so that we can start to consider new and more effective treatments.”

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