Meet a Riley Researcher: Dr. Ngoc Tung Tran

Two years ago, Ngoc Tung Tran, PhD, opened his lab at the Herman B Wells Center for Pediatric Research to take on the feat of helping people with the rare disease, Fanconi anemia. As an assistant professor of pediatrics at the Indiana University School of Medicine, Dr. Tran has confidence in his team’s capabilities to contribute to the treatment of the painful disease.

Fanconi anemia is a genetic condition that impacts the bone marrow and results in the decreased production of all types of blood cells. Patients with the disease have a mutated gene. Typically, blood stem cells reside in bone marrow that develop into red blood cells, white blood cells, and platelets; however, blood stem cells with these mutations die gradually and are challenged by many stress factors.

“At a certain time in the patient’s childhood, there will not be enough blood stem cells in the bone marrow, which means other types of blood cells can’t be produced,” Dr. Tran says. “Bone marrow failure can occur and leads to an increase of blood cancers such as leukemia.” Other side effects include cancers of the neck and head and of the female reproductive system, hearing loss, other anemias, infections, and bleeding problems.

In his lab, they are using a gene editing tool to correct the mutations in the blood stem cells. “Our aim is to correct the mutations by isolating them from the patients with the hope of transplanting it back,” Dr. Tran explains. “The corrected blood stem cells will then regenerate the whole blood system and rescue the bone marrow failure.”

Dr. Tran appreciates the collaborative environment at the Wells Center. The resourcefulness and support from other physicians and researchers in unmatched, making it easier for him to accomplish his goals. At the same time, the biggest limitation to research is funding. If he had unlimited funds, Dr. Tran would focus on developing translational research into a clinical trial.

Dr. Tran wants to extend his gratitude to Riley Children’s Foundation donors who make his research possible. “Your support for us on this journey will help us support those suffering from the disease and allow them to discontinue the genetic chain for generations to come.”