Photo courtesy of Dr. Juliana Anyanwu
Dr. Juliana Anyanwu helped run a clinical trial at Mulago
Hospital in Uganda to study the safety of the drug
hydroxyurea, a treatment for sickle cell disease.
During medical school, Fogarty Fellow Dr. Juliana Anyanwu was taken aback when a Nigerian friend suddenly asked her, "Do you know your status?" She looked blank, so he explained he was referring to whether or not she had inherited a sickle cell trait, information he said was essential to have before deciding on a mate.
Anyanwu, an American pediatrician with a Nigerian father, said their conversation really made her aware of the problem of sickle cell disease in Africa and the dearth of research about it. She soon developed a passion to help change that. While having one sickle trait is benign, children inheriting it from both parents typically face a host of health problems. Without treatment, the disease kills most children before they turn five. Such deaths are common in sub-Saharan Africa, where about three-quarters of the world's affected babies are born, according to the WHO.
Anyanwu successfully applied for a Fogarty fellowship to help run a clinical trial in Uganda to study the safety of a treatment for sickle cell disease - hydroxyurea. Fogarty's Global Health Program for Fellows and Scholars provides one-year, mentored clinical research experiences overseas for postdoctoral Fellows and pre-doctoral Scholars, helping to foster the next generation of clinical investigators and to build global health partnerships between U.S. and other researchers and institutions.
The drug hydroxyurea is commonly used in developed countries, but because it can curb the immune system it poses a potential concern for patients in countries with a heavy burden of malaria and other infectious diseases, combined with extremely limited health services. Children with sickle cell disease are known to be at particular risk for serious complications if they develop malaria, so it is important to know if hydroxyurea exacerbates this vulnerability. Uganda has a high level of both diseases.
The trial, funded by the Doris Duke Charitable Foundation and collaborators, is randomizing 200 children to receive hydroxyurea or a placebo. The principal investigators are Drs. Chandy John at the University of Minnesota and Christopher Ndugwa of Mulago Hospital in Uganda; both serve as Anyanwu's mentors.
Their sage advice has been crucial to her success. "You can't expect everything to go A-B-D-C-E and F," Anyanwu said. "There will be challenges and you learn from these challenges. It’s good to have someone who’s been there, done that, wisdom from someone else who has been through the experience you are trying to go through."
After arriving in Uganda, Anyanwu became immersed in overseeing management of the trial, which enrolled its first patient in September 2014. She ensured appropriate documents were in place, examined lab processes that would be required and prepared and edited an operations manual. She also needed to confirm everyone was on the same page. "I wanted to make sure that trust was in place, that everyone understood the goal of the trial and would work toward it as a team," she said.
The experience has helped her develop new skills and gain valuable insights. "It's been a real eye-opening opportunity. You're thrown into a new environment, a slightly different work culture, and you're trying to learn so many things," she noted. "I know that international health is what I am meant to do."
The study's results may have great implications and cause a paradigm shift in sickle cell disease management in Africa, Anyanwu said. If the trial shows hydroxyurea's benefits far outweigh the risks for children, the evidence could help persuade health officials to make it available, which could save lives and reduce pain and other symptoms. "I'm also hoping this trial will be a catalyst for other interventions for the disease and that this will emphasize the need for newborn screening here," she said. "And I hope it will also shed light on the need for more sickle cell research in sub-Saharan Africa, because it's immense."