Sickle-Cell Disease in Africa
An Interview with Tanzanian Researcher Julie Makani
BY HELENE BLANCHARD, NIA
Julie Makani, a Tanzanian investigator, just completed a three-month sabbatical at NIH as part of an effort to integrate Western and African knowledge to improve the care of people with sickle-cell disease (SCD). Makani first came to NIH on February 13, 2013, to give a talk for the Wednesday Afternoon Lecture Series (WALS) on “Sickle Cell Disease: What Can Africa Contribute?” in which she talked about leveraging existing resources in Africa and the United States to develop programs that integrate health care, education, and research.
SCD is a group of inherited red-blood-cell disorders in which the red blood cells become hard and sticky and are C, or sickle, shaped. These cells die early, clog blood flow, and can cause painful episodes, serious infections, organ damage, and anemia. Worldwide 300,000 babies are born with SCD each year, with more than 70 percent on the African continent. SCD is a major cause of infant mortality in sub-Saharan Africa. Tanzania has recognized SCD as a major public-health problem and is using SCD as a model to establish scientific and technological solutions that are locally relevant but have global significance.
When Makani was at NIH for the WALS lecture, she met with Clinical Center Deputy Director for Educational Affairs and Strategic Partnerships Frederick Ognibene, who oversees the Sabbatical in Clinical Research Management program. The sabbatical program, which was launched in 2009, is self-directed training for experienced investigators and managers and includes one-on-one meetings with and demonstrations by experts in clinical-research management as well as other training opportunities. So far 23 investigators from 16 institutions in the United States and abroad have participated. Makani applied and was accepted into the program in the fall of 2013.
In January 2014, Makani returned to Tanzania to her position as a tenured faculty member and consultant physician at the Muhimbili University of Health and Allied Sciences (MUHAS) in Dar es Salaam, Tanzania. MUHAS is the main clinical, academic, and research center in Tanzania. She is also a Wellcome Trust research fellow (http://bit.ly/1jT96ul) in the Nuffield Department of Medicine at the University of Oxford (Oxford, England). She plans to return to the NIH in mid-2014 to spend more time in the sabbatical program.
Before she left, the NIH Catalyst met with her to find out more about her work and how she thought the sabbatical at NIH might help. The following is an edited version of the interview.
CATALYST: How did you decide to do a sabbatical at NIH?
MAKANI: When I was at NIH for my WALS lecture, I met with several outstanding NIH scientists including NIH Director Francis Collins, NHLBI Director Gary Gibbons, NIDDK Director Griffin Rodgers, and Fogarty International Center Director Roger Glass. I learned that NHLBI has led an extensive research program on sickle-cell disease (SCD) since 1972 and has committed more than $1 billion to improving the understanding of the disease and identifying new methods of treatment, management, and prevention. Although many studies have been performed in the United States, Europe, and Jamaica, more research is needed to understand the specificity of the disease in Africa. NIH can influence SCD research by moving forward the research agenda globally.
CATALYST: What did you do during your sabbatical and what did you like especially?
MAKANI: I focused on various aspects of clinical-research management, such as data management, laboratory testing methods, and patient-recruitment issues. I liked the one-on-one training aspect, the opportunity to have discussions with NIH experts, and being able to tailor the program to focus on my topics of interest. I also liked the idea of doing a sabbatical at the United States’ largest hospital that is totally dedicated to clinical research and being able to collaborate with NIH researchers.
CATALYST: Who will you be collaborating with at NIH?
MAKANI: I hope to collaborate with researchers at NHLBI, including Deputy Director Susan Shurin, who was my host during my sabbatical. She helped me identify other potential collaborators. I met with Courtney Fitzhugh and Caterina Minniti at NHLBI to discuss genotyping SCD patients on different continents to look at epigenetic (environmental) influences on phenotype. I am also collaborating with Nancy Geller and Neal Jeffries, both in NHLBI’s Office of Biostatistics Research, on data analysis. In addition, I hope to continue discussions with other institutes such as the Fogarty International Center (its director Roger Glass visited Tanzania in February) and NHGRI, which is involved in the H3Africa initiative (Human Heredity and Health in Africa Consortium).
CATALYST: What might you be doing differently based on what you learned during the sabbatical program?
MAKANI: I will be developing partnerships in research, health care, and training; facilitating joint research projects between scientists in Tanzania and the United States; improving my scientific writing, thanks to courses I took at the NIH library; and proposing changes in the laboratory diagnosis of SCD based on my training on hemoglobinopathy diagnosis. I will also encourage colleagues to participate in NIH’s sabbatical program to receive further training in laboratory diagnosis, research administration, and management. In addition, we hope to formalize a partnership between MUHAS and the NIH Clinical Center to develop virtual courses and exchange programs.
CATALYST: What additional research is needed on SCD?
MAKANI: There is a lot to understand about this genetic disease. Although SCD is common, the ongoing research is not sufficient in comparison to the burden. For example, why do the pain crises vary so much in terms of severity, frequency, and duration within a population or even for a single individual? In addition, the disease has been known for a century now, but only one drug exists to treat it. We need to conduct research to diversify the treatment options and alleviate the burden of this pathology in Africa.
CATALYST: What do you see as the challenges in SCD patient care?
MAKANI: Infants and children with SCD are more at risk for infections. There are two critical needs: screening newborns to detect SCD early, and preventing infections by generalizing the use of penicillin and promoting vaccination. Those measures, which were developed in the United States, considerably reduced the mortality, but they have yet to be implemented in Africa. Blood transfusion is also key in SCD patient care and could be done using existing tools from the PEPFAR (The United States President’s Emergency Plan for AIDS Relief) program, such as care centers and trained health-care providers.
CATALYST: What else needs to be done?
MAKANI: It’s important to integrate health care, advocacy, research, and training in public-health issues. My goal is to use research to support the three others. Stakeholders have to work together. For example, advocacy is supported in Tanzania via the Sickle-Cell Foundation of Tanzania (http://lms.muhas.ac.tz/sicklecelltz) and the Ministry of Health. The efficient implementation of policies is crucial to the improvement of health care. Training of health-care providers could be the weakest link, especially regarding the treatment and management of SCD complications. The mortality in hospitals is still too high.
CATALYST: Is there anything you’d like to add?
MAKANI: While there are still challenges to improving SCD patient care and treatment, I want to emphasize the extensive efforts being made by researchers and public-health stakeholders in Africa. My main objective is to invest in science and use science to improve health in a way that can be applied far beyond the SCD context.