Samira Musah Awarded $1.5 Million in NIH High-Risk, High-Reward Grants

Samira Musah has received a New Innovator Award from the National Institutes of Health’s (NIH) High-Risk, High-Reward Research Program. An assistant professor of biomedical engineering and medicine at Duke University, Musah will use this highly competitive grant to explore how to use stem cells to repair damaged kidney cells, with the long-term goal of developing new methods to treat kidney disease.

Established in 2007, the NIH Director’s New Innovator Award supports highly innovative research from promising early-stage investigators. The program catalyzes scientific discovery by supporting exciting, high-risk research proposals that may be considered too ambitious in the traditional peer review process despite their transformative potential. Program applicants are encouraged to think outside the box and to pursue creative, trailblazing ideas in any area of research relevant to the NIH mission.

With joint appointments in Duke BME and the Division of Nephrology in the Department of Medicine, Musah’s research is centered around the use of patient-derived stem cells to model and understand the mechanisms of human kidney disease, a disorder that affects more than 15 percent of United States adults and 10 percent of the worldwide population, and costs more than $81 billion in annual Medicare expenditures.

Healthy kidneys filter blood, removing waste and extra fluid as urine to keep our bodies healthy. But when kidney function breaks down, dangerous levels of fluid and waste can build up in the body, damaging the heart, brain and immune system. Because many patients don’t exhibit early symptoms of kidney disease, the illness isn’t typically diagnosed until kidney function is severely impaired. This often means that patients must go on dialysis, which only delays the necessity of a kidney transplant. It is also difficult to develop new therapies and treatment options for kidney disease, as animal models often fail to faithfully replicate human kidney biology and drug responses.

By using pluripotent stem cells from patients, Musah aims to design environments that give cues to stem cells telling them to differentiate into specific kidney cells. By determining how to generate various types of kidney cells, her lab could then create more complex in vitro models that mimic the structure and function of the kidneys, potentially creating a platform for targeted drug development.

With the support of the New Innovator Award, Musah will advance this work by using her stem cell-derived kidney models to uncover molecular targets that can trigger cell and tissue repair. They will use these findings to develop synthetic molecular circuits that can automatically send messages to jumpstart healing in damaged tissues, helping to restore the kidney’s blood filtration abilities.

“If we’re successful, this line of research will provide opportunities to develop cell-mediated regenerative therapies for kidney disease by targeting the most vulnerable tissues in the organ,” explains Musah. “The breadth of this work is critical to advancing the field of nephrology and overcoming the limited therapeutic options for the overwhelming number of patients suffering from kidney disease around the world, and I’m tremendously grateful to the NIH and NIDDK for supporting our research with this award.”