Name: Joshua Waitzman, MD, PhD

Institution: Beth Israel Deaconess Medical Center

Grant: 2020 Ben J. Lipps Research Fellowship

Project Title: Structure and Molecular Mechanism of ApoL1

How would you sum up your research in one sentence?

• My research uses tools from structural biology and biochemistry to determine how gene mutations cause the kidney disease focal segmental glomerulosclerosis.

Provide a brief overview of the research you will conduct with help from the grant.

• Focal segmental glomerulosclerosis (FSGS) is a type of kidney disease that disproportionately affects people of recent African ancestry. While most cases of this disease are caused by mutations in the APOL1 gene, we do not have any treatments that target the disease-causing APOL1 protein. My research will determine the structure and function of the APOL1 protein. By determining the molecular mechanism of the protein that causes FSGS, my research will enable a search for drugs to help patients with this currently untreatable disease.

What impact do you hope your research will have on patients?

• Patients with aggressive cancers have treatments that are specifically targeted against the abnormal genes that cause their diseases. I hope to help develop similarly targeted treatments so that we can improve the health of patients with kidney disease.

What are your career goals at the end of the grant period? Five years out? Ten years out?

• I plan to establish my own independent research group focused on identifying mechanisms of and treatments for kidney diseases. Over the next few years, I will perform research, present my work and apply for funding, while continuing to see patients.

What inspired you to focus your research in this area?

• As a physician-scientist, I have been inspired by my patients, their families and the physicians and nurses who help care for them. Throughout my training, my patients and I have faced limited treatment options with significant side effects. By improving our understanding of the mechanisms of kidney disease and developing new therapies, I hope to help future generations of caregivers face easier choices. Great clinical and research mentors have encouraged me along my path to a career in Nephrology, including Drs. James Paparello, Susan Quaggin and Martin Pollak.

What are the major challenges to beginning a career in nephrology research today?

• Students and residents have less exposure to research and clinical care in nephrology. As a result, we see less interest in the field among trainees. I think engaging with trainees to get them excited about nephrology is critical to having more people begin careers in nephrology research. The research problems in nephrology are as interesting as those in other clinical areas (if not more so!) and have the potential to impact the care of the millions of people with kidney disease.

What advice would you give to others to encourage them to apply for this grant funding?

• Once you have developed a set of specific aims, show them to multiple mentors and get lots of feedback. My research plan improved a great deal because of the input of others.

Something you may not know about me is…

• I’ve played trumpet with Wynton Marsalis, the rock band Wilco and the world’s only ice-skating marching band.

In my free time I like to…

• Run, cook and spend time with my family.