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University of Michigan doctor, family urge action on rare kidney disease at House Health Policy Committee
Summary
A University of Michigan pediatric nephrologist and a mother of a teenager with recurrent FSGS testified to the House Health Policy Committee about the disease’s severity, limits of current treatments and need for earlier diagnosis and research pathways.
The House Health Policy Committee heard testimony from a University of Michigan pediatric nephrologist and a family Thursday about focal segmental glomerulosclerosis, or FSGS, a rare kidney disease that can lead to end-stage kidney failure.
Zubin Modi, a pediatric nephrologist and director of the pediatric nephrology research program at the University of Michigan, told the committee FSGS causes patients to lose protein in the urine and can result in swelling, infection, breathing difficulties and blood clots. "Some people will get better with medications that decrease your immune system, but unfortunately, many patients don't," Modi said.
Sashay Walker, the mother of a teenager with recurrent FSGS, described a multi-year diagnostic and treatment struggle for her son, Aiden, who was diagnosed at age 8 and has since had dialysis and two kidney transplants that failed because the disease recurred. "As of right now, at 17 years old ... we have tried every treatment available," Walker said. "This disease will not back off."
Nut graf: Committee members heard that there are no Food and Drug Administration-approved therapies for FSGS, that the disease affects both children and adults at similar rates of progression to end-stage kidney disease, and that researchers are pursuing regulatory pathways to permit clinical trials and potential drug approvals.
Modi said researchers and industry partners have compiled registry data and worked with the FDA to develop clinical trial endpoints, a step he said is needed to make trials feasible for drug sponsors. "We have been working with their statisticians and with their team to make sure that whatever we found ... would be okay with them," Modi said.
Representative Carter asked about causes and prognosis; Modi said the causes are incompletely understood. "Certain types of FSGS can be caused by secondary things like infections and medications. But most of the time ... it is considered to be an immune system regulated disease, that may have some genetic issues and may not," Modi said.
Several members praised Aiden for speaking to the committee. Aiden said his daily life is affected by treatments and procedures: "School is difficult. Just life is difficult in general... If we could get a cure, that'd be really great." Representative DeBoer and others thanked him for testifying.
Ending: Modi and witnesses asked the committee to note the research work underway and the need to preserve regulatory pathways for trials; no committee action on legislation related to FSGS was recorded during the hearing.
