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Legislature hears FSGS patients and researchers urge more screening and drug development
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Summary
A University of Michigan pediatric nephrologist and a mother and teen living with focal segmental glomerulosclerosis told the House Health Policy Committee about diagnostic delays, limited treatments and the need for research and telehealth access.
Legislators heard personal testimony and scientific context Tuesday about focal segmental glomerulosclerosis, or FSGS, a rare kidney disease that can cause protein loss, swelling and progression to end-stage kidney disease.
Zubin Modi, a pediatric nephrologist and director of the Pediatric Nephrology Research Program at the University of Michigan, told the House Health Policy Committee that ‘‘there are currently no FDA approved therapies for this disease’’ and described the condition’s course and treatment limitations.
Modi said FSGS affects both children and adults, can lead to dialysis or transplantation, and in some patients recurs after transplant. He described efforts to improve early identification, to expand telehealth access for patients in Northern Michigan and the Upper Peninsula, and to develop regulatory trial endpoints to help drug sponsors pursue approvals.
Sashay Walker, the mother of a 17-year-old patient identified in testimony as Aiden, described a prolonged diagnostic odyssey and repeated transplants that failed because of disease recurrence. Walker said her son ‘‘has recurrent FSGS’’ and recounted that he was on dialysis at age 8, has had two transplants that failed, and has undergone dozens of procedures.
Aiden spoke directly to committee members and said, ‘‘My experience has been difficult’’ and, ‘‘if we could get a cure, that’d be great.’’ Representative members thanked the family for testifying and asked the panel about causes, prognosis and regulatory progress.
Modi told Representative Carter and other members that causes are incompletely understood, that some FSGS cases are secondary to infection or medication but many are idiopathic and immune-related, and that research collaborations plus FDA engagement on endpoints are creating a possible regulatory path for new therapies.
Committee members applauded the witnesses and expressed support for continued research and patient access to specialized care.
The committee did not take formal legislative action on FSGS during the testimony session.
