Promising Treatments for Rare Chronic Kidney Diseases Under Evaluation at Joseph M. Sanzari Children’s Hospital

Drug treatments for orphan diseases with devastating kidney function impact in clinical trials

With the advent of genetic-based diagnostics and treatments and other diagnostic advances, Kenneth Lieberman, M.D., Division Chief of Pediatric Nephrology at Joseph M. Sanzari Children’s Hospital, prioritizes application of these new approaches for the rarest of kidney diseases, giving hope to children and families who previously often faced dismal 1-year mortality rates.

Seeing these practices shift lives in his clinical practice continues to motivate Dr. Lieberman. His primary focus is bringing new treatments for rare kidney diseases to clinical trial and advocating their use in practice globally when successful.

Dr. Lieberman and Joseph M. Sanzari Children’s Hospital are currently participating in the following clinical trials of treatments for rare kidney diseases:

• INShore trial studying Obinutuzumab for steroid-dependent nephrotic syndrome
• Amplitude trial for targeted therapy for APOL1-mediated kidney disease
• Commute trial evaluating home-administered anti-complement therapy for aHUS
• EPPIK trial of sparsentan for proteinuric glomerular disease in children
• Duplex trial of sparsentan for FSGS
• Valiant trial studying APL-2 for C3G

While 5 to 10% of the world’s population is affected by chronic kidney disease, only a handful of children might experience the conditions above annually. Yet treatments for rare diseases collectively and individually make a significant impact.

Atypical HUS (aHUS) following e-coli infection is one devastating rare disease for which Dr. Lieberman helped develop a successful treatment. The disease, which presents in only a dozen cases a year typically, has a genetic component and leads the complement system to damage the patient’s kidney. Dr. Lieberman worked with an industry partner to develop a “magic bullet” to turn off the complement system for this disease that previously had a 70% rate of 1-year mortality. He was also able to apply for compassionate use of this treatment in a pediatric patient whose complement system was causing similar issues after transplant, which proved successful.

These are the kinds of cases he said he finds extraordinarily gratifying and motivate his mission to continue seeking rare kidney disease diagnosis tools and treatments.

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