Hackensack Meridian Children's Health Division Chief, Kenneth Lieberman, M.D., Dedicates Career to Rare Kidney Disease Treatment Development

Seeking new diagnostics and treatments proves fruitful for in era of genetic innovations

Individually, orphan diseases struggle to gain clinical and research focus. But Kenneth Lieberman, M.D., Division Chief of Pediatric Nephrology at Joseph M. Sanzari Children’s Hospital, has dedicated his career to assimilating a large toolkit to address the rarest causes of chronic kidney disease in children.

He became involved with the National Organization for Rare Disorders and embraced the philosophy of addressing specific, rare diseases, which collectively add up to significant populations of children affected by kidney disease.

“Individual rare diseases are rare, but to have a rare disease is not,” he said.

Dr. Lieberman’s focus is on diagnostic tools and cutting-edge therapeutics, and making sure clinical trials are readily available when these new options arise. He is currently engaged in multiple clinical trials for drug applications in rare conditions at Joseph M. Sanzari Children’s Hospital, including Obinutuzumab for steroid dependent nephrotic syndrome, targeted therapy for APOL1-mediated kidney disease, APL-2 for C3G, home-administered anti-complement therapy for aHUS, and two trials of sparsentan, one for proteinuric glomerular disease in children and another for FSGS.

Sometimes the innovation is diagnostic, thanks to genetic analysis advances. Other times it involves unlocking the molecular mechanisms of genetic variances and applying new or existing drug treatments. Dr. Lieberman draws on his undergraduate training in engineering and a “mechanistic mind,” seeking understanding of kidney function and dysfunction novel functional solutions.

While he’s been offered industry roles at points in his extensive career, he values providing patient care too much to make that move, he said. His clinical practice is a continual source of motivation. Here, Dr. Lieberman has seen the impact of rare disease diagnosis delays—three to five years in some cases. He also has witnessed the life-changing effects of new treatments for kidney disease in real families’ lives. He has seen rare diseases shift from a 1-year mortality rate to nearly 100% treatment success rates with patients living normal lives.

“I liked the idea of being able to change the trajectory for a child. You can really have a lifelong impact,” Dr. Lieberman said.

After more than four decades in practice, he now sees children of former patients and often has new treatment options to offer that weren’t available during the parent’s childhood care.

Learn more about our pediatric nephrology innovations.