New targeted therapies could cut need for dual transplants in rare kidney disorders: Experts

Rare kidney disorders, while individually uncommon, represent a significant global health burden. Often striking at a young age, these conditions frequently progress silently toward kidney failure.

For decades, medical intervention was limited to symptom management, controlling blood pressure or reducing protein loss, rather than tackling the root cause.

However, a paradigm shift is underway. Advances in genetic research and biotechnology are ushering in an era of precision medicine, offering new hope to patients who previously faced a predetermined path toward dialysis or organ transplantation.

The silent threat of rare disorders

According to Dr. Vikram Kalra, Principal Director of Nephrology and Kidney Transplant at Aakash Healthcare Multi Speciality Hospital (Delhi), the primary challenge has always been the "quiet" nature of these diseases.

"Conditions like primary hyperoxaluria, C3 glomerulopathy, and atypical haemolytic uraemia syndrome are often diagnosed late, after damage has already started," Dr. Kalra explains. "For years, treatment options have been limited... but this is starting to change. New research is leading to the development of drugs that target the underlying mechanisms of these conditions."

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One such breakthrough is Lumasiran, an oral drug approved for Primary Hyperoxaluria Type 1. Dr. Kalra says "Early studies show it can reduce the production of oxalate—the substance responsible for kidney damage in this disorder, offering a more direct and effective treatment approach than ever before."

Breaking the cycle of organ failure

The impact of these advancements is perhaps most visible in the treatment of Primary Hyperoxaluria, a genetic disorder characterised by excessive oxalate production.

Dr. Prasad Dandekar, Consultant of Nephrology and Kidney Transplant at Yatharth Hospital (Greater Noida) said that primary hyperoxaluria remains a life-threatening genetic disorder where excess oxalate leads to recurrent stones and progressive kidney damage, often culminating in end-stage kidney disease.

“Traditionally, the biggest challenge… has been that even after a kidney transplant, oxalate deposition continues, often necessitating a combined liver-kidney transplant,” he said.

However, he added that newer targeted therapies are proving transformative. “These drugs work at the metabolic level to reduce oxalate production, thereby not only slowing disease progression but also potentially preventing kidney failure and eliminating the need for complex dual organ transplants,” Dandekar said, calling the development a major step toward safer and less invasive treatment options.

Experts say the shift reflects a broader move toward personalised medicine, where therapies are tailored to the specific cause of a disease. While challenges around cost, access and long-term safety remain, the emerging treatments are raising hopes of improved outcomes and a future where patients with rare kidney disorders may avoid dialysis or transplantation altogether.

A future of personalised medicine

The innovation extends beyond a single disease. New classes of drugs, including complement inhibitors and targeted immunotherapies, are showing positive results in conditions like C3 glomerulopathy. These treatments act on specific biological pathways to prevent kidney injury with surgical precision.

Furthermore, technologies such as RNA-based treatments and monoclonal antibodies are being deployed to target disease-causing genes or proteins at an earlier stage.

Dr. Kalra emphasises that this marks a broader move toward personalized medicine. "Instead of facing a future that may lead to dialysis or a kidney transplant, there is growing hope for treatments that can control or improve the disease," he says. While hurdles like cost and long-term accessibility remain, the potential for improved patient outcomes has never been higher.