Kidneys saved: Researchers have discovered a drug that reduces the toxicity of chemotherapy.

A recent study conducted by researchers at Mass General Brigham has found that glucarpidase, a drug approved by the U.S. Food and Drug Administration (FDA), may reduce kidney toxicity in patients undergoing high-dose methotrexate (MTX) chemotherapy. If this effect is confirmed in further trials, it could significantly impact the quality of life for individuals battling cancer, reports Mass General Brigham.

MTX, widely used for treating central nervous system cancers, is associated with serious risks, including acute kidney injury (AKI), liver damage, and low white blood cell counts. By converting MTX into inactive metabolites within 15 minutes after administration, glucarpidase offers a rapid biochemical solution to the issue; however, its actual clinical impact has remained largely unverified until now.

In a study published in the journal Blood, researchers analyzed records from 708 patients across 28 major American cancer centers, comparing the treatment outcomes of those who received glucarpidase with those who did not. The results indicated that the likelihood of kidney recovery was 2.7 times higher for patients receiving glucarpidase. The drug also expedited recovery time and reduced the risk of severe neutropenia or liver toxicity.

The study employed a method called "target trial emulation," using real-world data to simulate the conditions of randomized clinical trials, allowing researchers to obtain reliable evidence without the costs and logistical challenges associated with traditional trials. Dr. Shruti Gupta, the first author of the study and a junior physician at Brigham and Women’s Hospital, emphasized the unique role of glucarpidase as one of the few available antidotes for chemotherapy-induced toxicity.

Even though the drug was FDA-approved in 2012, its use has been inconsistent, partly due to the lack of comprehensive clinical studies until now. Dr. David Leif, the senior author and director of clinical and translational research in AKI at Brigham’s renal medicine department, highlighted the rigor and precision of the study's methodology, which included manual chart review to account for critical variables.

High-dose MTX chemotherapy, while effective for certain types of cancer, necessitates a balance between its application and significant side effects. The authors hope that with evidence supporting the benefits of glucarpidase, it will see broader clinical use. "FDA approval is just the first step. If people are not using the drug, then patients are not benefiting from it," says Dr. Leif, stressing the need to align practice with new research findings.

The ability of MTX to cross the blood-brain barrier has made it indispensable in treating central nervous system cancers, but it has also increased the risk of organ toxicity in patients. Besides the efficacy of glucarpidase, the study exemplifies a broader issue of managing the side effects of cancer treatments and drugs, which can severely impact patients' quality of life and adherence to treatment.

Important! This article is based on the latest scientific and medical research and does not contradict it. The text is purely informational and does not contain medical advice. For diagnosis, please consult a physician.