Experimental Treatment Grows Miniature Livers in Lymph Nodes, Offering Hope for End-Stage Liver Disease

SCIENCE

In a groundbreaking clinical trial, biotechnology company LyGenesis has successfully transplanted healthy liver cells into a patient's lymph node, marking a significant step towards treating end-stage liver disease. The trial, which began on March 25th, aims to help individuals whose livers are failing but have not received an organ transplant.

A Bold and Innovative Approach

The procedure involves injecting donor liver cells into a lymph node in the patient's upper abdomen. The researchers expect the cells to multiply over several months, eventually taking over the lymph node and forming a miniature liver capable of performing the blood-filtering functions of the failing organ.

"It's a very bold and incredibly innovative idea," says Dr. Valerie Gouon-Evans, a liver-regeneration specialist at Boston University who is not involved with the company.

Addressing the Organ Shortage Crisis

Liver disease claims the lives of more than 50,000 people annually in the United States. In its final stages, the accumulation of scar tissue prevents the liver from filtering toxins from the blood, leading to potentially fatal complications such as infection or liver cancer.

While liver transplants can be life-saving, the shortage of available organs is a critical issue. Approximately 1,000 people in the U.S. die each year while waiting for a transplant, and thousands more are ineligible due to the severity of their condition. LyGenesis' approach not only has the potential to help these patients but also to make use of donated livers that would otherwise go to waste due to the lack of a compatible recipient.

Promising Results in Animal Studies

The treatment has shown success in mice, dogs, and pigs. In one study, researchers restricted blood flow to pigs' livers, causing organ failure, and then injected donor cells into their lymph nodes. Within two months, miniature livers with a cellular architecture resembling that of a healthy liver had formed. Notably, the mini livers contained cells that transport bile, a digestive fluid produced by the liver, suggesting that the new organs were functioning properly.

The Road Ahead

LyGenesis plans to enroll 12 participants in the phase II trial by mid-2025 and publish results the following year. The trial will assess participant safety, survival time, and quality of life post-treatment while also determining the optimal number of mini livers needed to stabilize a patient's health.

However, some experts caution that mini livers may not address all complications associated with end-stage liver disease, such as portal hypertension, which can cause internal bleeding. Nonetheless, the hope is that these miniature organs can provide a bridge to transplantation or improve patients' health enough to undergo the procedure.

"That would be amazing, because these patients currently have no other treatment options," says Dr. Gouon-Evans.

Future Directions

Beyond the current trial, LyGenesis is exploring similar approaches to grow kidney and pancreas cells in animal lymph nodes. If successful, this technology could revolutionize the treatment of multiple organ failures.

Additionally, researchers are interested in investigating the use of a patient's own stem cells to generate the cells that seed the lymph nodes. This personalized approach could capture the diversity of liver cells and eliminate the need for immunosuppressive drugs.

As the first patient recovers from the groundbreaking procedure, the medical community eagerly awaits the results of this innovative trial, which could offer new hope to those suffering from end-stage liver disease.