It sounds straight out of science fiction, but clinical trials are underway to grow a miniature liver in a person’s lymph nodes.

The first-ever patient has been dosed with an allogenic regenerative cell therapy transplanted into their lymph nodes, in the hopes of treating end-stage liver disease.

LyGenesis announces first patient dosed in its Phase 2a clinical trial of a first-in-class regenerative cell therapy for patients with end-stage liver disease. (2024).

“This is an important example of translational medicine and really, truly a bench-to-bedside example of something that really was science fiction just a few years ago. To be able to grow a functioning ectopic organ that exerts potentially life saving  effects and to leverage so much natural biology in doing it. The goal here is nothing short of eliminating organ transplant waitlists, at least in the case of the liver,” Michael Hufford, PhD and CEO and Co-Founder of LyGenesis, the biotech company behind the clinical trial, told Medical News Today.

The clinical trial involves using an allogeneic cell therapy known as LYG-LIV-001, an investigational biologic product. It is made by isolating hepatocytes from a donor organ before suspending them in a solution to prepare them for transplantation.

Allogenic Hepatocyte Transplantation Into Periduodenal Lymph Nodes. (2023).

LyGenesis announces first patient dosed in its Phase 2a clinical trial of a first-in-class regenerative cell therapy for patients with end-stage liver disease. (2024).

“Ours is a unique therapy, because we're starting from donated organs that were unmatched for transplant. So it's an unusual cell source, but very advantageous. Here in the US…about 25% or so of livers are donated but unmatched. So we're now able to use some of those organs that otherwise would have not been used clinically,” Hufford said.

The cell therapy is administered to patients through an endoscopic ultrasound that injects the cell therapy into the patient’s upper abdominal lymph node. The procedure is noninvasive and takes about 10 minutes.

From there, the injected lymph node acts as an in vivo bioreactor, allowing liver cells to grow and proliferate to form functional liver tissue inside the lymph node, essentially creating an ectopic liver.

Eric Lagasse, PhD, was the first to demonstrate that the lymph node could be used as a site for the transplantation of cells and tissues.

In the case of end-stage liver disease, he says that transplanting cells into the lymph node instead of the liver has benefits.

“What we were doing previously was targeting the cell transplantation, which are the liver cells into the diseased liver. However, in the real world of liver disease, the liver is highly diseased, and transplanting cells directly into the liver is difficult, because you have an environment that is already dying. [P]utting a cell there in that environment is not generally good,” he tells MDLinx.

12 patients with end-stage liver disease will be involved in the clinical trial, across three dosage groups. The group with the highest dosage of therapeutic cells will have the therapy injected into five lymph nodes, for a potential total of six livers in one person: five ectopic organs, and one native organ.

The researchers expect that their therapy won’t be a good fit for those with acute liver failure or cancer, but they are hopeful that the therapy will offer options for others with end-stage liver disease, including those who may not be well enough for an organ transplant.

“It's done under light sedation. And that's a benefit because patients that would not qualify for a full organ transplant because they're simply too ill to survive the procedure are well enough to undergo the endoscopic ultrasound. So it's another reason we're excited. It's outpatient, low risk. That's another kind of innovation behind the therapy,” Hufford said.

More than 11,000 people in the United States are on the liver transplant waiting list, with demand exceeding supply.

Liver transplantation. (2022).

The researchers argue that their trial could pave the way to a potential solution to the organ shortage, not just for livers, but also for other organs.

“Not every person that needs a liver will get the liver. So there is really a high need of finding a new therapeutic approach for liver disease. So that is really something that is very important. You basically have a blueprint here of what could be done for organ regeneration, by being able to generate a fully functional organ,” Lagasse says.