Clinical trial will have patients grow multiple mini-livers internally
A new experimental treatment could help treat end-stage liver disease – by growing tiny new livers elsewhere in the patient’s bodies. The technique, pioneered by cell therapy company LyGenesis, is due to begin human clinical trials in the next few weeks.
The liver has a powerful regenerative capacity, able to repair itself from the constant damage it sustains as it works to rid the body of toxins. But alcohol intake or an unhealthy diet can impair that ability and lead to liver disease, the end stages of which can require liver transplants.
But the team at LyGenesis has been working on a creative alternative that would be much less invasive. Rather than replacing the liver, the technique would involve growing entirely new ones elsewhere in the body – mini-livers that can perform the same vital functions.
The process involves injecting healthy liver cells, taken from donated organs, into the recipient’s lymph nodes. There, they multiply and grow into functioning mini versions that can support the work of the remaining cells in the original liver. Previous tests in mice, pigs and dogs showed that the treatment improved their liver function, and can save the lives of many animals that would otherwise succumb to liver failure.
And now LyGenesis is preparing to test the technique in humans for the first time, in a phase 2a clinical trial. Beginning in the next few weeks, 12 adults with end-stage liver disease (ESLD) will receive batches of healthy liver cells. These will be delivered via endoscope and injected directly into the lymph nodes.
The trial participants will be split into three groups of four that receive different doses – either 50 million, 150 million or 250 million cells. It’s thought that for every 50 million cells a patient receives, they will grow one mini liver, meaning the highest dose group could end up with five extra livers. The LyGenesis team will monitor the patients for a year afterwards, assessing the effectiveness and safety of the treatment at the different doses.
Patients will need to receive immune-suppressing drugs to prevent their bodies rejecting the “foreign” mini-livers, much the same as those who currently receive whole organ transplants. However, LyGenesis recently announced a research collaboration with another company, iTolerance, to investigate how the latter company’s “microgel tolerance platform” could remove the need for immunosuppressants in patients receiving the new cell therapy.
Coaxing the body to grow mini versions of organs could not only end up being a less invasive treatment than transplants, but it could help reduce the strain on the organ donation system in other ways, too. The transplanted cells can be harvested from donated organs deemed unsuitable for transplantation themselves, with each organ theoretically able to provide enough cells for at least 75 people to undergo the treatment.
The team hopes to wrap up the trials in under two years, and if all goes well, the technique could later be adapted to other organs, such as the kidneys, pancreas and thymus. The team describes the process in the video below.