A first-in-class experimental drug that stimulates liver regeneration could be practice changing for surgeons who treat liver diseases, new research suggests.
The treatment, HRX-215 from German drugmaker HepaRegeniX, was safely tested in humans following success in animal studies, researchers reported in Cell.
The liver can regrow itself – a process regulated by an enzyme called MKK4 – but removing too much of the liver injures it beyond its ability to regenerate, the researchers found.
Inhibiting the activity of MKK4 with HRX-215 strongly enhances liver regeneration, they discovered in studies in mice.
In later experiments, 75% of pigs treated with HRX-215 survived the usually-fatal removal of 85% of the liver.
In the early human trial, HRX-215 was safely absorbed into the bloodstream with no ill effects in 48 healthy volunteers, paving the way for mid-stage trials to test the drug’s effectiveness, the researchers said.
In patients with liver cancers, complete removal of the tumors offers the only potential cure. When the remaining liver volume would be below a certain threshold, patients are at high risk of developing liver failure. Liver regeneration prompted by HRX-215 could make more extensive liver resections possible, the researchers believe.
The drug could eventually also help mitigate the organ shortage for liver transplantation by allowing more healthy volunteers to donate pieces of their liver, study leader Dr. Lars Zender of University Hospital Tuebingen in Germany said in a statement.
Adult recipients usually require a large piece of a living donor’s liver. With the potential for HRX-215 to improve regeneration of transplanted liver lobes, surgeons might be able to transplant smaller lobes from donors, making the donor surgery safer while preserving the benefit for the recipient.
