“Mini livers” to save lives

Since 2016, the D^r Massimiliano Paganelli, his wife, the D^D Claudia Raggi and their team are working to create “mini-livers” to save the lives of children and adults suffering from liver failure. Funding of tens of millions of US dollars has just been granted to enable them to reach the clinical phases.

In their laboratory, researchers grow stem cells to mimic those in the liver. “We push these cells to become different types from those found in the liver. These are small spheres called organoids. We bring them together so that they act like a tissue with biomaterials that we have developed and we recreate the conditions that we normally have in the liver,” describes Dr.^r Paganelli, pediatric hepatologist at the Sainte-Justine University Hospital Center.

Each sphere is invisible to the naked eye. Once these balls are brought together with biomaterials, together they have the size of a small leaf, explains the man who is an associate clinical professor at the Faculty of Medicine of the University of Montreal. “With cocktails of molecules, we will recreate a bit of the environment we have in the development of the uterus. And thanks to that, the stem cells become mini-livers. And these will talk to each other to really remodel liver tissue,” explains the researcher.

These mini-livers will then be installed in an implant, which will be inserted into a patient’s abdomen for a period of a few weeks. “It is a treatment which is transient, which allows the functions of the liver to be replaced”, specifies the D^r Paganelli.

Delaying deaths and saving lives

The liver is an organ that rebuilds itself efficiently, explains Dr.^r Paganelli. However, in many cases of liver failure, the days are numbered. “The problem is that the person dies before the liver has time to heal itself. And we replace the functions and accelerate the regeneration of the liver with our implant,” he describes.

This new technology called ReLiver^MC will therefore aim in certain situations to avoid an organ transplant altogether. In other circumstances, it will allow the patient to survive while waiting for a donor. “We know that the benefits of this implant can last several months. But the goal is really to keep it for a few weeks. This is the time we need for the liver to regenerate,” adds the doctor.

Potential treatments for other diseases

If he first wishes to provide better therapy for liver failure, the D^r Paganelli believes that the new technology developed by his company could ultimately be applicable to other ailments, such as alcoholic hepatitis or cirrhosis. “We also aim to use it to treat pediatric metabolic diseases. And all the problems we have after liver cancer repair for example. The liver which remains too small, we could help it to grow back. There are plenty of diseases that could benefit from this,” he says.

The researcher and his team are also studying the possible uses of this technology for different organs. “The potential is exactly the same for various diseases, which could be treated by the same approach. »

Clinical phases

It was in 2018 that the D^r Paganelli co-founded his company, Morphocell Technologies. With new funding of 40 million US dollars from private investors, the initiative will be able to begin clinical trials in the near future, rejoices the doctor.

Experiments have already been carried out on pigs, whose livers are very similar to those of humans. “Over the next two years, we want to reproduce them on a human scale,” hopes the hepatologist.

Currently, researchers are carrying out tests to have the safety of the method approved by Health Canada and the Food and Drug Administration (FDA) in the United States. “We could imagine that in 2027, we will be able to start treating patients,” says Dr.^r Paganelli.

If the doctor remains optimistic about his work, he nevertheless specifies that he does not want to give false hope to certain patients. “We believe enormously in these technologies. We think it will revolutionize treatments, but we have to go about it very rigorously. We don’t want people to refuse other treatments to wait for this one,” he emphasizes. While many biotechnology companies are based in the United States, the D^r Paganelli chose to maintain his discoveries in Quebec. “It’s important to attract talent and keep them here. If we develop these therapies ourselves and everyone ultimately benefits from them, in Quebec, we will be the first to have access to them. »

This content was produced by the Special Publications team at Duty, relating to marketing. The writing of the Duty did not take part.

To watch on video