What if a single injection could slow aging and prevent cardiovascular disease? And what if that same injection could treat potentially deadly autoimmune disease, all without any side effects?
While it's still in its early days, revolutionary treatment based on stem cells appears to do just that. But how does this treatment work, and who might benefit from it most?
Newsweek spoke to Professor Katarina le Blanc, one of the world's leading experts on clinical stem cell research.
"When I started out it was a small, sort of obscure field," le Blanc, who is a professor at the Karolinska Institutet, told Newsweek. "But then we had some findings about five years into the project that got a lot of attention—we discovered that these cells appeared to prevent inflammation, or at least dampen it in humans. And my little field just exploded. When I started, there were only 30 scientific publications in the field. Now there are over 98,000."
Le Blanc's work revolves around a group of cells called mesenchymal stem cells, or MSCs. These cells are undifferentiated, meaning that they can develop into multiple different cell types. MSCs are derived from adult bone marrow and, due to their interaction with the immune system, have the potential to revolutionize the way we treat many severe and often incurable diseases.
To understand this, we need to understand the behavior of the immune system. When we are infected or injured, our immune system responds by triggering a inflammatory response, sending more cellular soldiers to the site of the injury or infection to trap and destroy any germs and toxins and initiate the healing process.
This is obviously very important for our survival. However, if this persists over a long period of time, this inflammation can start causing problems.
"Inflammation is behind so many diseases," le Blanc said. "It's thought to play a role in diabetes, in stroke, in heart disease, in high blood pressure, and it's likely to be a part of aging too."
This low-grade, chronic inflammation can be caused by a range of factors, including low levels of physical activity, chronic stress, environmental toxins, an inflammatory diet, air pollution, tobacco products, and too much alcohol, among other things. But what if we could turn off this immune response?
"We already knew a lot about the immune system and how it is turned on [when we started this research,]" le Blanc said. "But what had been totally unrecognized is that, in healthy tissue, there is an anti-inflammatory signal too. When there is no infection, your immune cells have a break turned on—an anti-inflammatory signal to say that the immune system should not get activated."
What le Blanc and her team have discovered is that MSCs switch on this anti-inflammatory signal. "So, by injecting them, we're propagating nature's 'all is well' signal," le Blanc said. "It's really very simple, which was really very exciting and unexpected."
So far, we have focused on the issues that arise from low-grade, chronic inflammation. But overactivation of our immune systems can also cause very severe acute problems, as is the case in graft-versus-host-disease. This occurs in a subset of patients following the transplantation of bone marrow and/or blood stem cells for the treatment of leukemia and can be fatal for an estimated 1 in 3 patients affected.
So, how might these MSC injections work? "The cells are only around for about 48 hours, they don't stay," le Blanc said. "They don't like being taken out of the body and then put back in, so they die. But we now believe that stem cell death signals to the immune system to engulf these cells and further propagate this anti-inflammatory signal even though the cells are long gone, for about three months."
This goes against the team's initial hypothesis for the regenerative behavior of these stem cells. "We thought they were replacing tissue—that was the initial hypothesis," le Blanc said. "But then it turned out to be something very logical but completely different."
The transient nature of these MSC injections is particularly beneficial when it comes to regulation. "One of the big fears around MSCs, especially for regulators, was 'would the cells form tumors? Would they form the wrong types of tissues in the wrong parts of the body?' and the short answer was—no," le Blanc said. "They aren't there. But their signal is."
On top of this, the treatment has—so far—not resulted in any side effects. So, what's the hold up?
Well, until recently, these cells were very expensive to access, hindering their application in both research and therapeutic settings. However, in 2021, le Blanc founded a biotechnology company called Cellcolabs, which aims to produce high quality stem cells on an industrial scale and bring down the cost of this cutting-edge treatment.
It's a complex task—the cells need to be removed from donors, frozen, managed and prepared for patient transplantation. But the startup hopes to eventually produce 1,000 to 2,000 high-quality batches of stem cells per year, with the help of roughly a dozen young, healthy bone marrow donors. By making these cells more accessible, Cellcollabs hopes to accelerate the time frame in which they could be commonly used to treat patients.
However, not everyone is responsive to stem cell therapy. "What we find is about 50 percent of the patients have a complete response and recover from their disease, whereas 50 percent are non-responders," le Blanc said. "So, the research now is really to understand who these responders are and who will really benefit from this treatment."
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