Much hyped by the media, stem cells have tremendous power to improve human health. As part of the Cambridge Stem Cell Initiative, Ludovic Vallier’s research in the Anne McLaren Laboratory for Regenerative Medicine shows how stem cells can further our understanding of disease and help deliver much-needed new treatments.
The new technology consists of taking cells from skin and reprogramming them so that they become stem cells - cells that are capable of proliferating and differentiating into almost all tissue types."—Dr Ludovic Vallier
How do you study a human disease that has no equivalent in animals and where the human cells in question are so hard to grow outside the body they cannot be tested in the laboratory? The answer, until now, was with great difficulty. But by using a new stem cell technique, that is set to change.
Ludovic Vallier, who holds an MRC Senior Fellowship in the Anne McLaren Laboratory for Regenerative Medicine, Department of Surgery at Cambridge in collaboration with Professor David Lomas (Cambridge Institute for Medical Research and Department of Medicine), works on a group of devastating genetic diseases affecting the liver.
"We target metabolic diseases of the liver, diseases such as alpha 1 antitrypsin deficiency. It’s one of the most common single genetic disorders and the protein it affects - which is only produced by the liver - is really important because it controls activity of elastase in the lung. Without this control, people develop serious lung problems and the disease also affects the liver, so these patients develop liver failure," he explained.
The problem is that these diseases cannot be studied in vitro - in a dish - in the laboratory, he said: "You can’t take cells from the liver of these very sick patients, and if you could they wouldn’t grow, which means you don’t have any way of screening drugs that could help treat these diseases."
Without effective drugs, the only current treatment is a liver transplant. "There is a huge shortage of organs and transplantation involves taking immunosuppressive drugs, which is heavy treatment especially in already fragile patients," Vallier said. "And the disease is progressive so it’s very complicated to manage." Understandably, Vallier is excited that a new method of producing stem cells developed in Japan has given him and other researchers a way of studying these diseases and screening potential drugs to treat them.
"The new technology consists of taking cells from skin and reprogramming them so that they become stem cells - cells that are capable of proliferating and differentiating into almost all tissue types," he said.