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Fighting to shorten the liver transplantation waiting list

According to the American Liver Foundation nearly 6,500 liver transplants were performed in the United States in 2005 and another 1,848 patients died waiting for a donor to become available. Even more worrisome is that the number of patients waiting for livers to become available is increasingly every year.

Without going into detail of the different types of transplantation methods and complications, it suffices to say that the surgery is very complicated and even today carries a relatively high risk of mortality. The risks arise from the simple fact that the surgery is extremely invasive and requires significant manipulation of anatomy, but risks also prevail in the post operative period. Like all other organ transplantations, there is a risk of donor organ rejection by the recipient's immune system—this may occur immediately, in a short period of time, and in the most successful cases, slowly or perhaps not at all. Though rejection has been a significant factor to early death after transplantation in the past, better matching of immuno-compatibility and new generation of immunosuppressants have drastically prolonged the survival for transplant recipients.

Alpha-1-antitrypsin (A1AT) deficiency is an inherited disease that frequently leads to cirrhosis (severe and irreversible damage) of the liver in young patient and is an indication for liver transplantation. In a recent article published in the journal Nature, researchers discussed a novel technique where skin stem cells were taken from patients with A1AT deficiency and genetically altered them to correct the mutation. They then but these cells into rats and discovered that the cells had integrated into the livers of the rats and produced the previously deficient human A1AT. Though far from human application, this research may pave the way for later human trials in hopes of preventing end-stage liver disease in patients with this A1AT deficiency and thereby also decreasing the need for liver transplantation.

Much research remains to be done, and the assessment of the safety of modified stem cells is ongoing. Nonetheless, there is significant potential to help these patients, and simultaneously decrease the need for liver transplantation.

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