Ludwig Institute for Cancer Research

(Stockholm, January 27th) – While it seems that the whole world watches and waits for true human stem cells to be isolated, researchers at the Stockholm Branch of the Ludwig Institute for Cancer Research (LICR) and the Karolinska Institute have made a critical breakthrough in the genetic engineering of stem cells for transplantation therapy, according to a study published today in Cell.

“Theoretically, cell transplantation would treat Parkinson’s Disease by replacing the midbrain dopamine (DA) neuron cells that have degenerated,” says LICR’s Dr. Thomas Perlmann, a senior author of the study. “However, there are ethical and practical issues with transplanting fetal DA neurons, so a lot of interest lies in inducing stem cells to become DA neurons. The big challenge is how do we artificially induce a stem cell to become exactly the right type of replacement cell?”

The Swedish team identified and characterized the program of gene expression that occurs during the normal development of the DA neurons and identified two crucial genes, Lmx1a and Msx1. The team then proved their hypothesis inserting the Lmx1a gene into mouse stem cells and showing that midbrain DA neurons were generated. The results were confirmed by analyzing molecular markers in single cells.

According to senior co-author, Professor Johan Ericson of the Karolinska Institute, the study is a landmark in the development of a potential Parkinson’s Disease therapy, but still an early landmark. “These engineered cells have the correct identity, at a molecular level, for therapeutic cell transplantation. However, we have to show that this type of engineering also works in human stem cells, we have to show that these cells can be transplanted safely and effectively, and only then will we be able to test if the engineered cells have a therapeutic effect.”

For further details, please contact:

Ludwig Institute for Cancer Research:

Dr. Thomas Perlmann, Stockholm Branch
thomas.perlmann@licr.ki.se

Dr. Sarah White, Director, Office of Communications
swhite@licr.org
+1 212 450 1543 (Business hours - New York)
+1 917 974 7952 (After hours - New York)

Ludwig Institute for Cancer Research

The Ludwig Institute for Cancer Research (LICR) is the largest international academic institute dedicated to understanding and controlling cancer. With ten Branches in seven countries, and numerous Affiliates and Clinical Trial Centers in many others, the scientific network that is LICR quite literally covers the globe. The uniqueness of LICR lies not only in its size and scale, but also in its philosophy and ability to drive its results from the laboratory into the clinic. LICR has developed an impressive portfolio of reagents, knowledge, expertise, and intellectual property, and has also assembled the personnel, facilities, and practices necessary to patent, clinically evaluate, license, and thus translate, the most promising aspects of its own laboratory research into cancer therapies.