Stem Cell Breakthrough for Lou Gehrig's Disease (ALS)

A new technique for reprogramming cells has allowed scientists to grow neurons from cell samples donated by people suffering from Lou Gehrig's disease (ALS) that genetically match the bad cells in the spinal cords of ALS patients. This breakthrough may lead to an understanding of how the disease develops.

ALS, technically known as amyotrophic lateral sclerosis, is a progressive neurodegenerative disease that causes motor neurons in the brain and spinal cord to die, which can lead to paralysis or death. It is estimated that 30,000 people in the United States have the disease, according to the ALS Association.

Scientists at Harvard and Columbia universities have become the first to succeed in the laboratory creation of a supply of cells with the same genetic makeup as a patient with a specific disease. The same technique used for the creation of these cells can be used to study other genetic diseases in hopes of better understand what causes these diseases and allow for the testing of new drugs to battle against them. The accomplishment of creating these cells has been a fundamental reason for performing stem cell research since its inception.

Stem cells have the capability of developing into various cell types in the body and can act as a repair system within the body. These cells can continue to divide and replace other cells in the body as long as the body lives. Stem cells divide and each new cell can remain a stem cell or become another type of cell with a specialized function.

The researchers worked with a form of ALS that is caused by a defect in a single gene using patches of skin removed from the arm of an 82-year-old woman and her 89-year-old sister, who share a rare genetic mutation that causes only about 2 percent of ALS cases.

The research team used a technique that was developed two years ago by Japanese stem cell researchers. During the procedure skin samples were placed into a laboratory dish. The researchers then inserted four genes into the DNA of the skin cells, which reprogrammed the cells. These skin cells then formed cell colonies much like embryonic stem cells called induced pluripotent stem (iPS) cells. Then, the iPS cells were coaxed by signaling molecules into motor neurons. The resulting patient-specific cells will be studied to see how the development of diseases such as ALS occurs.

According to co-author Dr. Kevin Eggan of the Harvard Stem Cell Institute Eggan commented, "Since the cloning of Dolly the sheep and the first derivation of a human embryonic stem cell line by Jamie Thomson some 10 years ago, it's been the hope of scientists to generate stem cell lines that have the genes of a patient." He went on to explain, "This really suggests that it's going to be possible to make these cells from patients suffering from other diseases." Eggan noted that the research team plans to study the cells in the lab and compare them with cells of someone who doesn't have that disease next.

Another long term goal is to actually repair defects in the cells and then transplant them back into patients, however, the current technique used to create the iPS cells necessitates adding viruses and genes to the damaged cells that can cause cancer.