September 17, 2014

Anyone who has watched TV or been online over the past few months is bound to have seen someone performing the “ALS Ice Bucket Challenge.” From government officials, celebrities and athletes, to teachers and the guy next door, almost everyone got in on the viral campaign, which was shared through social media. This experience involved a video of a person or group of people dumping a bucket of icy, cold water over their head, and then calling on specific friends or others to do the same, as well as make a monetary donation to the ALS Association, all in an effort to bring awareness to the disease. (ALS, or Amyotrophic Lateral Sclerosis, is a neurodegenerative disease of the brain and spinal cord, characterized by muscle deterioration throughout the body, eventually leading to death of the patient. It is also known as Lou Gehrig’s disease, after the famed New York Yankee baseball player who died of the disease in 1941). Currently, there is no cure for ALS.

As of October, the donations from the ALS Ice Bucket Challenge have brought in more than $100 million to the ALS Association, which intends to use the money on education and research for a cure.

Can cord blood banking have a role in future therapies for ALS? It just might! One current treatment being researched is the use of stem cells to help reverse neural degeneration1. At the Cedars-Sinai Regenerative Medicine Institute in California, a team of doctors and scientists have developed an innovative technique that involves combining stem cells from an ALS patient with molecules of minute sections of a genetic material (called antisense oligonucleotides) that in essence, prevents the damaging effects of the disease by blocking the buildup of toxins from the damaged gene. The study basically created the disease in a dish and, with their new technique, treated the damaged gene. The defective gene in this case is C9ORF72, which has been identified as the most common cause of ALS, creates an excessive build up of nucleotides and changes the structure of ribonucleic acid from the gene. By preventing this toxic build up, researchers were able to reverse the damaging effects in their tests.

For this study, the researchers used stem cells derived from the patient’s skin cells. However, it opens the door to potential future therapies utilizing stem cells from other sources, including umbilical cord blood. The research team plans to continue with further, more extensive stem cell research and testing to hopefully one day have a cure for this disease.

1. Targeting RNA foci reduces pathology in iPSC-derived motor neurons from C9ORF72 repeat patients. Science Translational Medicine. Published October 23, 2013: Vol. 5 no. 208 pp. 208ra149, DOI:10.1126/scitranslmed.3007529. Found online: http://goo.gl/z0NMbS

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