Another ray of hope on the research front

Many of us associated with the Kennedy’s Disease Association (KDA) realize the importance of research. Because Kennedy’s Disease is a ‘rare’ disorder (only 1 in 40,000 have the defect), it does not receive the attention or the necessary funding needed to entice many labs across the world to focus their research on finding a treatment. We are a very small fish in a large ocean.

Fortunately, we do have a small group of dedicated researchers who work tirelessly towards that goal. Some of the labs in the United States that have Kennedy’s Disease research facilities are: National Institute of Health, Thomas Jefferson University, St. Jude Children’s Research Hospital, University of Michigan, Michigan State University, and the University of Washington.

About once or twice a year, one of these labs strikes gold and publishes a paper concerning their current research. In January of 2008, the KDA awarded a research grant to Maria Pennuto, Ph.D., from the National Institute of Health. Her research into the potential benefits of insulin-like growth factor -1 (IGF-1) looked promising at the time. Maria and her colleagues have just published the findings of their research. You can read more about it by following this link: http://www.news-medical.net/news/20090812/Promising-new-treatment-target-for-Kennedys-disease.aspx.

Here is a quote from the article:Importantly, augmentation of IGF-1/Akt signaling also improved motor performance, body weight, and muscle and spinal cord pathology, and extended survival in the SBMA mice. "Our study establishes IGF-1/Akt-mediated inactivation of mutant AR as a strategy to counteract disease in living animals and is the first to demonstrate that skeletal muscle is a viable target tissue for therapeutic intervention in SBMA," concludes Dr. Pennuto.

I realize it is ‘Greek’ to most of us, but in talking with a few other researchers, this appears to be another step forward and opens doors for additional research and possibly a clinical trial. Even though the KDA is a small organization, often we have provided the 'seed money' to allow promising research to continue until other funding becomes available.

For those of us who have met Maria, Isabella, and several other young researchers working to find a treatment for KD, we understand how fortunate we are that another generation has picked up the baton and continues to move towards the finish line (finding a treatment or cure). As long as someone is carrying the baton, we have hope.