As people continue to dump ice buckets and write 3 million checks, raising $100.9 million so far, we are left with the following questions:
What is the status of finding a cure to this horrible disease? And, how can citizens continue their support after the #IceBucketChallenge ends?
To answer these questions, let us focus on one promising line of research currently being conducted in the field of ALS and the prospects that exist for future powerful treatments.
Causes Unknown - Stem Cells as a Potential Treatment
The causes of ALS remain frustratingly unknown. For the 30,000 living with ALS, there is no existing therapy that can cure or slow their disease or extend their lives beyond a few additional months. (To get a sense of the life of an ALS patient, see ALS patient advocate Bo Stern's blog post.) Understanding these bleak prospects, what is the best path to a cure or therapy to mitigate this terrible disease?
The latest research using stem cell therapy has emerged as one potential new treatment to help patients regain muscle movements and slow the progression of the disease. The proposed treatments use a range of stem cell types including pluripotent stem cells--self-renewing cells that have the potential to turn into the tissue and organ cells that make up our body--to support dying motor neurons.
Clinical Progress in Animals; Human Clinical Trials in 2015?
The California Institute for Regenerative Medicine (CIRM) awarded nearly $54 million in funding to ALS-related research. These awards have gone to an array of projects. Some projects target understanding the disease and why the motor neurons die. Others focus on how to treat the disease and its progress. Dr. Clive Svendsen and his team at Cedar-Sinai Medical Center, funded by CIRM, is currently in pre-clinical studies with the hope of finding novel stem cell and growth factor treatments for the thousands of ALS patients who suffer from the disease every day.
Svendsen and his team have developed an approach using a "nerve fertilizer"--a growth factor called Glial Derived Neurotrophic Factor (GDNF)--to treat the deadly disease. The team will modify stem cells to produce GDNF. Then, he will use these modified stem cells as "Trojan horses" to carry the GDNF to afflicted areas to stimulate and repair dying nerve cells, and rejuvenate the same area with young brain cells.
Svendsen's combined stem cell and gene therapy trial is currently in pre-clinical studies. The nerve fertilizer combined with stem cells has proven effective at significantly increasing the survival of motor neurons in animal models.. Svendsen and his team are projected to start human clinical trials in the summer of 2015.
Sustained Support to Reach a Cure
The public's focus on this disease is outstanding and the huge amount of resources being donated to the ALS Association will help push forward critical research like Svendsen's. The Robert Packard Center for ALS Research at Johns Hopkins is another world-class institution on the leading edge of ALS research funding--developing projects such as disease models in rats that will help researchers further understand how ALS develops. Both the ALS Association and the Packard Center are worthy destinations for this outpouring of support.
But the amount raised alone is not a true measure of the success of this campaign. Rather, it will be the sustained interest and support for the life-saving research taking place throughout California, the United States and the world, and the impact that it will have on changing the lives of ALS patients and their families.
With that plea, allow me to join the millions of other caring citizens who have participated in the #IceBucketChallenge thus far and submit my own entry into this worthy endeavor. And, with that, I nominate and challenge my fellow participants to not let our participation be just a passing fad, but a continued commitment to stay informed, be supportive, and continue donating to ALS research and other therapy initiatives that will put an end to chronic diseases that ultimately destroy the health of men, women, and children in this country.
To learn more about ALS Research, Stem Cells, and Research being funded in California, please see the links below:
To learn more about ALS research, watch the video of CIRM's Google Hangout on the Progress toward Stem Cell Therapies for ALS with Dr. Svendsen, Dr. Karen Berry, a CIRM science officer, and Diane Winokur, an ALS patient advocate and a CIRM governing board member.
For more information about stem cell advocacy, please visit Americans for Cures' website.
And, to learn more about stem cell research being funded in California, please visit the California Institute for Regenerative Medicine's website.
A Note about ALS: Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease--is a fatal and relentless neurodegenerative disease that damages and destroys motor neurons in the brain and spinal cord. About 5,600 people are diagnosed in the United States each year. ALS has an extremely rapid progression; most of these people die within four years. For patients who have been diagnosed with ALS, the disease begins with smaller symptoms--uncontrolled muscle twitching, uncharacteristic clumsiness, nighttime cramps. Ultimately, patients inevitably lose all control of their bodies through complete paralysis. This means not being able to walk, eat, speak clearly, use the bathroom, or even make sense of the world. Again, to get a sense of what it is like to walk in the shoes of an ALS patient, take these ALS "empathetic experiences" issued by Huffington Post blogger and ALS patient advocate Bo Stern.