A few weeks ago, Victoria, Gwendolyn, and I were invited to an update event for a very promising stem cell therapy program headed by Dr. Hans Keirstead, Co-Director of the Sue and Bill Gross Stem Cell Research Center at UC Irvine. In short, this therapy has the potential to end, yes END Spinal Muscular Atrophy (SMA), ALS/Lou Gehrig's Disease, and acute spinal cord injuries by replacing the lost motor neurons that characterize these conditions with high purity human motor neurons derived from human embryonic stem cells; a process that Dr. Keirstead's team has perfected. The program has been in process for five years and is a collaboration between Families of SMA, UC Irvine, California Stem Cell, and Johns Hopkins University.
As an aside, while this specific program and the accomplishments of Dr. Keirstead's team stand on their own as groundbreaking research discoveries, it is important to note that Dr. Keirstead is no stranger to the stem cell world or pioneering research. In 2004, Dr. Keirstead led his team of researchers at UC Irvine to successfully develop a human embryonic stem cell derived treatment for acute spinal cord injuries. That treatment was approved by the FDA in January 2009 for clinical trials in humans. This spinal cord injury trial, which is currently in process and is being carried out by Geron Corporation, marks the first ever human embryonic stem cell trial approved in the United States. The motor neuron replacement therapy that will focus on SMA will most likely be the second — ever!
The SMA program is currently at a critical and exciting juncture. All of the pre-clinical efficacy studies, including the pivotal animal safety studies, have been completed, demonstrating that the cells work and that the motor neuron replacement should be a safe strategy in the treatment of diseases, such as SMA, characterized by motor neuron loss. Dr. Keirstead and his team are now preparing for a pre-IND meeting with the FDA in Q3-2009 with a targeted Phase I clinical trial in humans to begin in early 2010. Phase I will initially focus on babies with SMA Type I, Gwendolyn's Type, with other Types planned for later phases of the study.
Families of SMA and others have invested over $1.5 Million in the program over the past 5 years. While California Stem Cell will be responsible for most of the funding through the clinical trial process, the program needs an additional ~$500,000 as a bridge to that phase for staffing and studies to support the requirements leading into that critical Phase I clinical trial in early 2010. This research is unbelievably promising and could truly change the future for SMA and countless other diseases.
If you or anyone you know is interested in helping see this promising research make it swiftly to and through clinical trial or if you know any foundations that focus on this type of groundbreaking medical research, please email me directly for more information:
Bill Strong
[email protected]
Click here to read more about the utility of stem cell therapy in treating SMA.
The CNN video below is of Dr. Keirstead explaining the process of using stem cell therapies to treat spinal cord injuries: