- The SMA stem cell program is headed by Dr. Hans Keirstead, Co-Director at the Sue and Bill Gross Stem Cell Center at UC Irvine
- The SMA 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
- While this program will initially focus on SMA to prove the science, the treatment has the potential for dramatic breakthroughs for ALS/Lou Gehrig’s, acute spinal cord injuries, and many other conditions
- Dr. Keirstead has already been credited with successfully developing a stem cell derived treatment for acute spinal cord injuries; this program is currently in human clinical trials - the 1st ever stem cell trial approved in the United States
- For SMA, Dr. Keirstead has perfected a process that replaces lost motor neurons that characterize SMA with high purity human motor neurons derived from human embryonic stem cells
- All of the pre-clinical efficacy studies, including the pivotal animal safety studies, have been completed, demonstrating that the cells work
- They are now targeting a Phase I clinical trial in humans to begin in early 2010 with later phases to begin shortly thereafter
Slightly More Detail
The motor neuron replacement stem cell program is 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 cure 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.
While the motor neuron replacement program and the accomplishments of Dr. Keirstead’s team with respect to this specific program 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 initially focus on SMA will most likely be the second — ever!
Dr. Keirstead’s SMA stem cell 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 late 2009 with a targeted Phase I clinical trial in humans to begin in early 2010. Phase I will initially focus on babies under the age of 12 months with SMA Type I, with other SMA Types and older Type I’s planned for later phases of the study.
Click here to read more about the utility of stem cell therapy in treating SMA.