Phase II trials of a study designed to treat Amyotrophic lateral sclerosis (ALS) patients with injections of human neural stem cells administered in the spinal cord are now beginning, led by researchers at Emory and the University of Michigan.
ALS – or Lou Gehrig's disease – is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, according to the ALS Association website.
The disease attacks the body's motor neurons, the destruction of which renders the brain unable to initiate and control muscle movement. The ALS Association says this can lead to total paralysis and, eventually, death.
The disease affects approximately two out of every 100,000 people – primarily those in their 40s through 70s. The average life expectancy of an ALS patient after diagnosis is approximately two to five years, according to the ALS Association.
Nicholas Boulis, an assistant professor of neurosurgery in the Emory School of Medicine, administers the surgeries involved in the study. Death from ALS usually occurs because the patient loses the ability to breathe on his or her own, he said.
Although ALS patients can prolong life expectancy by undergoing a tracheotomy, which provides an air passage to assist breathing, and by using a ventilator, Boulis said this progressively worsens the patient's quality of life.
While there is no known cure for ALS, this new therapy may increase the life expectancy and quality of life for ALS patients.
"This is about trying to keep people off ventilators for longer," Boulis said. "It's about trying to preserve their ability to breathe as long as possible."
According to Boulis, initially the idea was to manufacture neurons that would be transplanted into the nervous system to eventually replace the function of motor neurons.
In this particular study, however, the stem cells are turning into cells that protect motor neurons rather than replace them, Boulis explained.
Boulis said experiments conducted with animals prior to the study showed that injecting neural stem cells into the spinal cord helped preserve motor neurons. Phase I of the study tested the safety of injecting stem cells into the spinal cord, including the safety of the devices used in surgery and the stem cells themselves.
In Phase I, Boulis said researchers began by injecting the cells into the lumbar region of the spinal cord, which is the region that controls the legs, of patients paralyzed from the waist down.
The researchers then proceeded to test patients who could walk. According to Boulis, the trials proved safe. The injections were then administered in the cervical region of the spinal cord, which is the region that controls breathing.
The study is now moving onto phase II trials. Boulis said this phase will test different dosages of injections and different concentrations of stem cells, in addition to testing the efficacy of these treatments.
Boulis said if phase II is successful, the study will move onto phase III, which will test the therapy in a much larger population. He said phase II of the trial is proceeding much faster than phase I, which indicates positive signs.
"I think it means that the FDA is gaining confidence in what we're doing," Boulis said.
The idea to inject stem cells into the spinal cord is rooted in the rising popularity of using stem cells in regenerative medicine, Boulis said. Jonathan Glass, director of the Emory ALS Center and principal investigator for the study, said that despite the enthusiasm surrounding the use of stem cells to treat diseases, no substantial research had been conducted until this study.
"Stem cells have a magical aura around them in many diseases but, in reality, they haven't been tested in any major way to see if they will work," Glass said.
Glass said this study was innovative in the way that it involved injecting stem cells into the spinal cord. According to Boulis, although the spinal cord is generally seen as vulnerable and delicate, this study demonstrates that scientists can inject various cell therapeutics into the spinal cords of people with neurodegenerative diseases. He said the results of the study so far have a global impact.
"It opens the doorway to many different kinds of therapeutics that might be effective," Boulis said.
– By Harmeet Kaur
