John Cornick, a resident of North Carolina, is not the first person to be diagnosed with amyotrophic lateral sclerosis — a neurodegenerative disease that robs its sufferers of voluntary muscle movement. But he is among the first to receive direct stem cell injections to experimentally treat the disease.
Leading a team in the first ever FDA-approved stem cell procedure, Prof. of Neurology Eva Feldman said she hopes the landmark study will bring aid to those suffering from the effects of ALS.
After years of work, Feldman, director of the A. Alfred Taubman Medical Research Institute, and her team have been able to apply their stem cell research to the phase one clinical trial of the study of ALS. The trial marks the first FDA-approved procedure in which fetal stem cells are injected into the spinal cords of adults.
“For 20 years, I could offer little hope to my patients with ALS,” Feldman wrote in an e-mail interview. “Now, there is truly tangible hope. We are testing a therapy that may allow us to halt the progression of this terrible disease.”
The trial has been ongoing at Emory University in Atlanta, Georgia since January. So far, three patients have received the injections of stem cells, but up to 12 patients may be enrolled in the study.
Neuralstem, Inc. — a publicly traded biotherapeutics company based in Maryland, which specializes in the use of neural stem cells as a form of biotechnology — is supplying the stem cells for the study. The company is funding the entire clinical trial at Emory University.
Richard Garr, president and CEO of Neuralstem, said the use of neural stem cells isolated from humans in the fetal stage of development is better suited to the study as opposed to embryonic stem cells.
Unlike the already differentiated neural stem cells, embryonic stem cells have the potential to develop into any type of cell found in the body with the proper stimulation.
Garr said the neural stem cells provided by Neuralstem do not require the additional manipulation necessary to coax embryonic stem cells to develop into neural tissue.
“Think about the incredible complexity of development and all of the information that is exchanged between the cells during the developmental process,” he said. “We think that that is kind of hard to replicate in a couple days in a (petri) dish. So we would say that these (neural stem cells) are more physiologically relevant. We don’t have to worry about what information these cells do or do not have.”
Garr added that, since ALS affects nerve cells, the use of neural stem cells in the study is warranted.
ALS is characterized by death of motor neurons in the spinal cord, which control muscle movement. Progression of the disease to the muscles of the diaphragm, which assist in breathing, often causes the death of ALS patients.
Feldman, who oversaw the surgical implantation, said ALS leaves neurons in a damaged state that can be supported by injected stem cells, according to a May 4 interview with CNN.
“When we inject stem cells in the spinal cord the stem cells surround those large nerve cells and allow those nerve cells to actually become less diseased and, in fact, those nerve cells begin to look healthy,” Feldman said in the interview.
The phase one trial will determine if injecting fetal stem cells into the spinal cords of adults is a safe procedure and will show whether it is an effective treatment for ALS, Feldman wrote.
According to Garr, it could take about six years to complete the first phase of the trial.
“I recently told a new patient that the situation is hopeful now. The future is very bright. Not just for ALS, but for patients with other neurological diseases, including Alzheimer’s, Parkinson’s and Huntington’s,” Feldman wrote.