Thursday, January 21, 2010
First U.S. Stem Cells Transplanted into Spinal Cord
Doctors injected stem cells from 8-week-old fetal tissue into the spine of a man in his early 60s who has advanced ALS, or amyotrophic lateral sclerosis. It was part of a clinical trial designed to determine whether it is safe to inject stem cells into the spinal cord and whether the cells themselves are safe.
ALS is a fatal neurodegenerative disease that causes the deterioration of specific nerve cells in the brain and spinal cord called motor neurons, which control muscle movement. About 30,000 Americans have ALS at any given time, according to the ALS Association.
There is no cure for ALS, which is better known as Lou Gehrig's disease, named after the New York Yankees' first baseman and Hall of Famer who retired from baseball in the 1930s after being diagnosed with the disease.
As the illness progresses, patients lose their ability to walk, talk and breathe. Patients usually die within two to five years of diagnosis, according the ALS Association.
Neuralstem Inc., a Rockville, Maryland-based biotech company, received approval from the U.S. Food and Drug Administration to conduct the clinical trial in September. The company is fully funding the research and provides the stem cells that are being injected into the patients.
Neuralstem announced the start of the clinical trial in a news release Thursday.
Longtime ALS researcher and University of Michigan neurologist Dr. Eva Feldman is overseeing the first human clinical trial of a stem cell treatment in ALS patients.
"We are entering a new era of cell therapeutics for ALS, and in my opinion, it is an new era of hope for patients with ALS," Feldman said.
At least 12 patients are expected to participate in this early research. They are to receive the stem cell transplants at Emory University in Atlanta, Georgia.
"This is the first study to see if the invasive injection into the spinal cord is safe for the patient," said Lucie Bruijn, science director of the ALS Association.
This first patient in the clinical trial received several injections of stem cells into the lumbar region of the spinal cord, the area that controls leg function, because most ALS patients first lose muscle function in their legs, according to Karl Johe, Neuralstem's chairman and chief scientific officer.
Bruijn says there have been a few other occasions outside the United States in which fetal stem cells have been injected into a patient, "but not necessarily using a very [rigorous] trial design." She adds that there were also a couple of small studies in Italy that injected other types of stem cells into a few patients but that this is the first FDA-approved trial in the United States.
"Our biggest hope for stem cells is to significantly slow the progression the disease," Bruijn said.
The ALS Association is not providing funding for this clinical trial, but it has supported the work of Dr. Nick Boulis, the Emory neurosurgeon who developed the surgical technique used to inject the stem cells.
Johe invented the technology that allows the company to manufacture billions of copies of stem cells that are taken from a single source of spinal cord cells: cells that were extracted from fetal tissue, which was donated to the company.
"The cells are human neural stem cells," Johe said, acknowledging that the introduction of stem cells is a very invasive procedure.
"What we are attempting is a novel approach by directly injecting them into the middle of the spinal cord, which to our knowledge has never been done before," Johe said.
Researchers plan to follow this and future patients participating in this trial for a long time to determine the safety of the procedure.