Friday, November 27, 2009
Emory wins 1st stem cell trial for ALS
Atlanta Business Chronicle - by Urvaksh Karkaria Staff Writer
Emory University will be the site of the first U.S. clinical trail that focuses on using stem cells to slow the progression of adults with Lou Gehrig’s disease.
Rockville, Md.-based Neuralstem Inc. (Amex: CUR) hopes to use neural stem cells from the spinal cord of a fetus to slow the progression of adults with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
The early-stage trial, which could include up to 18 patients, will test the safety of the injection process and the implanted stem cells.
“No one’s ever injected cells directly into the gray matter of the spinal cord,” Neuralstem President and CEO Richard Garr said.
ALS is a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement. ALS patients typically die within three years of diagnosis. About 30,000 people in the U.S. have the degenerative condition and about 7,000 are newly diagnosed each year. There are more than 500 Georgians with ALS.
Embryonic stem cell research is controversial locally and nationally. President Barack Obama signed an executive order in March lifting restrictions on federal funding for embryonic stem cell research. The Georgia Senate, however, OK’d legislation this year that would have shut down most forms of embryonic stem cell research in the state. However, the proposal, which failed in the Georgia House of Representatives, would not have prevented researchers from using new stem cell lines brought in from out of state or existing stem cell lines.
Unlike Neuralstem’s spinal cord-derived stem cells, most embryonic stem cells are derived from embryos that develop from eggs that have been fertilized in an in vitro fertilization clinic.
The internationally watched Neuralstem trial will put Emory’s ALS program — one of the largest ALS clinics in the country — on the map.
Emory was chosen as the site of the trial because it “has one of the best, if not the best, ALS clinicians and research groups,” Garr said. Emory neurosurgeon Dr. Nicholas Boulis developed the surgical techniques to implant the stem cells in the adult spinal cord.
The high-profile clinical trial will accelerate Emory’s translational research, said Dr. Jonathan Glass, principal investigator for the trial and director of the Emory ALS Center.
“It’s going to make us the center of attention for anybody who wants to do stem cell injections into the spinal cord for other diseases,” Glass said. “They’re going to come to us ... and say, ‘How do you do it?’ ‘What’s the best way to do it?’ and ‘Teach us how to do it.’ ”
The publicity surrounding the trial will also make Atlantans aware of the resources in their own back yard, Glass noted.
“When people get sick, some of them go to the Mayo Clinic,” he said. “The reality is that they have the best thing in town and maybe they need to see that.”
People are born with a specific number of spinal cord neural cells, which typically last a lifetime. In ALS patients, certain neural cells die early. When that happens, the spinal cord isn’t able to send messages to the body’s muscles, which in turn atrophy.
Neuralstem hopes its spinal cord-derived stem cells will protect healthy neural cells and repair those that have ceased communicating with the patient’s muscles. That loss of signal triggers muscle atrophy and eventual paralysis that ALS patients suffer.
“The promise of stem cells has been hanging out there for probably more than a decade,” Glass said. “Nobody has really tried it in a systematic way.”
Stem cells are able to find their way to the injured region and transform into nurturing cells, Glass said. “What I’m hoping for,” he said, “is that ... these [stem] cells will set up shop in this region of injury and provide some kind of nurturing effect that will protect the cells that are still there, and possibly even allow the sick cells to reconnect with the muscles.”
Neuralstem reported in the online journal Neuroscience that three rats paralyzed by a specific spinal cord injury returned to near-normal ambulatory function six weeks after having stems cells grafted to their spinal cords. Three others showed significant improvement after two months. In all the grafted animals, the majority of the transplanted stem cells survived and became mature neurons, Neuralstem said.
The Phase I human trial will test the safety of the procedure which involves delivering the stem cells to a delicate spot — the spinal cord. “Just looking at the spinal cord can hurt it,” Glass quipped.
There’s a lot more at stake than Neuralstem’s fortunes. “If we mess up,” Glass said, “we could take the whole stem cell therapeutic idea and kind of set it back 10 years.”
The trial, while small, is significant in terms of helping move the science forward and potentially develop a treatment for ALS, said Lucy Bruijn, chief scientist at The ALS Association.
“It’s a disease that’s desperately looking for a good treatment,” she said. “Stem cells have been extremely promising, but there haven’t been very many rigourous efforts, at least in the clinic ... to try this out.”
ALS involves a complicated systems of cells that connect the brain to the spinal cord, which then connect to muscles, Bruijn said. “Clearly, it’s a tricky thing as to where exactly do you put these [stem] cells to have the best benefit,” she said.
Using technology, developed at the National Institutes of Health, Neuralstem can expand stem cells taken from the donated fetus, up to a “billion-billion times.”
Neuralstem is able to produce enough cells to “transplant every spinal cord patient we have to ever transplant with these cells,” Garr said.
The company has invested more than $50 million in developing its stem-cell technology and getting ready for human trials. It expects to spend about $8 million to $10 million more to complete the clinical trial process.
If the Phase I trial is successful, Garr said, the next step would be a larger, possibly multi-site trial that would focus on the therapy’s effectiveness.
“Some day,” Garr said, “neural stem cells will be the universal delivery vehicle for all large-molecule therapies in the (central nervous system).”
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