Scientists Seek Stem Cell Cure For Spinal Cord Injuries

WAMU

WAMUScientists Seek Stem Cell Cure For Spinal Cord Injuries

By: Emily Berman // January 25, 2013

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Thomas Hazel PhD and Richard Garr @Neuralstem
Thomas Hazel, Ph.D, and Richard Garr pose in their neural stem cell laboratory, in Rockville, Md.

Neuralstem, a Rockville-based biotech company, has just been approved by the FDA to begin implanting stem cells into people with spinal cord injuries. While there’s a long scientific journey ahead, this trial could mean hope for paraplegic and quadriplegic patients all over the world.

The nervous system works, by sending electrical signals up and down the spinal cord. Degenerative diseases like Multiple Sclerosis, ALS, or Parkinsons impair the ability to send those signals. “There’s a gap,” says Richard Garr, co-founder and CEO of Neuralstem. He says there’s something that’s blocking signals from getting through.

Unlike our skin cells, the central nervous system doesn’t repair itself when damaged. “You’re born with a certain number of neurons, and that’s the way it is.” says Garr.

In 1998, Garr met Karl Johe, Ph.D. who had made a discovery while working at NIH. Around week 7 or 8, when the human embryo is the size of the tip of your thumb, there are cells in the brain area of the embryo that have all the information they need to become neurons. These are called ‘neural stem cells.’ Dr. Johe developed and patented techniques for extracting and multiplying these cells, then implanting them as ‘replacement neurons.’

“We’re actually putting in cells that are going to turn into neurons that are going to bridge the gap,” he says. “We’re creating new circuitry.”

Dr. Thomas Hazel, the head of Research at Neuralstem, explains one of the most important aspects of neural stem cells is that they can easily replicate. The lab received a donated tissue from a legally aborted fetus about 10 years ago, and they’ve been using those cells ever since.

The surgery recently approved by the FDA is much like an earlier trial, done on ALS patients. The surgeon injects neural stem cells directly into the patient’s spinal cord. Those stem cells grow into neurons, and if all goes according to plan, they help messages pass from the brain to muscles.

The ALS trial is waiting for phase 2 approval, but patients, on their own, are reporting some improvement. The spinal cord trial will take on eight patients who have experienced injury in the past 1 to 2 years in the thoracic spine, which is from the chest, down. Neuralstem will announce the partner hospitals in the coming months, and hope to start the surgeries in summer 2013.

Bespoke Stem Cells for Brain Disease

excerpt from article: “Over the last 2 years, stem cell therapies have done well in early clinical trials for sporadic neurological diseases, such as ALS and macular blindness. Such therapies have also shown promise for spinal cord injury, and just this week, the US Food and Drug Administration gave approval to the biopharma NeuralStem to begin a Phase I trial with fetal stem cells. It is the second US stem cell trial for spinal cord injury;

Click Here to read the online article

TheScientist

Bespoke Stem Cells for Brain Disease

Scientists use virus-free gene therapy on patient-derived stem cells to repair spinal muscular atrophy in mice.

By Nsikan Akpan | January 15, 2013

Most children with spinal muscular atrophy (SMA) will never jump rope, play tag, or even walk because a genetic deletion will provoke the gradual destruction of their spinal motor neurons. By correcting this mutation in stem cells derived from patients, Italian scientists have successfully curbed the progression of the disease in a mouse model. The results, published last month (December 15) in Science Translational Medicine, suggest that SMA sufferers may one day serve as their own donors for neuron transplants to treat their disease, according to a report.

“[It] is a beautiful study of the potential for using induced pluripotent stem cells (iPSCs) to treat genetic diseases,” said Lisa Ellerby from Buck Institute for Research on Aging, who was not involved in the study but is currently investigating the use of the technique to treat Huntington’s disease (HD).

Over the last 2 years, stem cell therapies have done well in early clinical trials for sporadic neurological diseases, such as ALS and macular blindness. Such therapies have also shown promise for spinal cord injury, and just this week, the US Food and Drug Administration gave approval to the biopharma NeuralStem to begin a Phase I trial with fetal stem cells. It is the second US stem cell trial for spinal cord injury; Geron abruptly halted the first in 2011.

Adding gene correction to the equation could expand their scope to treat a wide array of inherited disorders, Ellerby said. “As the field demonstrates that patient cells can be genetically corrected, we are closer to using this new technology to either model the disease or develop therapies for human patients.”

SMA is the leading genetic cause of infant mortality, killing one of every 6,000 babies born worldwide. The disease arises when a person fails to inherit a partially deleted version of the survival motor neuron 1 (SMN1) gene, which regulates multiple cellular processes involved with RNA metabolism. There is no cure.
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Somewhere along the evolutionary road to becoming human, the SMN1 gene was duplicated, resulting in SMN2, which can partially compensate for the dearth of SMN1 in SMA patients. Every SMA patient possesses an SMN2 gene. Those with multiple copies of SMN2 experience less severe SMA and typically survive into adulthood. But SMN1 and SMN2 are not identical: a single nucleotide difference impairs the pre-mRNA splicing of SMN2, such that its functional protein is produced at one-tenth the rate of the SMN1 protein.

Neurologist Giacomo Comi of the Univerity of Milan reasoned that if the single differing nucleotide of SMN2 were changed to mimic SMN1 in spinal neurons, perhaps the cells could survive. Rather than correct the SMN2 gene in endogenous SMA neurons, which may be dead or dying by the time the disease is recognized, Comi proposed replacing them entirely with iPSC transplants carrying a corrected copy of SMN2.

“The ideal therapeutic approach for SMA will be a combined strategy of molecular therapy to resolve the genetic defect and cell transplantation that can complementarily address signs of the disease,” said first author Stefania Corti of the University of Milan.

To accomplish this goal, Comi and his colleagues reprogrammed skin cells from SMA patients into iPSCs. The researchers then transfected the iPSCs with sequence-specific oligonucleotides that can repair genes with single base mutations. (In both steps, the researchers avoided using viral vectors because of the risks of tumor formation or harmful immune responses following transplantation.)  Finally, the genetically altered iPSC cells were differentiated into motor neurons and transplanted into mice that displayed symptoms of SMA.

SMA pups that received spinal grafts of “corrected” SMA-iPSC-derived neurons a day after birth showed significantly less spinal neuron loss and muscle atrophy. They were more physically active and stronger, as judged by open-field and grip tests. Neuron transplantation also extended lifespan by 50 percent.

The results suggest that the implanted neurons integrated into the spinal cord to alleviate motor dysfunction. Indeed, fluorescent histology revealed that the transplanted neurons formed neuromuscular junctions with muscle tissue near the spine. The implanted neurons also promoted the survival of the endogenous neurons still carrying the SMA mutation, indicating that the therapy provided neuroprotective benefits to the surrounding tissue as well. When grown in culture, corrected iPSC-derived neurons secreted more growth factors than uncorrected neurons, which may explain this transfer of vitality.

Of course, the research is very early stage, and many years of work will be needed to translate this success to the clinic, but “the implications are significant, not just for SMA disease, but also for similar neurodegenerative conditions like ALS and other neuromuscular diseases,” said Corti. “These data demonstrate the feasibility of generating patient-specific cells that are free of disease.”

S. Corti et al., “Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy,” Science Translational Medicine, 19:165ra162, 2013.

Neuralstem’s stem cells give spinal injury patients hope

Neuralstem’s stem cells give spinal injury patients hope

FDA gives Rockville biotech green light for initial trial

By Lindsey Robbins

This story was corrected on Jan. 15, 2013. An explanation follows the story.

Richard Garr NeuralStem CEO
Richard Garr NeuralStem CEO

Physicians, researchers, patients and their advocates in the spinal injury field are keeping a close eye on Rockville biotech Neuralstem as it prepares to launch a Phase 1 safety trial of its stem cell treatment for chronic spinal cord injury.

The Food and Drug Administration approved the trial Monday. Neuralstem plans to conduct the study on eight patients who are completely paralyzed at or below their spinal cord injuries.

“It’s important that people understand this is very different from other methods that have gone on before,” CEO Richard Garr said. “This is the real deal. We have compelling data. Cells are surviving, grafting and doing what we would expect they would do.” The FDA go-ahead follows Neuralstem’s report in October that rats given the stem cell product, NSI-566, seven days after suffering an ischemic stroke showed improvement in motor and neurological tests.

“Should this prove to be successful, it will allow for some regeneration of human spinal cord cells and for people to regain function. It will be an incredible breakthrough, with huge implications for the health care market,” said Paul Tobin, president and CEO of the National Spinal Cord Injury Association.

More than 10,000 people in the U.S. sustain spinal cord injuries each year, according to the Christopher & Dana Reeve Foundation. About 840,000 people have chronic spinal cord injury. Currently, the best treatment is mitigating secondary damage and providing environments and tools that support patients with these injuries, Tobin said.

While Tobin emphasized that the industry is still “far from a cure yet,” the Neuralstem treatment could be a tremendous step and appears to be worth exploring.

The primary objective of the study is to determine the safety and toxicity of human spinal stem cell transplants for treating paralysis and related symptoms due to chronic spinal cord injury, according to Neuralstem information. A secondary objective is evaluating graft survival in the transplant site.

All patients will receive six injections in or around the injury site, with the first four patients receiving 100,000 cells per injection and the second four receiving 200,000 cells per injection. The study will follow the patients for six months after the procedures.

Following Monday’s announcement, stock analyst Aegis Capital of New York raised its 12-month price target for Neuralstem to $4 from $3.50.

“Investors should note the fact that spinal cord injury is the clinical indication that most closely mirrors the situation in the preclinical rat model that yielded the ground-breaking data published in the [trade journal] Cell last year,” Aegis wrote in a report Monday.

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Aegis described itself as “cautiously optimistic” about Neuralstem’s treatment, but said the stem cell lines are still in “comparatively” early stages of development and that stem cell research, in general, faces uncertainty. Neuralstem’s stem cells are not from embryos.

Aegis also cautioned that Neuralstem had $9.9 million in cash and equivalents as of Sept. 30, with a monthly cash burn estimated as high as $1.5 million. Garr said Neuralstem is not planning fundraising in the immediate future.

Garr said the Phase 1 trial should begin this spring.

Other stem cell studies

Next month, Neuralstem plans to begin dosing patients with NSI-566 to treat paralysis from stroke in China, with a trial in Korea scheduled for the summer.

Neuralstem already has completed dosing in its Phase 1 trial at Emory University in Atlanta for amyotrophic lateral sclerosis. The trial will end six months after the last surgery. That therapy has received orphan status designation from the FDA, which confers certain advantages, such as a more streamlined process.

Though other companies have looked into this treatment, Neuralstem is the only one with an FDA-approved spinal cord injury trial using stem cells.

Geron Corp. in California had been conducting stem cell spinal cord injury trials in the U.S. but ended up shutting down that portion of its business in November 2011 and later sold it. StemCells is conducting trials in Switzerland.

Neuralstem officials said they hope success with this treatment allows for applications in chronic stroke motor disorder and multiple sclerosis.

“People should take hope,” Garr said.

Karl Johe, Neuralstem’s chairman and chief scientific officer, said the biotech’s data from other studies justify the new trial and the company’s confidence.

“In addition to the pre-clinical animal data, we have conducted 18 successful surgeries using the same cells and surgical device in our ALS trial,” Johe said in a statement. “That trial has demonstrated that the surgical route of administration and the cells are safe and well-tolerated and that the cells survive long-term in the patients. The successes of our human clinical experience, combined with the compelling data from the preclinical spinal cord injury animal studies gives us confidence that we are prepared to move into this additional indication for NSI-566.”

lrobbins@gazette.net

Explanation: The original version omitted a word from this quote by Richard Garr: “Cells are surviving, grafting and doing what we would expect they would do.”