Stem Cell Strides

An increasing number of stem cell studies are gaining FDA approval and making leaps toward hope in SCI research.

Studies in stem cells continue to lead the way in promising research for spinal-cord injury and disease (SCI/D).

With successful trials of stem cell transplantation in animals, patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and even patients with SCI, researchers are moving forward.

The Food and Drug Administration (FDA) continues to approve trials for patients with thoracic SCI and some researchers are beginning the transition to patients with cervical SCI. We have updates on several leading research studies as the developments continue to bring hope to the SCI/D community.

StemCells, Inc.

Researchers from StemCells, Inc. are nearing the end of a phase I study and may be making the exciting jump into phase II.

The phase I trial administered the company’s HuCNS-SC human neural stem cells to 12 patients with a complete thoracic SCI. The study started in Switzerland at the University of Zurich, but in the last year has received regulatory approval to initiate sites in Canada and the U.S.

“So at this point and time we are still analyzing the data. We dosed a lot of patients and it takes a while for the follow-up data to come in to be analyzed and to accrue,” says Stephen Huhn, vice president and head of central nervous system clinical research. “So we don’t have any new updates with regard to the status of the patients other than we have confidence in the ongoing safety to all of the interventions in the study so far.”

Phase I is expected to be completed in the first quarter of the year, but Huhn says they have already seen enough to begin planning a phase II trial. The control study will enroll patients in North America with cervical SCI in order to measure a clinical benefit from the stem cells.

Human Schwann cells from the Miami Project to Cure Paralysis being cultured in a dish.

“This is going to be a big jump up from our current study that started at a single site. Now we are going to be asking a more complex and a more meaningful question for the spinal-cord injury community in terms of the effect,” Huhn says. “So what will the effect of the cells be, and in particular, of course, what will be the effect in patients with cervical cord injury?”

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Neuralstem, Inc.

After completing trials in both rats and human patients with ALS, the FDA has approved Neuralstem, Inc. to begin a phase I trial in patients with chronic SCI.

The transplantation of Neuralstem’s human spinal stem cells showed a significant recovery of movement in lower-extremity joints of paralyzed rats during a study in conjunction with the University of California-San Diego. Neuralstem also successfully performed 18 surgical transplants of its NSI-566 stem cells — the same cells and application to be used in the phase I SCI trial — in patients with ALS. Since the successful phase I ALS trial, the company has treated the first patient in the phase II ALS trial.

“We believe that it is the right time to leverage what we have learned with ALS in spinal-cord injury,” says Neuralstem Chairman of the Board and Chief Scientific Officer Karl Johe, PhD, in a press release. The study will determine the safety and toxicity of human stem cell transplantation for treating paralysis and related symptoms in patients with a thoracic SCI.”

Up to eight patients will be enrolled in the trial, which will look at secondary endpoints. Johe says he hopes to measure some degree of functional recovery. Once surgery safety has been established, the plan is to transplant patients whose injuries are in the cervical (upper spinal-cord region). Neuralstem has not yet begun the trial, but hopes to in the first half of this year.

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The Miami Project

Doctors at the University of Miami’s Jackson Memorial Medical Center are currently treating and studying patients with recent complete thoracic SCI in a phase I safety trial. The study at the Miami Project to Cure Paralysis is the first study to transplant a patient’s own Schwann cells to gain FDA approval.

“I am delighted to announce that Miami Project scientists have injected autologous Schwann Cells in two subjects with acute (recent) spinal-cord injuries,” says Marc Buoniconti, president of The Miami Project, in a fall 2013 update. “Those subjects are reacting well to the transplant and have not demonstrated any adverse reactions. As we increase the number of subjects, we will be increasing the dose of Schwann cells in order to discover the optimal therapeutic dose level.”

Named after physiologist Theodor Schwann, Schwann cells are adult cells (not stem cells) found in the peripheral nervous system, including nerves going to muscles and sensory nerves from the muscles to the spinal cord. The support cells wrap around individual nerve fibers, making them essential for sending appropriate electrical signals through the nervous system. Researchers at The Miami Project have been studying the cells since the 1980s because they naturally help repair the peripheral nervous system and usually move to SCI sites.

As the trial is ongoing, the project can’t release any more information or results on the patients. It is, however, actively recruiting and screening nationwide for more patients to enroll in the study.

“We are confident that we will begin our chronic (those paralyzed a year or more) trial in 2014,” says Buoniconti.

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Harvard University

Researchers at Harvard University and the Riken Center for Developmental Biology in Kobe, Japan, have found a way to turn mature cells back into stem cells.

Based on findings from studies in which stem cells are isolated, lead scientist Charles Vacanti and his team realized scientists were taking an area of cells and exposing them to stress, which killed off the mature cells and only left the stem cells behind.

“We decided, is it the stem cells that remain or is the stress causing the mature cells to revert back?” says Vacanti.

The next step was to expose mature cells to chemicals from the stress of an injury and the chemicals scientists were using to isolate stem cells and see what would happen. Sure enough, they found the mature cell reverted back to stem cells, called stress-triggered acquisition of pluripotency (STAP) cells.

Vacanti has already done a small study with the cells on monkeys with spinal-cord injury. The only problem is scar tissue in the spinal cord that prevents the cells from repairing the spinal cord, adding an extra step to remove the scar tissue before transplanting the cells.

“As far as spinal-cord injury, I’d like to do a larger monkey trial to prove that this is effective and then apply for FDA approval to study humans,” says Vacanti.

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