Positive Results from Phase 2 Study in Spinal Cord Injury


Stem Cells, Inc., has released the six-month results from cohort I of an ongoing Phase 2 clinical trial of human neural stem cells for the treatment of chronic cervical spinal cord injuries. The data displayed significant improvements in muscle strength had occurred in five of the six patients treated. Of these five patients, four of them also showed improved performance on functional tasks that assesses dexterity and fine motor skills. Furthermore, these four patients improved in the level of spinal cord injury according to the classification system provided by the International Standards for Neurological Classification of Spinal Cord Injury or ISNCSCI.

Stem Cells, Inc., expects to release their detailed final 12-month results on this first open-cohort later this quarter.

Chief medical officer, Stephen Huhn, presented these data at the American Spinal Injury Association annual meeting in Philadelphia, on Friday, April 15.  Dr. Huhn also believes that the interim results are very encouraging and reason to be quite hopeful.

“The emerging data continue to be very encouraging,” said Dr. Huhn. “We believe that these types of motor changes will improve the independence and quality of life of patients and are the first demonstration that a cellular therapy has the ability to impact recovery in chronic spinal cord injury. We currently have thirteen sites in the United States and Canada that are actively recruiting patients. We have enrolled and randomized 19 of the 40 total patients in the statistically powered, single-blind, randomized controlled, Cohort II. We are projecting to complete enrollment by the end of September so that we can have final results in 2017.”

The present Phase 2 clinical trial is a multi-center enterprise that includes physicians and scientists at 13 different sites in the united States and Canada. Incidentally, these sites are presently actively recruiting patients.

Stem Cells, Inc., has enrolled and randomized 19 of the 40 total patients in this statistically powered, single-blind, randomized controlled, cohort II.

The Phase 2 study, “Study of Human Central Nervous System (CNS) Stem Cell Transplantation in Cervical Spinal Cord Injury,” will determine the safety and efficacy of transplanting the company’s proprietary human neural stem cells (HuCNS-SC cells) into patients with traumatic injury of the cervical region of the spinal cord.

Cohort I is an open label dose-ranging cohort in six AIS-A or AIS-B subjects. For those of you not familiar with the American Spinal Injury Impairment Scale (ASI A-E scale), here is a summary of the classification scheme:

ASI – A = Complete paralysis; No sensory or motor function is preserved in the sacral segments S4-5.
ASI – B = Sensory Incomplete; Sensory but not motor function is preserved below the neurological level and includes the sacral segments S4-5 (light touch or pin prick at S4-5 or deep anal pressure) AND no motor function is preserved more than three levels below the motor level on either side of the body.
ASI – C = Motor Incomplete; Motor function is preserved below the neurological level**, and more than half of key muscle functions below the neurological level of injury (NLI) have a muscle grade less than 3 (Grades 0-2).
ASI – D = Motor Incomplete; Motor function is preserved below
the neurological level**, and at least half (half or more) of key muscle functions below the NLI have a muscle grade > 3.
ASI – E = Normal; If sensation and motor function as tested with the ISNCSCI are graded as normal in all segments, and the patient had prior deficits, then the AIS grade is E. Someone without an initial SCI does not receive an AIS grade.
Cohort II is a randomized, controlled, single-blinded cohort in forty AIS-B subjects. Cohort III, which will only be conducted at the discretion of the sponsor, is an open-label arm that involves six AIS-C subjects.
The primary efficacy outcome will focus on changes in the upper extremity strength as measured in the hands, arms, and shoulders.  This trial will enroll up to 52 subjects.
StemCells, Inc. has demonstrated the safety and efficacy of their HuCNS-SC cell in preclinical studies in laboratory rodents.  Additional Phase I studies yielded positive human safety data.  Furthermore, completed and ongoing clinical studies in which its proprietary HuCNS-SC cells have been transplanted directly into all three components of the central nervous system: the brain, the spinal cord and the retina of the eye, have further demonstrated the safety of HuCNS SC cells in human patients.
StemCells, Inc. clinicians and scientists believe that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, there is the possibility of a durable clinical effect following a single transplantation.
The HuCNS-SC platform technology is a highly purified composition of human neural stem cells (tissue-derived or “adult” stem cells). Manufactured under cGMP standards, the Company’s HuCNS-SC cells are purified, expanded in culture, cryopreserved, and then stored as banks of cells, ready to be made into individual patient doses when needed.

Stem Cells Inc Spinal Cord Injury Trial Shows Sustained Improvements in Sensory Function


A cellular therapeutic company known as Stem Cells, Incorporated has been carrying out a Phase I/II clinical trial that was specifically designed to assess both safety and preliminary efficacy of their proprietary HuCNS-SC cells as a treatment for chronic spinal cord injury. Recently, Dr. Armin Curt, the principal investigator of this clinical trial, presented a summary of the safety and preliminary efficacy data from this Phase I/II study at the 4th Joint International Spinal Cord Society (ISCoS) and American Spinal Injury Association (ASIA) meeting which was held in Montreal, Canada.

Spinal cord injury patients are classified by a system that was developed by the American Spinal Injury Association (ASIA) and uses grades A through E on the American Spinal Injury Association Impairment Scale (AIS) to indicate the severity of the spinal cord injury. AIS Grade A injuries consist of a loss of all spinal cord function (sensation and movement) below the level of injury is lost. This is known as a complete injury. All the other AIS grades are considered incomplete. Patients with Grade B injuries have some sensation below the level of injury, but there is no movement below the injury.. In patients with AIS Grade C injuries, there is both sensation and movement, but most of the muscles below the injury cannot function against resistance and that includes gravity. Those with AIS Grade D spinal cord injuries have some sensation and movement, but more than half of the muscles below the injury can function against resistance. Finally those with AIS Grade E injuries have both normal sensation and movement, but there may be other signs of injury, for example, pain.

For this trial, Stem Cell Inc enrolled 12 subjects who had suffered from a severe spinal cord injury at the thoracic or chest level (T2-T11); seven AIS A and 5 AIS B patients.. In order to qualify for this study, all patients had to be classified as either AIS A or B and a minimum of 3 months from injury.

The trial involved internationally prominent medical centers for spinal cord injury and rehabilitation, and associated principal investigators; Dr. Armin Curt at the University of Zurich and Balgrist University Hospital, Dr. Steve Casha at the University of Calgary, and Dr. Michael Fehlings at the University of Toronto.

All subjects in this trial received HuCNS-SC cells by means of direct transplantation into the spinal cord and they were also treated, temporarily, with immunosuppressive drugs to prevent the immune system from rejecting the implanted cells. Patients were regularly evaluated for safety of the treatment protocol, and to determine if patients showed any change in neurological function. To determine this, patients were given a standard battery of movement and sensory tests before the surgery and at routine intervals after the procedure. Thus all patients were simultaneously enrolled in a safety evaluation and separate evaluation that tested the efficacy of the procedure as well.

In the safety analyses of these subjects, all the data demonstrated that the surgical transplantation technique and cell dose were safe and well tolerated by all patients. HuCNS-SC cells were injected directly into the spinal cord both above and below the level of injury and none of the patients in sequential examinations over the course of twelve months showed any abnormal changes in spinal cord function associated with the transplantation technique. Additionally, there were no adverse events that could be attributed to the HuCNS-SC cells.

Analyses of the functional data after twelve-months revealed sustained improvements in sensory function that emerged consistently around three months after transplantation and persisted until the end of the study. These gains in sensory function involved multiple sensory pathways and were observed more frequently in the patients with less severe spinal cord injuries. Three of the seven AIS A patients and four of the five AIS B patients showed signs of positive sensory gains. Two patients in the study progressed from AIS A, to the lesser degree of injury grade, AIS B.

“It has been a privilege to be a part of the first study to test the potential of neural stem cell transplantation in thoracic spinal cord injury,” said Dr. Armin Curt, Professor and Chairman of the Spinal Cord Injury Center at Balgrist University Hospital, University of Zurich. “The gains we have detected indicate that areas of sensory function have returned in more than half the patients. Such gains are unlikely to have occurred spontaneously given the average time from injury. This patient population represents a form of spinal cord injury that has historically defied responses to experimental therapies, and the measurable gains we have found strongly argue for a biological result of the transplanted cells. These gains are exciting evidence that we are on the right track for developing this approach for spinal cord injury. This early outcome in thoracic injury firmly supports testing in cervical spinal cord injury.”

Stephen Huhn, M.D., FACS, FAAP, Vice President, Clinical Research and CMO at StemCells, Inc., said, “This research program has the potential to revolutionize the therapeutic paradigm for spinal cord injury patients. The clinical gains observed in this first study are a great beginning. We found evidence of sensory gains in multiple segments of the injured thoracic spinal cord across multiple patients. Our primary focus in this study for spinal cord injury was to evaluate safety and also to look for even small signs of an effect that went beyond the possibility of spontaneous recovery. We are obviously very pleased that the pattern of sensory gains observed in this study are both durable and meaningful, and indicate that the transplantation has impacted the function of damaged neural pathways in the cord. The Company’s development program has now advanced to a Phase II controlled study in cervical spinal cord injury where the corollary of sensory improvements in thoracic spinal cord injury could well be improved motor function in the upper extremities of patients with cervical spinal cord injuries.”

Stem Cells Inc. Reports Additional Spinal Cord Injury Patients Transplanted with Neural Stem Cell Line Show Functional Improvements


StemCells, Inc. has developed a proprietary stem cell line called HuCNS-SC.  This stem cell line is a neural stem cell line, and neural stem cells can readily form neurons (the conducting cells of the nervous system), or glial cells (the support cells of the nervous system). In order to determine if these cells can regenerate spinal nerves in patients who have suffered a spinal cord injury, StemCells Inc. has commissioned a clinical trial to test their cells in human spinal cord injured patients.

Early indications showed that the HuCNS-SC cells were safe, but some patients have shows improvements in sensation.  Now StemCells Inc has issued an announcement that these initially reported improvements in only a few patients have also been confirmed in other patients.

According to Armin Curt, M.D., Professor and Chairman of the Spinal Cord Injury Center at Balgrist University Hospital, University of Zurich, and the principal investigator of their Phase I/II trial, the initial improvements that were observed in the first two patients treated with their HuCNS-SC neural stem cells have now been observed in two additional patients who have also been treated with these stem cells. These results come from an interim analysis of recent clinical data.

In a presentation to the Annual Meeting of the American Spinal Injury Association in San Antonio, Texas, Dr. Curt showed data on AIS B subjects who were transplanted with HuCNS-SC neural stem cells in the Phase I/II chronic spinal cord injury trial. This trial is different from the AIS A patients who have no mobility or sensory perception below the point of injury, since AIS B subjects are less severely injured, and are paralyzed but retain sensory perception below the point of injury. Two of the three AIS B patients who are participating in the study showed significant gains in sensory perception. The third patient remained stable.  These interim results confirm the favorable safety profile of these stem cells and the surgical implant procedure used to transplant them into the spinal cords of spinal cord injury patients.

Also included in Dr. Curt’s presentation was data from a total of five new subjects with a minimum six-month follow-up. In total, Stem Cells Inc. has now reported clinical updates on a total of eight of the twelve patients enrolled in its Phase I/II clinical trial that is testing this Company’s proprietary HuCNS-SC (purified human neural stem cells) platform technology for treating chronic thoracic spinal cord injury.

“Thoracic spinal cord injury was chosen as the indication in this first trial primarily to demonstrate safety. This patient population represents a form of spinal cord injury that has historically defied responses to experimental therapies and is associated with a very high hurdle to demonstrate any measurable clinical change. Because of the severity associated with thoracic injury, gains in multiple sensory modalities and segments are unexpected, and changes in motor function are even more unlikely,” said Dr. Curt. “In contrast, the cervical cord, which controls more motor function, may represent a patient population in which motor responses to transplant may be more readily anticipated.”

“We are seeing multi-segmental gains and a return of function in the cord in multiple patients. This indicates something that was not working in the spinal cord, now appears to be working following transplantation. This is even more significant because of the time that has elapsed from the date of injury, which ranges from 4 months to 24 months across the subjects with sensory gains,” said Stephen Huhn, M.D., FACS, FAAP, vice president, CNS clinical research at StemCells, Inc. “These results are exciting with respect to the expansion of this trial into patients with cervical injury because even a gain of one to two segments in cervical spinal cord injury patients can allow for additional function in the upper extremities.”