StemCells, Inc. announced that the first patient in the company's breakthrough phase I/II clinical trial in chronic spinal cord injury was successfully transplanted with the company's proprietary HuCNS-SC adult neural stem cells. The stem cells were administered at Balgrist University Hospital, University of Zurich, a world leading medical centre for spinal cord injury and rehabilitation. The transplant surgery was performed by a team of surgeons led by Dr Raphael Guzman, a visiting staff neurosurgeon also on faculty at Department of Neurosurgery, Stanford University, and Dr K Min, an orthopaedic surgeon at Balgrist University Hospital.
"I am pleased to be a part of this innovative clinical trial designed to help us assess the safety and potential efficacy of HuCNS-SC stem cells for spinal cord injury," explains Dr. Armin Curt, Principal Investigator. "The preclinical data underlying this trial provided compelling rationale to conduct a study of this nature in spinal cord-injured patients." StemCells, Inc. has published numerous preclinical studies demonstrating the therapeutic potential of the company's human neural stem cells for the treatment of acute and chronic spinal cord injury. These studies were conducted in close collaboration with Drs. Aileen Anderson and Brian Cummings of the University of California, Irvine.
The first patient transplanted in the trial, a 23-year-old German man, suffered a spinal cord injury in an automobile accident in April of this year. He sustained a complete loss of sensation and mobility from the waist down. When asked about his decision to enroll in this leading-edge study, he said: "This terrible injury crossed out almost all my life plans, and has led me to an unexpected path. Participating in this clinical trial not only gives me a sense of hope, but it also helps move this important research forward."
"With this first patient enrolled and dosed, we remain on track to meet our goal of treating the first cohort of patients by the end of this year," said Stephen Huhn MD, FACS, FAAP, vice president and head of the CNS Program at StemCells, Inc. "While the trial's first cohort will consist of patients with the most severe, complete injury, the second and third cohorts will progress to patients with less severe, incomplete injury. This unique trial design will allow us to evaluate the potential of our HuCNS-SC cells as a treatment for a broad spectrum of spinal cord injury patients. Even a small improvement could have a marked impact on quality of life for the millions of people who suffer from this debilitating condition."
The phase I/II clinical trial of StemCells, Inc.'s HuCNS-SC purified human adult neural stem cells is designed to assess both safety and preliminary efficacy. Twelve patients with thoracic (chest-level) neurological injuries at the T2-T11 level are planned for enrollment. The first three patients will all have injuries classified as ASIA A, in which there is no apparent neurological function below the injury level, the most severe level identified by the American Spinal Injury Association (ASIA) Impairment Scale. The second and third cohorts will be patients classified as ASIA B and ASIA C, those with less severe injury, in which there is some preservation of sensory or motor function. In addition to assessing safety, the trial will assess preliminary efficacy based on defined clinical endpoints, such as changes in sensation, motor and bowel/bladder function.
All patients will receive HuCNS-SC cells through direct transplantation into the spinal cord and will be temporarily immunosuppressed. Patients will be evaluated regularly in the post-transplant period in order to monitor and assess the safety of the HuCNS-SC cells, the surgery and the immunosuppression, as well as to measure any recovery of neurological function below the injury site. The Company intends to follow the effects of this therapy long-term, and a separate 4-year observational study will be initiated at the conclusion of this trial.
According to a study reported by the Christopher and Dana Reeve Foundation, nearly 1.3 million people in the United States are estimated to be living with chronic spinal cord injury. The chronic phase of spinal cord injury is considered to begin when inflammation has stabilized and recovery has reached a plateau, which is typically several months following injury. Currently, there are no effective treatment options for patients with chronic spinal cord injury, and treatment approaches have generally targeted the acute and sub-acute time points, which are within hours or days of injury. Given the unmet need in chronic spinal cord injury, restoring some degree of function for patients at time points beyond the acute phase could have a transformative impact on the field. StemCells hopes to address a broad population of spinal cord-injured patients by opening the window of opportunity for therapeutic intervention well after the acute injury and targeting a wide range of injury levels and degrees of impairment.
Balgrist University Hospital, University of Zurich is recognized worldwide as a highly specialized centre of excellence providing examination, treatment and rehabilitation opportunities to patients with serious musculoskeletal conditions.
StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery.
“You Have the Power to Donate Life – Sign-up today! to become an organ and tissue donor
Tell Your Loved Ones of Your Decision”
Tell Your Loved Ones of Your Decision”
Australia, register at Australian Organ Donor Register
New Zealand, register at Organ Donation New Zealand
South Africa, http://www.odf.org.za/
United States, organdonor.gov
United Kingdom, register at NHS Organ Donor Register
Your generosity can save or enhance the lives of up to fifty people with heart, kidneys, liver, lungs, pancreas and small intestine transplants (see allotransplantation). One tissue donor can help by donating skin, corneas, bone, tendon, ligaments and heart valves
Has your life been saved by an organ transplant? "Pay it forward" and help spread the word about the need for organ donation - In the U.S. another person is added to the national transplant waiting list every 11 minutes and 18 people die each day waiting for an organ or tissue transplant. Organs can save lives, corneas renew vision, and tissue may help to restore someone's ability to walk, run or move freely without pain. Life Begins with You