In addition to ischemic stroke, Athersys has established collaborative relationships with independent investigators that are evaluating the potential application for MultiStem to treat a range of other neurological indications, including Traumatic Brain Injury (TBI), neonatal hypoxic ischemia, spinal cord injury, multiple sclerosis (MS), and Parkinson’s disease.
Traumatic brain injury is the cause for more than 50,000 deaths in the U.S. each year, according to the U.S. Centers for Disease Control (CDC). The CDC also estimates that there are 275,000 hospitalizations for moderate to severe traumatic brain injury and nearly 1.4 million emergency room visits for mild traumatic brain injuries each year, and accounts for almost a third of all emergency room visits. These injuries yield significant direct and indirect medical costs - an estimated $60 billion in the U.S. alone, according to the CDC's website.
In September 2010, collaborators from the University of Texas Houston Medical center published results in the journal Experimental Neurology demonstrating that administration of MultiStem following traumatic brain injury reduced tissue damage and helped preserve the blood brain barrier. The paper, "Intravenous multipotent adult progenitor cell therapy for traumatic brain injury: Preserving the blood brain barrier via an interaction with splenocytes," describes a series of preclinical in vivo and in vitro experiments evaluating the effect of intravenous injection of MultiStem on neurovascular protection after traumatic brain injury. Traumatic brain injury causes a reduction in splenic mass that correlates with an increase in circulating immune cells that subsequently leads to increased blood brain barrier permeability, thereby worsening the neurological deficit associated with the injury. As outlined in the publication, the findings showed that the intravenous injection of MultiStem preserved splenic mass and the integrity of the blood brain barrier, which in turn, can potentially reduce the neurobehavioral deficit associated with traumatic brain injury.
On February 1, 2012 we announced that we were awarded a SBIR Fast-Track grant of up to $1.9 million from the National Institute of Neurological Disorders and Stroke (NINDS) to develop MultiStem for the treatment of traumatic brain injury (TBI).
According to the Christopher & Dana Reeve Foundation, there are currently more than one million people in the United States living with spinal cord injury. Most patients that suffer spinal cord injury are between the ages of 15 and 35. The long term cost of spinal cord damage is estimated to range from $500,000 to more than $3 million per patient, depending on the severity of the injury.
In another neurologic breakthrough, a preclinical study published in The Journal of Neuroscience in January 2011 demonstrated the regenerative benefit of MultiStem therapy after spinal cord injury. This was the first time administration of an adult stem cell therapy has been shown to be capable of modifying multiple aspects of the wound response following a spinal cord injury, while simultaneously altering the inflammatory response to mitigate secondary injury in the central nervous system and increasing the regenerative potential of the damaged neurons themselves. The study, conducted by leading researchers from the Department of Neurosciences at the CWRU School of Medicine and scientists at Athersys, presented data supporting the potential therapeutic benefit of MultiStem program for spinal cord injury. Researchers observed that administration of Multipotent Adult Progenitor Cells (MAPC) following spinal cord injury in rodent models prevented the retraction of neurons, a process referred to as "axonal dieback," reduced inflammation in the region of injury, and also promoted the regrowth of neurons.
In June, 2010 we announced that we and collaborators at the Center for Stem Cell and Regenerative Medicine (CSCRM) and Case Western Reserve University (CWRU), had been awarded $1 million through the Ohio Third Frontier Biomedical Program to support research into the treatment of spinal cord injury (SCI) with MultiStem(R), Athersys' proprietary stem cell product candidate. The project is intended to advance preclinical and translational research involving the use of MultiStem as a treatment for damage associated with SCI.
There are approximately 4.25 million infants born in the United States each year. A lack or disruption of oxygen during birth, (also referred to as neonatal hypoxic ischemia, or perinatal asphyxia) occurs in approximately 1 in 200 live births, impacting an estimated 21,000 infants per year. While many infants do not experience serious disabilities as a result of temporary ischemia, more serious cases can result in significant, life-long disability. Once serious hypoxic–ischemic (HI) injury occurs at birth, the resulting brain damage lasts throughout the individual’s lifetime, and no effective treatments are currently available.
Athersys and investigators at the Medical College of Georgia have collaborated to investigate the potential application of MAPC’s toward the treatment of neonatal hypoxic ischemia. In published preclinical studies, researchers have shown that animals that received MAPCs administered intravenously (or delivered via intracerebral administration) exhibited significantly improved motor and neurologic scores compared with those that received vehicle infusion alone. We intend to continue to evaluate the potential application of MultiStem for treating neonatal hypoxic ischemia, and a range of other ischemic injuries.
We are also evaluating the potential application of MultiStem toward the treatment of other forms of neurological injury and disease, including Multiple Sclerosis (MS) and Parkinson’s Disease (PD). MS is an inflammatory disease affecting the brain and central nervous system, in which the myelin sheaths surrounding the axons of the brain and spinal cord are damaged, leading to demyelination and scarring. The symptoms of MS are variable, but can include acute or chronic pain, muscle weakness, significant loss of motor skills, cognitive impairment, visual problems, and other neurological dysfunction. According to Reuter’s Business Insights analysis, MS currently affects an estimated 450,000 - 565,000 individuals in the U.S., and an estimated 2.5 million people worldwide. MS is typically characterized as one of four types: progressive-relapsing, secondary progressive, primary progressive, or relapsing-remitting. There are several therapies available to treat relapsing-remitting MS that are considered to be moderately effective, however, effective treatment of the progressive-relapsing and secondary-progressive forms of the disease has proven more challenging. There are no therapies currently available to treat primary progressive MS.
In October, 2011 we announced that we had been awarded funding from Fast Forward, LLC, a nonprofit subsidiary of the National Multiple Sclerosis Society, to fund the development of Athersys' MultiStem® adult stem cell platform for the treatment of Multiple Sclerosis (MS), including treatment of chronic progressive forms of the disease. The goal of the alliance is to accelerate the clinical application of MultiStem for patients diagnosed with MS. Fast Forward will commit up to $640,000 to fund the advancement of the program to clinical development stage.
PD is one of the most common neurodegenerative diseases, affecting approximately 650,000 individuals in the United States, and an estimated 1.5 million people in the U.S., core European markets and Japan, and approximately 4 million people globally. PD is caused by the progressive loss of dopaminergic neurons in specific regions of the brain, such as the substantia nigra. The major symptoms of the disease include tremors, suppressed voluntary movements, impaired balance and coordination, impaired memory, dementia and depression. Current therapies for PD include the administration of levodopa or other pharmaceutical agents, or surgical intervention (e.g. deep brain stimulation). These approaches are primarily designed to manage symptoms of the disease, however there is no cure for the disease currently available. Athersys has received a grant from the Michael J. Fox Foundation to study MultiStem as a potential treatment for PD.
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