Carroll J.E.,Georgia Regents University |
Expert Opinion on Biological Therapy | Year: 2011
Introduction: Due to the publicity about stem cell transplantation for the treatment of cerebral palsy, many families seek information on treatment, and many travel overseas for cell transplantation. Even so, there is little scientific confirmation of benefit, and therefore existing knowledge in the field must be summarized. Areas covered: This paper addresses the clinical protocols examining the problem, types of stem cells available for transplant, experimental models used to test the benefit of the cells, possible mechanisms of action, potential complications of cell treatment and what is needed in the field to help accelerate cell-based therapies. Expert opinion: While stem cells may be beneficial in acute injuries of the CNS the biology of stem cells is not well enough understood in chronic injuries or disorders such as cerebral palsy. More work is required at the basic level of stem cell biology, in the development of animal models, and finally in well-conceived clinical trials. © 2011 Informa UK, Ltd. Source
Agency: Department of Health and Human Services | Branch: | Program: STTR | Phase: Phase I | Award Amount: 224.72K | Year: 2014
DESCRIPTION (provided by applicant): This is a resubmission of a Phase I-Phase II Fast-Track application for the clinical use of MultiStem in patients with acute respiratory distress syndrome (ARDS). ARDS is defined as acute onset hypoxemia, bilateral radiographic pulmonary infiltrates and lack of atrial pressure hypertension. A novel and exiting possibility is the use of cells as part of the therapy in lung injury. We and other groups have demonstrated that exogenous infusion of isolated mesenchymal stem cells (B-MSC) prevents inflammation and aberrant repair after lung injury. These and other observations suggest that B-MSC is a potentially safe and effective therapeutic intervention in lung injury. Progress toward B-MSC as a cell therapy for ARDS in humans requires completion of preclinical studies and validation in animal models. We propose to evaluate the therapeutic effect of a GMP-produced human adherent bone marrow derived stem cell (MultiStem) in a sheep model of endotoxin-induced moderate-sever
Agency: Department of Health and Human Services | Branch: National Institutes of Health | Program: SBIR | Phase: Phase I | Award Amount: 212.80K | Year: 2015
DESCRIPTION provided by applicant One out of every fifty people living in the United States suffers from some form of paralysis a total of almost million Americans This proposal outlines the translational development of a cellular therapeutic already in clinical trials with a novel regenerative peptide to be used in combination for treatment of spinal cord injury SCI and associated dysfunction Preclinical data from Athersys has demonstrated that acute intravenous administration of bone marrow derived multipotent adult progenitor cells MAPC provides immediate and durable neuroprotection after SCI Benefit to locomotor recovery was seen beginning at one week post injury as well as significant sparing of white matter tracts Dr Silver and his colleagues have identified the glial scar as a significant impediment to long distance regeneration specifically inhibitory molecules known as chondroitin sulfate proteoglycans CSPGs After discovering the receptor and signaling pathways associated with this pathway Dr Silverandapos s laboratory has developed a novel peptide Intracellular Sigma Peptide ISP which modulates the key receptor in this pathway In a preclinical study long term subcutaneous treatment with this peptide initiated in the acute phase following spinal cord injury allowed for delayed recovery of locomotion and bladder behaviors following SCI Interestingly while ISP treatment resulted in recovery it had no neuroprotective effects In the month time frame of this Phase I project we propose to combine the use of MAPC with ISP treatment following a translationally relevant rodent model of contusive SCI with the therapeutic aims of i promoting enhanced neuroprotection and ii enhancing sprouting regeneration by altering the response of regenerating axons to inhibitory extracellular matrix We will evaluate the synergistic effects of treatment by analyzing improvements in motor hind limb recovery return of coordinated control of the lower urinary tract and quantification of neuronal sprouting regeneration Dr Silver and collaborators have been investigating the ability of ISP peptide to induce sympathetic neural regeneration and alleviate arrhythmia following myocardial ischemia reperfusion injury This data suggests that ISP could also be utilized in other disorders in which CSPGs play a critical inhibitory role such as traumatic brain injury multiple sclerosis and stroke The fact that MAPC has already proven efficacious in preclinical models of acute myocardial infarction multiple sclerosis traumatic brain injury and stroke dramatically expands the impact that this combinatorial therapy could have in the field of regenerative medicine If MAPC and ISP administration can be shown to be effective in ameliorating SCI associated deficits and dysfunction this therapy could relieve some of the economic burden on the direct and indirect costs associated with SCI related care and more importantly provide meaningful improvement to SCI patients Both treatments are non invasive and systemic making them highly attractive therapeutic options for clinical use PUBLIC HEALTH RELEVANCE This proposal outlines the translational development of a cellular therapeutic already in clinical trials with a novel regenerative peptide to be used in synergy for treatment of contusive spinal cord injury SCI and associated dysfunction We propose to test the combined use of two non invasive and systemic therapeutics in a rodent model with the therapeutic aims of i promoting enhanced neuroprotection and ii enhancing regeneration If successful this combinatorial therapy could relieve a significant portion of the economic burden on the direct and indirect costs associated with SCI related care and more importantly provide meaningful improvement to SCI patients
Athersys | Date: 2012-06-18
The present invention generally relates to a series of compounds, to pharmaceutical compositions containing the compounds, and to use of the compounds and compositions as therapeutic agents. More specifically, compounds of the present invention are tricyclic indeno-pyrrole compounds. These compounds are serotonin receptor (5-HT) ligands and are useful for treating diseases, disorders, and conditions wherein modulation of the activity of serotonin receptors (5-HT) is desired (e.g. anxiety, depression and obesity).
Agency: GTR | Branch: Innovate UK | Program: | Phase: Collaborative Research & Development | Award Amount: 2.00M | Year: 2015
MUST-ARDS (MultiStem® for the Treatment of Acute Respiratory Distress Syndrome) is a phase 1/2 clinical trial designed to investigate a novel adult cell therapy for ARDS. MultiStem is an allogeneic, adult, multipotent progenitor stem cell that is currently being investigated in phase 2 clinical trials to treat ischemic stroke and ulcerative colitis. Athersys, Ltd., a SME and clinical stage biotechnology company, has been developing the MultiStem cell therapy platform for multiple disease indications. MultiStem cells exhibit a drug-like profile in that they act primarily through the production of multiple factors that regulate the immune system, protect damaged or injured tissue, promote tissue repair and healing and enhance the formation of new blood vessels in regions of ischemic injury. Athersys and the Cell Therapy Catapult through MUST-ARDS determine if MultiStem can reduce mortality, shorten hospital stay and improve patient outcomes for those suffering from ARDS.