MIPS Project Detail:
Company
Company Description:
Cellphire Inc. develops stabilized platelets for advanced wound care, transfusion, and reagent products for clinical and research diagnostics. The company has stabilized and modified platelets into a hemostatic agent that can be stored well beyond the current limit of five days. Platelets cannot be stockpiled and shortages occur because of this extremely short shelf life. These shortages can interrupt the treatment of cancer patients and actively bleeding surgical or trauma patients.
Cellphire’s first platelet stabilization product is Thrombosome®™, an easily rehydrated platelet product in Phase 1 clinical study for use as a potential agent in radiation remediation. The NB work is being funded by the Biomedical Advanced Research and Development Authority, part of the Office of the Assistant Secretary of Preparedness and Response in the U.S. Department of Health and Human Services. Cellphire, through use of its own funds and IP, is also developing other applications and has been intrigued by the concept of utilizing stabilized platelets as drug delivery vehicles.
MIPS Project
Loaded platelets for treatment of brain trauma
Project #
5530.27
|
MIPS Round
57
|
Starting Date:
Feb 2016
MIPS Project Challenge:
The broad goal of this two-phase MIPS project was to investigate the efficacy of Cellphire’s platelet technology for delivering therapeutics to the site of traumatic brain injury (TBI).
Project Scope:
Researchers engaged in this project planned to determine, in vivo, if platelets loaded with preferred antiapoptotic miRNA constructs could travel to sites of brain hemorrhage, release microparticles containing specific miRNAs, and reduce the cell death associated with TBI, effectively serving as a drug delivery device for targeted genomic therapy in patients with TBI. As proposed in this work, miRNA-loaded platelets stabilized by freeze-drying would be a highly attractive for a drug delivery method with great clinical utility.
Results:
Overall, these mechanistic in vivo experiments investigated whether the systemic (intravenous) administration of human platelets, or Thrombosomes®, containing miR-23a, may be able to transfer (following their recruitment and activation at the injury site) the loaded miR as an active molecule into cortical neurons following brain trauma, reducing the decline in miR-23a levels, inhibiting dependent cell death pathways, and ultimately attenuating neurological deficits after TBI.
Phase 1 defined conditions for platelet loading (miR-23a) and activation. It also investigated whether preventing miR-23a declines using microvesicle-rich fraction after platelet activation attenuates the activation of BH3-only proteins and decreases neuronal cell death in vitro. Phase 2 investigated whether preventing miR-23a declines with the intravenous administration of human platelets, or Thrombosomes® containing miR-23a, attenuates trauma-induced miR-23a downregulation, activation of BH3- only proteins, and/or improves neurological outcomes after TBI in vivo. Overall, the proposed studies have established the foundation for the future use of company-generated Thrombosomes® as carriers for molecular modulators in clinical studies focusing on therapeutic applications in traumatic brain injury and beyond.
Principal Investigator:
Bogdan
Stoica
Associate Professor of Anesthesiology, University of Maryland School of Medicine
Project Manager:
Daniel
Johnston
Director of Non-Clinical R&D
Technologies:
Biotechnology / Genetic Engineering
