Duke University

“Expanded access, sometimes called “compassionate use”, is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.”

TEAMSS (Transforming Expanded Access to Maximize Support and Study) seeks to advance clinical care and translational research by improving patient access to experimental therapies. This federated, national consortium for Expanded Access interventions is a partnership led by the University of Michigan with Duke University, University of Rochester and University of Texas Southwestern. Expanded Access provides an opportunity for patients who either lack therapeutic options or who are ineligible for clinical trials to potentially benefit from the clinical use of experimental drugs, biologics, and medical devices.

### Expanded Access Protocol: Umbilical Cord Blood Infusions for Patients with Brain Injuries The primary purpose of this protocol is to enable infusions of sibling, autologous (a person’s own), or publicly banked umbilical cord blood for various brain disorders. This is not a research study, but rather an infusion that may be done while studies are ongoing to determine if cord blood infusion is effective in treating certain conditions. Patients up to age 26 with cerebral palsy, hydrocephalus, hypoxic brain injury, stroke, apraxia and other brain injuries may be eligible if they do not qualify for or are unable to participate in another active clinical trial at Duke.

Here we report our updated investigational experience with mesenchymal stromal cell (MSC) therapy with remestemcel-L in a multicenter expanded access protocol (ClinicalTrials.gov identifier NCT00759018) in 241 children with aGVHD who failed to respond to steroids with or without other secondary and tertiary immunosuppressive therapies.

Here we report our updated investigational experience with mesenchymal stromal cell (MSC) therapy with remestemcel-L in a multicenter expanded access protocol (ClinicalTrials.gov identifier NCT00759018) in 241 children with aGVHD who failed to respond to steroids with or without other secondary and tertiary immunosuppressive therapies.