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Phase 2 clinical trial begins for stem cell therapy to treat aging frailty

Longeveron LLC, a regenerative medicine company developing cellular therapies, has treated its first patient in a Phase 2b clinical trial evaluating the safety and efficacy of its human allogeneic mesenchymal stem cells (LMSCs) in patients with aging frailty syndrome. Aging frailty is a common geriatric medical condition that is serious and life-threatening, and for which there are currently no U.S. Food and Drug Administration-approved therapeutics available.

The clinical trial is designed to enroll 120 subjects from approximately 10 medical centers around the United States. The primary objective of the study is to evaluate the effect that LMSCs have on functional mobility and exercise tolerance in aging frailty subjects. Three different LMSC dose groups will be compared to placebo over 12 months in a randomized, double-blinded, parallel arm design. 

This trial is being conducted pursuant to an Investigational New Drug Application (IND) in conformance with U.S. Food & Drug Administration (FDA) regulations. Specifically, it will evaluate changes to the following:

  • Timed walking distance, walking speed and ability to balance
  • Upper extremity strength
  • Fear and risk of falling
  • Patient-reported ability to perform activities of daily living
  • Cognitive function
  • Clinical events such as falls or hospitalizations
  • Level of inflammation within the body

Allogeneic mesenchymal stem cells (MSCs) were previously tested in a Phase I/2 proof-of-concept study conducted by investigators at the University of Miami's Miller School of Medicine.  In that study, MSCs were shown to be safe and well-tolerated in frail, elderly subjects in a Phase 1 open label single ascending dose trial with a similar safety profile observed in the randomized, placebo-controlled Phase 2 study. Subjects treated with a dose of 100 million MSCs showed significant improvements in six minute walking distance and significant decreases in systemic inflammation, both relative to baseline.

"As individuals age, stem cell production and proliferation decreases, systemic inflammation increases and a person's ability to repair and regenerate worn out or damaged tissue diminishes," said Suzanne Liv Page, Longeveron chief operating officer. "In frail individuals this is particularly problematic.  Our hypothesis is that exogenously infused allogeneic mesenchymal stem cells that are derived from the bone marrow of a healthy young donor, and culture expanded in our lab, will have potent regenerative and restorative effects."

Participants in this study must be between the ages of 70 and 85, be diagnosed as mildly to moderately frail due primarily to aging and be able to walk between 200 and 400 meters over six minutes. Detailed information about the trial, subject eligibility and participating centers can be found by visiting and entering trial ID: NCT03169231.

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