Trial of Anti-Aging Stem Cell Treatment Displays Impressive Results
University of Miami researchers have published the results of their clinical trials involving a stem cell transplant that could diminish the conditions of age-related frailty.
There is increasing recognition of the health burden of frailty, a syndrome that increases in incidence with aging. Frailty is a medical syndrome characterized by age-related diminished physiologic function, endurance, and strength that can be secondary to a multitude of factors and is associated with a high risk of hospitalizations, worse clinical outcomes, dependency, and mortality.
Of note, the frailty syndrome is driven mostly by biological aging processes that include inflammation and stem cell dysfunction, as opposed to chronological aging. Early intervention may improve quality of life, reduce hospitalizations, and nursing home costs. Therefore, it is increasingly important to recognize the clinical onset of frailty and to develop effective therapeutic strategies.
Therapeutic interventions for aging frailty have mainly focused on exercise, nutritional supplements, and multidisciplinary methods. The goal of any potential therapy for frailty is to extend the well-being and ability of a patient to regenerate functionality.
And now, scientists at the University of Miami have chosen an entirely different approach to treat aging frailty
- an anti-aging stem cell treatment.
Buoyed by promising results of a Phase I study demonstrating the safety of mesenchymal stem cell therapy to treat frailty in older adults, researchers at the Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami Miller School of Medicine have taken it a step further.
Now Phase II randomized, placebo-controlled trial findings have verified their initial study, boosting the potential for this stem cell therapy to reverse or forestall the symptoms of frailty. Not only is there no currently approved medical therapy for frailty, the need is expected to grow substantially given the overall aging of the U.S. population.
“It’s a huge need — that’s one of the exciting things,” said Joshua M. Hare, M.D., the Louis Lemberg Professor of Medicine and founding director of ISCI. Frailty is estimated to affect about 10 to 11 percent of seniors, a considerable percentage of the 50 million people older than 65 years experts predict by the year 2050.
Mesenchymal stem cells (MSCs) are a particular type of adult stem cell generating a great deal of interest in the world of science. MSCs are currently being trailed as the treatment for no less than a dozen different types of pathological conditions from cancer to heart disease.
This new MSC treatment is targeted at reducing the effects of frailty on senior citizens. This is the first anti-aging stem cell treatment directed specifically at the problem of age-associated frailty to move closer to a final FDA approval stage.
The treatment derives human mesenchymal stem cells from an adult donor bone marrow and in these clinical trials involves a single infusion in patients with an average age of 76. Both Phase 1 and Phase 2 human trials have demonstrated the treatment to have no adverse health effects. Although the two human trials were ostensibly designed to just demonstrate safety they do offer remarkable results in efficacy as well, paving the way for larger, Phase 3 clinical trials.
In the first trial, 15 frail patients received a single MSC infusion collected from bone marrow donors aged between 20 and 45 years old. Six months later all patients demonstrated improved fitness outcomes, tumor necrosis factor levels and overall quality of life.
The second trial was a randomized, double-blind study with a placebo group. Again no adverse effects were reported and physical improvements were noted by the researchers as “remarkable”.
The next stage of the research is to move into an expanded Phase 2b clinical trial involving 120 subjects across 10 locations. After that, a final, large randomized Phase 3 clinical trial will be the only thing holding the treatment back from final public approval.
“These trials represent potential landmarks in the treatment of frailty,” the authors write in the paper. “Both studies are early-phase trials of a small number of participants, designed primarily to assess safety, so conclusions about efficacy need to be treated with caution. Even so, the results are striking and, at a minimum, pave the way for large randomized Phase III clinical trials.”
“I was very surprised and excited with the results,” Hare said. Replicating their results in Phase II randomized trials “gives me even greater confidence” in the findings. For example, both trials demonstrated a significant and meaningful increase in six-minute walk tests among older adults with frailty compared to their baseline.
“The idea that we can biologically modify frailty is very exciting,” Hare said. In fact, the research by Hare and his team demonstrate the beneficial effects of mesenchymal stem cell therapy in this population, including their anti-inflammatory effects, pro-regenerative capabilities, and anti-fibrotic actions (meaning they can decrease scar tissue). The investigators have also shown the therapy promotes new vessel growth and improve endothelial function.
“Mesenchymal stem cells are a multifactorial way of offsetting many of the key features of aging,” Hare said.
“The Interdisciplinary Stem Cell Institute is dedicated to looking for novel therapies for difficult-to-treat problems,” Hare said. “The culture here allows us to do investigator-driven trials, and 2017 has been a great year so far for us, with four stem cell clinical trials — these two on aging and two others addressing cardiovascular disease — including the TRIDENT study evaluating stem cell therapy for ischemic cardiomyopathy.”
