There are a lot of ways to promote healthy aging. Lifestyle factors and access to quality care certainly play a role. Other keys to aging are found in our cells and genes. At the UW Institute for Stem Cell and Regenerative Medicine (ISCRM), researchers are making discoveries that could lead to new treatments for diseases that can impair mobility, activity, memory, vision, hearing, and other abilities that are important for independence and wellbeing, particularly for older adults.
Many of these treatments may fall under the category of regenerative medicine, which means addressing the underlying causes of symptoms through emerging approaches like cell therapy or gene editing. Imagine being able to replace scar tissue in the heart with healthy muscle to restore normal function, or being able to protect neuronal health in the brain before Alzheimer’s disease takes hold.
ISCRM researchers are also looking at the machinery within our cells for whole-body strategies to help us age gracefully.
Meet the Mitochondrial All-Stars
Perhaps no structure in the human body has a nickname as firmly fixed in our minds as mitochondria, the organelles known to generations of students as the “powerhouses of the cell.”
Mitochondria earned that nickname because they convert nutrients into ATP, the energy cells use to carry out most of their work. But energy production is only part of the story. Mitochondria also help cells respond to stress, regulate metabolism and inflammation, control when damaged cells die, and send signals that shape cellular activity.
When mitochondria become damaged or less efficient, the effects can ripple throughout the body. Mitochondrial dysfunction has been linked to conditions including heart and neurodegenerative diseases, as well as to biological changes associated with aging.
Dr. David Marcinek is a University of Washington professor of radiology and laboratory medicine and pathology and an ISCRM faculty member. For much of his career, Marcinek has studied the relationship between mitochondria and aging, primarily in skeletal muscle and, more recently, in the heart, kidney, and brain.
In one study, Marcinek and Dr. Kevin Conley used magnetic resonance measurements to show that a single dose of a drug now known as elamipretide enhanced mitochondrial performance in older adults.
Elamipretide was developed by Stealth BioTherapeutics, now Mighty Therapeutics, a Massachusetts-based company focused on therapies for mitochondrial diseases. In 2025, the FDA granted accelerated approval for the treatment of muscle weakness in patients weighing at least 30 kg with Barth syndrome, a rare inherited disorder that disrupts mitochondria and can cause heart and muscle weakness.
Although elamipretide was developed to treat mitochondrial disease, the way it works has also made it a promising tool for studying the effects of aging.
“What’s really interesting about this drug is that it works through a structural interaction with the mitochondria,” Marcinek says. “Aging leads to a breakdown in the health of the mitochondria. We think elamipretide helps mitochondria work more efficiently by stabilizing the structure of the membrane, where much of the cell’s energy is produced.”
Over the past 15 years, Marcinek and his collaborators at Mighty Therapeutics have explored whether elamipretide might also support healthier aging. Now, a major competitive award will allow them to put that idea to a much larger test.
Launched in November 2023, XPRIZE Healthspan is a $101 million global competition administered by the nonprofit XPRIZE Foundation. Its goal is to develop and clinically test treatments capable of restoring muscle, cognitive, and immune performance in older adults by the equivalent of at least 10 years, with a stretch goal of 20 years.
In 2025, Mighty Therapeutics and Marcinek were selected as semi-finalists in the first round of the competition and received $250,000 for a pilot study that produced encouraging results. In August 2026, their team — dubbed the Mitochondrial All-Stars — learned it would receive an additional $1 million as one of 10 Milestone 2 Awardees. The funding will support a rigorous, placebo-controlled clinical trial scheduled to run through 2029.
The project leaders described the significance of the award in an August 2026 press release issued by Mighty Therapeutics.
“Our previous research supports the view that mitochondria are a key link between the molecular drivers of aging and age-related pathology leading to reduced quality of life,” said Marcinek. “With these feasibility data in hand, we can now move forward to design a gold standard, randomized, double-blind, placebo-controlled study to test whether targeting mitochondria with elamipretide enhances healthy aging by improving muscle, cognitive, and immune function in older adults.”
“This recognition from XPRIZE Healthspan affirms what we’ve believed since our earliest research: that targeting mitochondrial energetics is one of the most powerful levers we have in medicine,” said David A. Brown, Ph.D, Chief Scientific Officer at Mighty Therapeutics. “Impaired cellular energy production underlies every disease we seek to treat. Whether it presents early in life as a rare genetic condition or emerges decades later as a hallmark of aging, mitochondrial dysfunction is a central driver across pathologies. That view is what led us here, and this study led by Dr. Marcinek and his team is a meaningful step in extending mitochondrial research to advance healthy aging.”
The Connection to the Clinic
Because XPRIZE Healthspan aims to address muscle, cognitive, and immune health at the same time, the effort requires expertise across several fields. With that in mind, Marcinek turned to his ISCRM colleague Suman Jayadev, MD, who studies disruptions in cellular processes that contribute to neurodegenerative diseases and cares for patients facing challenges with brain health.
“As a specialist in cognition and a neurodegenerative disease clinician who works with older adults, Suman brings neuroscience expertise and experience with both patient care and clinical trials,” Marcinek says. “That gives our team an important translational dimension.”
Dr. Jayadev will help guide the clinical evaluation of participants, including identifying the most meaningful measures of overall health and physical performance. Working alongside colleagues analyzing molecular and physiological changes, she will also help assess how participants respond through a range of blood and other fluid collections, cognitive tests, and strength assessments. Because the study is still at an early stage, the goal is not only to detect changes in this study, but also to determine which measures are most useful for evaluating how elamipretide may affect health over time.
“Trying to bring potential therapies to patients has been incredibly challenging with brain-related disorders,” says Jayadev. “This project is an opportunity to follow the thread from the origins of the problem to the clinic, and to focus on maintaining health by nurturing resilience starting at the cellular level, even for patients who may have a brain disease already. Could this compound not only help with normal aging, but also give a metabolic boost to neurons that are exposed to disease risk factors, like amyloid plaque buildup? That’s what we’re excited to find out.”
Jayadev also emphasizes the importance of taking a holistic approach to healthy aging.
“The data have been showing us that there are direct connections between cardiac function and brain. We know that there is communication between the peripheral immune system and the brain immune system. And when we talk about resilience in the Alzheimer’s field, we’re talking about global systemic health and that is what influences how a disease or health manifests in the brain. I firmly believe that if we were to find a way to boost the health of the body, you would still absolutely see a benefit in the brain.”
With support from the XPRIZE award, the Mitochondrial All-Stars will conduct a Phase 2 double-blind, placebo-controlled clinical trial, in which neither participants nor researchers will know who receives elamipretide and who receives a placebo. The study will test whether one year of treatment can benefit muscle performance, cognition, and immune health in older adults.
Testing a single intervention across all three areas is central to the XPRIZE challenge — and closely aligned with Marcinek’s research.
“The hypothesis that drives a lot of the work in our lab is that as mitochondria age, their performance declines and cells experience more chronic stress,” Marcinek says. “That can make it harder for the body to respond to normal challenges, whether that means recovering from an injury, metabolizing a high-fat meal, or fighting off a virus. Our aim is to reduce that chronic stress and increase resilience in the muscles, brain, and immune system.”
Marcinek says another important aspect of XPRIZE Healthspan is the establishment of standardized criteria for measuring age-related changes across muscle, cognition, and immunity. Developed by a scientific advisory board, the measures are intended to give researchers and evaluators clearer and more consistent yardsticks for determining whether an intervention is making a meaningful difference.
Looking further ahead, Marcinek believes his affiliation with a stem cell institute could open another avenue for understanding how elamipretide works.
“If the drug is effective in clinical trials, we could potentially move into testing in 3D stem cell-derived models,” Marcinek says. “That could help us better understand what is happening in the mitochondria at the cellular level and identify the mechanisms contributing to positive health outcomes.”
Modeling Mitochondria in Stem Cell Models
Aging research is an important focus for other labs affiliated with ISCRM. Marcinek co-directs the University of Washington Healthy Aging and Longevity Research Institute with ISCRM faculty member Dr. Jessica Young, a Professor of Lab Medicine and Pathology. The Young Lab uses human induced pluripotent stem cells to study genetic risk factors for Alzheimer’s disease, in hopes of identifying potential therapeutic targets. Over many years, her team has shown that the loss of a gene known as SORL1 leads to debilitating neural traffic jams in brain cells, a process that happens many years before typical amyloid and Tau pathology develop, which sheds new light on why so many treatments targeting these later stage pathologies have hit dead ends in clinical trials.
In August, the lab received funding from the NIH to develop stem cell models of brain aging with Dr. Su-In Lee, the Boeing Endowed Professor in the Paul G. Allen School of Computer Science & Engineering. The team used single-cell RNA sequencing to map genes that are known to be drivers of aging. Chris Arian, a postdoctoral researcher in the Young Lab is now using CRISPR gene-editing technology to manipulate the expression of these genes. Among other readouts, he is looking closely at the relationship between mitochondrial function and dysfunction in cells that could affect brain health, analysis that will be supported by Dr. Jayadev. Young says the plan is to eventually use the model to test potential drugs, many focused on the mitochondria, that could someday move toward the clinic.
More recently, the Young Lab joined a multi-institutional consortium that will develop new tools to study links between mitochondrial breakdowns and Alzheimer’s disease. The group, which is led by University of Texas Health, will use longitudinal studies and biofluid samples to investigate sex differences in mitochondrial-driven metabolism. The research will also involve creating neurons from male and female patients to study these differences more directly and either suppressing genes involved in producing key metabolites or using small molecules that target mitochondrial function. The team will then assess mitochondrial activity using specialized assays that measure factors such as cellular respiration (Seahorse assays), membrane potential, and mitochondrial morphology.
Ultimately, the aging research supported through the XPRIZE competition and the kindred efforts to shed light on the mechanisms of Alzheimer’s disease using stem cells are fueled by a desire to turn discoveries in the lab into new treatments that improve wellbeing for people everywhere. Within ISCRM, Marcinek, Jayadev, and Young belong to a thriving, multidisciplinary community of scientists and physicians who are unlocking the secrets of our cells, and the components, like mitochondria, that allow them to function or cause them break down. Their goal: to confront the root causes of diseases affecting our hearts, kidneys, muscles, bones, and livers and to preserve quality of life as we age.