An investigational cell therapy being evaluated at UC Davis Health has received fast-track designation from the U.S. Food and Drug Administration (FDA) to address swallowing difficulties in patients who have undergone treatment for head and neck cancer.
The therapy, iltamiocel, developed by Cook MyoSite Inc., has been granted Regenerative Medicine Advanced Therapy (RMAT) designation, a regulatory pathway designed to expedite the development of regenerative medicine therapies that demonstrate potential to address serious conditions with significant unmet medical needs.
"We are thrilled to receive an RMAT designation for this technology which could be a game-changer for our patients who suffer from difficulty swallowing, many of whom are cancer survivors living with the consequences of radiation toxicity," said Dr. Peter Belafsky, director of the UC Davis Health Center for Voice and Swallowing and principal investigator for the clinical trials.
How Iltamiocel Works
Iltamiocel represents a personalized approach to treating oropharyngeal dysphagia (OPD), a condition characterized by difficulty swallowing that commonly affects head and neck cancer survivors. The therapy involves collecting muscle tissue from the patient's quadriceps during an in-office procedure. These samples are then processed to create a concentrated, cryopreserved suspension of the patient's own skeletal muscle progenitor cells.
The processed cells are later injected into the patient's tongue, where researchers believe they will augment existing muscle fibers to increase tongue strength and improve swallowing function. This autologous approach minimizes the risk of rejection since the treatment uses the patient's own cells.
More than 700 participants have contributed to the therapy's clinical development program thus far, highlighting the substantial research effort behind this innovative treatment approach.
Clinical Evidence Supporting the Designation
The FDA's decision to grant RMAT designation was based in part on promising results from the Phase I/II TODY clinical trial. This open-label study assessed both the safety and efficacy of iltamiocel, providing preliminary evidence of the therapy's potential benefit.
Ryan Pruchnic, managing vice president of Cook MyoSite, noted, "The UC Davis Health Department of Otolaryngology has been focusing on bringing new treatments for patients with oropharyngeal dysphagia. We were grateful to partner with them to provide iltamiocel, our investigational autologous skeletal muscle progenitor cell therapy, for the treatment of OD. We're looking forward to continuing research and finding a new option for these patients."
Addressing a Significant Unmet Need
Dysphagia represents a substantial health burden in the United States, with approximately one in 25 adults experiencing swallowing problems each year. For head and neck cancer survivors, treatment-related damage to swallowing muscles can lead to severe complications.
"This is a struggling patient population that currently doesn't have many good options to help them," explained Dr. Belafsky. "They can experience dramatic deficits in both speech and swallowing that may lead to malnutrition, dehydration, social isolation, depression, aspiration pneumonia, pulmonary abscess and even death."
Currently, no effective medical therapies exist to improve swallowing function in these patients, making the development of iltamiocel particularly significant for this underserved population.
The REVIVE Clinical Trial
Building on earlier promising results, researchers have now completed enrollment for the Phase II REVIVE trial, a double-blind, randomized, placebo-controlled study involving 62 patients. This represents the largest trial of its kind investigating an interventional treatment for patients with post-head and neck cancer treatment OPD.
The clinical trial is supported by an $11 million grant from the California Institute for Regenerative Medicine (CIRM), an organization established to accelerate regenerative medicine research for unmet medical needs.
In the REVIVE study, participants receive two sets of iltamiocel injections into their tongue, spaced 4-6 weeks apart. Each participant undergoes comprehensive monitoring through eight in-office visits and seven virtual visits over a 26-month period.
"The RMAT designation is great news for the team at UC Davis Health and for patients affected by dysphagia," said Paul Webb, senior science officer and program manager of therapeutics development at CIRM. "The designation is a major achievement and showcases exactly the type of collaboration between investigators, institutions, and patients that CIRM seeks to encourage."
Future Implications
Dr. Jan Nolta, director of the Stem Cell Program at UC Davis Health, emphasized the broader significance of this work: "We are steadfast in our commitment to pioneering innovative cell therapies, advancing the frontier of medicine to transform countless lives and offering new hope to those in need. We are hopeful this work could lead to a new treatment of this ailment."
The RMAT designation represents a significant milestone not only for the researchers and institutions involved but also for the millions of patients suffering from swallowing disorders who may benefit from this novel therapeutic approach. If successful, iltamiocel could establish a new paradigm for treating dysphagia in cancer survivors and potentially other patient populations affected by swallowing difficulties.
