FDA Grants Novel Therapy RMAT Designation for Dysphagia Following Head and Neck Cancer Treatment

• Iltamiocel, an investigational cell therapy, has been granted regenerative medicine advanced therapy (RMAT) designation by the FDA in patients with oropharyngeal dysphagia (OPD), a common complication following treatment for head and neck cancer.
• The autologous cell therapy is an in-office procedure being developed by Cook MyoSite Inc. and evaluated at UC Davis Health.
• Over 700 patients have been included in the therapy’s clinical development so far.

The FDA has granted iltamiocel RMAT designation in patients with OPD, a complication often following head and neck cancer treatment.¹

“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 technology,” said Peter Belafsky, director of the UC Davis Health Center for Voice and Swallowing and principal investigator of clinical trials investigating iltamiocel, said in a press release.

Iltamiocel is a personalized, regenerative cell therapy where muscle tissue obtained from the patient’s quadriceps is concentrated and cryopreserved into a suspension that is then injected into the tongue. The procedure is done in office. Investigators believe that using existing muscle fibers can help increase patients’ tongue strength.

“This is a struggling patient population that currently doesn't have many good options to help them,” Belafsky added in the press release. “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.”

About the REVIVE Study of Iltamiocel

3D rendering of hematopoietic stem cells

Iltmiocel is being investigated in the phase 1/2 REVIVE study (NCT05421689).² A total of 62 patients are enrolled in a double-blind, randomized fashion. Patients in the experimental arm will receive 2 sets of iltamiocel injections 4 to 6 weeks apart.

The study’s primary end points are incidence of adverse events and efficacy of the agent in improving anterior tongue pressure. Secondary end points include incidence of patient aspiration pneumonia, peak pharyngeal pressure, and patient-reported dysphagia symptoms. Patient-reported outcomes will be assessed at 4 weeks, 3 months, 6 months, 12 months, 18 months, and 24 months.

Patients at least 18 years old with primary symptoms of OPD following surgery, chemotherapy, or radiotherapy for the treatment of oropharyngeal squamous cell carcinoma are eligible for enrollment in the study. Treatment must have been completed at least 24 months before study enrollment, and patients must have failed to achieve symptom resolution following contemporary therapies.

Those who have previously been treated with an investigational therapy within 6 months, have severe fibrosis at the injection site, uncontrolled diabetes, compromised immune system, or another non-skin cancer within the past 24 months are not eligible for participation in the study.

The study is ongoing at UC Davis Medical Center and UC San Francisco Medical Center and has an estimated completion date of December 31, 2026.

REFERENCES:

1. Novel cell therapy receives expedited FDA designation. News release. UC Davis Health. April 15, 2025. Accessed April 16, 2025. https://tinyurl.com/3372vus5

2. Autologous muscle derived cells for treatment of tongue dysphagia (REVIVE). ClinicalTrials.gov. Updated December 9, 2024. Accessed April 16, 2025. https://clinicaltrials.gov/study/NCT05421689