Allegheny Health Network to Participate in Study of Investigational Gene Therapy Treatment for Radiation-Induced Dry Mouth

Wednesday, August 28, 2024

Press Releases Allegheny General Hospital

PITTSBURGH - Allegheny Health Network (AHN) announced today its participation in a study to determine the efficacy of a gene therapy, known as AAV2-hAQP1, in participants with radiation-induced xerostomia, commonly known as chronic dry mouth. The network's flagship academic and research hub, Allegheny General Hospital (AGH), is one of 10 sites nationally - and the only in Western Pennsylvania - to participate in this phase 2 study.

While undergoing radiation treatment, patients with head and neck cancer are prone to damage in the irradiation-sensitive salivary gland tissue, leading to salivary gland hypofunction and xerostomia. Xerostomia is a condition in which the salivary glands in the mouth don't produce enough saliva to keep the mouth wet. Individuals with chronic dry mouth are more likely to develop cavities, gum disease and mouth sores among other oral complications.

More than 170,000 patients in the U.S. are believed to have long-term moderate to severe radiation-induced dry mouth. While over-the-counter agents including lozenges, gums and artificial saliva often provide short-term relief, there is only one FDA-treatment currently approved specifically for radiation-induced xerostomia.

This clinical study, sponsored by MeiraGTx, LLC., seeks to enroll up to 120 patients who have dry mouth three years after completing radiation therapy in addition to other criteria. It is a phase 2 study which seeks to evaluate the gene therapy effectiveness. The first phase confirmed the safety of the treatment.

As a randomized, double-blind, placebo-controlled study, some participants will receive a placebo, or inactive treatment, while others will receive the investigational treatment. For those who receive the treatment, the gene therapy is inserted into a gland in the patient's mouth. It is a one-time, outpatient procedure which may result in some temporary mild discomfort.

AGH began enrolling patients on May 1 and will do so through the end of this year. AHN otolaryngologist Warren Swegal, MD, is a principal investigator for the study.

"We are thrilled to be among the small number of health systems nationally who are participating in the study of this promising treatment for the many head and neck cancer survivors who are living with chronic dry mouth," said Dr. Swegal. "We look forward to the findings that this phase of the study will uncover relative to the effectiveness of this innovative gene therapy."

Physicians and researchers at AGH helped pioneer this treatment through preclinical studies and continue to work at the preclinical and clinical levels to bring bench to bedside treatments. As part of its gene therapy program, AHN has previously led research regarding the use of ultrasound technology to deliver therapeutic DNA to restore saliva flow for patients living with xerostomia.

Criteria for participation in this phase 2 study can be found here. Those who are interested in participating should call (412) 330-6151.

###