29 Jan Gene Therapy Restores Hearing In Trial of Autosomal Recessive Pediatric Deafness
Posted at 00:00h
in Author Interviews, Brigham & Women's - Harvard, Genetic Research, Hearing Loss, Lancet, Pediatrics
MedicalResearch.com Interview with:
[caption id="attachment_61287" align="alignleft" width="180"] Dr. Zheng-Yi Chen[/caption]
Zheng-Yi Chen, D.Phil.
Department of Otolaryngology-Head and Neck Surger
Harvard Medical School
Boston, MA
MedicalResearch.com: What is the background for this study? Would you briefly explain the process and indication
Response: This clinical trial is to use gene therapy to treat a type of genetic hearing loss. Genetic hearing loss mainly affects children. One in 600 newborns can have genetic hearing loss. There is no drug treatment for any type of hearing loss except for cochlear implants, which have limitations. This study focuses on a type of genetic hearing loss, DFNB9, due to a missing gene called Otoferlin. Without Otoferlin, children are born with complete hearing loss and without the capacity to speak. The goal of the trial is to study if gene therapy is safe and efficacious in treating children so they can regain hearing and the ability to speak.
Dr. Li Li[/caption]
Li Li, M.D., Ph.D., M.P.H
Walter M. Seward Professor
Chair of Family Medicine
Director of population health
University of Virginia School of Medicine
Editor-in-chief of The BMJ Family Medicine
Dr. Li joined the U.S. Preventive Services Task Force in January 2021
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Speech and language delays and disorders can be challenging for children and their families and can lead to difficulties with reading and writing as children grow up.
The Task Force looked at the evidence on screening for speech and language delays and, unfortunately, there is not enough evidence to tell us whether or not it is helpful to screen all children 5 years old and younger for speech and language delays and disorders.
Dr. Di Ciano[/caption]
Patricia Di Ciano, PhD
Lauren C. Davis, MBS
Department of Medical Education
Geisinger Commonwealth School of Medicine
Scranton, PA 19409
MedicalResearch.com: What is the background for this study?
Response: Financial conflicts of interest (COIs) resulting from ties between academia and industry have been under scrutiny for their potential to hinder the integrity of medical research. COIs can lead to implicit bias, compromise the research process, and erode public trust (1-6). The American Psychiatric Association’s Diagnostic and Statistical Manual of Mental Disorders (DSM), standardizes symptom criteria and codifies psychiatric disorders. This manual contributes to the approval of new drugs, extensions of patent exclusivity, and can influence payers and mental health professionals seeking third-party reimbursements. Given the implications of the DSM on public health, it is paramount that it is free of industry influence. Previous research has shown a high prevalence of industry ties among panel and task force members of the DSM-IV-TR and DSM-5, despite the implementation of a disclosure policy for the DSM-5 (7,8). This study (9) determined the extent and type of COIs received by panel and task-force members of the DSM-5-TR (2022) (10). As the DSM-5-TR did not disclose COI, we used the Center for Medicare and Medicaid Services Open Payments (OP) database (11) to quantify them.
Scott Kaplan PhD
Assistant Professor of Economics
United States Naval Academy
Annapolis, MD 21402
MedicalResearch.com: What is the background for this study?
Response: Sugar-sweetened beverages (colloquially known as SSBs), which include sodas, fruit drinks, sports drinks, energy drinks, and sweetened coffee drinks, are the leading source of added sugars in the American diet, according to the CDC. They are associated with serious negative health outcomes, including type 2 diabetes, obesity, heart disease, kidney disease, 
Kalli Koukounas[/caption]
Kalli Koukounas, MPH
Dr. Lu Qi[/caption]
Lu Qi, MD, PhD, FAHA
Interim Chair, Department of Epidemiolog
HCA Regents Distinguished Chair and Professor
Tulane University School of Public Health and Tropical Medicine
Director, Tulane University Obesity Research Center
Director, Tulane Personalized Health Institute
New Orleans, LA 70112
MedicalResearch.com: What is the background for this study?
Response: Adding salt to foods is a behavior reflecting long-term preference to salty diets. High sodium intake is a major risk factor for chronic kidney disease.
In our previous studies, we have found that adding salt to foods at the table is related to various disorders including cardiovascular diseases, diabetes, and mortality. 
Amy Kennalley[/caption]
Amy Kennalley, MBS
First Year Medical Student
Department of Medical Education
Geisinger Commonwealth School of Medicine
MedicalResearch.com: What is the background for this study?
Response: Opioid use disorder (OUD) is a major public health crisis in the United States. Despite the availability of effective treatments, including the medication-assisted treatment (MAT) with methadone, many individuals with OUD do not receive the care they need. Over the COVID-19 pandemic, there were several policy changes related to take-home doses of methadone, which may have impacted access to this lifesaving medication.
The Drug Enforcement Administration's (DEA)'s Automated Reports and Consolidated Ordering System (ARCOS) is a database that tracks the distribution of controlled substances, including methadone distributed to opioid treatment programs (OTPs). This data can be used to assess trends in methadone distribution over time. Additionally, the Medicaid State Drug Utilization Data (SDUD) database provides comprehensive information on methadone prescribing to Medicaid patients.
By analyzing data from both ARCOS and SDUD, we aimed to provide a comprehensive picture of methadone distribution for OUD treatment in the United States. These findings1 are important because drug overdose deaths, primarily involving opioids, increased substantially (49%) from 2019 (70,980) to 2021 (~106,000).2
Dr. Klompas[/caption]
Michael Klompas MD, MPH, FIDSA, FSHEA
Hospital Epidemiologist
Brigham and Women’s Hospital
Professor of Medicine and Population Medicine
Harvard Medical School and
Harvard Pilgrim Health Care Institute
MedicalResearch.com: What is the background for this study? Can teeth be safely brush in patients who are comatose, intubated or have NG tubes?
Response: Pneumonia is thought to occur when secretions from the mouth get into the lungs. Since there are many microbes in the mouth, there’s a risk that secretions from the mouth that get into the lungs will lead to pneumonia. Toothbrushing may lower this risk by decreasing the quantity of microbes in the mouth.
It is indeed safe and appropriate to brush the teeth of someone who is comatose, intubated, or who has an NG tube. Indeed, our study found that the benefits of toothbrushing were clearest for patients receiving mechanical ventilation.
Dr. Angélica Cifuentes Kottkamp[/caption]
Angélica
Dr. Brousseau[/caption]
David Brousseau, MD, MS
Chair of Pediatrics
Nemours Children’s Health, Delaware and the
Sidney Kimmel Medical College at Thomas Jefferson University
