Genetics, Clinical Trials, and Gene Therapy
Family Decision Making
By Candace Lindow-Davies, H&V Headquarters
Making decisions for our children who are deaf and hard of hearing requires an incredible amount of research, education, networking, resources, and time. With all the decisions families often face early on in their child’s life, it’s no wonder parents can feel overwhelmed and need support.
Like any other choice, parents will need information that allows them to understand options to make a truly informed decision.
One newer decision heading toward parents comes from the field of potential gene therapy focused on improving hearing in children. Clinical trials are rapidly developing for gene therapy that targets a specific type of auditory neuropathy caused by the gene otoferlin. Like any other choice, parents will need information that allows them to understand options to make a truly informed decision.
Global Genes, an advocacy organization committed to helping individuals and families find information about rare conditions, dove deep into the topic in their article, “Making Informed and Shared Decisions About Genetic Testing and Clinical Trial Participation.” They introduced the Ottawa Personal Decision Guide. This Guide was designed to walk families step-by-step through:
- Exploring each option’s benefits and risks
- Understanding what matters most to the decision-maker
- Discovering where to turn for information, support and advice
- Responding to feeling pressured by someone to choose
- Taking action if you’re uncertain about your decision
Especially relevant for families in the Global Genes’ article addressing unique considerations of families making decisions for a child and the American Medical Association’s (AMA) recommendations for Pediatric Decision-Making. These considerations, plus the discussion and resources for understanding gene therapy and considerations for participating in clinical trials, are well worth reading.
Another resource for families is the handout, “Evaluating the Clinical Trial Option: Questions to Consider” by the Courageous Parents Network. This tool empowers parents by providing a series of questions to ask before consenting to participate and offers definition of frequently used terms, such as placebo, phase, informed consent, and clinical benefit. Among factors to consider are the impact on a child’s health, quality of time and time commitment, emotional impact, and the risk of waiting and risk of participation?
Perhaps most important of all is having open dialogue with the professionals already involved in our child’s care and to put clinical trials in context to the other efforts to maximize our child’s potential for growth in all areas of their life. Dr. Dylan Chan, MD, PhD, FAAP, Associate Professor, Pediatric Otolaryngology-Head and Neck Surgery, Director, Children’s Communication Center, University of California, San Francisco finds it helpful to approach conversations about clinical trials with families focusing on language needs and communication as the core, and all other topics are secondary. “Gene therapy for otoferlin has great potential and may be on the near horizon. For now, providing your child’s brain with a strong foundation in language through whatever means are available and appropriate now, including using hearing aids, cochlear implants, and visual language, will be the best way to set them up for whatever advances the future holds, including gene therapy.” ~