With its potential to eliminate and prevent hereditary diseases like cystic fibrosis and hemophilia, gene therapy has the potential for never-before-seen medical breakthroughs. Despite this, there has not yet been a single approved human gene therapy product available to the US market. Nonetheless, the amount of gene therapy research and development continues to skyrocket.
I recently had the pleasure of attending the annual World Orphan Drug Congress (WODC) hosted by Terrapin at the Washington Marriott Wardman Park in Washington D.C. Together with more than 1,000 attendees from big pharma, biotech, payers, patient groups, academics and government bodies, we heard from over 150 industry speakers on many topics, but primarily the unique challenges of gene therapy trials.
Barbara Wuebvels, VP of Patient Advocacy at Audentes Therpeutics talked about the challenges in Enrolling (Subjects) in Gene Therapy Trials.” While she talked about the challenges that present with any rare or orphan disease trial, including small patient populations, limited number of trial sites, and the burden of traveling on subjects and their families, she also highlighted challenges specific to patients participating in gene therapy programs. These challenges often include extensive logistical issues, such as the need for wheelchairs, electric-powered chairs, ventilators, portable generators, pressure mattresses, photo therapy lights, and more. Additionally, principal investigators and site staff are generally unfamiliar with gene therapy. Many times patients and their families don’t have an understanding of the mechanism of gene therapy in general, making enrollment in these programs particularly challenging.
This year’s WODC highlighted not only the insurmountable benefits of getting an approved gene therapy to the market, but also the challenges that still need to be overcome with gene therapy programs. As part of the staff at Colpitts Clinical, where we strive to relieve the burden of clinical trial enrollment on patients, I found this year’s program to be particularly insightful and impactful. Despite the fact that we are not where we need to be in terms of breakthroughs in gene therapy, I feel more optimistic now more than ever that if we continue to collaborate as an industry we might someday soon see benefits of gene therapy research that could truly be life changing for patients living with rare diseases caused by faulty genes.
By: Courtney Topham