PRIM&R is pleased to welcome Patricia Furlong as a keynote speaker at the 2016 Advancing Ethical Research Conference to be held November 13-16, 2015 in Anaheim, CA. Furlong will speak on November 15 on the theme of “SUBJECTS” Matter: Burden of Participation for Children in Clinical Trials.
PRIM&R: How does your advocacy intersect with research ethics?
Patricia Furlong (PF): Parent Project Muscular Dystrophy (PPMD)’s goal is to serve the Duchenne community. In all we do, we consider issues of justice and fairness. Ethical issues cut across all of our programs and everything we do—our defining principle being “do no harm.”
PRIM&R: How has your educational and career background informed your pursuit of a cure for Duchenne?
PF: My early career focused on Intensive Care, Cardiac Care and Renal Dialysis, working closely with the transplant team. My mid-career (prior to my sons’ diagnoses) focused on patient education. This journey set the stage to enhance my knowledge of disease and its impact on the individual diagnosed, as well as the ripple effect on the family and extended family. Clearly I learned principles of diagnosis, critical thinking, and the need to understand the pathophysiology of diseases. For the last 22 years, I have focused on Duchenne. Applying my early experience, I have been able to understand key elements of progress, drug development, regulatory science, data collection, and how all of these elements contribute to progress.
PRIM&R: How would you explain the process of finding a cure for Duchenne to someone unfamiliar with it?
PF: Explaining the process of finding a cure for Duchenne is difficult because we have no cure. The path, the journey is ever-evolving as new knowledge is gained, contributing to an existing body of understanding. While the fundamental defect is known—a mutation in a gene located on the X chromosome, which results in the loss of a fundamental structural protein in muscle—there is a great deal we do not yet understand. It is about a continuum of knowledge, of uncovering and explaining differences in progression, identifying biomarkers that exacerbate or limit disease progression. Finding cures requires collaboration of all key stakeholders to fully understand the underlying mechanisms of the disease and pursue a range of targets that singularly or in combination will halt progression at every stage and for every individual diagnosed.
PRIM&R: What do you want our audience to take away from your talk at the 2016 Advancing Ethical Research Conference?
I am hopeful that the audience will take away that everything matters when we are talking about children. Words matter. Body language matters. Informed consent must be translated to be understood. That information matters, specifically that individual’s data matters and that confidentiality, honestly, fairness, and justice matter. That the good of the patient is first and what is best for the patient should be our highest concern.
PRIM&R: What would you tell other patient advocates about why they need to understand and support research ethics in their advocacy?
I would recommend that support for research in ethics sets the stage for the journey toward treatment and cure.
PRIM&R thanks Ms. Furlong for sharing these thoughts with our community; we look forward to welcoming her to Anaheim for AER16!
Patricia Furlong is the founding president and CEO of PPMD, the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (Duchenne). Ms. Furlong, together with other parents of young men with Duchenne, founded PPMD in 1994, and their mission is to end Duchenne. They accelerate research, raise their voices in Washington, demand optimal care for all young men, and educate the global community. Duchenne is the most common fatal, genetic childhood disorder, and it affects approximately one out of every 3,500 boys each year worldwide. It currently has no cure. Today, she continues to lead the organization and is considered one of the foremost authorities on Duchenne in the world.