On November 15, 2018, the Nephrotic Syndrome Clinical Trial Recruitment Collaborative Summit was held in Washington DC. Over 120 senior-level stakeholders from around the world, representing diverse parties (FDA, ASN [American Society of Nephrology], biopharma companies, patients, clinical researchers, and NephCure) met in unprecedented collaboration to assure the recruitment success of the many current drug trials that will likely evolve into those future treatments. We asked two patient parents, David and Stephanie, who were in attendance at the summit to reflect on the innovative ideas, future treatments, and solutions which were discussed during the meeting.
Three years ago, our adult son was diagnosed with Focal Segmental Glomerulosclerosis (FSGS). This was an extremely difficult time for him and our entire family. As healthcare professionals ourselves, we combed through the medical literature and turned to our colleagues for answers. To our surprise, all we found were a few generic treatments for the condition, nothing disease-specific, based on clinical research that had appallingly low numbers of participants. There was only one clinical trial in progress and no glimmer of better treatments or cures in our future.
Through our searching, we found NephCure Kidney International and quickly attended a local outreach meeting. We were heartened by what we discovered: enthusiastic staff from the organization, a local researcher focused on FSGS, and other patients and families who were facing the same challenges. While we had not found a solution, we had found a community, and that gave us a great deal of comfort and encouragement.
Fast forward to 2018 when an amazing shift occurred—the arrival of 16+ clinical trials testing potential new treatment options for FSGS, IgA Nephropathy and other rare kidney conditions. Finally, the dream of more effective medicines with less harmful side effects became a closer reality for our son.
While this is incredibly encouraging news, it also presents significant challenges. Getting new treatments to the pharmacy shelf relies on willing patients to be part of clinical trials. History shows that filling trials for any disease is difficult, including those affecting millions of people such as cancer and heart disease. In the case of Nephrotic Syndrome, a rare condition, finding patient volunteers to participate in clinical trials is doubly hard. This is critical. Only last week when our son applied to participate in a major trial he learned it had just been closed due to lack of participants. Opportunity lost.
It was both the threat of unsuccessful trials and the promise of new and better medicines that prompted NephCure, together with members of the US Food and Drug Administration, the American Society of Nephrology, the Kidney Health Initiative, and the National Institutes of Health, to jointly develop the Clinical Trial Recruitment Collaborative meeting in Washington, DC on November 15, 2018. We were fortunate to have attended the ‘all-hands’ meeting where we witnessed firsthand the passionate and dedicated group of individuals laser-focused on filling clinical trials for Nephrotic Syndrome. It was encouraging and exciting to be in a room filled with 100 of the leading international researchers; clinicians; pathologists; staff members from the FDA, the NIH, and the ASN; representatives of pharmaceutical companies; patients; and family members coming together to find solutions to ensure these trials will not go unfilled. The sheer number of highly committed people in the room who truly cared about Nephrotic Syndrome treatments was inspiring.
With a clear understanding of the once in a lifetime opportunity unfolding before us, we left the meeting with a renewed sense of purpose and strong desire to spread the word to other patients and family members:
The time is now—with 16+ clinical trials in progress for Nephrotic Syndrome, 9 of which are specifically for FSGS, this day in time holds great promise for patients. It is imperative that all patients and families affected by these rare conditions heed the call to action by standing ready to be part of the clinical trial revolution.
Equally important, the Gateway Initiative has become a central hub for information, entry to clinical trials, and expert opinion resources. We encourage you to join this global movement by visiting KidneyHealthGateway.com, a user-friendly website created to help patients easily find the best clinical trial for their health condition. By taking this important step, you are not only finding trials right for you or your loved one—but also doing your part to move research forward and make better treatments a reality. In addition, NephCure has announced a strategic partnership with the IgA Nephropathy Foundation of America to combine resources and ensure that no potential new treatment fails due to lack of patient volunteers.
We hope to see better treatments and improved outcomes for our son and others like him, and one day, a cure. These once lofty goals may finally be on the horizon. We are grateful for the many dedicated individuals and organizations that are working to make this happen.
David and Stephanie