Rare Leader: Janice Schwartz, Executive Director, Pachyonychia Congenita Project
September 12, 2019
Name: Janice Schwartz
Title: Executive director
Organization: Pachyonychia Congenita Project
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Disease focus: Pachyonychia Congenita (PC) is a chronic, debilitating, ultra-rare autosomal dominant skin disorder. PC is caused by a mutation in one of five keratin genes: KRT6A, KRT6B, KRT6C, KRT16, or KRT17. The most prominent physical symptoms are painful calluses and blisters on the soles of the feet and/or palms of the hands. Other common features of PC include thickened nails, painful cysts, and white build up on the tongue. PC does not affect lifespan, but almost all patients experience nearly constant pain, causing many to crawl and use mobility aids such as crutches or wheelchairs.
Headquarters: Holladay, Utah
How did you become involved in rare disease: I married the right man, who had the right mom. Because I, and two of my children have PC, my mother-in-law, Mary Schwartz, was determined to find relief for our painful disease. Her initial thought was to give money to a researcher who would then find a cure. With the guidance of some special scientists and physicians, we realized it wouldn’t be quite that simple. We needed to find patients for studies and clinical trials and we needed an array of specialists to work together and facilitate research for PC, which at that time was not happening at all. In 2003, Pachyonychia Congenita (PC) Project was formed and I’ve been involved ever since. And although I was motivated in the beginning to help my own children with PC, I quickly became driven by love for my other ‘children’ of all ages who have PC.
Previous career: I was a stay-at-home mom of four children by choice, and served as a board member and patient advocate for PC Project.
Education: B.A. in Communications with an emphasis on advertising from Brigham Young University
Organization’s mission: PC Project’s mission is “Fighting for a cure for PC by helping and supporting patients, empowering research” and more recently, by partnering with industry professionals and drug developers who are interested in advancing research into marketable treatments for PC.
Organization’s strategy: PC Project’s strategy is marked by collaboration and unity. In 2004, PC Project established the International Pachyonychia Congenita Consortium (IPCC), a gathering of the brightest scientific and medical minds in the fields of keratoderma, skin, pain, genetics, anything that might be applicable to PC. All interested professionals are still invited to join the IPCC and attend our annual IPCC Symposium where research is presented, ideas are discussed, and new goals are set for the upcoming year. We’ve partnered with a biopharmaceutical company that’s developed a potential treatment for PC. This company has enlisted members of the IPCC for scientific and clinical help and for trial site coordinators. PC Project has specifically assisted in developing clinical endpoints for the study, provided patient perspectives, and helped with trial recruitment by communicating with our patient community, connecting patients with various trial site coordinators, and providing genetic testing. We can do all that because of the International Pachyonychia Congenita Research Registry, the most important asset for PC Project. With more than 2100 patients in over 60 countries, this patient registry is the link between our patient and scientific communities. The registry houses patient information that has changed what we now know about PC and has resulted in numerous studies for PC. Everything we do involves collaboration in order to achieve results.
Funding strategy: Our main funding source is from individual donors who are affected in some way by PC. We have two yearly campaigns. The first is a drive to increase monthly donors. The second starts with Giving Tuesday and runs until the end the year. We do apply for and receive some grants.
What’s changing at your organization in the next year: We plan to reach out to our patient community to discover skills and talents they would be willing to share and utilize to help advance our cause. By next year, we will know whether a current phase 2/3 clinical trial for a treatment for PC is successful. The results of that study, one way or the other, will determine some of our next steps. There are additional therapeutics to further explore for their potential applications for PC. We’d like to collaborate more with organizations of other rare skin diseases to see if there is potential crossover in treatments.
Management philosophy: I believe people need to feel successful and appreciated in their individual roles, and when they do, everyone wins. As we work together, each person contributes with their different talents, abilities and personalities. For me, it’s important to show gratitude to the people I work with, including my employees who work hard and effectively. In addition, we ask a lot from our patient advocates, our patient community and our board of directors, as well as members of our scientific, medical, and professional communities, many who freely assist us. I hope I can convey to them regularly how much their dedication means to me and how significant their service is. I am not a one-woman show. Any bit of success we have is absolutely a result of teamwork.
Guiding principles for running an effective organization: For us, it’s our mission that guides us. We are small, we are rare, and we are busy. I think about our mission statement often. If something doesn’t serve to fulfill a part of our mission, we typically don’t do it. We are also frugal. We try to make wise decisions with our funds. Most of our donations come from patients, their family members, or friends. I feel those funds are sacred and should be handled with care.
Best way to keep your organization relevant: Networking and collaborating with those who have the ability to help patients and advance research and therapeutics. This includes producing regular newsletters, supporting publications in reputable scientific journals, hosting annual patient support meetings and the annual consortium symposiums, and representing PC Project at relevant conferences. We’re also working to be more engaged with our various communities through social media.
Why do people like working with you: I think people like working with PC Project because we are collaborative, we respond quickly, and we truly care about individuals. Our labor is one of love. We don’t do this because it’s simply a job. Bottom line, we want effective treatments and eventually a cure for PC. We happily help and support anyone who shares those same goals. We are also the only organization worldwide to serve PC patients and facilitate PC research. We’re too rare to be splintered. If someone is interested in PC or has PC, we are the one-stop organization for them. Professionals like working with us because we share our knowledge and our registry data (de-identified). We provide patients for studies, and we promote collaboration from all corners of the world. Because of our patient registry, we know who our patients are, where they are and their exact affected gene and mutation—things we feel are important to know in our corner of the rare disease world.
Mentor: I have an array of mentors in my life. I learn different things from each of them. They include my mother-in-law, my parents, my husband, co-workers, all who tend to know more than me. I receive mentoring from those in the professional world—doctors, scientists and drug developers who freely give me guidance. I’m thankful to each one of them.
On the Job
What inspires you: Like probably all rare disease leaders, I’m inspired by the patients with their courage, their resilience, and their willingness not to let this horrible disease get them down. But I’m also encouraged by the scientists, clinicians, and drug developers, people who have become my true friends, who commit their time and energy to helping us simply because they genuinely care about those who suffer with PC.
What makes you hopeful: All the advances in science that have potential applications now and in the future for PC make me hopeful. If I didn’t believe that one day, we will have effective treatments for PC, I would not be dedicating my time this organization. I’m also energized by the hope of others – not just the patients, but all the people who donate their wisdom and resources to this cause. We see lots of little miracles all the time. Sometimes I worry and wonder, “How is this going to work out?” And then somehow, things do work out, usually because of the goodness of people.
Best organization decision: To build the ever-growing International PC Patient Registry and form the International PC Consortium of researchers and physicians. With a body of data from willing, genetically confirmed patients, PC Project is in a position always to collaborate with scientists, clinicians and drug developers in order to advance research and treatments for PC.
Hardest lesson learned: Because PC patients don’t die from this disease and because the disease is so rare, it’s harder to attract interest. The lot of our patients is to live with debilitating, constant pain – for now.
Toughest organization decision: My toughest decisions involve the best ways to use our funding past our basic programs and services.
Biggest missed opportunity: I guess I don’t know what the biggest missed opportunity was because I missed it. Seriously though, I try to listen to those who give me advice and determine what opportunities are best for our organization. For example, it was proposed PC Project do a Patient Focused Drug Development meeting where PC patients presented testimonials about the burden of the disease before key FDA officials. I listened to the counsel of people I respect, and it turned out to be a pivotal decision for PC patients.
Like best about the job: Feeling like I’m making a difference in the lives of patients who live with this painful, isolating and sometimes embarrassing disease. While we may not have a cure yet, I feel like patients and caregivers are edified and empowered through the programs and services of PC Project. I love that feeling of reassuring patients that they’re not alone in the world and that there are people out there fighting on their behalf.
Like least about the job: Needing to tell patients, and especially parents of PC children who reach out to us in hope or desperation, that we don’t have an effective treatment for them – yet.
Pet peeve: Complacency – especially when some PC patients don’t see the vision of joining the registry or even donating a few dollars to the cause. Fortunately, overall, we have an engaged and enthusiastic patient community. I wish each person touched by PC could know how much of a difference one person can make. Small and simple things make great things come to pass.
First choice for a new career: This is the only career I want. I couldn’t ask for a better one. However, someday I hope to put myself out of this job with a cure for PC.
Most influential book: The scriptures. There are a million life lessons in them.
Favorite movie: Pride and Prejudice and Marvel movies. I like happy endings and people who do the impossible.
Favorite music: Classic rock, hits from the 70’s, and Broadway musicals
Favorite food: Seafood
Guilty pleasure: Ice cream
Favorite way to spend free time: I love hanging out with my family, talking, and laughing.
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