Editor’s note: We asked patients, leaders of rare disease patient organizations and CSL Behring employees to cast ahead to a more hopeful 2021. We'll share their responses in a series of articles. Today we focus on the primary immunodeficiency community.
COVID-19 scared just about everyone, but people who have immune system problems were especially concerned. They already have trouble fighting off common infections. Primary immunodeficiency (PI) is a group of 400+ rare, chronic disorders in which part of the body’s immune system is missing or doesn’t work the way it should.
When the pandemic hit, the global PI community flew into action to provide expert guidance to patients. And patients reached out to one another virtually when in-person events were not possible. Fortunately, hope is always possible. Here’s what patients and leaders in the PI community had to say as 2020 came to a close.
John G. Boyle, President & CEO of the Immune Deficiency Foundation:
“The challenges of 2020 brought members of the IDF community closer together. Our community grew larger, as well. I am hopeful that a better and more global understanding of what it means to be immunocompromised will resonate in 2021 and beyond.”
Boyle played it straight with fellow patients from the start. In March, the leader of the U.S. patient advocacy organization said the COVID-19 pandemic was uncharted territory and he felt the uncertainty rippling through the patient community. At the time, he said his worried 10-year-old son asked him to stay home to be safe.
“I couldn’t tell him exactly how this particular situation will unfold, but I was able to tell him that there’s a lot that we do know about public health and that if we’re vigilant about the basics – especially hand washing – we’ll weather the storm together,” Boyle wrote in an email to the IDF community.
Patients wanted answers specific to their situations – which vary depending on which type of PI they have. While the IDF couldn’t provide medical advice, it did the best next thing by creating deep resources. They included regular video updates on its YouTube channel from Dr. Kathleen Sullivan, medical adviser to IDF and Chief of Allergy and Immunology at Children’s Hospital of Philadelphia. Another expert physician, Dr. Vivian Hernandez-Trujillo, also gave video updates in Spanish.
Johan Prevot, President of the International Patient Organisation for Primary Immunodeficiencies (IPOPI):
“COVID-19 erupted in our world unexpectedly, taking us by surprise, bringing about fear, casting doubts and uncertainty about the future. During such an unstable time the importance of having reliable, trustworthy and supportive partners around you is paramount.
The PID community has come together to face these challenges in a remarkable way…It has reaffirmed and demonstrated our belief that we are stronger when we work together. It has also shown us that each challenge presents an opportunity to drive change. Change so that the importance of prevention, of equitable access to diagnosis and care, of patient centeredness and of international collaborations is better recognized and integrated in health policies. This gives me great hope for the future and a renewed confidence in our ability to come together in the most testing of times.”
IPOPI, representing patients around the world, issued its first statement in February, aiming to provide reliable, evidence-based information and guidance to the community. In March, it joined with nine other groups from around the world to issue a statement on best practices for those in the patient community. And throughout 2020, IPOPI group collaborated with stakeholders to offer educational opportunities, awareness and advocacy campaigns, and to support research. IPOPI adapted by moving its work, communication and outreach methods online, Prevot said, allowing the organization to stay on track and fulfill its mission.
Vicki and Fred Modell, founders and leaders of the Jeffrey Modell Foundation:
“'Hopefully, history will record that the year 2020 was just a moment in time – albeit a devastating time filled with uncertainty, fear, anxiety and an unprecedented and extraordinary loss of life. In spite of the challenges, the Jeffrey Modell Foundation’s mission of hope, advocacy and action has not waned. In fact, the pandemic has re-energized us and called upon us to fulfill unmet medical needs and generously fund vital research that can be implemented now and well into the future.
This year confirmed that whatever obstacles are put in our way, we are unstoppable and will continue our journey to “do something,” until all patients with Primary Immunodeficiencies can have access to earliest possible and accurate diagnosis and equal access to treatments and therapies. Our hope for the near future, is that we can continue to advocate for patients with rare disorders and turn their tears to laughter and their fears to wonder.”
Whenever something affects the primary immunodeficiency community, you can expect Vicki and Fred Modell to show up. Driven by grief and love, the Modells started their foundation in 1987 following the death of their 15-year-old son Jeffrey, who died of complications from a primary immunodeficiency. Over more than 30 years of advocacy, they have raised millions of dollars and organized the medical community around the world, establishing JMF centers in 86 countries on six continents.
During the pandemic, the foundation shared resources with patients and supported the critical need for plasma donations. In November, the JMF Centre in Melbourne, Australia announced its researchers had published a study about the complexities of immunity to the virus that causes COVID-19.
Sharon Popik, CSL Behring’s Executive Director of Clinical R&D for Immunology:
"The R&D community has rallied in an unprecedented way to both continue life-saving research, as well as focusing on therapies for COVID-19 infection. I am optimistic that the SARS-CoV-2 vaccines will be rolled out in the next six months and everyone, but in particular the rare disease community, will be able to return to normal.”
Lynne Doebber, a lifelong learner and patient who has the primary immunodeficiency, common variable immunodeficiency (CVID):
“Science gives me hope. As a result of the COVID-19 pandemic, I hope that many young people whose lives have been adversely affected by this killer virus will develop an interest in science and devote their lives to research and development and bring about great advances in health. In addition, learning to combat COVID-19 has fostered a partnership between universities, pharmaceutical companies, international governments and people from all walks of life. By working together, sharing research and ideas rare disease will gain more awareness and attention. "
Stacy Ahearn, distance runner and patient living with a primary immunodeficiency:
“This has truly been the most challenging year living with PI to date. Hands down. My small world got a lot smaller for sure. I find I constantly run a mental marathon of staying positive and looking ahead. One of the best quotes I've seen is ‘Maybe some of the best days of your life haven't happened yet.’ I think about this all the time and am honored to watch science catch up to this awful COVID-19 pandemic.
I am so proud of vaccine development and collaboration from all the rare disease companies to quickly move ahead safely on to better days. Those good days are waiting and I can't wait to see what adventure is next. Thank you all for everything you do for patients like me. I am grateful always.”
