“Turning discovery into health” – NIH
In her opening remarks at NIH Rare Disease Day, Senator Amy Klobuchar signaled the call to our nation’s policy makers to “position the United States as a preeminent research country” and offered patients, researchers and attendees her “Story of Hope.1” February 29, 2016 marked a world observance for rare disease awareness, with millions united across the globe in a shared commitment to the mission of caring and need to spurn more progress and hope for approximately 7000 different types of rare diseases and disorders. In the US alone, this figure translates to 30 million people or 1 in 10 Americans.2 A federal investment in one of our nation’s most sacred institutions, the National Institutes of Health, comes at a critical time for patients touched by one of the most deadly and rare aggressive cancers – mesothelioma - with median survival rate of 12-14 months and only one approved treatment that extends average survival by three months.3
At NIH Rare Disease Day, I was proud to help amplify the voice of the patient and bring a presence for patients suffering by mesothelioma with my poster submission and table exhibit. My father recently passed after a 20 month battle with this disease, described as the “asbestos’ signature cancer,4” with symptoms that became manifest in multiple pleural effusions a year and a half prior to an official diagnosis confirmed on biopsy. Because of the ubiquitous nature of asbestos in many consumer, home and industrial products (with peak usage spanning the 1930s-1970s),5 hazardous and occupational exposures still pose a continuous threat to public health, especially for everyday working Americans and Navy Veterans. Although the disease may be latent for a number of years following exposure, risks to contaminated materials and asbestos carcinogens have become especially heightened in the aftermath of national tragedies, such as the cleanup and rescue efforts following 9-11 and Hurricane Katrina.6 There is great need to not only prevent more environmental exposures to this silent and hidden killer, but there is need to ban asbestos completely, improve diagnoses, and collaborate with specialists early on in the process to develop better treatments that save lives7 rather than mitigate the consequences of an already congealed tumor.
“Medical information alone is not enough for an informed moral decision.8”
My father was a civilian in the US Navy and an influential thinker in ethics, physics, and political theory. He contributed to the greater society in modeling what he valued in everyday life, and his character and commitment to help anyone and everyone was far reaching and much evidenced by the packed rooms of individuals from all walks of life who came to offer condolences following his passing. Dad was never much for words or writing, but that’s how he lived – by example. At NIH Rare Disease Day, I wanted to celebrate his biggest treasure – his legacy – and lend testimony to his enormous strength in facing adversity with this rare, life-threatening disease. He showed so much courage, humility and taught us to have compassion. I was very fortunate for NIH to have taken him under their wing. Although it was just for a short time, NIH/NCI offered my family hope and I wanted to extend a thanks back for their greatest act of compassion: giving exceptional treatment to my father who, unlike many other rare disease patients at the institutes, was not part of a study protocol.
We were initially set up for an NIH clinical trial, but the day before the study was to commence, a pre-test parameter came back that was not within FDA safeguards. When the NIH/NCI team (much familiar with my dad’s case) brought this news to us, there was no need for words; the grim and solemn faces told the story. Learning of the disappointing news - that one did not make study protocol - was like watching hope pass you by. My dad had a very diffuse tumor of the lining of the lung, thereby making treatment very difficult. The response to chemotherapy (with platinum based drugs) is not curative, and treatment measures often become palliative due to no maintenance therapy for this fatal and excruciating, painful cancer. The NIH clinical study was aimed to improve and synergize the standard chemo response to mesothelioma with a vaccine. This is what scientific leaders do – they offer a Story of Hope through dedication and discovery and a willingness to explore through the clinical trials processes. They are driven at promoting new treatments, faster treatments, and delivering better treatments that improve and save lives.
I was grateful NIH bought us time, and dad was fortunate to go on as a participant for a clinical study in a New York hospital. He was later removed from this investigative trial due to disease progression and subsequently was approved off-label treatment with OpdivoR (nivolumab), product of Bristol-Myers Squibb and FDA indicated for the treatment of other cancers - lung and renal cancer. In the end, there was no treatment that worked despite all the medical information and best research efforts. He suffered terribly, and I am still trying to put the face of meso behind me and turn this story of rare into one of resilience. However, the devastating disease manifestations are a part of the patient experience and a harsh, brutal and cruel reality. The sounds of death rattles still haunt me. The oxygen machine in the house was always a reminder that the end was near and he was tethered to tubing and tanks 24/7 for over 4 months. And, with disease progression, the simplest tasks were more and more burdensome. He could not walk, he had no energy – not even to make it across the living room to the specially installed stair lift. He could not swallow food or medicine due to the tumor pushing against the esophagus. Clothes were painful, even the slightest wrinkle would cause great discomfort. The chairs and bed were rigged with padding. Because of the location and growth of the tumor, he could not tolerate a sitting position. He preferred standing or being reclined, which made transportation especially difficult. Not only did the bumps in the road cause pain, but so did the simple task of just sitting in the car.
These were all visual and graphic reminders that there was no comfort to be found, made especially worse having traveled over eight thousand miles in the multiple roundtrips to New York from Maryland just to receive medication and pine for hope.
He drowned in his lungs, evidenced by increasingly shallow respirations as his body was shutting down. We were off the path to cures and on a path of terminal illness. How does one cope? Is there a Story of Hope at the crossroads of death?
“Translation is a team sport” – Dr. Austin, NCATS
Pope Francis once said, “There is no human life more sacred than another, just as there is no human life qualitatively more significant than another. The credibility of a health care system is not measured solely by efficiency, but above all by the attention and love given to the person, whose life is always sacred and inviolable.9” It might sound strange, but I could hear the echoes of Francis in the opening remarks of Dr. Christopher Austin, Director, National Center for Advancing Translational Science, (NCATS), challenging everyone with a “moral imperative: to have treatment for every patient with rare disease.10” Although NCATS has been described as a “distinctly different entity in the research ecosystem,11” the underlying factor – and the greatest resource factor in driving both the church’s social policy and at the root of science-driven policy – is the human resource factor. The service strategies of the Catholic Church have stood the test of time, with the first health care facility founded by the Ursuline Sisters in 1727.12 Today, expansion through charitable organizations and public-private partnerships has resulted in outreach to millions on domestic and global issues of pertinence to social policies. Supplanting the framework are the Scriptures, spiritual beliefs and sense of empowerment that further an institutional mission of caring and improve efficiency and effectiveness in the process. Using the Catholic Charities USA (CCUSA) social policy initiative to end poverty as a case example, successful implementation invokes a 3-C’s approach:13
· “Calling upon the government to improve public policies…and all in our nation to address the issues that impact the most vulnerable”
· Collaboration, “working with individuals and organizations across the country”
· Communities are best served when policy makers make educated and informed decisions and “engage those who are most impacted by government policies”
Similarly, the NIH patient-centric framework, supplanted with a philosophy “patients and researchers – partners for life” parallels the ecumenical approach to solving problems for some of the nation’s most vulnerable citizenry, those with rare disease. The “expression of the servant model of the Church14” is also embodied in the hallmark feature of Rare Disease Day, the handprint logo. The handprint is a universal symbol of sanctity, dignity, unity, and respect. Dr. Austin captured this culture of collaboration in his opening remarks stating, “Translation is a team sport.15” And, Dr. Petra Kaufmann, Office of Rare Diseases Research (ORDR) reinforced the key attribute to a successful NIH service strategy initiative in acknowledging that “patients drive the process16” for translating science into meaningful results. Although a team approach is integral to strengthening pathways, a balanced approach may be more prudent when pathophysiological needs cannot be met or understood, as in the case of my father. The mission of patient-focused compassionate care must also nurture the spirit. The art of healing might take a paradigm shift in culture towards the sick and dying, who are often deemed burdensome by a wasteful society. A culture of compassion is the interconnecting factor among researchers, patients, and even policy makers, as modeled by Senator Klobuchar and her testimony to the power of advocacy and saying, “I go to work every day to do the right thing.17” What I find most rewarding about participating at NIH Rare Disease Day is being part of this team, and I was really moved as recipient to several hugs for sharing my dad’s story. I wanted to extend a thanks back to NIH for being our partners and in words of my dad who made the ultimate sacrifice, “don’t give up on me.”
In memorandum, Jerome E. Weiland 2/21/45 – 12/22/15
1 Senator Amy Klobuchar, Rare Disease Day at NIH, February 29, 2016 Masur Auditorium, Building 10 NIH Clinical Center, Bethesda, MD
2 Global Genes, Rare Diseases: Facts and Statistics, https://globalgenes.org/rare-diseases-facts-statistics/ accessed 3/6/16
3 Mesothelioma Applied Research Foundation, “Talking Points” Advocacy Resource; NIH, quoted in Mesothelioma Fact Sheet, http://www.curemeso.org accessed 3/6/16
4 National Institute of Occupational Safety and Health (NIOSH) February 2007 report, “Asbestos and Other Mineral Fibers: A Roadmap for Scientific Research,” quoted in Mesothelioma Applied Research Foundation, “Asbestos in the Nation A Hidden Killer,” Asbestos Fact Sheet Advocacy Resource, http://www.curemeso.org accessed 3/6/16
5 Mesothelioma Applied Research Foundation, “Asbestos in the Nation A Hidden Killer,” Asbestos Fact Sheet Advocacy Resource, http://www.curemeso.org accessed 3/6/16
6 Mesothelioma Applied Research Foundation, “Asbestos in the Nation A Hidden Killer,” Asbestos Fact Sheet Advocacy Resource, http://www.curemeso.org accessed 3/6/16
7 Mesothelioma Applied Research Foundation, “Asbestos in the Nation A Hidden Killer,” Asbestos Fact Sheet Advocacy Resource, http://www.curemeso.org accessed 3/6/16
8 Maryland Catholic Conference, A Pastoral Letter from the Catholic Bishops of Maryland, “Comfort and Consolation Care of the Sick and Dying,” 2007, p9 www.mdcathcon.org
9 Pope Francis, September 20, 2013 quoted in Maryland Catholic Conference, A Pastoral Letter from the Catholic Bishops of Maryland, “Comfort and Consolation Care of the Sick and Dying,” 2007, pIV www.mdcathcon.org
10 Christopher P. Austin, National Center for Advancing Translational Sciences (NCATS) Director Remarks, Rare Disease Day at NIH, February 29, 2016 Masur Auditorium, Building 10 NIH Clinical Center, Bethesda, MD
11 NIH News Release, “NIH Rare Disease Day Event, on Leap Day (Feb. 29) will feature patients’ voices and international collaboration,” Tuesday, February 16, 2016 http://www.nih.gov/news-events/news-releases/nih-rare-disease-day-event-leap-day-feb-29-will-feature-patients-voices-international-collaboration
12 Catholic Charities USA, Working to Reduce Poverty in America, 2013 Catholic Charities USA, Alexandria, VA. http://catholiccharitiesusa.org accessed 2/15/2015
13 Catholic Charities USA, Working to Reduce Poverty in America, 2013 Catholic Charities USA, Alexandria, VAhttp://184.108.40.206/our-solutions/campaign-to-reduce-poverty/accessed 2/15/2015
14 Catholic Charities USA, Working to Reduce Poverty in America, 2013 Catholic Charities USA, Alexandria, VA. http://catholiccharitiesusa.orgaccessed 2/15/2015
15 Christopher P. Austin, National Center for Advancing Translational Sciences (NCATS) Director Remarks, Rare Disease Day at NIH, February 29, 2016 Masur Auditorium, Building 10 NIH Clinical Center, Bethesda, MD
16 Petra Kaufmann, Office of Rare Diseases Research (ORDR) Director Remarks, Rare Disease Day at NIH, February 29, 2016 Masur Auditorium, Building 10 NIH Clinical Center, Bethesda, MD
17 Senator Amy Klobuchar, Rare Disease Day at NIH, February 29, 2016 Masur Auditorium, Building 10 NIH Clinical Center, Bethesda, MD