Interview with an aPAP Advocate
Our last two blog entries focused on the rare lung disease autoimmune pulmonary alveolar proteinosis (aPAP). Treatment of aPAP is the focus of our investigational drug candidate, Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF). One entry provided a physician’s perspective on the disease and another gave a first-hand account of a patient’s journey (highlighting her path to diagnosis, challenges of living with the disease and her hope for a cure). Today’s entry, an interview with Dr. Tisha Wang, offers an advocate’s perspective.
About Dr. Wang:
Dr. Tisha Wang is a pulmonologist and critical care physician who practices at UCLA in Westwood, California. She treats adult patients with pulmonary conditions and works in the intensive care unit taking care of critically ill patients. She is director of the liver transplant intensive care unit and co-director of the pulmonary embolism response team, and is board certified in pulmonary disease, critical care medicine and sleep medicine.
Dr. Wang received her medical degree from the University of Texas Medical Branch at Galveston, graduating with highest honors. She then completed her internal medicine residency and her pulmonary and critical care fellowship at UCLA.
In addition to her clinical work, Dr. Wang conducts research on pulmonary complications of liver disease and a rare lung disease called pulmonary alveolar proteinosis, or PAP. She is clinical director and vice president of the patient-based PAP Foundation and is vice chair of the education committee for the American Thoracic Society. She is also an associate professor of clinical medicine in the David Geffen School of Medicine at UCLA and is the program director for the pulmonary and critical care medicine fellowship and the clinical chief of the UCLA pulmonary, critical care, and sleep medicine division.
As part of her work, Dr. Wang advocates for the advancement of women in medicine, for medical educators training the next generation of physicians, and for patients with rare lung disease.
Chief Executive Officer
An Interview with Dr. Tisha Wang:
Connecting aPAP patients in the U.S. has come a long way but is still a work in progress. One area of focus for the PAP Foundation is to help connect patients with each other and to help connect them to the clinicians and scientists who are conducting research on their disease. The Foundation has only been active since 2016 and our focus on connecting patients has really gotten off the ground in the last couple of years.
In the past, patients relied on old Yahoo chat groups to connect. Now they are able to participate in the “PAP Discussion Forum” on the PAP Foundation’s website. This is an open discussion forum for patients, doctors and the entire aPAP community to connect and chat about issues that are important to them and the disease. We are starting to leverage social media tools as well, such as Facebook, Twitter, and Instagram to better connect the aPAP community and encourage collaboration and discussion.
Additionally, we now hold national and local “Patient Education Days,” that were modeled off the LAM Foundation (an advocacy organization for lymphangioleiomyomatosis, a rare lung disease). These half-day meetings, which are led by a healthcare professional, a research coordinator and PAP patients themselves, are held at major academic centers and are an opportunity to connect patients, their families and healthcare providers. Importantly, we teach both the patients and the local pulmonary physician community about PAP―and the latest research within PAP―and then we introduce them to the PAP Foundation and its mission. We’ve held events in Los Angeles, Orange County, Cincinnati, Gainesville (Florida), Denver and New Haven. And we have plans for Patient Education Days in Dallas, Michigan, Seattle (and a second one in Los Angeles in the next year).
The Patient Education Days that are held locally are one of the Foundation’s biggest accomplishments. Additionally, to help create awareness for our cause, we received a Google grant to ensure that people who search “PAP” will get to our Foundation’s page. We just recruited a Social Media Director who will maintain that momentum by building out our presence on Facebook, Twitter and Instagram. Having a more substantial internet presence will better enable patients to connect to us, to each other and to physicians. It works, too. Not a month goes by without me getting a call from an aPAP patient or clinician.
For example, I’ve started seeing a pediatric aPAP patient from Orange County. Originally, her parents were flying her to Denver and Cincinnati for treatment. After they connected with the PAP Foundation, they became aware of me…and I’m just down the road from them. They didn’t really need to travel for treatment but had no idea because they weren’t aware of my practice. We want to create a network of resources to help all aPAP patients connect to healthcare providers in this way and find the help and information they so desperately need. We are also working to define local “Centers of Excellence” in cities so that people can more easily and efficiently get care for this debilitating disease.
We need to find more―no, we need to find ALL of the aPAP patients and their loved ones in the U.S. We also need to increase our ability to fund novel research so that we can achieve our ultimate goal of finding a cure for aPAP. We work with and encourage aPAP patients and their families to advocate for this funding. Like so many other rare disease advocacy organizations (e.g., The Cystic Fibrosis (CF) Foundation or LAM, which have well organized and active patient advocate groups) patients are able to provide the most accurate “voice” for this kind of work. Patients advocating on their own behalf put a face to the disease, and they can best articulate the challenges they face and the solutions they need. We all need to work together for a cure―we can’t do it without patient participation.
Education for healthcare providers is also a big push so patients are diagnosed correctly the first time around. We need to increase physician awareness of the disease and educate doctors on how to diagnose and treat it. aPAP is quite rare so most clinicians and healthcare providers have never even heard of it. Right now, a typical time horizon to an accurate diagnosis is about 18 months. Especially if the patient is young, they often get misdiagnosed with asthma or pneumonia and are prescribed multiple antibiotics that are ineffective in treating aPAP. Eventually they might get a CT scan of their lungs, which will indicate that something is wrong, and then get referred to a pulmonologist. The pulmonologist will often times recommend a surgical lung biopsy (which can be invasive and painful—and unnecessary as there is a simple blood diagnostic for aPAP). The whole process can be arduous and take a long time and, in the meantime, the patient is struggling and feels alone and isolated because no one can tell them what is wrong. The data indicate that when an aPAP patient is inaccurately diagnosed with another condition, such as pneumonia, it can delay an accurate diagnosis by 1.5 years.
At a Patient Education Day in Denver, I recently met a young girl who was a pre-teen when she developed symptoms of the disease. Rather than being diagnosed accurately, for years she was initially told that she had an eating disorder and was then labeled as having psychiatric problems. These misdiagnoses were made without anyone ever even getting a chest x-ray.
However, while the path to diagnosis is typically not easy (due to the rareness of the disease), I have heard of cases where patients get diagnosed right away. It all depends on how quickly they can find a healthcare provider that is aware of aPAP and its symptoms.
We receive an amazing amount of gratitude, directly from patients and their families, thanking us for connecting them to other patients, healthcare providers and researchers. aPAP patients have dreamed of this and now it’s becoming a reality. With such a rare disease, most aPAP patients don’t know a single other person with the disease, nor can they find a doctor who knows about it and can properly diagnose or treat it. The burden of the “unknown” is isolating, frustrating and scary for the patients. Connecting them to a network of people who understand what they are going through, can share experiences and provide a message of hope is life-changing. Patients no longer feel alone, misunderstood or isolated—they have created a community united by their disease and a desire to find a cure.
One vivid success story that I can recall is the kid I mentioned before who took so long to get diagnosed. She wanted to be connected to other teenagers so we introduced her to an aPAP patient who was also diagnosed as a teen. This older patient is now playing collegiate golf and does not even look like she has the disease from the outside. I’m sure it was extremely encouraging for the younger teen and gave her hope of a better future.
For any progress to be made in a rare disease, an advocacy organization is essential. In close partnership with people affected by the disease, the advocacy organization can help promote the needs and priorities of patients. Together we become a loud voice for a patient population that isn’t large in numbers, but strong in action. We’ve seen this collaborative partnership (between patients, healthcare providers and research organizations) work well in other disease areas, such as CF and LAM. In fact, the LAM Foundation has provided us with the most inspiration and guidance. They are a perfect example of what can be accomplished. Having been in existence for more than 25 years, they now have events happening everyday across the country, and they have helped get new drugs approved to better treat the disease. We have modeled many aspects of our work after the programming/structure of the LAM Foundation.
It’s important to point out that a patient advocacy organization cannot succeed without the efforts of patients. People living with aPAP (and their families) need to work for their own cause and there are many ways to get involved. Patient groups are instrumental in raising awareness for the disease, advocating for research and new treatments and fundraising. Their efforts and contributions are at the center of the ecosystem that is driving change and working toward a cure.
What changes would you like to see regarding:
Dr. Tisha Wang is not a spokesperson for Savara Inc. The views, thoughts and opinions expressed by Dr. Wang in the interview set forth herein are her own and may not reflect the views or opinions of Savara Inc., its affiliates, or its or their employees, directors, officers, successors, or assigns.