Savara Pharmaceuticals has recently interviewed Dr. Tisha Wang, who is a pulmonologist and critical care physician at UCLA. Dr. Wang is also an advocate for autoimmune pulmonary alveolar proteinosis (aPAP). Savara is interested in this disease as they are currently developing a drug called Molgradex, which is an inhaled form of treatment for aPAP.
About Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare lung disorder. It is a form of pulmonary alveolar proteinosis, and it is also the most common form. The majority of those affected are adults aged 20-50.
The cause of this disease is a malfunction of the immune system, in which antibodies block the GM-CSF protein from regulating the clearance of surfactant by alveolar macrophages. The surfactant then accumulates in the alveoli, which leads to an inability to breathe.
The severity of symptoms ranges, as some people may be asymptomatic while others experience progressive difficulty with breathing upon exertion. Other symptoms include a chronic cough, fatigue, weight loss, chest pain, and a general feeling of ill health. In severe cases, people cough up blood, have round and swollen finger tips, and have cyanosis.
Treatment of aPAP varies from person to person, depending on severity and age. Lung lavage is the most common treatment, which is a procedure where one lung is pumped with pure oxygen while the other is cleansed with a salt solution. Depending on severity this procedure may need to be done once or multiple times over several years. Another treatment that is currently being tested and may soon be available is recombinant granulocyte macrophage-colony stimulating factor (GM-CSF), which is given through the nose or by injection. The last resort for treatment is a lung transplant.
Dr. Wang’s Interview
Dr. Wang was asked questions about her work as an advocate, along with advocacy in general. She stated that in terms of aPAP patients connecting, the PAP Foundation focuses on ensuring that patients connect with each other. They have a discussion forum on their website, and they are also beginning to use other forms of social media to connect patients. Their newest effort in patient connections is “Patient Education Days,” which are half-day meetings that focus on introducing PAP patients with each other and healthcare professionals. This foundation is also working on growing their internet presence in order to connect patients to healthcare providers that are right for them.
She was also asked about what needs to be done in terms of advocacy. Dr. Wang firmly believes that all of the aPAP patients need to be located in order to connect them with other patients and healthcare professionals. Along with this connection, funding for research must be increased so that a cure can be found. Education is another area that Dr. Wang wants improved. As many patients are not correctly diagnosed initially, she stresses the importance of educating healthcare professionals so that are able to make the right diagnosis. Even though more work needs to be done in terms of advocacy, Dr. Wang says that so many patients are grateful for the work that has already been done. It is this gratitude that pushes her to do more.
Advocacy is necessary for rare diseases as there are not a large amount of people affected by these conditions. A connecting force is important to ensure that their voices are heard. Dr. Wang speaks of the patients who are powerful advocates and how the effort would be nothing without them.
The interview was concluded with Dr. Wang listing some of the improvements she would like to see. In terms of research, she believes that there needs to be more novel targets for drugs that are based on a better understanding of the disease. She also thinks that there needs to be more treatment options, and hopes that Molgradex is approved, as it provides an option besides lung lavages.
Dr. Wang also wanted to ensure that people know that aPAP can be diagnosed with a simple blood test rather than a biopsy.
Find the interview here.