By Francesco Bonella
In the last two decades enormous progresses have been made in the field of rare lung diseases, but there is still a long way to go. Pulmonary alveolar proteinosis (PAP) is an ultra rare disease with prevalence around 0.3 cases per 100.000, with no approved drugs for its treatment. From one side there is growing knowledge about the nature and the mechanisms leading to PAP development, and diagnostic methods as well as treatment options have been significantly implemented in the last years.
On the other side, from a patients perspective, there is paucity of information, scarce financial resources and lack of visibility making it very challenging for patients, relatives and treating physicians to deal with this disease. Although policy makers have started some initiatives at European level to create networks for rare diseases, fast tracks for the approval of orphan drugs and specific investment plans, at the local level an enormous amount of work has to be done.
Due to all the above-mentioned reasons, we decided to start our own initiative to inform patients affected by PAP and respiratory physicians. The intent of this online bulletin is to provide, on a regular basis, an update about advances on PAP from basic research to clinical trials, to report on the biggest congresses worldwide, patients’ initiatives, and also practical information about centres or networks dedicated to PAP.
Our Editorial Team is composed by physicians and researchers with expertise on PAP and patients willing to give their voluntary contribution, and will turn over every 12 months, in order to guarantee independency and transparency. The newsletter will be published every six months.
We will open the first issue of our newsletter by illustrating the state of the art on PAP, consisting of some progresses and many unmet needs, and we will present also clinical cases, to demonstrate the heterogeneity of PAP presentation and disease course. In addition, we will focus our attention on whole lung lavage (WLL), currently the gold standard treatment for this disease.
We hope that our small contribution will be of help for all PAP patients and interested physicians in order to share knowledge and practical information, connect each other more effectively, and improve visibility and political weight of this neglected and poorly understood disease.