A new gene therapy could make it easier to treat a serious lung disease: pulmonary alveolar proteinosis. Professor Gesine Hansen and her team received the 2013 Eva Luise Köhler Research Prize for Rare Diseases for their findings.
Diagnosis PAP. Three letters that can severely limit a person’s life. They stand for a complicated name: pulmonary alveolar proteinosis. In this rare and serious lung disease, the lungs gradually become ‘clogged’. This causes severe breathing problems – and can lead to death. Treatments already exist, but their effectiveness is limited. This could soon change, thanks to research by paediatrician and lung specialist Professor Gesine Hansen and her team.
The Disease
Pulmonary alveolar proteinosis is an incurable genetic disease. Children are born with it but appear healthy at first. PAP literally sneaks up on them and causes the child to grow and thrive poorly, suffer from frequent infections and later develop breathing difficulties. This is due to ‘scavenger cells’, medically known as macrophages, whose job it is to keep our lungs clean. They clear them of cell debris and materials. But in people with PAP, the macrophages do not do their job properly. They do not break down the unwanted substances properly. As a result, a protein- and fat-rich material builds up in the lungs – a kind of viscous mucus that clogs the organ. At present, the disease is mainly treated by lavage, a process of washing out the lungs under general anaesthetic, which can take many hours.
The Research
The problem with lung washing is that although many patients feel better, they are still not well. In addition, the treatment is very hard on the body and has to be repeated regularly. Professor Dr Gesine Hansen from the Hannover Medical School and her team, consisting of Professor Dr Nico Lachmann, Professor Dr Thomas Moritz and Professor Dr Christine Happle, have taken a new approach: They cultivated phagocytes from immature blood cells in the laboratory. They then introduced these cells directly into the lungs of mice suffering from alveolar proteinosis. The breakthrough: the phagocytes matured in the animals’ lungs and took over their function. The diseased phagocytes were simply replaced – and the mice’s lungs remained free of clogged mucus during the many months of observation. Gesine Hansen: ‘There is hope that this completely new concept of cell therapy will lead to long-term therapeutic success, so that the children will be significantly better in the long term.
The Future
Gesine Hansen and her team are currently planning the first clinical trial of this new cell therapy with their American cooperation partner Bruce Trapnell. We very much hope that we will make rapid progress and that the first children will benefit from the new therapy within the next few years,’ says the doctor. Our new therapy concept could also provide a completely new treatment option for many other diseases. We are working hard to help our young patients as soon as possible.
