Current research and trials

Since 1995 both laboratory and clinical research into LAM has increased dramatically. In particular the US LAM Foundation has successfully involved a number of centres in basic laboratory work to try to find the cause of LAM. Data on patients with LAM are being collected in several countries, including the UK.

Research in Nottingham (supported by LAM Action) involves both clinical and laboratory research. Clinical research, including several surveys of women with LAM, has provided a clearer picture of how lung function (breathing) changes over time, the role of early life events in those with LAM and more details of the long term progress in women with LAM. These figures and the LAM register help us to plan studies of treatment. In the laboratory, research is focusing on how changes in the two proteins that are abnormal in LAM, tuberin and hamartin, lead to the increased growth of LAM cells. Knowledge of the cellular events involved in LAM led to the suggestion that one treatment (Rapamycin) might be helpful in LAM, and subsequent confirmation in clinical trials.

These approaches provide hope that more effective and safer treatments can be found. Substantial progress has already been made and we expect to have more treatment options within the next five to ten years.

Clinical studies are needed to find out more about LAM and to test potential new treatments as they become available. To do this properly requires well designed clinical trials comparing one treatment with another or with a dummy. Because LAM is rare it is particularly important that knowledge, discussion of best practice and research ideas are shared. This is widely recognised by both those with LAM and scientists with regular international meetings and communication.

In Nottingham, LAM Action jointly funded (with the British Lung Foundation) only the second randomised clinical trial of a possible treatment.

Also, as a result of trials in the UK and USA of the drug Rapamycin for the lung and renal manifestations of LAM, Rapamycin was approved as a treatment for women with moderate to severe LAM in 2011. The trials had concluded that Rapamycin stablilised the disease in women whose LAM is more advanced.

LAM tissue is extremely rare and a precious resource for LAM researchers trying to understand the condition. Patients may want to consider donating their unwanted tissue (including lymphatic fluid) to LAM research here in the UK. To discuss tissue donation, please contact Havi Carel (havi.carel@gmail.com).