Summary of Lymphangioleiomyomatosis (LAM)
ICD-10 code for LAM is J84.81.
Lymphangioleiomyomatosis, better known as LAM, is a rare, cystic lung disease associated with mutations in tuberous sclerosis genes, renal angiomyolipomas, lymphatic spread and remarkable female gender restriction. The clinical course of LAM is characterized by progressive dyspnea on exertion, recurrent pneumothorax, and chylous fluid collections. Lung function declines at approximately 2-3 fold times rate of normal subjects, based on an annual drop in forced expiratory volume in one second (FEV1) of 75-120 cc in various reported series.
The Future - LAM Clinical Trials
LAM and TSC researchers have identified a wealth of potential molecular targets and experimental therapies that may be appropriate for testing in clinical trials. These include mTOR inhibitors (e.g., sirolimus, everolimus), Rheb inhibitors (e.g., farnesyltransferase inhibitors and statins), selective estrogen antagonists (e.g., fispemifene), tyrosine kinase inhibitors (e.g., imatinibmesylate), metalloproteinase inhibitors (e.g., doxycycline), angiogenesis inhibitors (e.g., bevacizumab), and lymphangiogenesis inhibitors (e.g., anti-VEGF-D antibody). Many of these drugs are FDA approved or in development for other indications. In the absence of a known effective treatment, participation in clinical trials should be encouraged. Widespread off label use of candidate therapies like those above will deprive patients of the opportunity to find an effective treatment, and condemn LAM to the same fate as dozens of other pulmonary diseases where the prospects for controlled trials have passed us by.
The LAM Foundation's Role
The LAM Foundation is committed to supporting the development of effective treatments for LAM. The Foundation has committed the majority of the $13 million raised to date to support 75 peer-reviewed research projects for the study of lymphangioleiomyomatosis (LAM), many of which have provided the scientific basis for current and future trials. Annual LAM scientific meetings organized and sponsored by The LAM Foundation have become the primary forum for presentation of landmark LAM research and scientific exchange. The National Heart, Lung, and Blood Institute has shown tremendous support for LAM by investing more than $20 million to support LAM conferences, a national registry of LAM patients and a LAM protocol that evaluates LAM patients. The stage is set. LAM is poised for great advances!