Treating LAM

While there is currently no cure for LAM, there are a number of treatments that may relieve symptoms or prevent complications from the disease.

First-Ever Clinical Treatment Trial

The Multicenter International LAM Efficacy of Sirolimus (MILES) Trial tested a drug called sirolimus as a treatment for LAM.  Sirolimus is currently approved to prevent the immune system from rejecting kidney transplants and has shown benefit in treating benign kidney tumors known as angioliopomas. The MILES Trial was the first randomized, controlled study designed to develop a therapy for LAM.  Treatment with sirolimus, also known as rapamycin, for one year resulted in a small but significant improvement in lung function (FEV1).

In March, 2011, the MILES Trial results were reported in the New England Journal of Medicine (NEJM).  In addition to the trial results, the NEJM also published an editorial which highlights the trial and the role of The LAM Foundation in research advancements.

Please click here to see the full New England Journal of Medicine article.

Available (Palliative) Treatment Options

There are several current treatments that are considered palliative. That is, they do not treat LAM, but aim to relieve symptoms or prevent additional complications from the disease. The response to treatment varies from patient to patient. Potential treatments include:

  • Medicines (e.g., diuretics, hormone therapy, bronchodilators that relax the muscles around the airways)
  • Oxygen therapy - as lung capacity declines, supplemental or full-time oxygen therapy may become necessary
  • Procedures to remove air or fluid from the chest or abdominal cavities and prevent it from building up again
  • Procedures to remove angiomyolipoma (AML), or benign kidney tumors
  • Lung transplantation - a procedure to replace one or both lungs.  This should be considered as a last resort due to the risk of serious adverse reactions, including major bleeding, pneumonia, pulmonary edema, long-term infections, and possibly painful scarring

Role of Hormones

Since LAM occurs almost exclusively in women of reproductive age, researchers believe the hormone estrogen might be involved in the abnormal muscle cell growth that characterizes the disease. Although there is no direct evidence that there is a relationship between estrogen and LAM, the treatment of LAM has focused on reducing the production or effects of estrogen. This could include estrogen or other hormone suppressing drugs. Additionally, doctors believe pregnancy may accelerate the progression of LAM. Women with LAM are urged to speak with a health care professional before getting pregnant.