Treating LAM

While there is currently no cure or effective treatment for lymphangioleiomyomatosis (LAM), research and clinical studies are underway to find treatments that will stop or delay the development of LAM. There are also a number of treatments that may relieve symptoms or prevent complications from the disease.

First-Ever Clinical Treatment Trial
In just 10 years, The LAM Foundation scientists have reported major breakthroughs, including the identification of the genetic basis of the disease. These research efforts have led to dozens of studies for the disease, including the first-ever clinical treatment trial for women with LAM, currently enrolling patients. 

The Multicenter International LAM Efficacy of Sirolimus (MILES) Trial will test sirolimus (rapamycin), the first drug to show promise 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. For more information about this study or to enroll, click here.

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.

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