Prior LAM Foundation Research
Click here to download Progress in LAM Research.
Click here to view the list of prior LAM Foundation awardees.
From knowing nothing about LAM in 1998 to the inception of a $3 million treatment trial in 2006, the efforts of lymphangioleiomyomatosis (LAM) researchers have led to major discoveries.
LAM Foundation Breakthroughs
In the first two years of funding LAM research, LAM Foundation scientists reported the first evidence that LAM and tuberous sclerosis are genetically linked. Our major breakthrough made two years later by LAM Foundation investigator, Dr. Elizabeth Henske, was proof that LAM is caused by mutations in tuberous sclerosis genes. In 2002, LAM Foundation scientist, Vera Krymskaya, PhD, demonstrated that abnormal smooth muscle cell invasion of the lung in LAM is due to the loss of growth control by tuberous sclerosis proteins and can be controlled using drugs that mimic the function of the missing proteins.
The scientific breakthroughs by LAM Foundation scientists have been pivotal events in LAM research and provide the scientific basis for first-ever LAM treatment trial in 2003!
Pilot Study using Sirolimus (Rapamycin)
The 2003 Cincinnati Rapamycin Trial was conducted to test the effect of the drug sirolimus (also known as rapamycin) on kidney tumors (known as angiomyolipomas) in patients with LAM or tuberous sclerosis. Every patient in the trial received rapamycin in escalating amounts from low, to intermediate, to full dose as kidney tumor size was monitored by MRI over the course of four months. The drug was continued for a total period of one year, at which point rapamycin was withdrawn and the kidney tumor size was monitored off of therapy. In addition to MRI of the kidney, secondary endpoints of quality of life (measured by questionnaire), were followed, as well as lung function, CT scans of the chest, brain MRIs and skin lesions in patients with tuberous sclerosis.
The pilot study was to determine whether the drug is safe and has promise in treating LAM. There are three reasons that the study does not have the statistical power to prove that the drug works for lung disease: 1) the numbers are too small, 2) the study was not designed to minimize biases that lead to erroneous conclusions (i.e., no placebo control) and, 3) the primary endpoint was kidney tumor size and not lung structure or lung function.
Results were published in the New England Journal of Medicine in early 2008. The pilot study suggests that sirolimus has promise for treating LAM, tuberous sclerosis and angiomyolipomas (kidney tumors). These basic and clinical research studies inspired the development of the MILES Trial, a pivotal trial to test the efficacy of sirolimus in LAM.
Read about the history of LAM and see how LAM Foundation research played a role in bringing attention to LAM.
Check out a list of previous studies and papers from leading LAM and pulmonology researchers.
Learn more about the symptoms, cause and treatment of LAM.
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