Francis X. McCormack
Pulmonologist who led the MILES trial, which showed that sirolimus (rapamycin) halts the decline in lung function in lymphangioleiomyomatosis, a rare mTOR-driven lung disease.
Professor and Division Chief, Pulmonary, Critical Care and Sleep Medicine, University of Cincinnati · Gordon and Helen Hughes Taylor Chair (since 2006) · Co-leader, Rare Lung Diseases Consortium
Portrait: University of Cincinnati
Lymphangioleiomyomatosis, or LAM, is a rare lung disease mostly in women. Cells with overactive mTOR form cysts that slowly destroy lung tissue. Because the problem sits in the mTOR pathway, rapamycin (sirolimus) was an obvious candidate drug, but it had to be tested properly.
Francis McCormack is first author of the MILES trial, a 12-month, double-blind, placebo-controlled study of 89 patients. Lung function fell by about 12 ml per month on placebo and held steady on sirolimus. When the drug was stopped, the decline resumed, so the benefit lasted only while patients kept taking it. Side effects were more common on sirolimus.
McCormack leads the pulmonary division at the University of Cincinnati and co-leads the Rare Lung Diseases Consortium with Bruce Trapnell. In the Atlas, MILES stands as one of the clearest human demonstrations that blocking mTOR can change the course of a disease.
Milestones in the Atlas
A selection, not a full bibliography: only papers that meet the Atlas inclusion criteria.
| Year | Evidence | Study |
|---|---|---|
| 2008 | H | Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis BIS2008 Sirolimus shrinks renal angiomyolipomas in tuberous sclerosis / lymphangioleiomyomatosis. |
| 2011 | H | Efficacy and safety of sirolimus in lymphangioleiomyomatosis (MILES) MCC2011 First author of the MILES randomized trial: 12 months of sirolimus stabilized lung function in 89 patients with LAM, with decline resuming after the drug was stopped. |
Co-authors in the Atlas 4
People with a profile here who share at least one study with Francis X. McCormack.
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- Lisa Young 2 shared studies Lung physician-scientist who helped run the first sirolimus trials in lymphangioleiomyomatosis (LAM) and now leads pulmonary medicine at the Children's Hospital of Philadelphia
- Bruce C. Trapnell Pulmonologist-scientist who directs the Rare Lung Diseases Clinical Research Consortium whose MILES trial led to approval of sirolimus for lymphangioleiomyomatosis (LAM); also known for work on pulmonary alveolar proteinosis.
- David N. Franz Led the EXIST-1 trial showing that the mTOR inhibitor everolimus shrinks the brain tumours (SEGAs) of tuberous sclerosis, and helped run the first sirolimus trial for its kidney tumours
- John J. Bissler Led the first clinical trial showing that the mTOR inhibitor sirolimus shrinks kidney angiomyolipomas in tuberous sclerosis, and the EXIST-2 trial of everolimus for the same tumours
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