Lisa Young
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
Chief, Division of Pulmonary and Sleep Medicine, Children's Hospital of Philadelphia · Professor of Pediatrics, Perelman School of Medicine, University of Pennsylvania · Vanderbilt University (from 2011) · University of Cincinnati and Cincinnati Children's Hospital (fellowship and six years on faculty)
Portrait: Children's Hospital of Philadelphia
Lymphangioleiomyomatosis (LAM) is a rare lung disease, almost only in women, in which abnormal muscle-like cells with an overactive mTOR pathway slowly fill the lungs with cysts. It can also occur in tuberous sclerosis complex, together with kidney tumours called angiomyolipomas. Lisa Young was a co-author of the two Cincinnati-led trials that first tested sirolimus (rapamycin) against these problems. In the 2008 trial (BIS2008), a year of sirolimus shrank kidney angiomyolipomas in patients with TSC or LAM, though they grew back after the drug stopped. The larger MILES trial (MCC2011) compared sirolimus with placebo in 89 women with LAM: lung function held steady on sirolimus while it kept falling on placebo, and the benefit faded once the drug was stopped. MILES led to sirolimus becoming the standard treatment for LAM.
Young studied at the University of Virginia, earned an MD at Duke University and trained there in both paediatrics and internal medicine. A double fellowship in paediatric pulmonology and adult pulmonary and critical care followed at Cincinnati Children's Hospital and the University of Cincinnati, where Young then spent six years on the faculty, the period of the two trials. In 2011 Young moved to Vanderbilt University and later to Philadelphia.
Young is now Chief of the Division of Pulmonary and Sleep Medicine at the Children's Hospital of Philadelphia and Professor of Pediatrics at the University of Pennsylvania. Research topics include LAM and other rare lung diseases such as Hermansky-Pudlak syndrome and neuroendocrine cell hyperplasia of infancy, as well as lung development, injury and repair.
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 Co-author of the Cincinnati trial showing that sirolimus shrinks kidney angiomyolipomas in TSC and LAM. |
| 2011 | H | Efficacy and safety of sirolimus in lymphangioleiomyomatosis (MILES) MCC2011 Co-author of the MILES trial, in which sirolimus stabilised lung function in women with LAM while it was taken. |
Co-authors in the Atlas 4
People with a profile here who share at least one study with Lisa Young.
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- Francis X. McCormack 2 shared studies 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.
- 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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