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
Professor of Pediatrics, Univ. of Cincinnati · Founding Director, TSC Clinic, Cincinnati Children's · MD, pediatric neurologist
Tuberous Sclerosis Clinic, Cincinnati Children's Hospital Medical Center (Cincinnati, Ohio, USA) ↗
Portrait: Cincinnati Children's Hospital Medical Center
Led EXIST-1 (FRA2013, n=117): everolimus shrank SEGA brain tumors ≥50% in 35% vs 0% placebo — an alternative to high-risk neurosurgery. Collaborator on Bissler's 2008 sirolimus trial (BIS2008), groundwork for EXIST-1/2.
Founded and directs one of the longest-running, highest-volume TSC clinics in the US.
Milestones in the Atlas
A selection, not a full bibliography: only papers that meet the Atlas inclusion criteria.
| Year | Evidence | Study |
|---|---|---|
| 2006 | H | Rapamycin causes regression of astrocytomas in tuberous sclerosis complex FRA2006 First author: in five people with TSC, oral rapamycin shrank growing brain astrocytomas, and in one patient the tumour regrew when treatment was paused. |
| 2008 | H | Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis BIS2008 Collaborated on the earlier sirolimus trial establishing groundwork for EXIST trials. |
| 2010 | H | Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis KRU2010 In patients whose TSC1/TSC2 mutations cause brain tumors, everolimus shrank tumor volume by 30%+ in three-quarters of patients. Open-label and uncontrolled (n=28); randomised confirmation came later (FRA2013). |
| 2013 | H | Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial FRA2013 Led EXIST-1, proving everolimus shrinks SEGA brain tumors — cleanest randomized evidence blocking mTOR treats a genetic mTORopathy. |
| 2016 | H | Adjunctive everolimus therapy for treatment-resistant focal-onset seizures associated with tuberous sclerosis (EXIST-3): a phase 3, randomised, double-blind, placebo-controlled study FRE2016 Senior author of EXIST-3, the phase 3 trial in which add-on everolimus reduced treatment-resistant seizures in TSC in a dose-dependent way (response 28% and 40% vs 15% on placebo). |
Co-authors in the Atlas 14
People with a profile here who share at least one study with David N. Franz.
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- Darcy A. Krueger Child neurologist who led the first trial of an mTOR inhibitor for brain tumours in tuberous sclerosis complex
- David Lebwohl Medical oncologist and drug developer who led the clinical development of everolimus (Afinitor) at Novartis through approvals in several cancers and tuberous sclerosis, and later served as Chief Medical Officer of the gene-editing company Intellia Therapeutics
- Elena Belousova Child neurologist and epilepsy specialist in Moscow who heads a department of psychoneurology and epileptology and has been a principal investigator in international drug trials for tuberous sclerosis complex
- 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.
- Jacqueline A. French Epilepsy clinical-trial specialist; first author of EXIST-3, the placebo-controlled phase 3 trial in which everolimus reduced treatment-resistant seizures in tuberous sclerosis complex.
- 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
- Kerry R. Crone Pediatric neurosurgeon in Cincinnati; senior author of the small case series in which oral rapamycin shrank brain astrocytomas in patients with tuberous sclerosis complex.
- 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
- Michael D. Frost Co-investigator on the EXIST-1 and EXIST-2 trials showing that the mTOR inhibitor everolimus shrinks tuberous sclerosis tumours in the brain and kidney
- Paolo Curatolo Child neurologist at Tor Vergata University in Rome who has spent much of a long career on tuberous sclerosis complex, the genetic disease of overactive mTOR, with a focus on epilepsy and brain development in children
- Petrus J. de Vries Child and adolescent psychiatrist at the University of Cape Town known for work on TAND, the behavioural, learning and mental-health problems of tuberous sclerosis complex, and co-author of major everolimus trials in TSC
- Sergiusz Jóźwiak A child neurologist known for work on tuberous sclerosis complex and a senior author of the EXIST-1 trial of everolimus.
- Tarek Sahmoud Physician and epidemiologist who worked as a clinical program head in Novartis oncology drug development during the everolimus trials, and later as chief medical officer at several cancer drug companies
- Vicky H Whittemore Neuroscientist and NIH program director for epilepsy and ME/CFS research who spent over two decades working for patient organisations, including the Tuberous Sclerosis Alliance
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