Sergiusz Jóźwiak
A child neurologist known for work on tuberous sclerosis complex and a senior author of the EXIST-1 trial of everolimus.
Department of Child Neurology, Medical University of Warsaw
Department of Child Neurology, Medical University of Warsaw (Warsaw, Poland) ↗
Portrait: Wikimedia Commons
Sergiusz Jóźwiak is a child neurologist at the Medical University of Warsaw in Poland. He works on tuberous sclerosis complex (TSC), a genetic disease in which the mTOR pathway is permanently switched on. He has co-edited a collection of articles on TSC diagnosis and management.
EXIST-1 was a phase 3 trial in 117 people with TSC and brain tumors called subependymal giant cell astrocytomas. Patients got everolimus or placebo in a 2 to 1 ratio. In 35% of those on everolimus the tumors shrank by at least 50%, compared with none on placebo, and side effects were mostly mild to moderate.
Because TSC is caused by overactive mTOR, this is among the clearest randomized results that blocking mTOR helps in a genetically defined disease. It does not show the same benefit in people without such a mutation.
Milestones in the Atlas
A selection, not a full bibliography: only papers that meet the Atlas inclusion criteria.
| Year | Evidence | Study |
|---|---|---|
| 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 Senior author on EXIST-1, the phase 3 trial (n=117) in which everolimus shrank TSC-related brain tumors by 50% or more in 35% of patients versus none on placebo. |
Co-authors in the Atlas 6
People with a profile here who share at least one study with Sergiusz Jóźwiak.
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- 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
- 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
- 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
- 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
- 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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