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Lessons for cancer drug treatment from tackling a non-cancerous overgrowth syndrome

Abnormal activity of the enzyme PI3K can drive cancer growth, and mutations in a PI3K subunit can sometimes lead to non-cancerous overgrowth. A cancer drug that inhibits PI3K dramatically reduces such overgrowth.
  1. Robert K. Semple

    1. Robert K. Semple is in the Centre for Cardiovascular Science, University of Edinburgh, Edinburgh EH16 4TJ, UK.

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  2. Bart Vanhaesebroeck

    1. Bart Vanhaesebroeck is in the UCL Cancer Institute, University College London, London WC1E 6BT, UK.

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Researchers who investigate rare genetic conditions live in hope that the discovery of disease-causing mutations will lead swiftly to tailored treatments. Sadly, this is not often the case, because genetic defects usually cause impairments that are difficult or impossible to tackle using available medicines. Writing in Nature, Venot et al.1 now provide a rare exception to this rule. In severe non-cancerous overgrowth syndromes caused by mutations in an enzyme called PI3K, they show the beneficial effects of a PI3K-inhibitor drug that was initially developed to treat cancer. Their results bring the possibility of a transformative therapy for people with overgrowth conditions one step closer.

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Nature 558, 523-525 (2018)

doi: https://doi.org/10.1038/d41586-018-05365-w

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Competing Interests

Bart Vanhaesebroeck is a consultant to Karus Therapeutics (Oxford, UK), iOnctura (Geneva, Switzerland) and Venthera (Palo Alto, California).

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