The Role of Propranolol as a Repurposed Drug in Rare Vascular Diseases.
Author: Cuesta, Angel M.; Gallardo-Vara, Eunate; Casado Vela, Juan; Recio-Poveda, Lucía; Botella, Luisa-María; Albiñana, Virginia
Abstract: Rare Diseases (RD) are defined by their prevalence in less than 5 in 10,000 of the general
population. Considered individually, each RD may seem insignificant, but together they add up to
more than 7000 different diseases. Research in RD is not attractive for pharmaceutical companies since
it is unlikely to recover development costs for medicines aimed to small numbers of patients. Since
most of these diseases are life threatening, this fact underscores the urgent need for treatments. Drug
repurposing consists of identifying new uses for approved drugs outside the scope of the original
medical indication. It is an alternative option in drug development and represents a viable and riskmanaged
strategy to develop for RDs. In 2008, the “off label” therapeutic benefits of propranolol were
described in the benign tumor Infantile Hemangioma. Propranolol, initially prescribed for high blood
pressure, irregular heart rate, essential tremor, and anxiety, has, in the last decade, shown increasing
evidence of its antiangiogenic, pro-apoptotic, vasoconstrictor and anti-inflammatory properties in
different RDs, including vascular or oncological pathologies. This review highlights the finished and
ongoing trials in which propranolol has arisen as a good repurposing drug for improving the health
condition in RDs.
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