RT Journal Article
T1 The Role of Propranolol as a Repurposed Drug in Rare Vascular Diseases
A1 Cuesta Martínez, Ángel
A1 Gallardo Vara, Eunate María
A1 Casado Vela, Juan
A1 Recio Poveda, Lucía
A1 Botella, Luisa María
A1 Albiñana, Virginia
AB 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 risk-managed 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.
PB MPDI
SN 1422-0067
YR 2022
FD 2022-04-11
LK https://hdl.handle.net/20.500.14352/71816
UL https://hdl.handle.net/20.500.14352/71816
LA eng
NO Ministerio de Economía y Competitividad (MINECO)
NO CSIC (National Research Council of Spain)
DS Docta Complutense
RD 20 ago 2024