Hospital Sant Joan de Déu
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Found a molecule that could be used to block tumors developed in patients with Von Hippel-Lindau disease

Células en 3D
Wednesday, September 25, 2019

CSIC scientists have recently published a study in the journal Scientific Reports in which they demonstrate the therapeutic effect of a compound against Von Hippel-Lindau disease. This hereditary syndrome predisposes to the development of tumours, the most frequent being retinal, cerebellum, spinal hemangioblastoma, renal cell carcinoma and pheochromocytoma or tumour of the adrenal medulla. 

Von Hippel-Lindau patients have a mutation in the VHL gene that encodes for a protein that controls cell formation and multiplication. That's why the VHL gene is called a “tumour suppressor gene”. People affected by von Hippel-Lindau, therefore, have mutated that suppressor protein, making them prone to develop certain types of tumours, both benign and malignant. So far, there is no drug that works efficiently to alleviate these effects. Continuous surgery is the only way for patients to control the disease. 

Dr Luisa-María Botella's group has studied the effect of an orphan drug, propranolol, and another compound, ICI118,551, which specifically blocks adrenergic receptors β2. The researchers observed in cell cultures that ICI118,551 has the same antitumor properties already known in propranolol. The advantage of this new molecule is that, by specifically binding to the receptors β2, it does not produce the side effects seen in propranolol. This drug uniquely binds both the receptors β2 and β1, and this characteristic generates hypotension and bradycardia, undesirable effects for patients with hypotension.

This collaborative study, involving the Spanish Alliance of Von Hippel-Lindau families and neurosurgeon Daniel Aguirre, shows very promising results and brings us ever closer to an effective and safe treatment for patients with Von Hippel-Lindau. 

Access to the original article: The β2-adrenergic receptor antagonist ICI-118,551 blocks the constitutively activated HIF signalling in hemangioblastomas from von Hippel-Lindau disease. Scientific Reports.

Last modified: 
09/25/2019