Synthetic molecules deliver drugs directly to neuroblastoma

Thursday, 06 June, 2019

Synthetic molecules deliver drugs directly to neuroblastoma

A multidisciplinary team from diverse research centres in Madrid, including Universidad Politécnica de Madrid (UPM) and Universidad Complutense de Madrid (UCM), has developed synthetic molecules which are able to deliver drugs and diagnostic agents directly to neuroblastoma cells. Published in Angewandte Chemie International Edition, their work should increase the efficiency of cancer therapy and reduce unwanted side effects.

Neuroblastoma is a very aggressive paediatric tumour with a complex diagnosis when detected in the metastatic state. Current treatments consist of the administration of chemotherapeutic agents in order to kill cancer cells and slow the disease progression. Unfortunately, these drugs lack specificity against cancer cells and cause numerous side effects and high systemic toxicity, compromising the chances of recovery and quality of life of the patients.

In order to find new solutions to improve the treatment, various research centres and hospitals in Madrid commenced a study that is already showing good results. Researchers synthesised a family of molecules that are able to specifically bind a protein found in the cell membrane of more than 90% of neuroblastoma cells, the norepinephrine transporter (NET).

“These molecules have a small central structure composed by natural amino acids that work as scaffolds specifically designed to fit in the recognition centres of the NET protein in a similar way that a key fits the lock,” said Alejandro Baez, a researcher at UPM. “This way, the process of recognition is highly selective since the binding only occurs with the suitable ‘padlock’ which is only located in the surface of neuroblastoma cells, thus avoiding the healthy cells.”

The effectiveness of these molecular scaffolds was tested in lab mice suffering from neuroblastoma, revealing their capacity to transport drugs and diagnostic agents. The researchers are now working on applying this technology to the transport of antitumour drugs in neuroblastoma models similar to those that appear in humans.

The researchers are also studying the use of these systems for the improvement of early clinical diagnosis techniques of this disease. They expect that the future results will allow them to study applications in human within 2–5 years.

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