Alnylam joins Spanish team to deliver successful RNAi

VHIO Director of Clinical
Research Josep Tabernero--Courtesy of VHIO

Researchers at Barcelona's Vall d'Hebron Institute of Oncology (VHIO) in Spain and U.S. biotech Alnylam Pharmaceuticals ($ALNY) have shown that, with a novel delivery technique, they can actively "silence" cancer genes with molecules using the selective interference molecule, RNAi.

RNAi has been known to temper tumor cells' growth by shutting down genes that become hyperactive in cancer patients. But administering drugs using the approach is problematic--the drugs must penetrate cells in high enough concentrations to be effective but also retain their structural integrity.

To overcome this obstacle, VHIO and Alnylam are using a lipid nanoparticle to deliver the RNAi drug ALN-VSP, which targets and suppresses the two genes that code for vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP). Both of these proteins have been shown to induce cancer.

In a Phase I clinical trial of 41 patients with cancer that had metastasized in the liver, the drug demonstrated promising results, according to VHIO. But researchers have struggled to translate Phase I results into late-stage success in the RNAi-delivery arena. Big Pharma behemoths like Novartis ($NVS), Merck ($MRK) and Roche ($RHHBY) have cut back on their RNAi lines the last few years due to the difficulty of getting the drugs where they need to be.

But Alnylam and VHIO are confident in the preliminary trial results. VHIO Director of Clinical Research Josep Tabernero said in a statement: "This is the first evidence to show that RNAi can be administered to cancer patients effectively, leading to significant tumor response."

- here's the release