siRNA is one of the molecules of future medicine, with some success at early clinical trials. How will this molecule affect the future of therapeutics?
Twelve years ago, a novel mechanism for shutting down genes has been described in a nature paper. The mechanism described made use of molecules called siRNA – Small Inhibitory RNA – whose potential for the development of new therapies for diseases like cancer has been recognized immediately.
Last year, Dr. Dan Peer managed to create liposomes – vesicles made of fatty acids – that carried siRNA to the immune cells of mice. The siRNA were delivered selectively into the T cells and altered their function. Two weeks ago, a similar feat was accomplished by Caltech Scientists in a phase I clinical trial in cancer patients who were treated with siRNA-carrying nanoparticles. The nanoparticles were targeted to go into tumors and deliver the siRNA which inhibited the formation of a cell-growth protein rinonucleotide reductase.
The results of the clinical trial show that the nanoparticles successfully targeted the tumors and injected their siRNA delivery into the cancerous cells. A biopsy of the tumors revealed that the mRNA encoding the protein ribonucleotide reductase has been degraded, which in turn led to a loss of the protein.
The development of new therapeutics based on siRNA molecules seems to be on its way. However, there are still many problems with this approach. First off, siRNA are quite costly to manufacture, which will mean the medicine won’t be obtainable for most patients. Other than that, the siRNA can cause a severe immune response if not specifically targeted at the intended cells.
In the upcoming ILSI-BioMed 2010 week, Dr. Ora Horovitz will discuss novel delivery systems and the delivery of siRNA and other therapeutic molecules. I hope it will be an interesting lecture, and am eager to hear her thoughts on this molecule of the future.
A review of the future of siRNA, according to experts
Caltech’s new siRNA delivery system
An article in Hebrew about siRNA and Dr. Dan Peer
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