Can MicroRNA Reverse Heart Disease?

Includes: ALNY, IONS
by: Eben Tessari

Regulus Therapeutics, a joint venture between Alnylam (NASDAQ:ALNY) and Isis (ISIS) formed to discover, develop, and commercialize microRNA-based therapeutics, announced the publication of new research in the journal Nature on the role of a microRNA, known as miR-21, in heart failure.

Apparently, this is the first time a microRNA drug has reversed or halted a disease (not biomarkers for the disease, but the disease itself) in an animal model. Interesting science no doubt, but before we anoint microRNA therapy as the hottest area in drug development we should keep in mind a few things:

1.) A rodent heart, while a decent quick and dirty model, is vastly less complicated than even a dog or pig heart. Proof in these models would be much more convincing. Of course, you do the rodent model first… I’m just saying we shouldn’t put too much stock in curing a disease in an inferior animal model.

2.) Dosing by injection to the chest 3x a day is rather inconvenient. I would think patient compliance might be an issue (tongue in cheek). Of course, the formulation will be improved but the main issue with all of these oligo / RNAi type drugs is the delivery. Great research tools… terrible drugs and until I’m proven wrong, I invest based on a search for drugs (reward), not fancy lab techniques (risk).

I’m not trying to be a hater here, but when a company puts out a press release saying they have positive animal data, I’m always skeptical of the motives. The study is published in Nature… that should be all the publicity you need.