Can Petunias Cure Cancer?

Five years ago, the most respected US scientific magazine in the world--Science--called RNA interference (RNAi) the breakthrough technology of the year. And it was only about three years before that when the magazine published a paper about a radically new mechanism that cells can use to control protein production that was discovered by plant geneticists David Baulcombe and Andrew Hamilton.

Double-stranded RNA has an almost mystical ability to efficiently and potently silence any gene in plants, worms, fruit flies, or (most recently) mice. What Baulcombe and Hamilton discovered in plants was then reproduced a couple years later in mammals by Tom Tuschl at the Max Planck Institute in Germany, using mice. In essence, you could use short interfering RNA strands (siRNA) and stop specific genes from reproducing.

While the initial work was done simply to see if you could alter purple petunias’ gene sequence to get a more purple petunia, the experiment moved in a different direction and RNAi was discovered. From there, science has gotten to the point where most biomedical scientists would likely say RNAi is one of the most significant discoveries in medicine in decades.

Simply put, being able to silence genes effectively means you can stop many cancers from growing. Can you imagine stopping malignant cancers in their tracks? How about stopping and destroying tumors, especially inoperable tumors? That’s the kind of thing we’re talking about here. The implications are huge.

Every major pharmaceutical company is searching our development partnerships with small specialized firms that are already populating the RNAi niche. Most of the firms are developing unique methods of getting the siRNA to the target cells. But this is a mad race for capitalization as well. That means some of these firms are looking for investors but don’t really have much of a business plan.

I checked out a few Web sites and some of the companies talk about RNAi but don’t go into what their product is or how it works. Or, more important, they don’t explain why their products are so unique they’ll absorb all the demand or even stifle any second wave competition. Just being first in Food and Drug Administration (FDA) trials doesn’t mean your product is the best or that your company is worthy of a super-premium buyout.

Below are a few links to some companies I've found in this niche sector. The first one, Starpharma (www.starpharma.com), has been a favorite of mine for some time, and I cover it in my paid service Real Nanotech Investor. The second company, Alnylam (www.alnylam.com), is something I just stumbled upon as I was doing research for this article, but its pedigree is intriguing and may be worth some serious interest. But I’m going to have to take some more time with it.

As for the latter two, Intradigm (www.intradigm.com) and Calador Pharmaceuticals (www.caladorpharmaceuticals.com), have hit at least my yellow flags. The former’s claim to fame is its first in trials in the RNAi sector. The latter has licensed technology from the California Institute of Technology for development in the field and is partnered with Arrowhead Research, a publicly traded nanotech aggregator.

While they both have solid partners, their strategies articulated very well, which either means they haven’t really thought about how they’re going to pull off the business side, or they’re just hoping someone will dump a lot of money in their laps for being at the right place at the right time.

The reason I like Starpharma in this space is because it's not selling siRNA technology, it's selling delivery vehicles for RNAi. Its dendrimers make uniquely appealing delivery devices for siRNA.

You see, the biggest problem with RNAi as it stands now is the siRNA isn't very consistent in its gene silencing abilities and it doesn't always make it to its intended site before the body's natural defenses sense an intruder and destroy it. Nanotech is extremely helpful in fashioning particles that are small enough to go undetected by most immune system defenders. Getting the siRNA to be more consistently effective may take more time.

I was on a flight once with an epidemiologist from a leading university who specialized in studying worms and flies. It may seem tedious, but because their life cycles are so short, they genetically modify in weeks what it would take decades to observe in human genetic transformation. Plus, much of the initial research in siRNA was done on worms. His take was while there's much promise, siRNA is very fragile, and until siRNA can become more robust or dropped in "behind the lines," this whole RNAi thing may simply be a tempest in a teapot.

Either way, it's a moot point for Starpharma, since its simply selling dendrimers to the researchers who are looking for the most effective way to deliver RNAi. This lab business will be booming, especially because initial studies indicate that dendrimers are very good at getting their payload--in this case siRNA--to where it has to go in the body because the body's immune system doesn't view dendrimers as outside invaders.

This is a boon to RNAi researchers since they can stop worrying about the delivery challenge to RNAi therapy and concentrate on siRNA efficacy. For Starpharma and other dendrimer manufacturing firms, this means lots of new business and a growing long-term client list.

Expect to hear more about siRNA or RNAi coming years, but don't get caught up in the hype. This is at best five to 10 years out, so any company you plan to invest in now is going to be a development-stage company for a while. That means you have some time to see how the landscape develops for this "miracle cure."

Don't get greedy--you're not going to miss the boat on this one, especially if you already know the story.

For more information on siRNA and RNAi, please visit The National Cancer Institute's Web site, and check out the Journal of the National Cancer Institute.