Novartis Research Foundation's FMI Director Susan Gasser: An Interview With "The Pink Sheet" DAILY (Part 2 Of 2)

The Director of the Friedrich Miescher Institute for Biomedical Research talks about the orphan drug space, and big pharma's growing interest in it.

The Friedrich Miescher Institute for Biomedical Research, based in Basel, Switzerland, is the discovery branch of the Novartis Research Foundation, which options its research findings to Novartis. The institute was established as an independent foundation in 1970 by two separate companies, Ciba and J.R. Geigy, which were later combined and merged with Sandoz in 1996 to create Novartis. In the second installment, Director Susan Gasser talks to "The Pink Sheet" DAILY about big pharma's growing interest in the orphan disease space. In the first part, she talked about the FMI's research focus and relationship with Novartis (Also see "Novartis Research Foundation's FMI Director Susan Gasser: An Interview With "The Pink Sheet" DAILY (Part 1 Of 2)" - Pink Sheet, 22 Sep, 2010.).

"The Pink Sheet" DAILY: Novartis has talked a lot about focusing early research on small market indications and rare diseases to better understand mechanisms of action before pushing into larger indications. Is that a philosophy that FMI adheres to?

Gasser: We are looking at rare diseases, particularly rare diseases that have genetic inheritable defects. If you can develop a drug to restore a specific target that is effective in a rare disease, that is a proof of principle. You can then go from there to more common diseases that have perhaps the same pathway observed. For a basic biomedical scientist, it's a really good way to study disease because it is something we have clear genetic definition of.

NIBR President Mark Fishman's bet is that once you get these drugs and they're effective against even small populations, it will open doors to more common diseases. It's a strategy I adhere to.

At NIBR, Fishman has been a proponent of first identifying a molecule's pathway and method of action, then developing the drug for a targeted disease mediated by that pathway, before moving into broader indications (Also see "Novartis Has Proof Of Concept For New R&D Model, And NMEs To Show It" - Pink Sheet, 8 Dec, 2008.).

"The Pink Sheet" DAILY: What do you think about indications that big pharma is more interested in getting involved in the orphan drug space?

Gasser: It's definitely scientifically defensible, and time will tell if that's going to be the new mode of discovery. The other real advantage of it, not from the scientist point of view, but from a drug development point of view [is that] if you have a very precise indication in a subpopulation of the world ... you're going to get that approved for that indication very easily.

One of the reasons getting the drugs for cardiovascular disease, diabetes approved is challenging is that you are working with many different causes.

[The pharmaceutical industry] needs drugs in the pipeline that it knows are going to get approval because it has a very targeted disease to cure. It is going to cure it, and then is going to see, what have I learned from that? Can I go a step from there to a more frequent or more common disease? From a scientists' point of view, that strategy is great. It's something very well defined. From the point of view of financial strategy, I think it is there. The only thing we don't have a lot of proof for is how many of these rare disease models will be stepping stones.

"The Pink Sheet" DAILY: What orphan disease research are you doing?

Gasser: I'm working on Emery-Dreiffus Muscular Dystrophy. It's a rare genetic disease, which affects a protein of the nucleus of the cell. It's only manifested in the muscle ... We've been working on the question why is it muscle-specific. It's a protein that determines how the genes are organized in the cell nucleus. It's found in every cell that is in your body, and the interesting question is why does it affect muscle and can we change it in some way ... We can recreate this orphan disease in a model organism and then in a short time make a lot of progress on figuring out how to reverse the effects. We wouldn't develop the drug, but if Novartis is interested, we could give them some tools to do some screens and try to develop something. I'm not sure they are interested actually because we have to see how much a rare disease like that can be generalized.

"The Pink Sheet" DAILY: Muscular dystrophy is a big area for drug development though.

Gasser: Muscular dystrophy is big. Again, this is a rare form of muscular dystrophy, but if something comes from that, it may be a foot in the door for more common muscular dystrophy. That's the strategy one takes nowadays.

-Jessica Merrill ([email protected])