The patients view: Patients with a (re)purpose







How patients can help pharma repurpose existing drugs

 

Pharmaceutical companies are now heavily focused on finding new uses for the assets that have made them healthy profits over many years.

These assets-chemical compounds, genes, not to mention the considerable eco-system (processes, partners, scientific research) that enables drugs to be developed-are often past the date by which they can generate big profits.

But rather than let these assets go to waste, pharmas are looking for new opportunities.

So the new word we are all getting used to is repurposing'; in simple terms, finding new uses for these now familiar drugs.

This, of course, makes eminent sense, since huge investments have been made to bring these assets to a state where they benefit patients.

If, with some tweaks and modifications, they can now be made to benefit other diseases then, in theory, much can be gained for minimal new investment.

This repurposing could perhaps also generate new drugs much faster, as some of the clinical trial stages might be fast-tracked.

Recently, I noted a news item that showed how one non-pharma patient organization is picking up on this idea.

The Myelin Repair Foundation (MRF), a California-based non-profit seeking ways to repair the myelin damage from multiple sclerosis, claims to have identified 150 drug targets, of which more than a quarter are already on the market as treatments for other conditions.

Slightly more than another quarter comprise drug targets that are in discovery or in preclinical stages of development, once again for conditions other than MS.

So, out of the total number of potential compounds that the MRF think could be useful, about half are already out there' being used or trialed for other conditions.

This is just one example, but if it were replicated even in a small way with other diseases it would have powerful implications.

If half of the drug targets that could be useful to my disease were already out there and in use, the potential options for cures would suddenly look a whole lot more promising than they did yesterday.

There are obvious objections.

The key one is identifying those 150 potential drug targets in the first place.

The Myelin Repair Foundation, through the inspired vision of its founder Scott Johnson, controls an extensive and dedicated group of scientists who have done just that.

Identifying the initial group of targets is therefore the difficult bit; analyzing these against what's being considered for other diseases is arguably much easier since it's largely an information analysis task.

But it's still not easy.

The average patient advocacy group, especially those for rare diseases, doesn't usually have access to the science resources needed to identify compounds that might be repurposed.

But I suspect that this challenge is something that patient groups could step up to.

Just as Wikipedia found, if you set up a website to crunch this type of analysis, there are probably many retired scientists or younger hopefuls who would relish this kind of challenge.