Re(verse) Purposing to deliver MS drugs

I was giving a talk about animal models and their role in developing drugs to MS and I was asked by someone from Finland to post on this subject.

As you may know only two drugs (out of about two thousand tried) have their routes in animal MS models and these are glaterimer acetate and Tysabri, where their action was first found in MS models.

However in recent time I would bet that in most cases, MS drugs have been tried in MS models as part of the development pathway. Furthermore, we have to be aware that the development pathway for a drug is along time and many of those drugs active in animals may be things for the future.

But how do we get drugs to people with MS?

We can invent a drug and take it through the full development process (See Drug the Board Game). This takes about 10-15 years with pharma driving this. For my symptom control drug without sedative side-effects (work not yet published)… has been 12 years so far (first experiment done 25:11:2002 and we had to invent and make the compound first) and we still have phase II/III and licensing to go.

The quick way to develop drugs is to repurpose a drug. Here the idea is to take a drug that has been through the full development process for another disease so it is known to be safe in humans and then use the drug in MS to show it works and is safe and Bobs your uncle you have an MS drug…so that’s the theory.

The Wellcome Trust and the Department of Health in the UK and the National Institute of Health have been putting millions into this process. However, I wonder if any of them have really thought the process through. Can we really repurpose drugs for MS?

There are two ways of repurposing the academic way and the pharma way.


The academic to take a cheap drug and then do a trial as academics are good at doing trials…but then what?

For a rapidly life threatening condition it is easier to push things forward…take the new anti-ebola virus drugs. For Chronic diseases the hurdles are higher.

Academics sit on committees like the FDA and have said to industry we don’t believe your trial so go away and do another one and then hold a licence and have a safety monitoring structure and this all adds to the cost and the time taken to develop drugs.
Industry are going to say to academic neuros….. fine do a trial, but it should be a level playing field and so academics need to do the process properly just as pharma are made do it.

Could an academic get funded to do the same trial twice? I doubt any grant agency would support this. Some academics think that you do a trial and ever one will believe their data and prescribe the drug.

Is this Hope or False Hope or Bob Hope?

More likely it will show pharma what drugs could work in MS and they will then go into their drug libraries and come up with a new patented drugs or will repurpose the pharma way.

However, what have academic Neuros delivered…..lots of trials…but so far no drugs in recent history, unless pharma are involved!

So here is the challenge for all academic Neuros doing clinical trials…how do you get your repurposed drug to people with MS?

If it can’t be realistically done, is it ethical to start?
If it can’t be done, what needs to put in place to make this happen?
This may need changes in Government policy

If Governments would protect industry such that they can recoup their trial costs etc with a generic drug there will be an incentive to really repurpose drugs.

However, MS pharma have been repurposing drugs for years and this is the MS way.

The pharma way to repurposing is to find a drug that is safe in humans and then get a patent on it.

This may mean taking a drug that works in another disease and re-engineer them to make them work in MS.

This repurposing approach is so successful that MS pharma is repurposing its own drugs. Such that we have pegylated interferon to replace interferon and Copaxone three times a week (tiw) to replace Copaxone once a day (qd).

Funny that these drugs arrive as the patent for the founder drugs expire….Will we get a new tysabri once every 6 months in the future or the anti-CD49d pill?

However, the pharma way to repurposing can be more simple, take a drug and make it hang round in the system for longer e.g. Fampryidine from 4-amino pyridine.

They may spend millions taking a generic drug then re-engineering it to make a patented drug. For example making a pro-drug which converts into the active metabolite or just developing the active metabolite like, Aubagio.

What does this say…well the generic parent drug will be active also eg. Leflunoamide

Should academics show this, to deliver cheap MS drugs to the world? They could do the reverse development (reverse purposing) and take an active pharma drug and show that the generic drug works.

However, if they use the academic way …what will this deliver based on past record? As many of the readers of the Blog are Neuros…This is your challenge that needs change.

There was a comment last week chartising Pharma Neuros, but as they point out at least they can say that they are helping delivering treatments to people. Can academic neuros get this reward for their labours?

How do you change the academic way so that it actually delivers useful treatments for people with MS?

This is important as we are in a process of hunting for neuroprotectant and repair agents using repurposing

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  • Was this meant to make me laugh or cry? Everything you write here rings so true from what I know. The repurposing lately has conveniently happened as the patent clock ticks down to zero. Infuriating? Yes. It took me a while to understand the complex relationship of our neurologists and Pharma here in the US. Without pharma, I am sure there would be very little our neuros could offer us. As angry as I get at their profit taking at my expense as a person with MS and with good insurance, I am also realistic and know they are the only ones able to offer me hope to coexist with my MS. It is a true love/hate relationship and I wish there were a way that academic research could break through the hold Pharma has on us and create meaningful and usable treatment knowledge.

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