Research Question; The One-Off Cure

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Whilst Trying to Gag and Complaining About Prof G to our Bosses You Wrote: “Under what conditions could anyone [even a Neurologist] propose a one-time cure [not a more effective maintenance and management drug regime] for MS without falling victim to pharmaceutical influence? How do MS families work through their elected representatives to ensure such conditions exist?”

The considerable costs of clinical trials, which is an essential part of drug development mean that they are difficult to undertake without pharmaceutical company support. However, the infrastructure is already there to undertake investigator-led studies that are motivated by science and not profit. Indeed there are many studies ongoing at the moment that are fulfilling this aim.


To come up with the idea and get funding to show proof of principle for a one-off cure is readily achievable, without the pharmaceutical industry knowing anything about it, so they can’t influence it. Doing the clinical trials are the costly bits.

There are agencies such as the MS Societies (which you can lobby to undertake studies and indeed do so given the evidence of some of the studies they are supporting) and the Medical Research Council in the UK and NIH in the USA, and others, that have the capacity to fund such studies. However, these studies will take a considerably longer period of time to adequately fund and get off the ground compared to the speed that big pharma can do it. Therefore, if you have a one-time cure then it is feasible to undertake studies, without being in the pocket of current companies.

In animal models this one-time cure is probably already achievable, but in MS it is more complex, but the earlier you start treatment the greater is the chance of success. Team G is in the process of testing such a strategy, developed in our lab! However, if you need to generate a new drug, manufacture of clinical grade chemicals is hugely expensive. You will probably need to set up a company to manage the funds and the study, so do you become the pharmaceutical industry?

Would pharmaceutical companies go for a one-time cure, well maybe if the price is right. If you look at Alemtuzumab at the moment then you may be only getting a couple of courses of drug. This may be not that far from a one-off treatment. We will have to wait and see if it is a cure. We have seen in previous posts how much Pharma may be planning to charge. This may have been very different had Merck Serono not withdrawn their oral version of cladribine (movecto), which would have probably been sold at a considerably cheaper price. Cladribine would have been taken in a similar way to Alemtuzumab, and may be as effective as Alemtuzumab in some groups of patients, but lacks some of the serious adverse side effects of Alemtuzumab. There may be a cancer risk with cladribine but this is going to be a possibility with any immunosuppressive drug of this nature.

However, the success of oral cladribine, would have woken people up to the fact that intravenous cladribine would be just as good and would be several times cheaper and could be supplied by any generics drug company, as intravenous cladribine is off-patent. This could kill the golden goose that MS has become for companies, as you could have a cheap, effective drug. So you MSers should lobby your MS Societies to investigate this and get it licensed! Cladribine is not a toxic evil, but a potential opportunity that should not be missed.

There are enough people out there, me included, that would bite your arm off for the chance to find and bring a one-off cure to people with MS, irrespective of what the pharmaceutical industry would do. If the pharmaceutical industry come up with it, it will cost, but they have been doing one-off vaccinations that are cures and saves millions of lives for many, many years. The difficultly in not the conspiracy, it is finding the one-off cure, to do that it would help to know the cause, then you can Think Prevent.

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MouseDoctor

10 comments

  • This a good post. I liked reading it.

    Do you think what the Cambridge Centre for Remyelination is doing, as in Prof. Franklin's work on RXR-gamma, may be the potential panacea for MS, and thus, a one-off cure?

  • Please can you post something about your thoughts regarding pharmaceutical companies in the UK not being able to protect their investment by not being allowed to patent the results of their research.

    Are you sympathetic or do you think the big pharmas can go take a hike?

    Also, what does this ruling mean for those of us with MS?

    Ta.

  • Professor Robin Lovell-badge wrote an article in the Daily Mail today saying that ruling did not even mention the potential for stem cells to treat conditions like MS. It merely talked about the ‘commercialisation’ of embryos as if we were trading in body parts.

    Lovell-badge says that the UK is going to fall behind now as a result of this ruling..

  • Re: "Do you think what the Cambridge Centre for Remyelination is doing, as in Prof. Franklin's work on RXR-gamma, may be the potential panacea for MS, and thus, a one-off cure?"

    I must remind you that all the initial work on RXR was done in rodents. Not all discoveries in rodents pan out in humans. Until the biology has been shown t be the same in humans, which is likely, it is difficult to make a call on this. So no predictions possible at this stage.

    Another warning one of the problems with RXR biology is that it is used by many systems within the body, therefore there is likely to be off target effects from small molecules targeting this factor. The latter may be a problem, although it is likely you will only take the medication for a short period of time to stimulate myelination and then it could theoretically be stopped.

  • Re: "Please can you post something about your thoughts regarding pharmaceutical companies in the UK not being able to protect their investment by not being allowed to patent the results of their research."

    I am not sure this is correct; patent law is generally EU wide so patents are easy to enforce under EU law.

    "Are you sympathetic or do you think the big pharmas can go take a hike?"

    I don't think big pharma should take a hike. It costs ~$800M (2004 figures) to develop a new drug. With some economists reporting that this figure has now gone above $1 billion. There is no way we can develop new drugs without Pharma. So we need to engage them with MS and keep them engaged. On the other side of the coin Big Pharma needs to clean up its act; some companies have a reputational problem and the MS field is no exception. But we can't tar all companies with the same brush, which is why I would prefer not to make sweeping generalisations.

    A bigger problem is the lower drug costs in the EU compared to the USA. At the moment the US is subsidising the price of drugs across the world. At some stage this will have to stop. I can't see the US consumer agreeing to this indefinitely. This means the costs of drugs in Europe, and in particular the UK, will increase in the future. This may affect NICE's cost-effectiveness calculations for new treatments. I suspect that we won't be able to prescribe drugs some of the newer drugs under the NHS because of this problem. I sincerely hope that this does not happen.

  • Re: "… it is likely you will only take the [RXR] medication for a short period of time to stimulate myelination and then it could theoretically be stopped."

    If the drugs work, which you believe is likely, then that to me sounds like a promising one-off cure for MS, no?

  • With regard to the getting myelin back onto nerves (as an aim of the myelin repair centres). This has to be a desired outcome. However, surely one must need something else to deal with the problem that caused the myelin to go in the first place so it is unlikely to be a one-off cure in itself. However it could be part of the jigsaw.

  • The thought that if funding doesn't come through for stem cell workers in the UK, they will move to US as mentioned in the daily mail. This could be applied to many scientists in the UK.

    There is a funding squeeze and many things are moving eatwards (China/India). Hence the increasing disappearence of pharma in the UK

  • Good points MouseDoctor however perhaps moving into developing nations with ballooning economies is simply a business decision for large pharma… locations like China represent huge exploitable markets where demand for product coexists with an increasing ability to pay. Further, cost of product development / approval / manufacturing is less in such locations… it doesn't cost $1 billion dollars to get a drug approved there.

    China is a nation of 1.3 Billion people where 80% of males smoke… so if smoking is associated with increased risk of MS, well you get the picture.

  • Good point, I agree with you and accept your comments

    Maybe these countries are an emerging market hence the relocation. However I still think that many African Nations produce the best long-distance runners but their shoes are still probabably made in taiwan. Does it really matter where things are made relative to the market verses the cost of production of the goods?

    Economics can be used to justify many things but in terms of MS the demographics have yet to show that MS is a disease of the Far East

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