I want to see treatments in the FDA pipeline. I don’t care about published research, it’s too slow, too cumbersome, and too political. There is not enough funding for the perfect research papers we want to see published; let’s all be realistic. We need Pharma onboard. We need to take action.
But how do you get Pharma on board if we have no idea what we want them to produce? I have been to talk at a big Pharma company just to let them know what is going on in the ME/CFS research world. They were interested enough to toss some ideas around but there was not the slightest indication that they would start a development programme. There is no lead. Pharma jump in when academia has found a lead. By and large Pharma people do not have any imagination. They want to be told what to synthesise. We can only do that if we have solid published science. It is not the publishing that is slow and cumbersome, it is finding a lead that is worth publishing, which is pretty much the same as worth developing.
John Chia has already got pharmaceutical companies on board, by explaining to them the need for good enterovirus antivirals. In the past, pharmaceutical companies have not wanted to invest in developing enterovirus antivirals, because they were only aware of one enterovirus infection that antivirals might be clinically useful for, enterovirus 71, which being a relatively rare and short-term infection, will not make much profit for the companies. Well, that and rhinovirus, an enterovirus that causes the common cold. But Dr Chia has courted and explained to pharmaceutical companies that ME/CFS is linked to chronic enterovirus infection, and that for treating ME/CFS, enterovirus antivirals would probably have to be taken on a long term basis, perhaps indefinitely, in order to treat the patients. That suddenly makes a good anti-enterovirus drug very profitable, with there being 17 million ME/CFS patients worldwide. The pharmaceutical companies were not previously aware of the likely role of enterovirus in ME/CFS, but Dr Chia made them aware of this. Apparently a couple of years ago, some pharmaceutical companies started advertising for jobs for enterovirus experts, indicating that these companies may have got the message about enterovirus, and may have started to develop an antiviral. Chronic non-cytolytic enterovirus infection is not only linked to ME/CFS, but also to type 1 diabetes, chronic myocarditis, dilated cardiomyopathy, and others. So potentially an enterovirus antiviral may provide major benefits in all these diseases.
Honestly, if I have to drive this agenda single handed, then I will. It’s really up to Pharma to listen or ignore me. I have sat back for way too long on patient forums, waiting for things to change, and nothing changes. Again and again, we have been told by many researchers that ME research needs about $250M annually. The problem is that ME research is not funded that way nor are there any plans in the works to do so. So I can continue to choose and sit around and wait for THAT day, assuming it comes in my life time, or I can take a risk now and give it my best shot. Re Pharma, I am prepared for complete and utter failure. But I am persistent, because I know that it just takes one person to listen, to make a difference.
Unfortunately I did not see the job advert myself, but read an article in which the author claimed that some pharma company or companies were advertising for enterovirus experts, and the author concluded this was a sign they were going to develop an antiviral. You might start with writing to Janssen Pharmaceuticals, a company that has in its portfolio a drug called Staphypan, which in clinical trials lead to major benefits in ME/CFS. Staphypan was used in clinical practice for many years by thousands of ME/CFS patients in Sweden, and is safe, cheap and effective, putting lots of patients into remission. It is probably the most effective drug ever used for ME/CFS. Unfortunately due to regulatory reasons (GMP requirements), Janssen stopped manufacturing this drug in 2005, leaving lots of ME/CFS patients who were taking Staphypan in the lurch. I can see no good reason why Janssen cannot find a way to circumvent the regulatory requirements, and bring this drug back to market. The letter I wrote to Janssen Pharmaceuticals is copied in this post; details of Janssen's reply are given in my posts just below. Basically Janssen told me there is nothing they can do to re-start manufacture of this drug, which was stopped due only to regulatory issues. But I am pretty sure if they found Staphypan could treat say ebolavirus, they would very quickly find a way around the regulatory rules, and start manufacturing this drug again. Many ME/CFS patients in Sweden were able to continue with their normals lives because of Staphypan. So here we have an example of a successful, cheap and safe drug that was used for many years by thousands of ME/CFS patients, allowing many to go back to work and live a normal life. Yet Janssen say they cannot find a way to get around the new GMP regulations in order to bring Staphypan back to market. If anyone wants to write to Janssen, let me know, and I will give you the email of my contact there. Better still, it might be an idea to organize a petition, so that we can send the petition to Janssen. That should carry a lot more weight than just an email from a single patient. If you want to read the whole story about the Staphypan vaccine treatment of ME/CFS and its history, here are some links: Article on Professor Carl-Gerhard Gottfries (discoverer of the Staphypan vaccine treatment for ME/CFS) Prof Gottfries's studies and clinical trials of the original Staphypan vaccine treatment for ME/CFS Brief Story of Prof Gottfries and his Vaccine Prof Gottfries — Various Video Interviews
Sorry, but I feel the process of relying on the academia method with it’s lack of government funding is not working for ME. Maybe it works for other illness, but I don’t see it working for ME in my lifetime. Other paths must be created.
I know you re keen to get pharma involved, but remember pharma is a business. If they think they can make money they will be all over it in an instant. If there is too much risk they will stay away. A lot of the big pharma companies don't do much new research now because there is too much risk, too many drugs fail at phase II or phase III trials after years and millions of dollars of work. So it is up to the startups and incubator companies to to the initial research. Then if the drug shows promise big pharma step in and buy the startup. Big pharma have licensing and sales expertise to bring the drug to the market. So it suits both entities. The best bet in the near term is actually a small startup, which is most likely to come from a university. In all likelihood, a university that has been funded by government or patients.
We all want progress yesterday, but we can't wish it into existence, we need to make it happen, and how is the big question. If i want to build a rocket and fly to the moon i can't say lets build it out of parts in my toolbox and we can get there next week, i would need to know how much force is needed to escape the atmosphere, how much gravity is on the moon, how much fuel do i need, how to make a rocket get off the ground, how to keep it from blowing up, how much radiation protection do i need, how much oxygen to get there and back, and a million other details. NASA did not will this knowledge into existence, they had to gather many extremely creative minds to figure it out, do countless tests and calculations, build many prototypes, they lost many brave astronauts and so on. The monetary cost to go to the moon was enormous. If we want progress we need to figure out how to make it happen, we need money, talented researchers, resources, labs and so forth. If you have a better way we are all ears. I want to know the identity of this molecule, thats the research we need. It may be the key to unraveling the disease mechanism, assuming it is the primary driver of our exhaustion, identify it, find its origin and find out why its being produced and how to either inactivate it or suppress its production. Then again it could be a dead end. Either way i think it should be pursued because if its a big piece then we have accomplished something very useful, if its not then Dr Davis needs to move on.
I admire your enthusiasm @MErmaid, but what are you going to ask them to do? Remember that virtually all drugs produced by pharmaceutical companies were initially invented in academia. Or at least academics worked out what the target was and the basic chemistry to be worked on. If you go to a pharmaceutical company and just say 'cure this' they will just look blank. You say that academia has failed but the only half decent trials we have had in ME recently have been the academic trials in Norway. Pharma has shown LESS interest than academia. The Pharma associated trials have tended to be poorly designed and inconclusive. I also think it can only do harm if the pharmaceutical industry is bombarded with poorly thought out theories or shaky evidence. Dr Chia may think enteroviruses are important but everyone else in the research community finds the evidence unimpressive and assumes they are probably irrelevant. If staphypan was really any use the physician using it should have done some proper trials. I am pretty sure the regulatory problems with it boil down to the fact that there is no evidence it works and the authorities are no longer prepared to let doctors use something prepared as a vaccine, which may have different regulatory requirements, for treating something else. Perhaps look at it this way. I am much more convinced than anyone I know in Pharma that we need treatments for ME/CFS. I know pretty much most of the available science. If you are going to convince anyone in Pharma then it ought to be easier to convince me first - what are we going to study? We are at the stage of needing to study patients because we do not know enough to study things in test tubes yet. People in Pharma by and large know nothing about ME patients, so how are they going to be able to help us?
I find it hard to believe that the pharma is interested in finding a medicine that will cure a disease like ME. They are much better off selling many painkillers, digestive aids, tranquillizers, mood stabilizers etc. And that's whats happening at the moment, they are happy with it. It's good business. They don't want an umbrella cure which requires a diagnosis system. This is not happening anyway. I don't think it's a coincident that the establishment's state funded research is psycho-bubble oriented.
Yep - if £5M had been made available to biological research back in 2007 thereabouts, we might be at that point now. But hey ho ... no 'harm' done . I think it is the scientific equivalent of passive aggression - harm done by consciously blocking beneficial actions.
I don't really buy what I think you are saying. In RA the government never funded any of the good research. We scraped together charity money and bits from here and there. Governments are not instructed by the electorate to fund each disease according to need. Maybe they should be but they never have been. Most medical research is done by people with a personal interest in a problem funded by charity. The problem for ME has been the lack of charity money as much as anything. I agree that £5M was wasted, but similar amounts are wasted on biological research that is clearly going nowhere. An in the pharmaceutical industry billions are wasted on useless animal models and all sorts of nonsense. I doubt we would have been any better off if the government had put up £5M specifically for immunological research into ME in 2007 and advertised for applicants. The grant would have gone to some politico working on some fad or other like TH1/Th2 balance and poured it down the drain. Throwing money at scientific questions is not that useful in my experience. It is great for spin off biotechnology questions, like how to find a cure for HIV, once you know what HIV is, but we are not in that situation.
The new hepatitis C antiviral drugs like sofosbuvir which fully eradicate the virus and cure this disease indicate that pharmaceutical companies will provide cures. Yes, these new drugs cost $1000 per pill, so that a full course of treatment totals up to around $80,000, but that is necessary in order to recoup the R&D costs.
I still find it hard to believe that the pharma is interested in finding a medicine that will cure a disease like ME, even after reading what you wrote here. I'm not quite sure hepatitis C is a disease like ME. First of all, is ME really only and solely caused by a virus and an anti-viral medicine is going to cure it? Are we sure about this? Even so, this one example (sofosbuvir)is enough to reach a judgement that pharma is interested finding curative medicine -considering majority of autoimmune and many other illnesses have not got curative medicine solutions in general? I am also shocked to the price of this medicine you give it as an example. Is it made out of gold? If there is a very expensive solution for ME this will not be a solution to masses who have ME, this will be another problem that ME community, NHS's, governments etc will have to deal with. As long as medicine is a business there will be no solution to many health problems, especially diseases like ME which are very complicated and still unknown. Business need to be sustainable otherwise it is not business. I was trying to tell this more than anything else.
I appreciate the time you spent composing your posts, not just to me, but the hundred or so I read over the years directed at other members. This is something I need to pursue now, because I wasted the last 2+years not doing enough. In fact, there are too few people taking action. The severely ill can’t advocate for themselves. The moderately ill, are just barely squeaking by each day. The mildly ill seem to be mostly absent. So it’s up to a handful in the moderate/mild category to drive changes while dealing with the criticism/push-back from the ME Community. IMO it would be more constructive to support and encourage the handful of us who are trying to make changes. There is nothing easy about being a PwME. The hardest challenge for me is not ME itself, but trying to find a crack, or make a dent in a research “system” that seems to fails us. I can’t share the details, at this time, of why I feel so strongly.
Dear @MErmaid, I am sorry if you feel strongly that things are wrong. But I assure you I am trying to give all the support I can. Part of that is trying to get people to clarify their ideas so that we do not blunder into things and just put people off. I am genuinely asking what you think we should tell the Pharma people to do. Pharma people in general do not have much imagination so they are not going to work that out for themselves. Approaching Parma is in principle a good idea because it is good to try any route. I have done it myself, as I indicated. But what i don't want to happen is for them to go off and work on an 'animal model' that actually has nothing to do with the human disease and just wastes vast amounts of time and money. There is no 'research system'. There are people wasting public money, yes. There are also people like the Biobank and CureMe groups working as hard as they can do get some sensible leads. What exactly do y mean by 'make changes'? I have working pretty hard to shift the balance of research in the right direction for the last four years. If there is more to do then I am all for it, but I am unclear what 'changes' you want.
Pfizer announcement on their decision to exit neuroscience research. https://www.pfizer.com/news/feature...our_neuroscience_r_d_decision?linkId=46901181
