Thank you for your support. I have been thinking about this for 2 years, and have done some research along the way regarding who to target, and the timing involved. I am referring to the US. There seems to be a trickle down approach, that is failing us. My government is throwing out a few crumbs here and there to a handful of researchers/scientists. I don’t see any continuity or a serious long term commitment. I only see continued failures as each year passes. I predict the future will look much like the past, unless something changes. I want to be an impetus of change. I am not encumbered by the past nor feel burdened with protecting the hierarchy or pecking order of academia. I don’t care what University is studying ME, nor what title appears after a researchers name. Past Nobel prize winners do not impress me. I only care about results, which to me means, getting the first ME drug inserted into the FDA pipeline ( and fully approved). Jen made a film and started MEAction. Julie wrote a book. Its my turn to step up and do something wonderful with the skills, ability, and knowledge I bring to the table.
Exactly. It seems that the majority of us have survived, so far, which only supports the “lack of urgency” theory. I don’t see any funding actions that align with “urgency”. It’s seems that someone at some future date just might get back to us, someday. IMO, this translates to “not in my lifetime”.
We heard this loud and clear at the last ME Rally in the US. PwME need to raise the majority of the funds; the government will kick in a few dollars after we raise the money. I think there was video made of the local representative that spoke that day. But I have heard this message before; sadly it’s not new information.
I disagree. Yes, I have to prove to a business that a hefty profit can be made. I have taken this into account with the strategy I created. I would much rather work with corporations, over academia or the government. I understand how corporations run, because the majority of the decision making seems logical to me. I don’t understand how the academia’s run or how government operates; I have no desire to acquire this knowledge.
No idea, really. A smaller company, less known, may be more inclined to take risk while expanding it’s drug portfolio. It needs to create a name for itself, so it can sell more shares of stock, and thus gain more capital. It won’t be able to absorb the cost of a huge failure, unless huge piles of cash are sitting around; which is not a typical scenario. A large corporation’s CFO, will seek to keep enough cash around to absorb R&D failures. Large companies usually swallow up smaller companies so they can own the patent/ technology outright and eliminate any competition. After a buyout, the founders of the smaller company make millions, which they can invest in another start up, or retire.
Hello @MErmaid this thread has left me thinking and contemplating both your post and @Jonathan Edwards answers. I am thankful for his willingness to share his thoughts and experience and for engaging with the patients in a forum setting. I appreciate your determination and I encourage you to pursue your idea, because trying something gives us a chance to move forward. Not trying anything gives us no chance at all other than being vulnerable to the many pittfalls of research, and there are so many of them. The research we ahave right now, around the world is disjointed. Scientists are scrambling for funding. Clinicians are far too few and clearly do their own things, practicing according to their pet theory, or simply following Cochrane reviews, or making money out of providing unproven treatments which are not covered by insurance, may it be private or socialized health. The International ME association does not seem particularily active or helpful. 30 years following the Tahoe outbreaks, patients are still waiting. We are still stigmatized as a patient group. Most physicians still doesn’t know how to diagnose this. No one seem to care that we do not belong into any medical specialty and the dammaging implications of this. Moreover, not many seem to even care of studying the natural history of the disease, which could provide important clues should this study be done correctly. (For instance, the brain imaging study from Australia). What is most striking to me is that things are more likely to remain as they are if patients do not get involved and take act. Millions of patients from around the world left behind. Many dying from their own hands. So many so terribly sick they are in bed in a dark room, and if they are lucky there are family members caring for them. Too often, there is no one. The suggested strategies, if there are any, may have worked decades ago for a certain field, but there is no warranty that it will work for our stigmatized, neglected, underfunded disease. Taking a wait and see approach, we have done this for decades. Each year I celebrate my sick-versary is worse than the last. So what are we to do? Wait and see if things will get better, see if we will get better funding this year? See if the doctors will take us seriously now that x,y,z paper has published? We have been at crossroads for decades. i welcome any attempts that will position us closer to access to biomarekrs and treatments.
Do you actually know anyone who works in Pharma? I had a friend who got to meet the CEO and some chief scientist in a pharma start up. They were working on a targeted therapy for specific types of cancer. Several of the researchers had personally lost family members to cancer. These were very smart people, they could’ve worked on Wall Street or in a tech start up or developing super candy Bird crush or some fluff where you have a much better chance of making money. As it is the company has been spending tens of millions for a number of years, and hasn’t seen a dime in revenue. Will only get revenue if they make it past the third stage FDA trials and then they have only about 10 years, because the US patent of runs out, and a generic pill mill in India will start cranking it out. Trademarks and copyright for things like Mickey Mouse were extended to 99 years, but inventions that actually do something are patented only for 12 (or 13). Many times and inventor gets no royalties because people who wish to buy the invention just wait until the patent expires. If that invention is going to cost you $100 million to develop, then guess what, very few people are willing to do it
Thank you!! Futhermore, we need to encourage each other to think “out of the box” and follow up with action. INaction is killing us. After reading Anne’s parting letter, I felt like throwing up, and have everyday since. Anne’s absence propelled me to make a firm commitment for change. I hope more people step up.
Agree, history has proved itself, that sitting and waiting for someone or something else to solve our problems doesn’t work. That’s why I firmly believe changes will come about from PwME led activities. I am very sorry Take a risk. Find your passion. Jump in feet first.
That's not correct. A granted drug patent holds for 20 years. Certain safety certificates (exclusive marketing) can lead to an extension of up to 15 years. But I slightly remember the number 50 - don't know why...and I don't find a justification for that. (US and EU patent law are a bit different, but the main aspects are the same.) Patent law and design/trademark laws are completely different, and so is the scope of protection.
What are you going to ask them to do, action for the sake of action won't get a cure, diseases are treated by figuring out whats causing/sustaining them and/or discovering a way to affect the disease mechanism. Our civilization is predicated on science, its nowhere near perfect as all of us can very well attest to but the human body is an organic biochemical mechanism and works based on interactions with the environment (and chemistry/biochemistry). In order to change things to our desired goal we must either define the problem and work on a solution or luck out and have a solution fall out of the sky. Sometimes that does happen, Rituximab was discovered by coincidence and may work for a subset, and we can increase our odds by throwing things at a problem (Dr Davis's drug assay for example) but typically you solve a problem by defining its cause. Even the drug assay may fail because its used on the wrong target, only testing it on blood may be the wrong approach, again a disease mechanism makes itself needed, if its caused by say an immune malfunction then drugging a patients blood under a microscope is missing the more likely target. Its nice to think we can will a cure into existence or bring it about by raw determination but it just does not work that way. We need will or determination to get where we want to go but it needs to be applied in the correct way This is my concern as well, burning our possible bridges is a bad idea. Believe me, I understand your frustration and sometimes just taking action makes us feel better doing something feels a lot more productive then doing nothing. You seem to be on a mission here and i'm not going to say you should not be driven, what i am saying is you need to figure out how to make your goal happen, not just charge at something because you feel you have to do something forceful
I don’t know exactly how to respond. I am getting the impression you have little confidence in my ability to pull this off? That’s fine, I was actually expecting a lot more push back. Have a nice day
Actually, someone else started this thread, it was not me. If anyone has any positive feedback or suggestions, please let me know.
There is no permanent cure, and doubt there ever will be. Treatments must be on going, which is the best that’s available now. There is no guarantee of 100% recovery. Most likely many drugs will be need FDA approval, because we are not all wired in the same way. My goal is to create a new pathway and get one ME drug into the FDA pipeline, and see that it gets fully approved. The drug I am thinking of, won’t help everybody. But after reading forums for 2+ years, it will be helpful to a subset, because people post about the subject often. After that, I have other goals I want to achieve, involving improving health care. Currently, I am coming up to speed on how drugs are fast tracked. The FDA is being more flexible in some cases. There is a lot to learn, and I try to tackle it in chunks. I located the exact person and Pharma I plan to target. I am currently working on a letter. I plan to try a few approaches to get my foot in the door; this is where persistence and determination pay off. I can’t guarantee or promise a result. My guess is that the best case scenario would be an FDA approval in 2019. The worst case is that I make zero progress. I am not asking for money or anything like that. It’s my time and energy. There is no hype or false promises. I am honest, hard working, intelligent, and driven. I owe no one anything. It’s me wanting to make a difference. But I am not 100%, I have ups and downs, so I will do the best I can.
Hi @MErmaid, I hesitate about responding to your plan to approach drug companies because I do not want to upset you by appearing negative. I admire your determination to make a difference. I know nothing about your background or qualifications so I have no idea whether you are a scientist with a viable proposition to put to a company or a dreamer whistling in the wind. Please don't take that as an insult. I am simply stating the fact that I don't know where your proposal is coming from. I don't think we can help you when you are unwilling to spell out just what your plan is. I don't think that's negativity, more bemusement. You have not given us anything concrete to respond to, either with helpful suggestions or equally helpful critique. I do understand that if you indeed have a viable concrete proposition you feel the need to keep the details confidential. But if that's the case, there's no way we can comment helpfully. I wish you well.
