Prion Diseases: Madness and Memory
 

I just read Madness and Memory - The Discovery of Prions - A New Biological Principle of Disease by Nobel Laureate Stanley Prusiner, M.D., published 2014. He gives convincing evidence that PD, AD, ALS, all the neurodegenerative diseases are prion based. A 2011 thread on this site: "Breakthrough! Alpha-synuclein responsible for progression" begins the discussion.

Prions are proteins with no DNA or RNA, not viruses. Simplistically put, they can mis-fold and go rogue, taking over normal proteins and wiping us out. He ends the book with a couple of sobering points: "Once so mysterious, degenerative brain diseases are beginning to reveal the mechanisms by which they compromise nervous system function." and "For the wars on cancer and heart disease there are a myriad of advocates and pipelines filled with hundreds of promising drugs--for neurodegenerative diseases, the ranks and the pipelines are virtually empty."

It is sobering to hear a scientist so key to unlocking the mystery of brain disorders basically acknowledge what many patient advocates have felt for some time now. It only reinforces how we would probably make more headway if these diseases were not treated as separate entities but rather on a continuum by both researchers and patients. There needs to be a larger representative foundation focused on neurodegenerative disorders as a group. However, there is little tolerance for reality in patients, I find. If you are not optimistic and inspirational every moment you can be shunned in social groups online. Not going to make any progress unless we can change that dynamic.

Thanks!
 

Thank you, Nan and Laura, for pointing out the truth. And I do believe if the truth were to be picked up by enough people, we would have already found a cure for Parkinson's. The truth is this:


There is a lot of talk about patient empowerment, but I haven't really seen true patient empowerment. Truly empowered patients (in this case people with PD, which includes caregivers) are given enough power by those holding the purse-stings to take risks. And taking risks is what it takes to make things happen.

This is what occurs in research - someone has a sponsor (it takes money to take risks), then they apply that money to an idea (in research, we call this the hypothesis). When you have a solid hypothesis, then you turn that over to the experts - which would be the medical technologists, the clinicians, the research nurses, the statisticians, the investigating physicians, and last but not least - the patient.

Who knows more about living with PD than the patient and the one who cares for the patient? No one! The PWP is te real expert. I can think of hundreds of trials which would not have gone sour if those reporting the stats would have listened, truly listened to what the patient was saying. Who wants a cure more than the patient? No one!

What can we do about the fragmented organizations that are representing the unified voice of the PWP? Let's start using these forums to brainstorm ideas, point out where we fall short in listening to those who live with the disease, and DEMAND PATIENT EMPOWERMENT!

OK. The ball is in your court. Take the serve and start playing like it's for real or we lose. And after 20 years with this disease and playing advocate the best way I know how, I am ready to try another way of playing this game.

Any suggestions?? Remember, the players are the same. It's just the rules that must change.

Peggy

Thanks Laura, you are so right about the need for collaboration among the neurodegenerative disorders groups, foundations, organizations and researchers. Maybe the place to start is collaboration among the patients. If we spoke with one voice, we're likely to get better cooperation from the other entities.

There is some movement in this direction with MJFF. They've partnered with the Alzheimer’s Association and The W. Garfield Weston Foundation to create and fund a new research grant program — Biomarkers Across Neurodegenerative Diseases — that will support initiatives including, but not limited to, those that:
- analyze datasets to test hypotheses related to aging and neurodegenerative disorders;
- seek to identify panels or pathways that may play a role in disease mechanisms, such as around inflammation;
- pursue shared or disparate biochemical markers of disease risk, onset or progression;
- or assess potential commonalities across the disease spectrum, including around other neurological disorders such as Lewy body dementia.

It's a start, although there is still a long way to go......

Peggy, thank you so much for this much needed post. Everything you wrote is spot on correct. There is a lot of lip-service given to patient empowerment or patient-centric, but I see us being used more as show pieces rather than as true partners or advisors. As you have said, we know more about our conditions than any researcher, fund raiser or research sponsor. Yet, as a group, our input is at best, minimal, and at worse, non-existent. What amazes me most is how many truly talented people with PD I've met over the past few years, yet how little this talent is used to help find a cure and better symptomatic relief. Sometimes I get the feeling that all of these other entities are saying "he has a brain disease, get him on a panel or project but make sure he remains quiet".

With regard to research, the best way we can get some more control over our destiny is to control the one thing we have that all these other groups need. VOLUNTEERS. Without our participation, the studies will stall and die. We, collectively, have the ability to decide what research goes forward and what doesn't, because, no matter how much money is raised and offered in sponsorship, without us as volunteers, nothing goes forward. I'm not saying we have to dictate terms where we have no knowledge. What I am suggesting is that EVERY study done on and with PD patients should be first reviewed with a patient advisory committee. We should have input into the need for the research, its design, how volunteers are selected, and HOW VOLUNTEERS ARE TREATED THROUGHOUT THE TRIAL, including reasonable expense reimbursement. Finally, we should have access to results and reports.

You have much more input into the entities we are speaking about than most of us, so I would love to hear some of your thoughts.

Nan Cyclist quotes Prusiner that the drug pipeline for PD is "virtually empty".

Conductor71 writes "It is sobering to hear a scientist so key to unlocking the mystery of brain disorders basically acknowledge what many patient advocates have felt for some time now."

Peggy writes "I am ready to try another way of playing this game. Any suggestions?? Remember, the players are the same. It's just the rules that must change."

Tupelo3 writes "We, collectively, have the ability to decide what research goes forward and what doesn't, because, no matter how much money is raised and offered in sponsorship, without us as volunteers, nothing goes forward."

There seems to me to be something self-contradictory talking about taking control. If we're ready for it, we should just take it.

Aunt Bean took control by growing fava beans. Not fighting for years for a grant and then to delegate the work to some university research group. Who: will most likely publish a paper we can't read, because it's behind a pay-wall; will recommend further research, which most likely won't be done, because it can't be patented; and, if they do make a breakthrough, rather than make the most of what they've got, will most likely make enough tweaks to get patent protection. It will most likely take years getting regulatory approval, coming too late to be of any use for many of us; and, once they've got a patent and get it to market, will most likely be so expensive that many PwP will be unable to afford it.

I think it important that we are not anti-science and not even anti-big science. Big science will need to continue to do the research that does not lend itself to being done by patients (surgery for DBS, for instance) But much research (symptom measurement, exercise, effect of caffeine, etc.) can be done well by PwP.

As I've said so many times before we don't lose our skills as managers, engineers, chemists, programmers and a myriad of other areas the moment we're diagnosed with PD.

Make a start now: take the side-to-side tap test. Repeat daily. Then when you find that some food or activity is having an effect, good or bad, you'll have an objective measurement of the effect. Share that with us.

http://www.parkinsonsmeasurement.org/PDMeasure/

And, I bet, the act of taking control will itself be therapeutic.

John