research in the doldrums
 

Sep 15 2010 (Vol. 30, No. 16)
Parkinson Disease Research in Doldrums

• Patricia F. Dimond, Ph.D.

Discontinuation of PD Trial Highlights Hazards Inherent in Developing Drugs for This Indication


A recent GlobalData report stated that Parkinson disease (PD) drug development in general, focused as it is on the dopamine pathway, is “increasingly hampered by a lack of innovation.” Compounds in late-stage development aim at the symptomatic treatment of the disease, as do current medications. Any company developing treatments for PD will need to overcome prevailing product weaknesses including psychiatric side effects and obsessive compulsive behaviors as well as limited efficacy, the report pointed out.


http://www.genengnews.com/gen-articl...doldrums/3417/

[QUOTE=paula_w;712349]Sep 15 2010 (Vol. 30, No. 16)
Parkinson Disease Research in Doldrums

• Patricia F. Dimond, Ph.D.

Discontinuation of PD Trial Highlights Hazards Inherent in Developing Drugs for This Indication


Paula,

thanks for finding confirmation of our gut feeling about this issue. When you see it in print it's even more depressing. So we must look in new directions.

katherine

Doldrums
 

Establish an Independent PWP Voice
Independent PWP should be a patient advocate group independent of established charities and aware of the value of medical research to both public health
and to the global economy. Experienced patient advocates taking the voice of PWP to clinicians the public pharmaceuticals and the medical research community strategically led on behalf of PWP patients.

Effective Patient Input
“Decisions about me not without me”

Doldrums and Depression weve no time for such luxury ladies.

Networks such as this forum are a valuable tool for all PWP and perhaps they should be given credible status as a resource worthy of recognition and inclusion by clinicians and researchers and managers of health systems

Improving Health Care PWP for ourselves.our future
The future must be with electronic and patient held records. Electronic records must involve some form of international co-ordination in the development processes or the tragic waste of shared information continues, security and access to data should protect but not obstruct.

The possibilitys are incredible but the obstacles are endless

Better data quality and collecting, potential to speed up research initiatives development and outcomes.
Earlier assessment and treatment for problems with medications, better access and co-ordination of care between clinicians.On and on.

Accredited researchers should be required to share results of any research surely its about progress saving lives not protectionism.Any chance?

How do we do it?

who said pd research is not innovative?
 

have they read about this?












What's the poop on fecal transplants for PD?


By Brian K. Fiske, PhD, Associate Director, Team Leader, Research Programs, Michael J. Fox Foundation for Parkinson's Research (MJFF)
Some recent news around fecal transplants for treating severe C. difficile/colitis mentions a trial in people with PD showing some potential benefit as well. Does anyone know more about this approach and how/why it might have PD potential? Is there a higher prevalence of C. difficile in people with PD or could such an approach as fecal transplants help to normalize gut function and improve ldopa pharmacokinetics to reduce fluctuations and dyskinesias, etc.?

http://www.pharmatimes.com/Article/1...impresses.aspx


At least something in the pipeline.

TG

agree completely
 

Most of the suggestions in your list are set up already. We have a champion for electronic records and we have an independent org ready. I think the first step is to set up a method of communication and suggest Skype because it's free and we wrote a book using it.

Our first priority is to finish our book, but i see no reasons why we couldn't set up communication; the more there are of us, the more projects will be going simultaneously.

Then we can determine what the most pressing needs are and go about speaking, researching, interviewing and informing people . We are going to use our PCC site to expand on topics in depth, interview people, blogs, news, data collection, and just continue in a creative format similar to our book. Once we start talking, ideas will be abundant. IMHO

Thanks for your input!! keep it coming!

MJFF point of view
 

I know they have their own perspective, but Katie Hood responded to the doldrums article saying, "It's not true!"

Also, one item on the agenda for the MJFF Patient Advisory Council meeting next week is "expanding patient contributions to research solutions"

We continue to gain influence. I'll keep you posted on the meeting.

I agree!
 

Carey, thanks for sharing Katie's perspective. While I don't have her level of expertise or connections to research, I am inclined to agree with her based on what I have researched.

There are many novel treatments being explored looking at other neurotransmitters and a vaccine in development! The bigger issue is why do so many of these potential treatments languish or stall out in the pipeline. It seems to me there are myriad options on the horizon, they just never seem to see the light of day.

Maybe the focus should be on why these non-dopaminergic alternatives never seem to get anywhere. Is it because investors see anything beyond the "gold standard" as too risky? We are impacted by people who have a lot of money but don't fully understand the disease enough to know what to wisely invest in. Neurologix, is short of funding for phase III trial monies for its GAD treatment. This speaks volumes, I think. The innovation is there, the risk taking of venture capital is not, so it would seem.

Just my take on it, I could be wrong....

Laura

Laura

sounds good
 

Each one of us could be reading this from a different perspective. The media has a mind slant with its own slippery slope. We are used to being ignored. BUt now it's beyond going back. We want to say how we are defined. Why should someone who doesn't have it, define it?

Oh no, Paula. I am in complete agreement with you on the patient perspective that is missing. I just think in looking beyond dopamine, there is progress. No matter the type of treatment, there is a huge, gaping void in the involvement of patient perspectives. Heck, but then again I think that if they would have started a huge database of all of us giving complete medical histories, our life stories, all our demographics and then some, they would have been able to run checks for patterns and narrowed down environmental culprits, at the very least by now. That is something crude, basic, and simple that may have yielded important clues.

Laura

oh i was talking to whoever
 

oh i was just directing some random thoughts. not to anyone just everyone.

It's all in the perspective
 

The problem lies in the the perception of PD by most, especially those who manufacture and market the treatment.

The gold standard of therapy (dopamine replacement for 40+ years) is the holdup. In spite of its side effects (on/off phenomena after 5-10 years of usage, dyskinesia, dystonia, etc.) , levodopa (Carbidopa/levodopa) works - maybe too well). In a matter of minutes, one can observe an invalid, akinetic PWP transform into a funtional, near "normal" person by swallowing a pill. But these same observers don't stick around to see what follows, and we as PWP aren't telling them. I actually had a CEO or one of our orgs say "Alzheimer's is a horrible disease; sufferers end up like a prisoner in their bodies." I reminded this person that PWP end up the same way - and most are aware of their "jail" sentence, where ALZ patients aren't.

We are being passive. We aren't making enough noise. We need to polish up our marketing skills. John Q. Public just sees that the pill we took an hour ago works. We should be in the public arena showing everyone, especially potential sponsors, what follows.

In second place as the most common neurological illness, there are approximately 4 million people world-wide with PD. In first place is Alzheimer's with only a little over 5 million. But ALZ seems to get loads of attention.

I have tried to analyze the reasoning behind the public's high interest in other neurological illnesses and have a few theories, but maybe you can help brainstorm. It isn't numbers - with only a small difference in ALZ's population. Is it because PWP are seen most of the time in their "on" state? Does it hurt our image to not talk more about non-motor symptoms, like depression? Are we hiding in our homes when we begin to freeze and festinate (taking baby steps)? And finally, does the fact that there are three major orgs for PD and only one for diseases with meganumbers, like diabetes and cancer hurt or help our image?

Do you have any answers?

Peggy

but we look so good...
 

I think a big part of the problem is that, as you say, the public sees us when we are functioning. Ok maybe we shake a bit, or drag a foot, or speak slowly. But when outside our homes, we do our best to be as “normal” as possible (at least I do). If I am feeling particularly bad, frankly I stay home because just riding in the car is torture for me.

My cousin has MS. And when she is functioning well, people yell at her for parking in handicapped parking spaces. Hasn’t that happened to many of us? And I have been berated by postal service employees for using on line printed customs forms rather than hand writing out those forms in 5 layers! Sure they are contrite when I tell them about PD, but why should we go through this?

I know that Sheryl & I are out there speaking, but we’re speaking at PD conferences. I think it would be terrific if we could speak at neurology conferences. But how to go about that when there were still objections by some neurologists to having patients attend the WPC in Glasgow?

Something else that may work is for non-parkies to see “the lighter side of PD.” When PDPlan4Life is at the podium, Sheryl tells her hilarious stories about life with PD. PWPs nod and laugh because “yes, that’s what it’s like.” And the “temporarily healthy” laugh as well, while shaking their heads, but ultimately they remember her stories. As any trainer will tell you, the audience remembers personal stories, and they remember humorous personal stories best.

And yes we need to publicize Parkinson’s, but how to go about doing that? How about some PSAs? If you haven’t watched the videos from the WPC, that’s a great place to start. Take a look at the 3 short PSAs from Norway – hilarious! OK - what I had forgotten is we have the power of the web. We can upload videos to YouTube, to blogs, to websites, to facebook, and to hundreds of other social media. We have at our fingertips a powerful and free medium to use -- social networking to share our message(s).

I hope that this thread & the Advocacy 101 thread give people ideas. The more voices, the better.

Jean

Yes, Jean!
 

I agree with all you said - we have work to do!
Quoting you: I think it would be terrific if we could speak at neurology conferences. But how to go about that when there were still objections by some neurologists to having patients attend the WPC in Glasgow?

I believe before we can be invited to these neurology meetings, we need to do a better job of selling our professionalism. I'm not saying that we aren't professioonal in what we do - we just can't seem to get the world of neurology to see us as equal team players (if not higher - lol). We can begin by writing comments everywhere they talk about PD (my dream is to someday write for Huffington).

Remember when the GDNF issue first surfaced? I remember that you and I were published in the comment section of Lancet Neurology. I suppose I am suggesting that we comment in scholarly periodicals (I wish the Fox Foundation would allow us to post in their online research forums). We need to write Op Eds, letters to editors, and stuff like that and show that we aren't just "patients," but want an active role in our advocacy walk.

I'm sleepy now, so I'll go dream of other ways to get in with the neurolog incrowd . Did you ever do a Google search on yourself? It's amazing what is picked up!

Peggy

Social media is the way!
 

Peggy,

I am a firm believer in grassroots movements. And creating a grassroots movement using the power of social networking will be a force to be reckoned with.

I'm still researching different social media and how we can work together, but I'm convinced this is the way to spread our message. Just as the print media is rapidly fading away in favor of Internet-based media, we should use social media to spread our message. We are not limited to trying to get our message on television. We have free access to social media: video sites, podcasts, communities, blogs, message boards, photo sites, twitter ... and more!! The mind boggles at what we can accomplish.

This is possible. It just takes people who are willing to take first one step, then another. To take action, not just talk about taking action. So for everyone who is interested in pd advocacy, why look into this? One person can do it. A handful of people can do it. What is your message? Can you make a plan on how to share it? And then just do it?

I will be doing what I can. I hope others in the Parkinson's community will consider this.

Jean

Michael J Fox defines PD for many - the relatively mild writhing Ldopa Dyskinesia ... along with him saying that there's nothing he can't do despite having PD.

Mike has done a lot for us, but I wish he'd show the general public what PD disability really is - Bradykinesia, Tremor etc.

MikeTTF

We can do this for ourselves,I am already trying to set this up ,the clues are still there Laura.A friend with PD made the following statement and his words are simle but true.
'The patient is the key to the cause and the path to the cure
The cause is hidden in my body.
I believe that every patient can contribute to the understanding of this disease”
I believe that if we join together with millions of fellow victims we can make a significant difference'

Empower patients we can only do that ourselves.

I agree with Paula and all of you who understand that it is the patient's job to define the disease. There are many ways to take direct and public action that makes a difference. Through social networking, public speaking, organizing, promoting, writing, and serving on advisory councils as many here do now, we all have influence.

The crucial connection that must be made and firmly established is with the research community - to inform the direction of their research, to praise and encourage when they "get it;" to educate and persuade when they don't. Arrogant scientists remind me of the stereotypical man driving the car, hopelessly lost, but dismissing the passenger with the map. They'd rather continue down dead end roads than ask for directions.

We can't, however, drive the car or arrive at the destination by ourselves. We require champions with money to fund research that makes sense. Nobody is doing that better than MJFF at this moment in time. They deserve our support and need our attention. They "get" it - but we can't relax and let them, or us, fall asleep at the wheel. Or take us to Cleveland when we really need to go to Toledo.

Alzheimer's
 

Peg - as to your question about the attention Alzheimer's gets -- it's because it is 5 to 10 times more prevalent than PD, and in many opinions (mine included), a million times worse. It is a disease destined to devastate a generation. Please read this op-ed by Sandra Day O'Connor from the NYTimes on Oct. 27.

Also, I believe that any advances made in Alzheimer's research will benefit PD as well. It is their model that MJFF is following in the design of the PPMI biomarkers study.

Link to op-ed
http://www.nytimes.com/2010/10/28/op...r's&st=cse

Excerpt

Starting on Jan. 1, our 79-million-strong baby boom generation will be turning 65 at the rate of one every eight seconds. That means more than 10,000 people per day, or more than four million per year, for the next 19 years facing an increased risk of Alzheimer’s. Although the symptoms of this disease and other forms of dementia seldom appear before middle age, the likelihood of their appearance doubles every five years after age 65. Among people over 85 (the fastest-growing segment of the American population), dementia afflicts one in two. It is estimated that 13.5 million Americans will be stricken with Alzheimer’s by 2050 — up from five million today.

words matter
 

How wonderful to wake up and read so many great suggestions for helping ourselves be the catalyst for the change we want to see. My one caution is that words matter. What we put out there will be used to define us. I am NOT a victim. The many definitions of this word imply that victims are powerless. The day that we accept that we might as well curl up and die.

vic·tim (vĭkˈtĭm)
noun
One who is harmed or killed by another: a victim of a mugging.
A living creature slain and offered as a sacrifice during a religious rite.
One who is harmed by or made to suffer from an act, circumstance, agency, or condition: victims of war.
A person who suffers injury, loss, or death as a result of a voluntary undertaking: You are a victim of your own scheming.
A person who is tricked, swindled, or taken advantage of: the victim of a cruel hoax.

I agree whole-heartedly that MJFF "deserves our support and needs our attention." My point is that there are things we can and should do ourselves.

Jean

It was a word in a statement that I remebered and liked, the whole had meaning to me
We cannot always assume that the words we hear mean the same to other readers and critisism is essential and not in anyway offensive in my personal opinion.Often what people say means something very different to them than it did to you., Victim yes I agree was not a good choice,another word I dislike is Sufferer,I dont regard myself a either, but I do hope that one word did not datach from the overall meaning .opinions about people can be spoiled by your interpretation of what was said, rather than making the effort to get under the skin of the detail and try to understand what they really meant.

tailor words to your audience
 

I understand and agree with your underlying point, especially when communicating one on one. My point is that we have to choose the right words for the audience we're trying to influence. Researchers, legislators,and drug company reps will not take the time to understand what we REALLY mean. Our message must be clear and concise.

Sheryl

Jean - I am agreeing with you on all fronts! We must be the ones to define the disease.

I think my earlier post is more of a reflection on my own personal path. I have long been an advocate for patient independence - that we need to clearly represent ourselves and not be co-opted by any one entity.

I still believe that - we need to promote anyone out there who we think is doing the best for us. But the longer I am involved with MJFF, the more impressed I become because, as they learn more, they evolve and adapt quickly. They don't fall back on old, established ways because they are not old and established. When they see a good idea, they pursue it. It's difficult for me to resist their sheer resource power when they also have the fundamental intellectual curiosity and understanding of the disease. I'm at the point when my challenge to the PD establishment is, "can you do this better than Fox?" -- they are my standard for informed action. It makes me a little uneasy, but also brings me more comfort than I've had in years. I no longer believe that those who are trying to help us are entirely clueless.

Our voice remains our own - unique and powerful. I'm going to use mine to push any idea or agenda that improves our lot. Fox more and more seems to be the one that listens and takes action.

Carey, you are well known. You are on the MJFOX patient advisory council. You can contact the leaders at MJFOX and initiate and have a dialog about issues you feel are important. I'm very glad about that. But the rest of us are not in that position.

Jean

Jean - I know that - I am extremely aware that I represent every person who can't be in that room with me. It means that when it would be more comfortable to say nothing, I always err of the side of being annoying by asking one more question or making one last point. I don't take my situation for granted.

At the same time, everyone should be doing just what you are doing so well - speaking out wherever and whenever you can. It will take all of us.

I'm just saying that I'm finding it harder to remain neutral when one organization appears to have a better grasp of the situation from the patient point of view than the others. And that bothers me because I've always valued my neutrality. Just revealing my private angst publicly!

we must be unique and innovative too
 

The devil is in the details, as we are now plodding thru the editing and the endless specifics of the book. I'd like to reiterate that we can write how this book ends because the book came mostly from right here. This is an interactive time to make the future happen.

This forum is the original social networking. It has certainly changed in the last decade.

I don't want to speak for everyone, rather I need everyone to talk for me. The more voices the better. How should we bring this book to a conclusion? It will continue on the site and, we have ideas.

What do we really want - where is the table - who else is at the table?
what do patients specifically want? How would you summarize the current status of research? I don't think the doldrums statement applies to MJFF. And if Katie is going to look for ways to use us then i'm expressing my gratitude to her and anyone else who is planning to use patients more towards the solution with a repeat of this video. [below]

These are the kinds of productive threads that we like to put in the book.

I suggest that we all just say what we feel; all are accepted. Be honest and check your reasons for what you say before you say them. iF we reviewed all the posts, would the reasoning behind each be self interest or helping the cause? [in this thread not all threads] Stay true to yourself and your own choices. All have talent but teamwork means very low self interest; it's not about getting credit, "it's not about me" - this is truly a collective. we are planning to publish the book in other languages and could have other countries with their own section on the site.

ok so Katie Hood, this video is for you and the added meeting agenda item about using patients in the solution. i know we can help but we need access to dialogue with researchers. oR have researchers post here. we could offer info and things might move faster. we are good at researching and outstanding at making connections. But most of all, we know how it feels.

example: through my own doing i've requested meds, adding first PT, then joining the health club and going [very importantly] with my closest teaching friend 3 days a week. i had written myself off a few years ago...truly felt i was dying, well i was but from malnutrition and lac of exercise. i went down to 113 lb. one doesn't see the emaciation -it didn't matter because it was meds taking away my appetite and bad teeth being pulled and chewing and swallowing problems.

Now i can jog for an entire aquafit class and i recommend physical therapy - pd qualifies for a higher cap if there even is one. an area of progress. The point is i had to figure out my meds and discover that PT is extended for pwp myself.

i have no doubt that getting us all together would yield so many ideas that we should take up linda herman's call for a cure by the next world congress and turn it into a celebration [including a parkinson prom] please!

to katie and mjff for efforts to include patients. it is better than the last time i was on your meeting agenda list...lol......i'm so over it now .....and a thank you.

http://www.knightarts.org/uncategori...80%A8%E2%80%A8

We do need to be more vocal
 

I so very much appreciate that we have MJF and the MJFF looking out for us. The very fact that Debi Brooks bothers to check in with us tells us that we have a staunch ally in that organization. The problem for me is that they operate within the confines of an imperfect system...one that is not ready to see patients at the table as collaborators. I know that MJFF is making huge strides in improving how research is done and in actually applying it and making changes right here and now.

However, I often feel with MJFF that the focus is on the future of Parkinson's. I think I know why that is, but I feel frustrated that it seems like we who have had PD for awhile will not really benefit from all the exciting stuff they have going on with biomarkers. It is absolutely fundamental that they identify this disease before motor symptoms start...I get that, but what about those of us who are newly diagnosed or who have lived with this much longer? MJFF funds many, many drug studies that may benefit us, but what happens to them? We get back to that imperfect system and for those who will not benefit from biomarkers, we are still in stasis because MJFF works within that system.

I have long been in support of forming a renegade, grass roots, in your face, activist group of patients modeled after ACT UP of the AIDS community. ACT UP is fifteen years old, and I daresay they have advanced AIDS research and patient rights light years ahead of us...we are here 50 years later with the average person thinking that we are stooped over and shuffling with a tremor, or worse, some even think that dyskinesia IS the disease. I would love to see performance art combined with activism and the element of surprise so we would have organized, random acts of raising awareness "events" at Central Park, LAX airport, the shopping mall, Picadilly Circus, etc. all taking place at once. Sergey Brin is on our side, why can't we seemingly "hijack" Google for a day with a message about PD? I don't know...it seems at times we are only bound by our creativity.

Not only do we need to educate others in general about what it is like to feel trapped inside one's own body and tethered to little yellow pills, we need to be a thorn in the side of the AAN and the FDA. The AAN does not help matters by refusing to evolve the field of neurology even at the most basic level like changing what Parkinson's is called or named; maybe they are not responsible for making changes like this possible, but they seem the most likely group. We all know it is a syndrome or a spectrum disorder; it is well-documented in the neurology journals; they know it is as well. Isn't it time, we heard from key neurologists effort to move what is known about Parkinson's forward?

We have PAN, but we need way more. As Peggy said, we need to find our collective voice and use it.

Laura

current sufferers suffer
 

Laura - I'm not sure what you are looking for. Besides PPMI, MJFF is focusing on trophic factors, alpha synuclein, and dyskinesia (for current sufferers). Debi knows that the attention they are giving to biomarkers (PR, not research attention) right now, in order to promote recruitment, is taking away from what they are attempting to do for those who have lived with PD for years.

Regardless, until they can figure out the true nature of the disease, they can't solve the problems - we are stuck with controlling symptoms and their side effects. Do you think more research should be spent on this? It is a legitimate point of view.

It may be that we are just victims (sorry, Sheryl !!) of history. We've often looked at AIDS activism as a model for patient participation, but it only works so far - AIDS had the added urgency of being infectious and more swiftly deadly.

Part of our burden as activists is to get society at large - and even most health care professionals - to care about people living with a slowly progressing chronic disease. We're not easy to fix; doctors like to fix people. It's a conundrum. I'm not sure our target is solely the research community - it also includes those responsible for quality health care.

I agree we have much work to do in the area of information dissemination and education - especially targeted to general practitioners and neurologists - they are way behind in their PD knowledge and care practices. Believe it or not, this is also a topic of discussion with patients at MJFF.

not a bad idea
 

laura,

I like the idea of kidnapping google for a day. They change the letters all the time. Maybe asking them to make a PD google and using it once a month (same date each month/like the 1st or the last day of the month. They don't have to explain their involvement, just adopt the illness as their "personal" cause like Jerry Lewis and the annual Labor Day MD telethon. Has he ever divulged his reasons for doing it?

I've wondered why...
 

I've wondered why it hasn't been used for this purpose already unless maybe it is, ironically, simply too personal? Maybe there is a corporate policy against that sort of thing...though since he is the big kahuna he can do what he wants.
Anyway, it is such an obvious venue for Brin, I figure their must be a reason he does not go that route.

Okay, here's some info. The change in the logo is called a "Doodle" - Google actually holds an annual competition for children grades K-12 to design one.
In looking at their list of Doodles, it appears they go for random, off beat sort of things, so I could see PD fitting in for a April awareness month thing, but it doesn't look they would make it a permanent once a month rotation. I think they have a pretty smart marketing gimmick. People are all abuzz over Doodles because they are random, not patterned (like you don't see one for every given holiday), and designed to pop up in a rather spontaneous way. The fun is not knowing when one will appear and what it will commemorate. It's a rather unique way to create buzz, but making it a regular feature would take all that away.

Still, since so many people take note it would be grand to have it acknowledged once even. The question is given that limited exposure and that the message needs to be conveyed through an image, what would PWP want to emphasize? Heck, even just a play on the "Got milk?" ads with a "Got Dopamine?" headline or neurotransmitter pic would with a short video clip that can be viewed would be something that millions would be talking about.

Laura

google doodle for a day
 

Laura - I think that is the question - If we gained millions of peoples attention for just one day, what is the message? What would move the ball forward? What would be meaningful?

If we had a written or spoken message, how would that be translated into a visual graphic?

When the doodle shows up, can it link to more info or does it have to be self explanatory (like John Lennon on his 70th birthday)?

I think some times the mysterious ones get a lot of attention because people start asking, "what the ??!" and a buzz gets started.

se past google doodles
 

You can see all of the Google doodles for past 10 years

jean

An interesting article on the front page of today’s Buffalo News ,” Finding hope in a spider's bite : Venom holds a key to possible therapy for cruel illness, “ describes efforts by a local grandfather to help move an innovative, promising treatmment for Duchenne muscular dystrophy through the pipeline , by funding the pre-clinical research needed to reach clinical; trials. Hiis grandchild is suffering with the disese and is running out of time.

The full article is at:
http://www.buffalonews.com/city/article244438.ece

BTW an article published by UB in Jan 2010, stated that this treatment, “GsMTx4 appears to have additional applications. Robert Plunkett, associate professor of neurosurgery, has shown that the peptide stimulates neuronal growth and may be useful in the treatment of Parkinson’s disease….. “

Excerpts From the current article on Duchenne’s--
“…By age 10, affected children, almost always boys, usually need braces to walk.
By their teen years, most depend on a wheelchair.
And as they grow into manhood, they lose their ability to breathe. Few survive beyond their 30s.

There is no cure, and the standard treatment of steroids can slow -- but not stop -- the progressive muscle weakness, forcing families into a race against time.
All of which is why Jeffrey Harvey, a Clarence stockbroker, couldn't sit still when his grandson, also named Jeffrey, was diagnosed with the disease more than a year ago.
Harvey began to look for potential new treatments, surfing the Internet for anything with promise. Out of curiosity, he searched in his hometown, which led him to a laboratory at the University at Buffalo and to a tarantula spider named Rosie.

The story of his search highlights a novel potential treatment for muscular dystrophy -- one of several therapies on the horizon. It also shows how challenging it can be to bring a drug from the laboratory to a patient's bedside….”
"There is just no drug for these kids other than the steroids to strengthen the muscles, and the steroids have serious side effects," said Harvey, whose grandson is now 3.
“UB biophysicists had found a protein in tarantula venom that showed promise against muscular dystrophy, but the idea was greeted skeptically. No one responded when they shopped it around to pharmaceutical companies earlier in the decade.

Then Harvey called.,,”

Harvey and the researchers formed a small biotech company that hopes to do what the big drug companies wouldn't do,,.
"I want my grandson to be able to have this drug," Harvey said…

“In September, the Food and Drug Administration gave GsMTx4 orphan drug status, a designation for experimental treatments of rare conditions that helps speed development.
Now, comes the difficult part -- seeking an estimated $3 million to fund testing before the drug goes into trials in children.

"The thing is, unlike a lot of others, we already have the drug. If we had the money to get it through preclinical testing, we might have it on the market in three years," Harvey said.
That's not much money or time in the world of drug development, where companies typically spend hundreds of millions of dollars and more than a decade on one product..”

Maybe this is part of our answer. Bypass the drug companies...Scientists, working with patients and families . .. Understand the urgency of getting treatments to patients sooner, not later... Welcome patients at the table, instead of banning them from scientific meetings.

Are the Doldrums based on business or scientific oppotunities?

We don’t know if the Tarantula cure will pan out, but at least its being given a chance based on the science, not sitting on a lab shelf, because of a business decision.

There are some really interesting thoughts coming out here.....

The thing of getting people to look at PD seems to be difficult, it is hard to present PD in a way that takes us away from stereotypes, nobody seems to have been able to come up with anything other that MJF or Ali to represent us.

the doodle idea is great,whats needed is a fantastic recognizable image that hits the spot.

I sometimes think that we need to take it out of the familiar and into a different context that will shock - something like a brief video in which kids play pwp world - slow shuffly unsmiling freezing falling over speaking quietly...... if we were kids we would stand out a mile........... and people would be shouting for something to be done.... we could do that, kids, grandkids might love to be roped into doing us!

Back to the future,we want research to give us a future,wellness with illness and ultimatley a cure.Much is happening that will be of benefit to us,I applaud that.
But throughout research history people have given up there lives,for some of the greatest discoveries.The days when guts luck instinct and imagination were your only tools.These things you cant quantify and plan,unpredictables with no measured outcomes but risk
The greatest risk today is in investing and loosing money,not lives.Improving lives,has become secondary to protecting market strategies and making profit.The greatest profit to us all would be improved health,for the benefit of living and the global health economy.Patent is king of monopoly ,right or wrong there are some things we cant change,but there is much we can.It amused me recently to read an ethical debate on the subject of a famous pharoah and wether his DNA analysis could be released without informed consent from him or a direct descendant,3000yrs dead my memoirs can be safely published then,pehaps, but I must make provision for the eventuality.

I cannot, for the life of me, understand this wall between patients and the scientific/medical community. With the Net it would be so simple to have an ongoing roundtable dedicated to the mutual education of the two groups based on mutual respect and acceptance of each one's ignorance. I know that it would require some innovative thinking to make the two worlds mesh.

Maybe a structure where a group of "moderators" served as translators and filters? The latter would address basic questions that could be answered by simple research on Google outside the forum. Once past that threshold, however, it should be a brainstorming format with the understanding that challenging the established wisdom is an integral part of it.

Now your talking
 

Absolutley no reason why not.
Have you ever thought about the neuros perspective,every one of us going through his door will come out with a different regime of meds, its not easy to get it right more reason than ever to be partners.

QUOTE=reverett123;713755]I cannot, for the life of me, understand this wall between patients and the scientific/medical community. With the Net it would be so simple to have an ongoing roundtable dedicated to the mutual education of the two groups based on mutual respect and acceptance of each one's ignorance. I know that it would require some innovative thinking to make the two worlds mesh.

Maybe a structure where a group of "moderators" served as translators and filters? The latter would address basic questions that could be answered by simple research on Google outside the forum. Once past that threshold, however, it should be a brainstorming format with the understanding that challenging the established wisdom is an integral part of it.[/QUOTE]

This may sound ludicrous . . .
 

. . . but would it be possible that some doctors feel intimidated (not necessarily the same as threatened) by the knowledge base some PWP groups possess? That would make them have to keep on their toes and mind their P's and Q's. This would likely be an area where they might become red-faced if put nose-to-nose with knowledgeable patients. I am not categorizing all doctors as believing they are on a higher rung of the medical community ladder, but some do have the "holier than thou" attitude.

Seriously understand that I am NOT saying some of us know MORE than a great number of neurologists, because we have never been clinicians. However, WE are the ones living day-to-day with the one disease that most always presents itself as highly ambiguous (even mysterious at times) and sometimes baffles even the best neurology experts.

Sometimes it takes a fresh perspective to shine the light on a mysterious disease such as Parkinson's. As those who have the disease, we have nothing to lose and everything to gain by shining our flashlights in dusty, spider-web infested corners; most doctors are in just the opposite position. The distinction comes from what one perceives as the "prize" . . . are they looking for a blockbuster therapy . . . or are they looking for a cure?

I am convinced that we will one day run across a rebel who is willing to take a risk and step out on faith as to why, how, where, and when this neurological body system breaks down and turns into PD. In fact, I believe he/she is already working toward that designation.

Will the REAL Parkinson's reseacher please stand up?
Peg