[paula_w]

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/

[violet green]

[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

[swept]

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?

[paula_w]

have they read about this?

By Cristina Luiggi
Same poop, different gut

For physicians and researchers alike, fecal transplants present an opportunity to gain insight into disease

[Published 3rd November 2010 02:47 PM GMT]

Most people might find the idea of having another person's feces injected into their intestine hard to stomach, but for those with intractable gastrointestinal problems, another person's bodily waste is all that's standing between a lifetime of severe illness and a full recovery.

This therapy — known as fecal transplants, bacteriotherapy, or human probiotic infusions — has taken to the limelight in recent

Read more: Same poop, different gut - The Scientist - Magazine of the Life Sciences http://www.the-scientist.com/news/di...#ixzz14PiuRU1D

http://www.pdonlineresearch.org/disc...transplants-pd

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.?

swept this isn't directed at your post...i will respond to it but was working on this one when you posted.

[tulip girl]

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


At least something in the pipeline.

TG

[paula_w]

Originally Posted by swept

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?

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!

[indigogo]

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.

[Conductor71]

Originally Posted by indigogo

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.

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

[LindaH]

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.

[lindylanka]

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!