Showing posts with label research initiatives. Show all posts
Showing posts with label research initiatives. Show all posts
Monday, June 25, 2012
Protecting the People Who Test New Parkinson’s Treatments
We all want better treatments for Parkinson’s. Tuesday’s announcement that one of PDF’s Research Centers - Rush University Medical Center, in Chicago, IL - received full accreditation from The Association for the Accreditation of Human Research Protection Programs, Inc. (AAHRPP) is another step in the right direction.
Fewer than 200 institutions around the country are fully or partially accredited by AAHRPP.
Before any new treatment, for PD or another condition, is approved it must first be tested in people – in clinical research studies – to ensure it is safe and effective. The AAHRPP accreditation means that Rush is following the highest standards when it comes to protecting those people.
As we discuss at length at trainings for PDF’s Parkinson’s Advocates in Research program, testing an experimental treatment always involves some risk – the reason a treatment is not yet available to everyone is that we don’t yet fully understand how it works. But we need to minimize those risks as much as we can and protect the rights of participants.
AAHRP’s work is part of that progress. It tests research centers on a number of criteria to make sure the institutions are up to par. To do its part, PDF has been involved in several initiatives, including collaboratively working on a Research Participants Bill of Rights, several pieces of which are featured in our educational booklet here.
If you would like to learn more about research studies and how you can ensure people with Parkinson’s are involved, learn more about our PAIR program or join our ongoing online course,which runs every Thursday in June (the recordings will be posted in July).
Friday, June 15, 2012
The Importance of Brain Banks for Neurological Research
Would you consider donating your brain for science?
A few years ago, Diana Barnwell wrote an article, entitled, My Last Gift. She chronicled her decision to donate her brain … and the complicated issues that arose when making her decision.
Studying actual human brains – donated by individuals upon their death, as a contribution to science – is a vital way to understanding neurological diseases such as Parkinson’s. After all, the brain is the only mysterious organ that we can’t study during a person’s lifetime.
This is why it was so devastating to scientists around the world, particularly those at the Harvard-affiliated McLean Hospital, when the institutions’ brain bank suffered a freezer dysfunction last week. The institution lost 150 brains that had been donated for research.
They say this constitutes one-third of the world’s largest collection of autism brain samples. The brain bank also housed brains designated for Parkinson’s research.
How does brain donation work? It varies at institutions around the country – from their requirements for donating to whom they consider eligible. For instance, some institutions require a donor to be seen several times a year at their center, while others require just one visit. Most times, much like any kind of organ donation, the decision to donate needs to be made well ahead of time.
For a listing of Parkinson’s brain banks in the US, browse PDF’s website, which includes our own PDF-funded brain bank at Columbia University Medical Center.
Back in 2010, Diana said her decision to donate was, “a magnificent legacy!” Do you agree?
A few years ago, Diana Barnwell wrote an article, entitled, My Last Gift. She chronicled her decision to donate her brain … and the complicated issues that arose when making her decision.
Studying actual human brains – donated by individuals upon their death, as a contribution to science – is a vital way to understanding neurological diseases such as Parkinson’s. After all, the brain is the only mysterious organ that we can’t study during a person’s lifetime.
This is why it was so devastating to scientists around the world, particularly those at the Harvard-affiliated McLean Hospital, when the institutions’ brain bank suffered a freezer dysfunction last week. The institution lost 150 brains that had been donated for research.
They say this constitutes one-third of the world’s largest collection of autism brain samples. The brain bank also housed brains designated for Parkinson’s research.
How does brain donation work? It varies at institutions around the country – from their requirements for donating to whom they consider eligible. For instance, some institutions require a donor to be seen several times a year at their center, while others require just one visit. Most times, much like any kind of organ donation, the decision to donate needs to be made well ahead of time.
For a listing of Parkinson’s brain banks in the US, browse PDF’s website, which includes our own PDF-funded brain bank at Columbia University Medical Center.
Back in 2010, Diana said her decision to donate was, “a magnificent legacy!” Do you agree?
Thursday, June 14, 2012
Easing Dyskinesia: PDF-funded Research from 2007 Leads to Testing of Experimental Drug
Earlier this week, PsychoGenics Inc., announced that an experimental drug for Parkinson’s, eltoprazine, seemed to reduce dyskinesia in people with Parkinson’s in early studies. Dyskinesias are the twisting and writhing movements that occur as PD progresses – a common side effect of the medication levodopa (Sinemet®).
Back in 2007, it was PDF-funded researcher Manolo Carta Ph.D., along with Anders Björklund, M.D., who performed the pre-clinical research that led to the identification of this drug. At the time, while many researchers were looking at dopamine neurons as the culprit behind dyskinesias (through their interaction with levodopa), Dr. Carta’s proposal suggested something different – serotonin neurons. His proposal led to a one-year research fellowship funded by PDF.
After completing his year of research with PDF funding during which he laid the foundation for this approach, he and his colleagues were able to investigate an experimental compounds that might help with dyskinesias. With the support of other funders, they have now been able to study the effects of eltoprazine in people in this early stage trial – to see whether it is a viable drug treatment.
The study results announced earlier this week indicate positive news about eltoprazine’s potential to ease dyskinesia and possibly some non-motor symptoms of PD.
However, the drug will have to undergo rigorous testing – in additional people with Parkinson’s in phase II and phase III clinical trials – before we know if it’s safe and effective. While we don’t yet know the fate of this drug, the results help us learn more about dyskinesias so we can find a solution in the future.
PDF believes it’s important to fund ideas like this so that scientists can have both the freedom to explore novel ideas for Parkinson’s and the time they need to gather data that can prove the promise of their ideas. In Dr. Carta’s work, this proved to be the case.
We believe this philosophy – funding creativity early on – will help researchers prove their case to other funders that can help them to further develop their ideas ... ultimately into new treatments and a cure for Parkinson’s.
What are your thoughts? Do you or a loved one need a drug for dyskinesia?
Please share your thoughts below, and as always, call our HelpLine at (800) 457-6676 with any questions you or a loved one may have about Parkinson's.
Back in 2007, it was PDF-funded researcher Manolo Carta Ph.D., along with Anders Björklund, M.D., who performed the pre-clinical research that led to the identification of this drug. At the time, while many researchers were looking at dopamine neurons as the culprit behind dyskinesias (through their interaction with levodopa), Dr. Carta’s proposal suggested something different – serotonin neurons. His proposal led to a one-year research fellowship funded by PDF.
After completing his year of research with PDF funding during which he laid the foundation for this approach, he and his colleagues were able to investigate an experimental compounds that might help with dyskinesias. With the support of other funders, they have now been able to study the effects of eltoprazine in people in this early stage trial – to see whether it is a viable drug treatment.
The study results announced earlier this week indicate positive news about eltoprazine’s potential to ease dyskinesia and possibly some non-motor symptoms of PD.
However, the drug will have to undergo rigorous testing – in additional people with Parkinson’s in phase II and phase III clinical trials – before we know if it’s safe and effective. While we don’t yet know the fate of this drug, the results help us learn more about dyskinesias so we can find a solution in the future.
PDF believes it’s important to fund ideas like this so that scientists can have both the freedom to explore novel ideas for Parkinson’s and the time they need to gather data that can prove the promise of their ideas. In Dr. Carta’s work, this proved to be the case.
We believe this philosophy – funding creativity early on – will help researchers prove their case to other funders that can help them to further develop their ideas ... ultimately into new treatments and a cure for Parkinson’s.
What are your thoughts? Do you or a loved one need a drug for dyskinesia?
Please share your thoughts below, and as always, call our HelpLine at (800) 457-6676 with any questions you or a loved one may have about Parkinson's.
Monday, March 26, 2012
Notes from a Day Spent Listening to Scientists Judging PDF Research Applications
Last Friday, March 16, was the occasion for reviewing applications for PDF’s International Research Grants program and Research Fellowships programs. Before us were some 30 proposals from some of the best young (and not-so-young) scientific investigators in the world.
As I sat there in the meeting as an observer, listening to the members of our scientific review committee as they made their comments and pronounced their judgments, I found myself scribbling notes on what seemed to be the principles on which they were basing their decisions. What they were saying, it seemed to me, said a lot about how we go about choosing the projects we will fund. Here are some of the ideas I picked up:
Relevance to Parkinson’s Disease. Every proposal that we fund – without exception -- must show promise in its potential to advance our understanding of PD, or charting the path to its cure.
New Ideas, New Investigators. To maximize the leverage of the program, successful applications will be one of two kinds. One is the innovative pilot project that shows promise for leading to a larger-scale endeavor that will be eligible for funding from the NIH (drawing on PDF’s “leverage” function). The other is the Parkinson’s-related application from an exemplary scientist whose past contributions have been largely in areas other than Parkinson’s – and who could be inspired by means of the PDF grant to turn his or her attention to PD.
Demonstrating Results. The renewal of an earlier award to the same scientist depends on the investigator’s ability to show “significant progress” since the first grant – that is, you don’t get a second grant if you can’t show that you used the first grant well.
Establishing Credibility. To make it through the grant review process, good ideas aren’t enough; the applicant needs to be able to demonstrate – both in his/her personal accomplishments and in the reputation of the lab in which the work will be done – a stellar track record in producing innovative and useful science.
Including Advocates in the Research Process. The meeting included three members of Parkinson’s Advocates in Research (PAIR), a PDF program in which lay leaders in the PD community are prepared to take on a variety of roles advocating for clinical research. An example of their contribution came early in the day, when one of these advocates raised a question about the validity of animal models in Parkinson’s research, which generated a spirited exchange among the scientist members of the committee.
I hope you are as impressed as I am with this little vignette of PDF’s research culture!
I conclude with a quotation from a memo on the mission of the program from Dr. James Beck, who is our Director of Research Programs at PDF and staffs the grants review committee. In thanking the scientists in the room, he said, in effect: “The two groups that will benefit most from your decisions today are not even here. One is the world of talented young scientists whose work will be made possible through your efforts. The other is the community of almost one million in the U.S. who live with Parkinson’s. In behalf of these two communities, PDF thanks you!”
Amen, James.
As I sat there in the meeting as an observer, listening to the members of our scientific review committee as they made their comments and pronounced their judgments, I found myself scribbling notes on what seemed to be the principles on which they were basing their decisions. What they were saying, it seemed to me, said a lot about how we go about choosing the projects we will fund. Here are some of the ideas I picked up:
Relevance to Parkinson’s Disease. Every proposal that we fund – without exception -- must show promise in its potential to advance our understanding of PD, or charting the path to its cure.
New Ideas, New Investigators. To maximize the leverage of the program, successful applications will be one of two kinds. One is the innovative pilot project that shows promise for leading to a larger-scale endeavor that will be eligible for funding from the NIH (drawing on PDF’s “leverage” function). The other is the Parkinson’s-related application from an exemplary scientist whose past contributions have been largely in areas other than Parkinson’s – and who could be inspired by means of the PDF grant to turn his or her attention to PD.
Demonstrating Results. The renewal of an earlier award to the same scientist depends on the investigator’s ability to show “significant progress” since the first grant – that is, you don’t get a second grant if you can’t show that you used the first grant well.
Establishing Credibility. To make it through the grant review process, good ideas aren’t enough; the applicant needs to be able to demonstrate – both in his/her personal accomplishments and in the reputation of the lab in which the work will be done – a stellar track record in producing innovative and useful science.
Including Advocates in the Research Process. The meeting included three members of Parkinson’s Advocates in Research (PAIR), a PDF program in which lay leaders in the PD community are prepared to take on a variety of roles advocating for clinical research. An example of their contribution came early in the day, when one of these advocates raised a question about the validity of animal models in Parkinson’s research, which generated a spirited exchange among the scientist members of the committee.
I hope you are as impressed as I am with this little vignette of PDF’s research culture!
I conclude with a quotation from a memo on the mission of the program from Dr. James Beck, who is our Director of Research Programs at PDF and staffs the grants review committee. In thanking the scientists in the room, he said, in effect: “The two groups that will benefit most from your decisions today are not even here. One is the world of talented young scientists whose work will be made possible through your efforts. The other is the community of almost one million in the U.S. who live with Parkinson’s. In behalf of these two communities, PDF thanks you!”
Amen, James.
Friday, March 16, 2012
Grant Review at PDF
Today is a big day for many at PDF — it is our annual grant review. Of course, those most affected by what goes on in the big conference room will not be there. There are two groups who depend on tomorrow's outcome: the scientists who have given us their best ideas for our scientific advisors to judge and the people who live every day with Parkinson's and are counting on these great ideas to make a difference in their disease and their lives. I am confident that the team of scientists helping — members of our scientific advisory board and other experts from around the country — will select only the best science today.
All in all, nearly 25 reviewers have pitched in their time to help whittle down the 200 letters of intent to 63 full grant proposals to, now, only the top 20 or so grants. Because of time, it is these top 20 applications that will actually get discussed.
And for the second time, PDF is doing what no other PD organization in America is doing—making certain those people living with PD have a literal seat at the table and participate in the decision making process. These people, PDF's Research Advocates, part of the Parkinson's Advocates in Research program, insure what we will fund is not just the best for science's sake but for the sake of people too. Something in which we, as an organization, take great pride and you as a community should too.
Tomorrow would be difficult to organize without the obvious help of my colleagues at PDF. But equally important is the help from everyone who will not be joining me in the room — you, the Parkinson’s Community. Your generous support of allows us to do what we do every day: support research and ideas that will improve the lives and futures of people touched by Parkinson’s. Our research program would simply not be without you. Thank you.
All in all, nearly 25 reviewers have pitched in their time to help whittle down the 200 letters of intent to 63 full grant proposals to, now, only the top 20 or so grants. Because of time, it is these top 20 applications that will actually get discussed.
And for the second time, PDF is doing what no other PD organization in America is doing—making certain those people living with PD have a literal seat at the table and participate in the decision making process. These people, PDF's Research Advocates, part of the Parkinson's Advocates in Research program, insure what we will fund is not just the best for science's sake but for the sake of people too. Something in which we, as an organization, take great pride and you as a community should too.
Tomorrow would be difficult to organize without the obvious help of my colleagues at PDF. But equally important is the help from everyone who will not be joining me in the room — you, the Parkinson’s Community. Your generous support of allows us to do what we do every day: support research and ideas that will improve the lives and futures of people touched by Parkinson’s. Our research program would simply not be without you. Thank you.
Tuesday, July 13, 2010
A Tale of Two Research Strategies
From James Beck, Ph.D., Director of Research ProgramsI hope you saw the news item PDF posted last week in which we announced $1.2 million in funding from two of PDF’s investigator-initiated grants programs for 13 Parkinson's research projects.
I want to share with you the approaches behind these two programs – both because they are philosophies of which I am particularly proud and more importantly, because we at PDF think these philosophies may also yield “the next great idea” for PD.
First among these approaches is PDF's goal to fund grassroots research, which we do through our International Research Grants Program, or IRGP. Here, PDF puts out a call for proposals that are rooted in the creativity of individual scientists — folks who are experts in their field with first-hand knowledge of the science of PD. The creativity of these scientists is brought to bear to tackle a set of problems on the path to a cure. Each scientist formulates his/her ideas into testable hypotheses and the merits of those ideas are then judged by an independent panel of PD researchers convened by PDF. PDF then extends support - up to the limit of available resources made possible by our donors - to the best ideas in our effort to affect a cure and help those living with the disease.
This approach to finding and funding the best science is certainly not new, though in the business world it is akin to the very popular method of “crowdsourcing” ideas...where new ideas are not generated by an organization, but by a community. In the world of PD, this approach of supporting ideas from individuals has led to the biggest advances in managing PD – levodopa and DBS.
Thus, PDF aims – by leveraging its initial research investment into supporting great ideas generated by the “crowd” of scientists – to help bring about the next big thing in PD.
Another related approach to research employed by PDF is to invest in talented young researchers. As the first not-for-profit organization created to focus on PD, PDF has an established commitment to fighting PD. Because the battle has yet to be won, we absolutely must invest in the future to ensure PD’s defeat. So, through our Fellowship Program, we are working to make certain that the best and brightest young talent joins and continues the fight. In this program, PDF funds young investigators to support them at a critical juncture in their scientific training – whether it be basic science or clinical research.
Since PDF’s grants are awarded exclusively on scientific merit, it is amazing to see how many young investigators were able to generate ideas that successfully competed in this year’s grant competition. Less than half (66) of the 150 total scientists who applied were young scientists fresh from their doctorates…yet, of the eight grants awarded this year, six went to support the Fellows in this category. This is quite an achievement. It provides great promise for the future of Parkinson’s research.
For more information on projects we’re funding, browse this year’s abstracts. We look forward to reporting on the results of this research and the impact we hope it makes upon science and our understanding and treatment of PD. Stay tuned.
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