Showing posts with label speeding research. Show all posts
Showing posts with label speeding research. Show all posts

Monday, August 19, 2013

Old Drugs – New Cures?



From James Beck, Ph.D., Vice President of Scientific Affairs

Using old drugs as new cures seems like a surefire winner. It may be. However, after attending a recent meeting outside London hosted by the Cure Parkinson’s Trust, a small yet impactful British charity, it is clear that this path is neither clear nor easy. A committee of experts at the meeting evaluated and prioritized dozens of existing compounds – many are drugs used to treat other diseases – based upon their potential to stop Parkinson’s disease.

From left, James Beck, Tom Isaacs, and Steven DeWitte at
Cure Parkinson's Trust meeting at Cumberland Lodge.

Repurposing drugs for a new use like this has the potential to shave significant time from the drug development process for Parkinson’s. But existing drugs come with existing problems. And these problems cannot always be overcome. For instance, a recent science article covered by PDF suggested that a chemotherapeutic agent, nilotinib, might be useful for treating PD. However, it is not clear what the proper dose should be (the article suggested a low one would be effective). Moreover, with potential side effects that include sudden death, the committee felt it was not ethical to consider placing people living with PD in potential danger.

Still, there are drugs that appear to be safe and well tolerated, like statins used to lower cholesterol and metformin used for type II diabetes to name a few. These drugs are not chosen at random: By surveying thousands of people, epidemiology studies have been able to link drugs people take for other conditions (like these) with a lower risk of developing PD. There are also data from laboratory studies that suggest how these types of drugs may help. Even here, though, the path forward is not so clear. The mechanism of action, that is how might some of these drugs actually affect PD in people, is not known. This is not always a stumbling block; but when combined with drugs that are likely to have modest effects and low odds of success, it can be a problem, especially with limited resources.

Recognizing these issues, the Trust’s prioritization committee took a hard look at each potential drug candidate and recommended a handful as most promising. These few culled from a list of two dozen compounds will now advance towards evaluation in clinical trials. Hopefully, that phase will begin soon.

Tuesday, July 16, 2013

Righting the Clinical Research "Enterprise"


By Ronnie Todaro, M.P.H., Director of National Programs

When opening the Sunday Review section of the New York Times this past weekend,  I did a double-take as a topic of much discussion here at PDF was featured front and center - trumping commentary on the state of the economy, international relations and the latest political debate.

"Do Clinical Trials Work?," by Clifton Leaf serves as a primer on some of the key issues that prevail within the clinical research enterprise - the tension between scientific inquiry and patient priorities; the odds that study sponsors take when betting on the success of a potential therapy; and the disconnect between those who participate in a study and those who may actually benefit from the treatment being tested. It also serves as yet another wake-up call on why people who are living with a disease, be it cancer, Alzheimer's or Parkinson's, need to be formally included in research decision-making.

I can't help but wonder, would clinical research be in a different state if people who actually had the disease were sitting around the table, eye-to-eye with researchers, study sponsors and federal regulators? Would their presence and perspective change the priorities and processes of clinical research? Would it create the sense of urgency that occurs when the personal is married with the professional?

Of course, we here at PDF would  answer in the affirmative as would our 200 Research Advocates across the country.  The bottom line - we can right this "Enterprise" - but only if we recognize who should be at the helm - the patient.

Tuesday, July 3, 2012

Understanding the Progression of Parkinson's

Can you predict the progression of Parkinson's? ... or change its course?

Last Tuesday, during PDF's PD ExpertBriefing, "Understanding the Progression of Parkinson's," presenter Dr. Ron Pfeiffer had a few suggestions:
  • Dr. Pfeiffer provided facts about PD progression - about the percentage of people with PD who develop certain symptoms or leave their jobs after a certain number of years - but also emphasized that these are general facts.  Each person's journey with Parkinson's is very different.
  • He noted that there are no treatments proven to reverse PD, but he also emphasized there are actions people can take, such as exercising and joining support groups, that may improve life with PD. (In fact, there is extensive research into exercise right now.)
He also stressed that the picture of Parkinson's he painted during his presentation ... is how the progression Parkinson's looks now.  It's not necessarily the picture of PD in five, 10 or 15 years.  So how can we change it as quickly as possible?  Here's are some suggestions from PDF:
  1. Advocate for PD Research: There may not be a cure for Parkinson’s, but you can be a part of the solution.  Join more than 150 PDF Research Advocates who are speeding the development of new treatments through the Parkinson’s Advocates in Research program.  In the Midwest?  We'll announce dates for our in-person three-day training in your region later this month and invite you to apply.  Don't have the time to commit to being a PDF Research Advocate or don't see a training nearby you right now? Take our four-part online course (available in mid-July) to advance your knowledge, and work with our Research Advocates in speeding new treatments. 
  2. Inform Others About Parkinson’s: Parkinson's disease is not well understood.  Spread the word in your community and bring the latest educational information to families touched by Parkinson’s.  Browse PDF’s Awareness Toolkit to find tips for raising awareness that work for you, whether writing a letter to the editor or setting up a display in your library.
  3. Fundraise for Research: PDF just announced $5.3 million in funding for Parkinson's research. We were only able to do so because of your support.   Support a PD organization, or join PDF Champions, the inspiring individuals who are raising funds for PDF’s programs, to improve the lives and futures of people touched by Parkinson’s.  Whether you run a lemonade stand that raises $20 or a golf tournaments that raises $50,000, you help to move the cause forward.
These are our ideas. What do you do individually to take charge of your Parkinson's?  What ideas do you have for the community to make progress now?