Showing posts with label National Institute of Health. Show all posts
Showing posts with label National Institute of Health. Show all posts

Tuesday, February 4, 2014

NIH, Industry and Non-Profits Join Forces to Speed Validation of Disease Targets


John Coakley, Sr., President of the KDA, sent out the following press release from the NIH along with this comment: 

“What a great program! 

We can begin to think about the Kennedy’s Disease Association  ‘partnering' with others in the future. It's not as 'far-fetched' as you might think. Let's all look ahead five years and open our minds to unlimited possibilities.”



For Immediate Release: Tuesday, February 4, 2014, 8 a.m. EST 
NIH, INDUSTRY AND NON-PROFITS JOIN FORCES TO SPEED VALIDATION OF DISEASE TARGETS

The goal is to develop new treatments earlier, beginning with Alzheimer's, type 2  diabetes, and autoimmune disorders.
 
The National Institutes of Health, 10 biopharmaceutical companies and several non-profit organizations today launched an unprecedented partnership to transform the current model for identifying and validating the most promising biological targets of disease for new diagnostics and drug development.

The Accelerating Medicines Partnership (AMP) aims to distinguish biological targets of disease most likely to respond to new therapies and characterize biological indicators of disease, known as biomarkers. Through the Foundation for the NIH (FNIH), AMP partners will invest more than $230 million over five years in the first projects, which focus on Alzheimer's disease, type 2 diabetes, and the autoimmune disorders rheumatoid arthritis and systemic lupus erythematosus (lupus).

A critical and groundbreaking element of the partnership is the agreement that the data and analyses generated will be made publicly available to the broad biomedical community.  The three- to five-year, milestone-driven pilot projects in these disease areas could set the stage for broadening AMP to other diseases and conditions.

"Patients and their caregivers are relying on science to find better and faster ways to detect and treat disease and improve their quality of life," said NIH Director Francis S. Collins, M.D., Ph.D. "Currently, we are investing a great deal of money and time in avenues with high failure rates, while patients and their families wait. All sectors of the biomedical enterprise agree that new approaches are sorely needed."

"The good news is that recent dramatic advances in basic research are opening new windows of opportunity for therapeutics," continued Dr. Collins. "But this challenge is beyond the scope of any one of us and it's time to work together in new ways to increase our collective odds of success. We believe this partnership is an important first step and represents the most sweeping effort to date to tackle this vital issue."

As a result of technological revolutions in genomics, imaging, and more, researchers have been able to identify many changes in genes, proteins, and other molecules that predispose to disease and influence disease progression. While researchers have identified thousands of such biological changes that hold promise as biomarkers and drug targets, only a small number have been pursued. Choosing the wrong target can result in failures late in the development process, costing time, money, and ultimately, lives. Currently, developing a drug from early discovery through U.S.  Food and Drug Administration approval takes well over a decade and has a failure rate of more than 95 percent. As a consequence, each success costs more than $1 billion.

"The AMP rallies scientific key players of the innovation ecosystem in a more unified way to address one of the key challenges to Biopharma drug discovery and development," said Mikael Dolsten, M.D., Ph.D., President of Worldwide Research and Development at Pfizer. "This type of novel collaboration will  leverage the strengths of both industry and NIH to ensure we expedite translation of scientific knowledge into next generation therapies to address the urgent needs of Alzheimer's, diabetes and RA/lupus patients."

AMP has been more than two years in the making, with intense interactions etween scientists in the public and private sectors, progressive refinement of the goals, strategy development support from the Boston Consulting Group, and scientific project and partnership management by the FNIH. Through this effort, AMP partners have developed research plans and are sharing costs, expertise, and resources in an integrated governance structure that enables the best informed contributions to science from all participants.

Monday, May 21, 2012

Petition to expand NIH Public Access Policy

NIHThe KDA received the following request from Sharon F. Terry, President and CEO, of Genetic Alliance. I signed the petition because this has been something that has bothered me for some time. Read Ms. Terry’s letter and the petition (also shown below) and please consider adding your support to it. Thank you.
___________________________

Dear friends,


Through the groundswell of our network, we've opened up access to the results of federally funded NIH research – let's take that another step!


As you know, the Obama Administration has been actively considering the issue of Public Access to the results of Federally Funded research this year. We have a brief, critical window of opportunity to demonstrate our strong commitment to expanding the NIH Public Access Policy across all U.S. Federal Science Agencies. The Administration is currently considering which policy actions are priorities that will they will act on before the 2012 Presidential Election season swings into high gear, an we need to ensure that Public Access is one of those priorities.


To help accomplish this, today, Monday, May 21st, a petition calling for Public Access to Federally Funded Research has been posted to the White House's "We the People" site.  When the petition garners 25,000 signatures within 30 days, it will be reviewed by White House staff, and considered for action.
To reach this number of signatures, we need you sign the petition at wh.gov/6TH and to do all that you can to activate your networks to do the same.  Qualified signers must be at least 13 years old, have a valid email address, and can come from inside or outside of the U.S. 


We are asking you to please not only sign onto the petition, but to spread the word far and wide. Consider a blog post, an email to constituencies, a tweet, a Facebook share, an action in your library  - anything that tells as many people as possible "I support this petition, I'm signing this petition, and I you to sign it also."  This is the kind of action that will have real consequences when we reach the 25,000 signatures goal - the White House takes this petition site very seriously, and is particularly cognizant of public opinion as the Presidential election draws closer.


Thanks in advance for your support for this effort - together we can expand the NIH Public Access Policy to all US Federal Science Agencies! 
______________________________

The petition reads:

We the people
WE PETITION THE OBAMA ADMINISTRATION TO:


Require free access over the Internet to scientific journal articles arising from taxpayer-funded research.


We believe in the power of the Internet to foster innovation, research, and education. Requiring the published results of taxpayer-funded research to be posted on the Internet in human and machine readable form would provide access to patients and caregivers, students and their teachers, researchers, entrepreneurs, and other taxpayers who paid for the research. Expanding access would speed the research process and increase the return on our investment in scientific research.


The highly successful Public Access Policy of the National Institutes of Health proves that this can be done without disrupting the research process, and we urge President Obama to act now to implement open access policies for all federal agencies that fund scientific research.
We the People … Full URL to the petition: https://wwws.whitehouse.gov/petitions/%21/petition/require-free-access-over-internet-scientific-journal-articles-arising-taxpayer-funded-research/

Sunday, July 17, 2011

NIH now accepting applicants for clinical trial on Kennedy’s Disease and Exercise

Saturday’s KDA chat room had a special guest.  Christopher Grunseich, MD, Neurology, from the National Institute of Health joined the chat to explain the current clinical trial on exercise. 

Dr. Grunseich explained that NIH is currently accepting patients for this trial.  The doctor also provided information on the type of candidates they are hoping to recruit, the length of the trial and other pertinent information. 

NIH
A. Objective:
  • To see if a 12-week program of either strength exercise or stretching exercises will improve strength, function, or quality of life in people with SBMA
  • This trial is a ‘single-blind’ study.
  • NIH hopes to recruit 50 qualified candidates
B. Duration:
  • The subjects will be in the study for a total of 16 weeks- 12 weeks of exercise and then a 4 week follow up period. The person would go to NIH at the beginning and then again 12 weeks later.
  • NIH will pick up the costs for travel expenses.
C. Eligibility Criteria:
  1. Genetically confirmed SBMA.
  2. Ambulatory and walk a distance of at least 50 feet with or without a walker.
  3. Able to stand for 10 minutes without the use of any assistive devices.
  4. Willing to travel to the NIH at the beginning and end of the study.
  5. Willing to participate in telephone monitoring.
  6. AMAT (adult myositis assessment tool test) score of less than 41, but greater than 14.
  7. Male.
  8. Willing to participate in all aspects of trial design and follow-up.
  9. Access to a computer with an internet connection
  10. Able to do all of the exercises according to the standards of the study examiners at the beginning and end of the study
  11. Willing to forgo starting an additional exercise plan for the 12 week duration of the study
  12. Age greater than 18 years
D. For More Information:

Friday, June 10, 2011

Clinical Research Study on Kennedy’s Disease

For several months now we knew a clinical study was coming.  This week NIH posted the following information on their website.

I feel this is an important study since I am an advocate of the benefits of exercise.  If you are interested in participating in this study, the contact information is near the bottom of this page. 

NIH Clinical Research Studies

  Active Accrual, Protocols Recruiting New Patients

Effect of Functional Exercise in Patients with Spinal and Bulbar Muscular Atrophy
Number: 11-N-0171
 
A.  Summary: Background: Spinal and bulbar muscular atrophy (SBMA) is an inherited disorder that affects men. People with SBMA often have weakness throughout the body, including the muscles they use for swallowing, breathing, and speaking. We do not know if exercise helps or harms people with SBMA.

B.  Objective:
-To see if a 12-week program of either strength exercise or stretching exercises will improve strength, function, or quality of life in people with SBMA

C.  Eligibility:
-Participants will be men 18 years of age or older who have genetic confirmation of SBMA.
-They must be able to walk at least 50 feet with or without an assistive device such as a cane or a walker and stand for 10 minutes without using an assistive device.
-They must have access to a computer with an Internet connection.

D.  Design:
-At the first visit to NIH (2 days), participants will have a medical history taken and undergo a physical exam. They will also have blood tests and an EKG, and complete questionnaires about mood, health, and exercise. Tests of muscle strength, balance, and endurance will also be done.
-Participants who qualify for the study will receive instruction about either strengthening or stretching exercises. They will do these exercises at home one to three times a week for 12 weeks.
-They will wear a small activity monitor while they exercise and record their exercise in a diary.
-At the end of 12 weeks, participants will return to the NIH for 2 days. They will undergo the same tests as they had on the first visit.
-Participants will receive follow-up phone calls and e-mails during the study and for 4 weeks after the last visit.

E.  Sponsoring Institute:
National Institute of Neurological Disorders and Stroke (NINDS)
 
F.  Recruitment Detail
Type: Participants currently recruited/enrolled
Gender: Male
 
G.  Eligibility Criteria:
INCLUSION CRITERIA:
1. Genetically confirmed SBMA.
2. Ambulatory and walk a distance of at least 50 feet with or without a walker.
3. Able to stand for 10 minutes without the use of any assistive devices.
4. Willing to travel to the NIH at the beginning and end of the study.
5. Willing to participate in telephone monitoring.
6. AMAT score of less than 41, but greater than 14.
7. Male.
8. Willing to participate in all aspects of trial design and follow-up.
9. Access to a computer with an internet connection
10. Able to do all of the exercises according to the standards of the study examiners at the beginning and end of the study
11. Willing to forgo starting an additional exercise plan for the 12 week duration of the study
12. Age greater than 18 years
          EXCLUSION CRITERIA:
1. Medical condition which would preclude exercise such as COPD, congestive heart failure, and cardiac arrhythmias.
2. Presence of an additional comorbid condition such as stroke, myopathy, or radiculopathy which also results in weakness.
3. Beginning a separate exercise program involving at least two weekly sessions of 20 minutes of exercise each within two months of the start of the trial.

Contact(s):
Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793
Electronic Mail:prpl@mail.cc.nih.gov
Link to the article:  http://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2011-N-0171.html