Tag Archives: clinical trial

Life According to Sam

SamNext Monday, October 21 at 9:00 pm EST, HBO will debut LIFE ACCORDING TO SAM, the story of Sam Berns, a young boy with an extremely rare and highly-rapid aging disease called Progeria, and his family’s 13-year fight to find a cure.

This film tells a story not only about the love of physician parents trying to save their son – it also portrays a winding journey throughout the scientific process and the pivotal role that medical innovation plays in our lives. In the film we see children with Progeria gather in Boston from around the world – all expected to live no longer than 13 years – to receive treatment and participate in a first-of-its-kind clinical trial.

The film’s Oscar®-winning Directors Sean Fine and Andrea Nix Fine have earned multiple awards for LIFE ACCORDING TO SAM, winning the “Best of Fest” at the AFI Docs Festival in Washington, DC; the Audience Award at the Nantucket and Martha’s Vineyard Film Festivals the Best Storytelling Award at Nantucket and Best Feature Documentary at the Woods Hole and Rhode Island International Film Festivals.

The Progeria Research Foundation, a Research!America member, is leading the fight to find better treatments – and ultimately the cure – for Progeria, the rare and fatal “rapid aging” disease affecting children around the world.

You can learn more online, visit their Facebook page and follow them on Twitter.

Advertisements

A Weekly Advocacy Message from Mary Woolley: “Ordinary Americans” at risk

Dear Research Advocate:

Yesterday, I joined Diane Rehm and other guests on her nationally syndicated radio program to discuss how sequestration impacts “ordinary Americans.” I was struck by how deep and distressing the damage is, in so many sectors, including but not limited to our own. Yet somehow the pain is not acute enough to force action.

What strikes me is how low our collective expectations have sunk when it comes to reinvigorating U.S. economic growth and prosperity. Our nation can do better; why don’t we maintain high expectations and hold our elected officials accountable for setting the policy stage to accomplish them? Policy makers should protect discretionary spending, make hard tax and entitlement reform decisions, and commit to policies that foster economic growth and societal (including health) progress. Part of that equation is ample, stable federal funding for medical research and policies that spur private sector medical innovation.

A glimmer of hope for policies creating such an environment can be seen in the Senate agricultural appropriations bill, which provides the FDA an increase of $96 million above FY13 and full access to collected user fees, previously subject to sequestration. The House also provides a modest increase for FDA. But don’t jump for joy; these increases still leave FDA worse off than FY12 and grossly underfunded. That we think of this as an increase is another example of lowered expectations. Meanwhile, the Senate 302(b) allocation levels for FY14 appropriations were adopted today. As expected, the Labor/H budget is significantly higher than the House’s. The Senate and House numbers are so far apart that reconciliation seems unlikely; the odds are still on flat funding minus sequestration. This is a low-expectation, low-outcome scenario, to be sure, and we should all fight against it. If we don’t, “ordinary Americans” will continue to suffer. Continue reading →

Invest in America’s health

By Olivera J. Finn and Robert E. Schoen

An excerpt of an op-ed by Olivera J. Finn, PhD a distinguished professor and chair of immunology at the University of Pittsburgh School of Medicine and Robert E. Schoen, MD, MPH professor of medicine and epidemiology at Pitt’s School of Medicine and Graduate School of Public Health published in the Pittsburgh Post-Gazette.

Olivera J. Finn

Olivera J. Finn, PhD

Robert E. Schoen, MD, MPH

Robert E. Schoen, MD, MPH

Every day, physicians and scientists see the hope and promise that medical research brings to patients and families. For nearly 70 years, research funded by the National Institutes of Health has increased understanding of the causes of disease, contributed to longer life expectancy and improved the health and well-being of all Americans. With such a proud record of economic and social benefit, it is shocking that the House Appropriations Committee has proposed a drastic cut of nearly 20 percent to NIH funding in 2014. This outrageous proposal must be stopped.

Research is a dynamic process. New, life-improving advances are constantly within reach — but only with uninterrupted effort, commitment and funding. NIH Director Francis Collins says these cuts would be a “profound and devastating” blow at a time of unprecedented scientific opportunity. Continue reading →

New National Public Opinion Poll Shows Majority of Americans Would Participate in Clinical Trials if Recommended by Their Doctor

Only Small Percentage say Health Care Professionals Have Ever Talked to Them about Medical Research

ALEXANDRIA, Va.-June 12, 2013 – More than two-thirds (72%) of Americans say it’s likely they would participate in a clinical trial if recommended by their doctor, but only 22% say a doctor or other health care professional has ever talked to them about medical research, according to a new national public opinion poll commissioned by Research!America. A wide majority (80%) say they have heard of a clinical trial – more than half (53%) through the Internet and only 24% from a doctor or other health care provider.

Only 16% of those polled say they or someone in their family have ever participated in clinical trials. Respondents believe individuals don’t participate because of a lack of awareness (53%), a lack of trust (53%), concerns that it’s too risky (51%), adverse health outcomes (44%), little or no monetary compensation (35%), privacy concerns (27%), and worries that it takes too much time (27%).

The findings point to the important role of health care providers in talking to their patients about clinical trials. “It is critical for providers and health systems in the U.S. to recognize the importance of generating knowledge about which treatments are best through participation in clinical trials,” said Robert Califf, MD, vice chancellor of clinical and translational research at Duke University Medical Center and board chair of the Clinical Research Forum, a co-sponsor of the poll. “Advances in common diseases like Alzheimer’s and diabetes, as well as rare diseases, depend on physicians and other members of the health care team offering their patients a chance to participate in clinical trials.” Continue reading →

TB Vaccine Trial Paves the Way for Future Research

On February 4, Aeras released the results of a clinical trial of one of their TB vaccine candidates. The trial was conducted in South Africa with nearly 3,000 infants and while the vaccine was safe and well-tolerated, ultimately it was not found to provide protection against TB. Although the results were not what researchers had hoped, the trial was the first of its kind and proved that a large-scale clinical trial to test a TB vaccine in infants can be successfully run in a country with a high TB burden like South Africa. Researchers also pointed out that there are twelve other TB vaccines in clinical development and the infrastructure built through this trial can be used to test these candidates in the future.

Instead of being disappointed with the results, the global health community must move forward in the fight against TB with renewed urgency.  TB kills 1.4 million each year and over 12 million suffer from TB infection. Cases of drug-resistant TB are on the rise and the existing treatments are extremely complex and expensive, meaning that only 3% of drug-resistant patients receive proper treatment. The economic cost of TB is also enormous – in South Africa alone, TB control costs nearly $300 million each year. New drugs, diagnostics and vaccines are urgently needed to saves lives and money.

-Morgan McCloskey, global health intern

Promising Results for NIH Dengue Vaccine

On January 23, the NIH announced that a Phase I clinical trial for a dengue vaccine candidate has yielded promising results. Dengue is a potentially lethal virus which causes severe fever, headaches, and rashes. WHO estimates that 50 to 100 million cases of dengue occur worldwide each year, including here in the U.S., and has recently warned of the possibility of a global dengue epidemic.

The results of the trial, in which 90% of participants developed some immunity to the virus, represent a significant breakthrough in the development of a safe and effective dengue vaccine. The vaccine costs just $1 to produce, making it cost effective and ideal for future distribution to developing countries. The vaccine will enter Phase II clinical trials shortly and is yet another example of the importance of federal funding to advance global health research.