Next 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.
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 →
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
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.