Acetaminophen does not aggravate young children’s asthma

asthma
A head-to-head comparison with ibuprofen refutes a link between acetaminophen and asthma exacerbations.

Your toddler is screaming in pain. Her forehead is burning. You rush to your local drugstore. What do you get — Tylenol or Motrin? And by the way, she also has asthma.

Recently, many parents have been under the impression that acetaminophen (Tylenol, etc.) may do more harm than good in young children with asthma.

“There’s been a lot of ‘smoke’ about this, based on a lot of retrospective observational data,” says Wanda Phipatanakul, MD, MS, of Boston Children’s Hospital’s Division of Allergy and Immunology.

The studies in question concluded that the common over-the-counter remedy can cause asthma exacerbations. Reviewing these studies, one author concluded, “Until future studies document the safety of this drug, children with asthma or at risk for asthma should avoid the use of acetaminophen.”

The Acetaminophen Versus Ibuprofen in Children with Asthma (AVICA) trial, led by Phipatanakul for the National Heart, Lung and Blood Institute’s AsthmaNet now sets the record straight.

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Citizen science: Genetic bone disease fuels a teen’s passion for research

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When I was just 3 months old, I was diagnosed with fibular hemimelia, a rare genetic condition that affects about 1 in 50,000 people. It manifests itself as the lack of the fibula bone, a key structural bone in the lower leg that provides major stability in the ankle and knee.

Fibular hemimelia leads to a severe leg length discrepancy — which, in my case, would have amounted to over 6 inches without treatment. Prior to my time at Boston Children’s Hospital, the go-to cure was amputation — replacing my lower leg with a series of prostheses.

Luckily, at the time of my diagnosis, leg-lengthening surgeries were just being approved in the U.S. My parents couldn’t bear to part with my leg, so over the course of 18 years, I have undergone 13 procedures to combat my leg-length difference, starting at age 5. This early exposure to the medical field, coupled with encouragement from teachers, led to a passion for science.

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Overcoming pain by tackling the fear factor

chronic regional pain syndrome
Grace Cahners prior to fear-based therapy (photos: Leigh Cahners)

When 11-year old Grace Cahners broke her foot in July 2015, she received the usual support boot, then casting and several weeks of physical therapy (PT). But instead of getting better, her pain intensified over the course of five months, forcing her to miss the first 54 days of sixth grade. She lost her normally sunny disposition and became crippled by fear.

Grace was diagnosed with complex regional pain syndrome (CRPS), a chronic pain condition in which the brain sends an over-abundance of pain signals to the affected limb. Not a newcomer to pain – Grace was diagnosed with psoriatic arthritis at the age of 13 months – she was using a wheelchair by December.

Leigh Cahners, Grace’s mother, knew that full-day narcotic pain medications and traditional PT would not restore Grace’s ability to walk. “I knew we needed another approach,” she says.

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Tracking the public health effects of the Rio Olympics: HealthMap

Olympics Brazil Zika Health Map
Athletes around the world are converging on Brazil. What effects will this have on Zika and public health generally? (Nuno Lopes/Pixabay)

This past week, the 2016 Summer Olympic and Paralympic Games began in Rio de Janeiro, Brazil, with more than 11,000 athletes and 500,000 international fans expected to arrive. As a major mass gathering, the Olympic Games are always vulnerable to disease outbreaks. This summer, all eyes in public health are on the concurrently occurring Zika virus and the under-reported H1N1 influenza outbreak in Brazil.

According to the European Centre for Disease Prevention and Control (ECDC), visitors to the 2016 Games are most at risk for gastrointestinal illness from waterborne pathogens and mosquito-borne infections, including dengue, chikungunya and Zika virus. So far in 2016, we have seen an estimated 165,000 cases of Zika virus, 1,345,286 cases of dengue, 137,808 cases of chikungunya and more than 6,500 cases of H1N1 influenza, with an additional 1,233 deaths from H1N1 — all in Brazil alone.

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Bridging academia and industry: Q & A with scientist and entrepreneur Timothy Springer

Timothy Springer on entrepreneurship

Biological chemist and molecular pharmacologist Timothy A. Springer, PhD, is poised at the nexus of academia and industry. As an academic — currently at Harvard Medical School, the Program in Cellular and Molecular Medicine at Boston Children’s Hospital and the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center — he has used monoclonal antibodies as research tools to unravel key mysteries of the immune system. As an entrepreneur, his discoveries — and those of others he has backed — have successfully launched seven companies. Drawing from his own entrepreneurship experience, he now aims to create his own innovation center, focused on accelerating antibody science toward drug discovery while helping nurture and mentor young scientist entrepreneurs. Vector sat down with Springer for his insights.

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Timothy Springer: scientist, serial entrepreneur and social advocate

Tim Springer resized

At the dawn of his career, immunologist, biological chemist, molecular pharmacologist and seven-time biomedical entrepreneur Timothy Springer thought science was a bad idea. “I was suspect of the purposes that science had been put to,” he says, “making Agent Orange and napalm.”

It was 1966, and Springer was a Yale undergrad thinking, “What the hell good is this Ivy League education? The best and brightest, the Ivy League-educated people, totally screwed up in getting us into the Vietnam War.”

So he dropped out. For a year, he lived on a Native American reservation in Nevada for Volunteers in Service to America (VISTA). He helped the Tribal Council draft resolutions, launched a 4-H club and lobbied for paved roads so kids could go to school.

Finally, he returned to school at the University of California, Berkeley — trying anthropology, sociology and psychology. Switching to biochemistry his junior year, Springer asked his advisor, scientific visionary Daniel Koshland, Jr., former editor of Science, “Do you think I can do this — graduate with a degree in biochemistry?”

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More clinical trials in kids? Nearly half are unfinished or unpublished

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Of 559 interventional trials in children, 19 percent were stopped early and 30 percent of completed trials remained unpublished several years later, finds a new study. (Vmenkov/Wikimedia Commons)

Recent laws like the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act are encouraging clinical trials in children. Yet, as with adult trials, these trials commonly stall out or, if completed, remain unpublished several years later, finds a study published online today in Pediatrics.

“Our findings may speak to how commonplace discontinuation and non-publication are in medical research in general,” says Natalie Pica, MD, PhD, a senior resident at Boston Children’s Hospital and the study’s coauthor. “We need to make sure that when children participate in clinical trials, their efforts are contributing to broader scientific knowledge.”

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Can asthma be nipped in the bud?

asthma
A multicenter randomized trial is testing omalizumab (Xolair) in wheezy toddlers. (FDA/Wikimedia Commons)

Worldwide, asthma affects an estimated 300 million people, and is expected to surpass 400 million by 2025, according to the World Health Organization. About 1 in 10 U.S. children have asthma, and research shows that the vast majority of them also have allergy. Could that provide a clue to its prevention?

Starting at 2 to 3 years of age, susceptible children start to become sensitized to pollens, mold spores and other airborne allergens. They begin to produce IgE antibodies, which not only trigger allergic reactions but also impair their anti-viral immune responses — potentially leading to more viral infections that can further hasten their progression to asthma.

A multicenter clinical trial, led by Wanda Phipatanakul, MD, MS, of the Division of Allergy & Immunology at Boston Children’s Hospital, now aims to test whether the anti-IgE drug omalizumab (Xolair) can short-circuit this process.

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Citizen science: Giving patients a voice in drug development

citizen science patient voice drug development

There’s a natural tension between wanting the FDA to ensure safety and efficacy before a drug enters the market and wanting to speed up what many view as a glacially slow approval process. The rare disease community tends to fall in the second camp, and has become increasingly vocal in calling for more clinical trials, more flexibility in their design and redefinition of what constitutes a benefit.

ALS advocates, for example, have called for a parallel track, “in which FDA provides an early approval based on limited data, and then continues the learning process in a confirmatory clinical trial and if needed, patient registries to collect additional data from patients receiving the drug outside the clinical trial…”

Recent legislation is encouraging patient engagement in drug development, especially for conditions with profound unmet medical needs. In its 2012 iteration, the Prescription Drug User Fees Act (PDUFA) introduced public meetings to get input from the patient community, captured in a series of informative white papers.

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Getting academic diagnostic discoveries to market: 6 tips from industry

diagnosticsdiagnostics (008) Loading of QIAsymphony platform“Wouldn’t it be great if we could come up with a noninvasive diagnostic assay to detect pancreatic cancer at an earlier, more treatable stage?” asked Lori Aro of Myriad Genetics. Her company has been trying to do so for years. So why hasn’t it happened?

Aro, senior director for new product planning at Myriad, outlined the business obstacles at a recent panel hosted by Boston Children’s Hospital’s Technology and Innovation Office (TIDO).

First, who are the target patients for a pancreatic cancer test? Skinny diabetics, patients with chronic pancreatitis, patients with hereditary cancer risk — or all three? “Those three patient types all sit in different doctor’s offices,” said Aro. Simultaneously reaching endocrinologists, gastroenterologists and high-risk patients would be an insurmountable challenge, Myriad concluded.

Second, the assay would likely need to be validated in all three patient populations, with confirmatory imaging. Could the test populations be large enough to make the results statistically significant?

Third, a new test wouldn’t change care, as there is no treatment for pancreatic cancer. In fact, no current data show that earlier diagnosis improves survival. So who would pay for it?

Aro’s story exemplifies just some of the challenges in developing a new diagnostic test.

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