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What we’ve been reading: Week of March 23, 2015

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Single-Dose Cures for Malaria, Other Diseases (MIT Technology Review)
Pills that deliver a full course of treatment in one swallow could, or “super pills,” could simplify the treatment of diseases such as malaria and potentially produce cost savings that stretch into the $100 billion a year range, according to Bob Langer, PhD, from the Massachusetts Institute of Technology.

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Meet the researcher behind “heart on a chip”

Pu and wife and waterfallFrom a series on researchers and innovators at Boston Children’s Hospital.

With all of the recent buzz about precision medicine, it’s no wonder that William Pu, MD is gaining recognition for his innovative application of stem cell science and gene therapy to study Barth syndrome, a type of heart disease that severely weakens heart muscle. Pu’s research was recently recognized by the American Heart Association as one of the top ten cardiovascular disease research advances of 2014.

Can you describe your work and its potential impact on patient care?

We modeled a form of heart-muscle disease in a dish. To do this, we converted skin cells from patients with a genetic heart muscle disease into stem cells, which we then instructed to turned into cardiomyocytes (heart-muscle cells) that have the genetic defect. We then worked closely with bioengineers to fashion the cells into contracting tissues, a “heart-on-a-chip.”

How was the idea that sparked this innovation born?

This innovation combined the fantastic, ground-breaking advances from many other scientists. It is always best to stand on the shoulders of giants.

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What we’ve been reading: Week of February 9, 2015

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(Photo: thomaslife/Flickr)

Vector’s picks of recent pediatric healthcare, science and innovation news.

Encryption wouldn’t have stopped Anthem’s data breach (MIT Technology Review)
Hackers got their hands on the personal information and Social Security numbers of 80 million people when they broke into the network of health insurer Anthem health. But encryption alone wouldn’t have been enough to keep those data safe.

Could a wireless pacemaker let hackers take control of your heart? (Science)
Medical devices like pacemakers, insulin pumps and defibrillators are getting ever smaller and more wirelessly connected. But are those connections secure enough?

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What we’ve been reading: Week of February 2, 2015

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Vector’s pick of recent pediatric healthcare, science and innovation news.

The problem with precision medicine (The New Yorker)
President Obama’s recently announced plan to invest $215 million in precision medicine – which uses DNA testing to personalize medical care- has many in the medical community cheering. Others, however, are concerned that DNA sequencing is still far from optimized and many of the best doctors remain unfamiliar with how to appropriately integrate genetic results into their care plans.

Schools may solve the anti-vaccine parenting deadlock (The Atlantic)
The recent outbreak of measles in the U.S. shed light on the growing number of parents “opting out” of vaccinating their kids.  Public schools are fighting anti-vaxxers in the courts- and precedent is on their side.

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Medical device panel outlines pathways to market

Physicians often dream of creating new devices to help their patients, but few are able to bring a device to market. At a panel discussion earlier this month at Boston Children’s Hospital, an entrepreneur, a venture capitalist and medical device industry experts offered advice for inventors who want to make their medical device a commercial reality. Here’s some of what they had to say.

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Can breast cancer cells tell each other to metastasize?

Extracellular vesicles exosomes microRNA breast cancer metastasis
Breast cancer cells might be able to give each other the ability to metastasize using microRNAs packaged into extracellular vesicles similar to these exosomes. (Photo: Kourembanas Laboratory, Boston Children's Hospital)

Not all cancer cells are created equal. In fact, to call a cancer a cancer, in the singular, is something of a misnomer. Really, a patient could be said to have cancers, as every tumor is actually a mixture of cells with different mutations and capabilities.

One of those capabilities is the ability to escape the main tumor and spread, or metastasize, to other sites in the body. Not every cancer cell has this ability. But just like bacteria can share the ability to resist antibiotics, at least some cancer cells may be able to share the ability to spread.

According to a study by Judy Lieberman, MD, PhD, of Boston Children’s Hospital’s Program in Cellular and Molecular Medicine, breast cancer cells that can metastasize can tell those that can’t to turn that ability on. That conversation takes place via small pieces of RNA called microRNAs, delivered in microscopic packages called extracellular vesicles.

According to Lieberman, not only do her team’s data give insight into the metastatic process, they might also reveal the first example of cancer cells teaching each other.

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Frozen poop pill offers a less invasive treatment option for emerging infectious disease

GI_6716_FecalTransplantGraphic_v3_ThumbnailThe fecal microbiota transplantation (FMT) movement is catching the attention of scientists, researchers and the media nationwide. Currently, fecal transplantation delivers pre-screened, healthy human donor stool to a patient via colonoscopy or by nasogastric tube. It’s prescribed as an effective alternative to long-term antibiotic use in treating debilitating infectious diseases such as Clostridium difficile, also known as C-diff.

But new research published in Journal of the American Medical Association says there is a third, less invasive, less expensive option to treat C-diff: poop in a pill.

“This ground-breaking paper shows that with encapsulated, frozen donor stool, fecal transplantation can be used to successfully treat recurring C-diff infection in 90 percent of cases,” says George H. Russell, MD, MS, pediatric gastroenterologist in the Inflammatory Bowel Disease Center at Boston Children’s Hospital and co-author of the Massachusetts General Hospital-sponsored study. “[The study] provides proof-of-concept that invasive means do not need to be used to deliver the fecal transplant.”

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15 health care predictions for 2015

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2014 continued to see massive evolution in health care—from digital health innovations to the maturation of technologies in genomics, genome editing and regenerative medicine to the configuration of the health care system itself. We asked leaders from the clinical, research and business corners of Boston Children’s Hospital to weigh in with their forecasts for 2015. Click “Full story” for them all, or jump to:
The consumer movement in health care
Evolving care models
Genomics in medicine
Stem cell therapeutics
Therapeutic development
New technology
Biomedical research

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Pathways to market for medical devices

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You’ve got a great idea for a new medical device. After you’ve created the device and proved its usefulness in a clinical setting—a challenge in itself—the next step is getting your device to a commercial partner who can mass-produce and market it. Working through all of the regulatory hurdles, projecting the market for your product and figuring out your product’s long term potential can seem overwhelming.

“The more you know, the more prepared you will be,” says Pedro del Nido, MD, chair of the Department of Cardiac Surgery at Boston Children’s Hospital and principal investigator on the FDA-funded Boston Pediatric Device Consortium. “The more prepared you are, the more likely you will be successful.”

On January 6, 2015, from 5:30 to 7:30 p.m., del Nido will lead a panel discussion at Boston Children’s about moving medical devices from idea to commercial partnership,

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Quality assurance for genome editing

Chromosome breaks translocations gene editing CRISPR TALENs ZFNs zinc fingers Frederick Alt
When chromosomes break, the ends can join together in a number of ways, some of which can cause trouble. A new QA method could help researchers avoid making problematic breaks when using gene editing technologies like CRISPR.

Labs the world over are jumping onto the gene editing bandwagon (and into the inevitable patent arguments). And it’s hard to blame them. As these technologies have evolved over the last two decades starting with the zinc finger nucleases (ZFNs), followed by transcription activator-like effector nucleases (TALENs) and CRISPR—they’ve become ever more powerful and easier to use.

But one question keeps coming up: How precise are these systems? After all, a method that selectively mutates, deletes or swaps specific gene sequences (and now can even turn genes on) is only as good as its accuracy.

Algorithms can predict the likely “off-target” edits based on the target’s DNA sequence, but they’re based on limited data. “The algorithms are getting better,” says Richard Frock, PhD, a fellow in the laboratory of Frederick Alt, PhD, at Boston Children’s Hospital. “But you still worry about the one rare off-target effect that’s not predicted but falls in a coding region and totally debilitates a gene.”

Frock, Alt (who leads Boston Children’s Program in Cellular and Molecular Medicine, or PCMM), fellow Jiazhi Hu, PhD, and their collaborators recently turned a method first developed in Alt’s lab for studying broken chromosomes into a quality assurance tool for genome editing. As a bonus, the method—called high-throughput genome translocation sequencing (HTGTS)—also reveals the “collateral damage” gene editing methods might create in a cell’s genome, information that could help researchers make better choices when designing gene editing experiments.

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