Stories about: Market trends

New Human Neuron Core to analyze ‘disease in a dish’

Human Neuron CoreLast week was a good week for neuroscience. Boston Children’s Hospital received nearly $2.2 million from the Massachusetts Life Sciences Center (MLSC) to create a Human Neuron Core. The facility will allow researchers at Boston Children’s and beyond to study neurodevelopmental, psychiatric and neurological disorders directly in living, functioning neurons made from patients with these disorders.

“Nobody’s tried to make human neurons available in a core facility like this before,” says Robin Kleiman, PhD, Director of Preclinical Research for Boston Children’s Translational Neuroscience Center (TNC), who will oversee the Core along with neurologist and TNC director Mustafa Sahin, MD, PhD, and Clifford Woolf, PhD, of Boston Children’s F.M. Kirby Neurobiology Center. “Neurons are really complicated, and there are many different subtypes. Coming up with standard operating procedures for making each type of neuron reproducibly is labor-intensive and expensive.”

Patient-derived neurons are ideal for modeling disease and for preclinical screening of potential drug candidates, including existing, FDA-approved drugs. Created from induced pluripotent stem cells (iPSCs) made from a small skin sample, the lab-created human neurons capture disease physiology at the cellular level in a way that neurons from rats or mice cannot.

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What we’ve been reading: Week of March 16, 2015

 

(government_press_office/Flickr)
(government_press_office/Flickr)

Scientists Call for a Summit on Gene-Edited Babies (MIT Technology Review)

Tools like CRISPR could give us the power to alter humanity’s genetic future. A group of senior American scientists and ethicists have called for a moratorium any attempts to create genetically engineered children using these technologies until there can be a robust debate.

Meet the healthcare company that won Mark Cuban’s heart at SXSW (MedCity News)

CareaLine, founded by the parents of a young girl who died of cancer, won over audience members’ hearts and investors’ wallets during SXSW 2015’s Impact Pediatrics competition.

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SXSW Interactive 2015: Our future selves, a maturing health tech industry and why failing is productive

SXSW Impact Pediatric HealthJudy Wang, MS, is a program manager in the Telehealth Program at Boston Children’s Hospital.

In 2012, when I attended the South by Southwest (SXSW) Interactive conference for the first time, health tech was still an emerging field. It was the first year the world’s leading conference for emerging technology and digital creativity made any effort to include health tech programming, and the first time its Accelerator pitch event included a category for health tech startups.

Only three years later, SXSW Interactive (March 13­–17, 2015) has grown to include almost 50 events related to health and medical technologies. Martine Rothblatt, CEO of the biotech company United Therapeutics, gave a keynote titled “AI, Immortality and the Future of Selves” that was both inspiring and provocative. She spoke to a world in which our 24/7 selves are increasingly being captured digitally. Audience questions captured by Twitter pondered the ethical implications of what Rothblatt called “mind clones”: future mechanical beings digitally programmed with our mannerisms, habits and memories.

This year also featured the Impact Pediatric Health pitch competition, captured in this Storify.

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Can the collaborative economy work in health care?

Airbnb Uber model health care
Airbnb and Uber have disrupted the hotel and taxi industries by finding and tapping unused assets. What's in store for medicine?

David Altman is manager of marketing and communications in Boston Children’s Hospital’s Technology and Innovation Development Office.

Robin Chase, co-founder of Zipcar and current CEO of Buzzcar, envisions collaboration as the future of the world’s economy. Her concept, PeersIncorporated, brings excess capacity of consumer goods or assets—such as unused time or untapped data—to online platforms and apps where consumers (“peers”) provide insights that drive business growth.

Speaking recently at Boston Children’s Hospital, Chase elaborated on the concept of excess capacity, which is the basis of Buzzcar. Typically, families pay an average of $9,000 a year—$25 a day—for cars they use only 5 percent of the time. That unused time represents value and economic potential. Buzzcar’s platform harnesses that unused capacity, allowing multiple peers to supply and book cars on an easy-to-use website at a low cost.

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Five new developments in hemophilia

Ellis Neufeld hemophiliaEllis Neufeld, MD, PhD, is a hematologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

From new longer-acting drugs to promising gene therapy trials, much is changing in the treatment of hemophilia, the inherited bleeding disorder in which the blood does not clot. Hemophilia Awareness Month comes at a time of both progress and remaining challenges.

1. Many more treatment products are being introduced, including some that last longer.

People with hemophilia lack or have defects in a “factor”—a blood protein that helps normal clots form. Of the approximately 20,000 people with hemophilia in the U.S., about 80 percent have hemophilia A, caused by an abnormally low level of factor VIII, and most of the rest have hemophilia B, caused by abnormally low levels of factor IX. Many patients with severe hemophilia give themselves prophylactic IV infusions of the missing factor to prevent bleeding (which otherwise can lead to crippling joint disease when blood seeps into the joint and enzymes released from blood cells erode the cartilage).

Hemophilia factors traditionally have such a short half-life that we tend to treat patients every other day with factor VIII and twice a week with factor IX. The first two longer-lasting products came onto the market within the past year, and more are on the way. So now, with factor IX, it is possible to get an infusion just once a week and not bleed. This is really changing how we think about the disease. So far, the longer-acting factor VIII products are not yet long-lasting enough to make as dramatic a difference in the frequency of infusions. And creating really long-acting factors remains a challenge.

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

What Vector has been readingVector’s picks of recent pediatric healthcare, science and innovation news.

23andMe and the Promise of Anonymous Genetic Testing (New York Times)
Four debators weigh in on direct-to-consumer genetic testing, asking: Is it good for consumers? Is it good for science? And what about privacy? Worth a read.

Internet of DNA (MIT Technology Review)
Emerging projects in Toronta, Santa Cruz and elsewhere are working toward being able compare DNA from sick people around the world via the Internet to identify hard-to-spot causes of disease—analogous to using the “Compare documents” function in Word.

Engineering the perfect baby (MIT Technology Review)
Since the birth of genetic engineering, people have worried about designer babies. Now, with gene editing and CRISPR, they might really be possible. Bioethicists and scientists weigh in on what “germ line engineering” would mean.

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The Precision Medicine Initiative: A child-centered perspective

child aiming bow & arrow Shutterstock croppedPatrice Milos, PhD, is president and CEO of Claritas Genomics, a CLIA-certified genetic diagnostic testing company spun off from Boston Children’s Hospital in 2013.

A child is sick, showing symptoms her parents cannot identify. Something is seriously wrong, but what? The family turns to Boston Children’s Hospital for answers. Yet, even with today’s medical advances, a precise diagnosis often remains elusive.

The Human Genome Project has sparked innovation over the last 14 years, and as President Obama’s Precision Medicine Initiative asserts, today genome science offers patients new hope for answers.

Initially, cancer will be the major medical focus of this initiative, as cancer is a genetic disease—a genomic alternation of the patient’s normal tissue DNA.

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

Children what we've been reading Flickr thomaslife https://www.flickr.com/photos/thomaslife/4508639159
(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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Pitching pediatric innovation at SXSW Interactive

Pitching pediatric digital health innovationsJudy Wang, MS, is a program manager in the Telehealth Program at Boston Children’s Hospital.

A major theme at Taking on Tomorrow 2014 was the difficulty in making the business case for innovation in pediatrics, since the market size is small relative to the adult market. Muna AbdulRaqqaq Tahlak, MD, CEO of Latifa Hospital in Dubai, was among many who urged innovators to collaborate and aggregate their data to make the most impact.

It’s in that spirit that the upcoming Impact Pediatric Health Startup Pitch Competition (March 16) was born. Hosted by organizers of the South by Southwest Interactive (SXSWi) conference in Austin and the four top pediatric hospitals in the country—Boston Children’s, Cincinnati Children’s, Texas Children’s and Children’s Hospital of Philadelphia—the event will identify the most promising digital health and medical device innovations for pediatrics.

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