In zebrafish, a way to find new cancer therapies, targeting tumor promoters

The lab of Leonard Zon, MD, has long been interested in making blood stem cells in quantity for therapeutic purposes. To test for their presence in zebrafish, their go-to research model, they turned to the MYB gene, a marker of blood stem cells…

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Super suppressor: Boosting a gene that stifles tumor growth

Most of the time, cancer cells do a combination of two things: they overexpress genes that drive tumor growth and they lose normal genes that typically suppress tumors. No two tumors are exactly alike, but some combination of these two effects is usually what results in cancer. Now, for the first time, researchers have shown that it’s possible to treat cancer by delivering a gene that naturally suppresses tumors….

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A life-saving adjustment in IV nutrition cleared by the FDA

In 2004, a surgeon and a hospital pharmacist went against the prevailing dogma. They began revising the IV nutrition formula being given to children unable to take food by mouth. In doing so, they saved many lives. Yet, it wasn’t until last month that their intervention, a new fat emulsion called Omegaven, gained formal approval from the Food and Drug Administration…

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Virtual reality tool lets kids voyage through their own bodies

Traditionally, doctors share the findings of invasive tests using printouts that are highly text-based and filled with medical jargon. Some may have static thumbnail illustrations, but all in all they’re not especially patient friendly…

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Intestine chip models gut function, in disease and in health

The small intestine is much more than a digestive organ. It’s a major home to our microbiome, it’s a key site where mucosal immunity develops and it provides a protective barrier against a variety of infections. Animal models don’t do justice to the human intestine in all its complexity…

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Building a better bubble: Engineering tweaks bring safe IV oxygen delivery closer to reality

Everything from food aspiration to an asthma attack to heart failure can cause a patient to die from asphyxia, or lack of oxygen. For more than a decade, the Translational Research Laboratory (TRL) of Boston Children’s Hospital’s Heart Center has been pursuing a dream…

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Six technologies we backed in 2017

Boston Children’s Hospital’s Technology Development Fund (TDF) to designed to transform early-stage academic technologies into validated, high-impact opportunities for licensees and investors. Here are the projects we funded in 2017…

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Science to care: Q&A with Boston Children’s Hospital’s new Chief Scientific Officer

When Boston Children’s Hospital decided to hire its first chief scientific officer (CSO) in eight years, the institution sought an individual who could spotlight the hospital’s robust scientific enterprise and effectively connect it to clinical medicine and industry…

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Zinc chelation: A better way to regenerate the optic nerve?

For more than two decades, researchers have tried to regenerate the injured optic nerve using different growth factors and/or agents that overcome natural growth inhibition. They’ve had partial success, sometimes even restoring rudimentary elements of vision in mouse models…

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A guide to market sizing for healthcare innovators

We recently published some helpful tips on how to create a business model that accelerates and operationalizes a healthcare innovation. But a business model — and the unique value proposition you’ll offer to your users or customers — cannot exist on its own…

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A gene therapy advance for muscle-wasting myotubular myopathy

For more than two decades, Alan Beggs, PhD, at Boston Children’s Hospital has explored the genetic causes of congenital myopathies, disorders that weaken children’s muscles, and investigated how the mutations lead to muscle weakness…

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Gene therapy: The promise, the reality, the future

Gene therapy stalled in the early 2000s as adverse effects came to light in European trials (leukemias triggered by the gene delivery vector) and following the 1999 death of U.S. patient Jesse Gelsinger…

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Developing a startup: bringing your healthcare innovation to market

We recently provided guidelines for selecting a platform and developing a Minimal Viable Product to take your digital health innovation beyond the prototype stage and create meaningful iterations…

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Effective vaccination of newborns: Getting closer to the dream

In many parts of the world, babies have just one chance to be vaccinated: when they’re born. Unfortunately, newborns’ young immune systems don’t respond well to most vaccines. That’s why, in the U.S., most immunizations start at two months of age…

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2017 Innovators’ Showcase spotlights healthcare decision support

Healthcare innovations will be on display next week — April 12 — at Boston Children’s Hospital’s Innovation & Digital Health Accelerator’s annual showcase…

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Can virtual reality headsets save vision in people with lazy eye?

Three to five percent of the population has amblyopia, a.k.a. lazy eye, in which a healthy eye never “learns” to see because isn’t used. This usually happens because of a focusing problem or subtle misalignment of that eye…

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Two resilient dogs point to new targets for Duchenne muscular dystrophy

Two golden retrievers that had the genetic mutation for Duchenne muscular dystrophy (DMD), yet remained healthy, have offered up yet another lead for treating this muscle-wasting disorder…

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Precision drug delivery systems could ‘trigger’ an age of nanomedicine

What if we could deliver biocompatible nanoparticles into the body and then activate them to release drugs exactly where they are needed, without causing side effects elsewhere…

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Under the hood of healthcare innovation: Gajen Sunthara and leveraging EHRs

Gajen Sunthara, MSc, has two innovation passions: healthcare policy and electronic health records (EHRs). With professional experiences spanning technology, business and government, he finds himself in a position to effect change in a way that few others can…

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Rare disease therapies: Three strategies to bridge the gap between research and industry

Right now, there are about 7,000 rare diseases affecting 10 percent of Americans. Only five percent of these diseases have any FDA-approved treatment options…

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Under the hood of healthcare innovation: Jared Hawkins and the digital phenotype

Jared Hawkins, director of informatics at Boston Children’s Innovation and Digital Health Accelerator (IDHA), brings a formidable skill set to his work…

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Building a clinician-centric healthcare accelerator

Who better to innovate in healthcare than doctors, nurses and others on the front lines? They know what’s broken. They want to fix it. And they understand healthcare’s complexity. Some have taken part in hackathons and pitch competitions

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A new inlet to treating neurological disease

The blood-brain barrier was designed by nature to protect the brain and central nervous system (CNS) from toxins and other would-be invaders in the body’s circulating blood…

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Mutated botulinum neurotoxin B: A stronger player in the Botox world?

Famously associated with smoothing out wrinkles, botulinum toxin — better known as Botox — has been in use for 40 years now. Initially approved as a treatment for crossed eyes and then facial wrinkles, its on- and off-label uses today extend to urinary incontinence, migraines, perspiration, spasticity and even depression…

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Three challenges precision medicine faces before it can scale up

Doctors, scientists, consumers, entrepreneurs and others came together recently for the Precision Medicine 2017 symposium at Harvard Medical School, now in its third year. This year’s theme was “breakaway business models.”…

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Building emotional strength with Mighteor: Will’s story

Will, a 13-year-old from Wisconsin, lives with high-functioning Asperger’s and faces difficulties recognizing and managing his emotions. He doesn’t like to talk about emotions he perceives as negative, and becomes upset when he doesn’t meet the high standards he sets for himself…

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Using ultrasound to trigger on-demand, site-specific pain relief

According to the CDC, 91 people die from opioid overdoses every day in the U.S. Here in Massachusetts, the state has an opioid-related death rate that is more than twice the national average…

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To monitor health, simply trip the ‘nanoswitches’

“Nanoswitches” — engineered, shape-changing strands of DNA — could shake up the way we monitor our health, according to new research…

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Monitoring mitochondria: Laser device tells whether oxygen is sufficient

Shining a laser-based device on a tissue or organ may someday allow doctors to assess whether it’s getting enough oxygen, a team reports today in the journal Science Translational Medicine

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Can rare pain syndromes point the way to new analgesics?

As the opioid epidemic deepens and drug overdoses increase, effective non-addicting painkillers are desperately needed. Efforts to discover new pain pathways to target with new drugs have thus far had little success

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Pre-treated blood stem cells reverse type 1 diabetes in mice

Type 1 diabetes is caused by an immune attack on the pancreatic beta cells that produce insulin. To curb the attack, some researchers have tried rebooting patients’ immune systems with an autologous bone-marrow transplant, infusing them with their own blood stem cells…

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Shunt-flushing device for hydrocephalus gets FDA clearance; could help patients avoid extra surgery

Children with hydrocephalus often have shunts implanted to drain the excess cerebrospinal fluid that builds up inside their brain. Unfortunately, shunts have a tendency to plug up. This potentially life-threatening event necessitates emergency surgery to correct or replace the shunt…

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What’s trending in neurological drug development?

Momentum has been growing in the field of neuroscience in our ability to understand and treat various disorders affecting the brain, central nervous system, neuromuscular network and more…

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Getting academic diagnostic discoveries to market: 6 tips from industry

“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…

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A long wait, but worth it: Vonvendi’s Boston origins

It’s no secret that it can take years, even decades, for a biological or medical discovery to move from the laboratory to the bedside. The Pharmaceutical Researchers and Manufacturers of America estimates that it takes on average at least 10 years (and $2.6 billion) to develop a new medicine from start to market…

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A filtration technology poised to cure sepsis

Sepsis is the most common cause of death in infants and children worldwide, and its incidence is increasing. Damage is caused not only by the bloodstream infection itself but by the systemic inflammatory cascade it triggers — which has been difficult to control without also causing long-lasting immune suppression…

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A calmer rodent is a better rodent for pain medication research

The global market for pain medications is huge — some estimates predict it will hit $41.6 billion by 2017. However, the costs of pain medicine development are huge, too; it takes roughly $900 million to bring a new analgesic compound to market…

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Cloud-based complex care platform empowers clinicians and families alike

For children with complex medical needs, care coordination across medical specialties is a major pain point, as is communication across multiple provider systems. And patients aren’t the only ones feeling the burden…

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Can the collaborative economy work in health care?

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…

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AudioHub app: Bringing hearing tests into the 21st century

There are 36 million Americans with hearing loss. Nearly 15 percent of children ages 6 to 19 have some level of hearing problems, according to the CDC, and the elderly population’s need for audiologic services is growing…

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Seeding medical innovation: The Technology Development Fund

Since 2009, Boston Children’s Hospital has committed $6.2 million to support 58 hospital innovations ranging from therapeutics, diagnostics, medical devices and vaccines to regenerative medicine and healthcare IT projects…

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Magnetic needle tackles suturing in challenging surgical fields

The current method of suturing used in surgery—stitching with a needle and thread—has been around for thousands of years. Kaifeng Liu, MD, a research fellow at Boston Children’s Hospital, hopes to reimagine this fundamental operating room practice…

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Leveraging bacteria biofilms for vaccines

Malaria. Cholera. Now Ebola. Whatever the contagion, the need for new, or better, vaccines is a constant. For some of the most devastating public health epidemics, which often break out in resource-poor countries, vaccines have to be not only medically effective but also inexpensive…

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New therapeutic development models build researchers’ commercialization savvy

Academic researchers and physician innovators are great at making research discoveries and developing inventions at an early stage. But if you were to fund them to turn their research findings into a product, would they have the expertise and experience needed to be successful…

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Digital health: The next blockbuster

At the recent 2014 Biotechnology Industry Organization (BIO) International Convention, the message was clear: Digital health is the new blockbuster. For the first time ever, BIO spotlighted digital health, with a specific focus on how digital health is influencing the pharmaceutical and biotech industries…

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Stopping blindness: The drug-eluting contact lens

Growing up, my grandmother’s eyes were always a problem. For years, she was losing her central vision to glaucoma, and numerous surgeries and treatments did not seem to help. Later in life, she could not see my face but could always tell who I was when I was close…

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The Accelerating Medicines Partnership: Transforming biomedical consortia and R&D

Successful therapeutic development requires multiple stakeholders along the path from discovery to translation to clinical trials to FDA approval to market availability. At various points along this path, academia, industry, government, hospitals, nonprofits and philanthropists may work together…

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Biogen Idec wins FDA approval for long-lasting hemophilia drug based on Boston Children’s technology

A few weeks ago Vector brought you the backstory of how a clotting factor for hemophilia was made to last longer in the blood, allowing injections to be pared to once every week or two, rather than two to three per week…

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Turning tumors against themselves to stop metastasis

With cancer, it’s generally not the primary tumor that kills people, but metastasis—the spread of cancer to locations far from the original tumor…

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From thalidomide to Pomalyst: Better living through chemistry

In times past, a pharmaceutical chemist’s main focus was to synthesize novel molecules to treat diseases. Today, an increasingly popular alternative is to re-engineer an existing drug—and continually improve it even after FDA approval…

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