Stories about: Market trends

Building precision medicine: Power to the patients

Tools to build precision medicinePrecision medicine involves the development and application of targeted therapeutics based on patients’ genomes, lifestyles and environments. The recent conference on precision medicine at Harvard Medical School highlighted a few challenges in scaling up this process.

To help further precision medicine, the Obama administration and NIH launched the All of Us program, registrations for which are slated to start later this year. Its aim is to collect health data from one million Americans.

But the conference also highlighted several tools that patients can use proactively to collect, share and analyze their own data and use it to improve their own health — and contribute to precision medicine as citizen scientists.

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Three challenges precision medicine faces before it can scale up

Different aspects of precision medicine therapyDoctors, 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.” What are challenges in developing targeted treatments based on clinical and genetic data, and how do we overcome them?

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

Jared Hawkins Boston Children's Hospital

What does it take to change healthcare for the better? In the second of a two-part series on digital health innovators at Boston Children’s Hospital, we profile Jared Hawkins, MMSc, PhD. Like Gajen Sunthara, MSc, featured in part one, Hawkins was named among MedTech Boston’s 40 Under 40 Healthcare Innovators for 2017.

Jared Hawkins, director of informatics at Boston Children’s Innovation and Digital Health Accelerator (IDHA), brings a formidable skill set to his work. With a PhD in Immunology from Tufts University School of Medicine and an MMSc in Biomedical Informatics from Harvard Medical School, his background combines biomedical research (immunology, virology, oncology, genomics) with data science, visualization, computational modeling and software development.

His current work spans an equally diverse range of topics, touching on population and public health, patient experience, decision support and pharmacogenomics. A faculty member in the Computational Health Informatics Program, Hawkins is wired into the digital health ecosystem. He serves as a scientific advisor and co-founder of Raiing Medical (home temperature and fertility tracking) and is the head of engineering and co-founder of Circulation (non-emergency medical transportation via Uber).

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Webchat to highlight what’s new in pediatric brain tumors

pediatric brain tumors, child MRI

Last September, the National Center for Health Statistics reported that brain tumors have overtaken the much more common leukemia as the leading cause of death from pediatric cancer. Although progress has been made and the promise of more progress is on the horizon, the cure rate for childhood brain tumors lags behind a number of other pediatric cancers.

As pediatric neuro-oncologist Peter Manley, MD, of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center told Live Science, new research on cancer genomics “is so impressive that my feeling is that we will continue to see a decline in deaths.”

To mark Brain Tumor Awareness Month, Mark Kieran, MD, PhD, clinical director of the Brain Tumor Center at Dana-Farber/Boston Children’s, will host a webchat on Monday, May 22 (3:30 p.m. ET). The live chat will highlight the latest research and treatments for pediatric brain tumors. Here’s a look back at some recent developments:

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Protein science 2.0: Amping up antibodies

Institute for Protein Innovation antibody libraries
The Institute for Protein Innovation, launching next week with $15 million in grants and philanthropy, aims to develop comprehensive, open-source libraries of antibodies targeting human proteins.

It began with the proteins. Before Watson and Crick unraveled DNA’s double helix in the 1950s, biochemists snipped, ground and pulverized animal tissues to extract and study proteins, the workhorses of the body.

Then, in 1990, the Human Genome Project launched. It promised to uncover the underpinnings of all human biology and the keys to treating disease. Funding for DNA and RNA tools and studies skyrocketed. Meanwhile, protein science fell behind.

While genomics unveiled a wealth of information, including the identity of genes that lead to disease when mutated, researchers still do not fully understand what all the genes really do and how mutations change their function and cause disease.

Now proteins are promising to provide the missing link.

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Lessons from the data: Applying machine learning for clinical decision support

machine learning clinical decision support

Mauricio Santillana, PhD, faculty member in the Computational Health Informatics Program at Boston Children’s Hospital, had an idea as he witnessed the volume of continuous real-time data generated in the pediatric intensive care unit (PICU). He realized that tapping the data on patients’ ever-changing vital signs, with the help of machine-learning algorithms, could support clinical decision-making and predict (and help head off) up-coming health issues.

He started a dialogue with the hospital’s Innovation & Digital Health Accelerator, and now collaborates closely with clinicians in the PICU to create machine-learning algorithms that can help them provide the highest level of care.

“It’s fairly recent that clinicians realized people with backgrounds in math and statistics can be very helpful in a clinical context,” says Santillana

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Gene therapy: The promise, the reality, the future

gene therapy
(Graphs courtesy Alexandra Biffi, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center)

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. But after 30 years of development, and with the advent of safer vectors, gene therapy is becoming a clinical reality. It falls into two main categories:

  • In vivo: Direct injection of the gene therapy vector, carrying the desired gene, into the bloodstream or target organ.
  • Ex vivo: Removal of a patient’s cells, treating the cells with gene therapy, and reinfusing them back into the patient, as in hematopoietic stem cell transplant and CAR T-cell therapy.

A recent panel at Boston Children’s Hospital, hosted by the hospital’s Technology and Innovation Development Office (TIDO), explored where gene therapy is and where it’s going. Here were the key takeaways:

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A healthcare Innovator’s Roadmap: 4 steps for developing a business model

healthcare innovation business plan

First in an on-going series of Innovator’s Roadmap posts from Boston Children’s Hospital’s Innovation & Digital Health Accelerator (IDHA). Matt Murphy is Innovation Lead at IDHA.

Seeing an idea go from the lab or clinic to the wider world is exciting. However, clinicians, researchers and administrators don’t always have the time or resources to take their innovations to the next step — that is, build them to scale. At Boston Children’s Hospital, the Innovation & Digital Health Accelerator (IDHA), comprised of 50+ researchers, business strategists and technologists, is dedicated to just that: We identify and vet high-priority health technology innovations at the hospital and provide the resources, funding and momentum to accelerate their development and commercialization.

To date, Boston Children’s has spun off more than 25 startup companies developed directly from clinical and research pain points. Some startups, like Neuromotion and Circulation, stand on their own. Others, including Epidemico, have been acquired by industry leaders. Through this experience, IDHA created the Innovator’s Roadmap – a comprehensive resource for taking ideas from concept to commercially available, impactful, economically sustainable products.

In this first installment, we look at the critical first step: understanding and justifying the business value of a technology or service by developing a business model.

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Pediatric brain tumor genomics arrives, as the need for new therapies grows

Allison was the first pediatric brain tumor patient in the world to receive a treatment targeting the BRAF mutation, originally developed to treat adults with melanoma who have the same mutation.

Precision cancer medicine – the vision of tailoring diagnosis and treatments to a tumor’s genetic susceptibilities – is now ready to impact the care of a majority of children with brain tumors. The molecular “signatures” of brain tumors were first characterized in 2002 in a study led by researchers at Boston Children’s Hospital. This has led to the creation of new tumor subgroups and changes in cancer treatment: For example, a current clinical trial is testing the anti-melanoma drug dabrafenib in a variety of brain tumors with the same BRAF mutation – including metastatic anaplastic astrocytoma and low-grade glioma.

In the largest study of its kind to date, investigators at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center genetically tested more than 200 brain tumor samples. They found that many had genetic irregularities that could guide treatment, in some cases with approved drugs or agents being evaluated in clinical trials.

The findings, reported online today by the journal Neuro-Oncology, also demonstrate that testing pediatric brain tumor tissue for genetic abnormalities is clinically feasible.

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

David Williams, MD
David Williams, MD

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. David Williams, MD, president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and director of clinical and translational research at Boston Children’s, was the ideal choice.

An award-winning researcher, Williams trained in the clinic but also pursued basic science, developing techniques for introducing genes into mouse and human blood cells. He focused on blood stem cell biology, leukemia and gene therapy to correct genetic blood disorders, becoming a 16-year Howard Hughes Medical Institute Investigator, a Member of the National Academy of Medicine and a Fellow of the American Association for the Advancement of Science. He has secured multiple patents for techniques still in use today.

Williams spoke about his vision as CSO to align basic research and clinical care at Boston Children’s and the challenges ahead.

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