Stories about: Market trends

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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2017 predictions for biomedicine

2017 predictions for biomedicine

David Williams, MD, is Boston Children’s Hospital’s newly appointed Chief Scientific Officer. He is also president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and director of Clinical and Translational Research at Boston Children’s. Vector connected with him to get his forecast on where biomedical research and therapeutic development will go in the year ahead.

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7 digital health predictions for 2017

digital health predictions

What does 2017 have in store for digital healthcare innovations? Vector connected with clinical, digital health and business experts from the Innovation & Digital Health Accelerator (IDHA) at Boston Children’s Hospital and asked for their predictions.

Overall? “Expect to see a reshaping of the patient journey, more patient-centric care and more clinically impactful technology in 2017,” says John Brownstein, PhD, Chief Innovation Officer at the hospital. “We’re also looking forward to digital health offerings being met by industry-wide adoption as patient-centric care is provided and reimbursed.”

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Parents are generally open to placebo use in children – with caveats

placebo use in children

Placebos are a key ingredient of any controlled clinical trial, the yardstick against which experimental drugs are measured. Placebos are also increasingly used as a treatment in their own right, as studies show that they make people feel better through a “mind-body” effect. But do parents find placebos acceptable for their children? A study published today by The Journal of Pediatricsled by Boston Children’s Hospital, found the answer is mostly yes, provided ethical guidelines are followed.

“The question of placebos is more complex when it comes to children, since parents must make medical decisions on their behalf,” says Vanda Faria, PhD, a research fellow at Boston Children’s Hospital’s Center for Pain and the Brain and first author on the study. “Large placebo responses have been seen in a variety of pediatric conditions, and parent’s perceptions can influence how well placebos work. At the same time, little is still known about the potential harms of prolonged drug therapy on children’s development. Sometimes, the best intervention might not involve pharmacotherapy.”

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News Notes: Research and innovation news

A few things that caught Vector’s eye during the week:

FDA drug evaluation staff: An unmet need

overflowing inbox at FDA

New laws or initiatives to expedite drug approvals, especially for orphan drugs, have prompted the Food and Drug Administration to create new positions to review the applications. Unfortunately, these slots are proving hard to fill. Kaiser Health News reports that the FDA has more than 700 job vacancies in its Center for Drug Evaluation and Research (CDER), which approves new drugs. The problem? Pharma pays more — “roughly twice as much as we can,” said CDER director Janet Woodcock at a recent rare-disease summit. The 21st Century Cures Act, still awaiting Senate approval, could help by allowing the FDA to offer higher salaries and relaxing postgraduate degree requirements for some positions.

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BabySeq: Early results of newborn genomic sequencing are mixed

BabySeq
While a previous study indicated parents were very interested in newborn sequencing, just 7 percent of those approached have enrolled in BabySeq so far.

It seems like a great idea. We all have our genomes sequenced at birth, and any findings that suggest a future medical problem are addressed with early interventions, optimizing our health and extending our lives. But are parents of newborns ready to embrace the vision? Yes and no, according to interim results of a first-of-its-kind randomized trial of newborn sequencing. Findings from what’s known as the BabySeq Project were presented last week at the American Society of Human Genetics (ASHG) 2016 Annual Meeting.

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Health care wants YOU: 9 opportunities, 4 pearls for digital health startups

digital health startups - reverse pitches
A visual summary of reverse digital health pitches (illustration: CollectiveNext).

Boston’s digital health world is humming with tech talent, idealistic health care professionals and business-savvy accelerator organizations. The passion was palpable last week as 300-plus people gathered at MassChallenge’s latest Pulse@Check digital health meetup, hoping to turn their health care ideas into reality.

The event, hosted by Boston Children’s Hospital’s Innovation and Digital Health Accelerator (IDHA) and Cerner, a lead developer of health care IT systems, presented numerous opportunities and tips for digital health startups.

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