From the category archives:

Drug discovery

Monique Yoakim Turk Technology Development FundMonique Yoakim-Turk, PhD, is a partner of the Technology Development Fund and associate director of the Technology and Innovation Development Office at Boston Children’s Hospital

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. What a difference six years makes.

The Technology Development Fund (TDF) was proposed to Boston Children’s senior leadership in 2008 after months of research. As a catalyst fund, the TDF is designed to transform seed-stage academic technologies at the hospital into independently validated, later-stage, high-impact opportunities sought by licensees and investors. In addition to funds, investigators get access to mentors, product development experts and technical support through a network of contract research organizations and development partners. TDF also provides assistance with strategic planning, intellectual property protection, regulatory requirements and business models.

Seeking some “metrics of success” beyond licensing numbers and royalties (which can come a decade or so after a license), I asked recipients of past TDF awards to report back any successes that owed at least in part to data generated with TDF funds. While we expected some of the results, we would have never anticipated such a large impact. Full story »

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From a series profiling researchers and innovators at Boston Children’s Hospital

He’s a big thinker focused on harnessing the hyper-small. Daniel Kohane, MD, PhD, is a leading drug delivery and biomaterials researcher, leveraging nanoparticle technology and other new vehicles to make medications safer and more effective.

It’s not quite what he had in mind as a child. He dreamed of studying life forms in remote galaxies.

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But when he became aware of the constraints of relativity, he re-focused his ambitions, ultimately concentrating on innovations in drug delivery. Here’s what he told us. Full story »

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Pain in a dish nociceptors

Neurons from patients could lead researchers to better drugs for chronic pain.

Chronic pain, affecting tens of millions of Americans alone, is debilitating and demoralizing. It has many causes, and in the worst cases, people become “hypersensitized”—their nervous systems fire off pain signals in response to very minor triggers.

There are no good medications to calm these signals, in part because the subjectivity of pain makes it difficult to study, and in part because there haven’t been good research models. Drugs have been tested in animal models and “off the shelf” cell lines, some of them engineered to carry target molecules (such as the ion channels that trigger pain signals). Drug candidates emerging from these studies initially looked promising but haven’t panned out in clinical testing. Full story »

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Naomi Fried, PhD, is Chief Innovation Officer at Boston Children’s Hospital.

Return on innovation ROI

When people hear about ROI, they often think of financial returns and “return on investment.” But, in my world, ROI is actually “return on innovation.” While the return on innovation can be financial, it can also take many other forms. Here are my top five. Full story »

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Rare disease panelWhen a rare disease affects you or your family, it doesn’t seem rare. Add them all up, and rare diseases aren’t all that uncommon. What’s rare is for patients to receive effective treatments.

“There are 7,000 rare diseases, and under 400 approved drugs,” says Peter Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD), “so there’s a huge opportunity there to try to develop more drugs.”

Saltonstall spoke today with five other panelists at Boston Children’s Hospital’s Global Pediatric Innovation Summit + Awards in a session titled, “Rare diseases: Lessons from the path less chosen.” David Meeker, MD, president and CEO of Genzyme, moderated. Full story »

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Global Pediatric Innovation Summit Awards big dataWhere is the next generation of therapeutic innovations going to come from? Population-level genomic studies? The fitness trackers on everyone’s wrist? Mining electronic medical records? People’s tweets, Yelps and Facebook posts?

How about all of the above?

What all of these things have in common is data. Lots of it. Some of it represents kinds of data that didn’t exist 5 or 10 years ago, but all of it is slowly beginning to fuel the pharma sector’s efforts to create the next blockbuster drug or targeted therapeutic.

At least, it should be. Full story »

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biofilm vaccine cholera

Through genetic engineering, this Vibrio cholerae biofilm can be loaded with extra antigens, creating a super-charged but inexpensive vaccine.

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. That means easy to produce, store and deliver.

Paula Watnick, MD, PhD, an infectious disease specialist at Boston Children’s Hospital, has a plan that stems from her work on cholera: using a substance produced by the bacteria themselves to make inexpensive and better vaccines against them.

Cells do all the work

Bacteria produce biofilms—a sticky, tough material composed of proteins, DNA and sugars—to help them attach to surfaces and survive. Full story »

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Organs-on-chips drug testing drug discovery mechanobiology microfluidics Wyss Institute Vascular Biology Program

(Credit: Wyss Institute)

With the launch this summer of Emulate Inc., organs-on-chips—a disease-modeling platform we’ve covered several times on Vector—made the jump from academic to commercial development.

Though developed at the Wyss Institute for Biologically Inspired Engineering, the chips’ story actually began more than 20 years ago in Boston Children’s Hospital’s Vascular Biology Program (VBP). It’s a story that brings together characters from multiple fields and emerges from one fundamental concept: that mechanical forces are critical to the function and fate of cells, tissues and organs. Full story »

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neurobehavioral mouse assays

(gegenart/Shutterstock)

A mouse surrounded by computer screens turns its head when it notices lines moving across one of them, as a camera captures this evidence of visual acuity. A chamber similarly equipped with video cameras tests social interaction between mice. A small swimming pool, with shapes on its walls as navigational cues, lets scientists gauge a mouse’s spatial memory. A pint-sized treadmill, with a tiny camera to watch foot placement, measures gait.

Here in the Neurobehavioral Developmental Core at Boston Children’s Hospital, managed by Nick Andrews, PhD, the well-tended mice also have opportunities to play: “If you have a happy mouse,” says Andrews, “researchers get better, more consistent results.” Full story »

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Listeria intracellular bacteria infection T-cell immunity granzymes granulysins

Listeria bacteria on a plate. The biology of HIV/AIDS suggests T-cells have a hitherto unrecognized way of killing pathogens like these.

The immune system, despite its immense complexity, really has only a few ways to kill bacteria:

  • Neutrophils and macrophages can capture and digest extracellular bacteria (ones that live free in tissues and the bloodstream).
  • Peptides (protein fragments) can punch holes in bacterial membranes or cross the membranes to disrupt bacterial processes.
  • T-cells can kill cells infected by intracellular bacteria (ones that take up residence within cells).

It’s this last mechanism that I want you to pay attention to. The conventional wisdom has long held that T-cells can only kill intracellular bacteria indirectly by eliminating the cells they’ve infected. But a paper by Judy Lieberman, MD, PhD, of Boston Children’s Hospital’s Program in Cellular and Molecular Medicine, reveals that T-cells have a hitherto unnoticed way of directly killing intracellular bacteria And she only found it because of HIV/AIDS. Full story »

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