Stories about: translational medicine

Orphan diseases: Bringing academia, industry, and government into the game

To make headway in translating the growing body of genomic knowledge into new treatments for orphan or rare diseases, we have to bring everyone – academia, industry, government, patients, etc. – into the game. (Navin Rajagopalan/Fotopedia)

“If you build it, he will come,” the ghosts of baseball players past tell a farmer in Field of Dreams. But it’s not that easy. To put people in the seats you have to have all of the right pieces: the right team, including players and managers; the right park, one that works for both the team and the fans; and a passion for being the best at the game.

In the field of rare diseases, not only are institutions like Children’s Hospital Boston stepping up to the plate, but industry and government are joining the game, bringing expertise, guidance and infrastructure. Together, they’re starting to turn basic biomedical discoveries – many made possible only through dedicated patients and their families – into lifesaving treatments.

The shape of the game came into clear view last week at the 2nd Annual Rare Disease Symposium at Children’s. This daylong event, hosted by Children’s Technology Innovation & Development Office (TIDO) and the Manton Center for Orphan Disease Research, united scientists, clinicians, regulators, funders and industry. It showcased not only the depth of the team Children’s has fielded for orphan disease work, but also facilities and support available through industrial and governmental partners to conduct translational research on orphan diseases.

Without a doubt, the word of the day was genomes.

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Reinventing therapeutic development: Systems pharmacology

As Children’s Hospital Boston holds its 2nd Annual Rare Disease Symposium today, here’s a reminder that drug development is incredibly difficult: many initially promising drugs ultimately prove ineffective or have unacceptible toxicity—typically at a late stage of a clinical trial. But it needn’t be that way. In this 10-minute video, introducing its Initiative in Systems Pharmacology, Harvard Medical School makes the argument that focusing on a drug’s action on a single cellular target is a flawed approach — rather, we need to understand and predict its activity in all body systems.

Today’s symposium, co-sponsored by the Technology and Innovation Development Office and the Manton Center for Orphan Disease Research, will address therapeutic development and for rare, often fatal diseases, focusing on moving discoveries to clinical trial and models for partnerships between academia, industry, the FDA and NIH, foundations and patient advocacy groups. Use this link to watch the proceedings live starting at 9 a.m. You can also follow the symposium on Twitter, using the hashtag #CHBRare. Click here for the full agenda.

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Brains, babies and battlefields: Taking pediatric neurocritical care to the bedside

Evacuation of a soldier injured by a roadside bomb, June 17, 2011, Kandahar province of Afghanistan (DVIDSHUB/Flickr)

From the time he was 11, Robert Tasker knew he wanted to be a doctor. The son of a serviceman, he was drawn to battlefield surgery, evacuations and managing traumatic injuries. Instead, he ended up on a different kind of battlefield, where what’s at stake are the highly vulnerable, still developing brains of infants and children – and where it’s critical to be mobile and show up on time.

Tasker directs the Pediatric NeuroCritical Care program at Children’s Hospital Boston, the first of its kind in the world. His goal is to protect brain function not only in children suffering direct head injury, but children undergoing major surgery, children with stroke, children hospitalized for critical illness, children on ventilators, children with nervous-system infections like meningitis and more.

Born in Hong Kong and raised throughout the globe,

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BioPharm 2011: Stakeholders’ changing roles in therapeutic development

Can we get this family to function? (Photo: eyeliam/Flickr)

My summary of BioPharm America 2011: We are a family and we just need to work together. As stakeholders in developing new treatments, we each have our own shortfalls and strengths, we’re under pressure, and our roles are changing over time.

Here’s the panelists’ take on the different players.

Big pharma: The old business model is broken. Pharma is cutting R&D and other programs that aren’t generating enough return. Companies now approach markets differently, said Angus Russell, CEO of Shire. A new product doesn’t have to be a first-line therapy to justify market entry; there’s a business case for selling a targeted drug to patients who don’t respond to generics and have no other solution.

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Vector’s upcoming coverage of BioPharm 2011

Vector and Children’s Hospital Boston’s Technology and Innovation Development Office (TIDO) at will be enthusiastic participants in next week’s BioPharm America conference (Sept 7-9).  In between networking with industry colleagues and pitching technologies developed at Children’s, we’ll be providing blog coverage and live-tweeting from the sessions under the hashtag #BPA11.

Since BioPharm is in Boston this year, we’re also heavily involved as presenters. Bruce Zetter of Children’s Vascular Biology program will speak about cancer biomarkers on a Wednesday panel titled Lessons Learned in Personalized Medicine in Oncology. Leonard Zon, director of Children’s Stem Cell Program, will describe the development of stem-cell-boosting drugs as part of a Friday panel titled Stem Cells and Drug Discovery: Confronting the Translational Imperative.

And Erik Halvorsen, director of Children’s TIDO, is a panelist in Wednesday’s session titled: Pharma’s Interest in Collaborating Early: Flavor of the Month or Viable Business Model?

Halvorsen elaborates on this theme in an interview for the online life sciences journal Partnering News.

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Enhancing the “virtuous cycle”: Focusing academic-industry partnerships


Therapeutic development at academic research hospitals
The virtuous cycle of treatment development, and how it can be enhanced by partnership with industry.

When the clinical and research enterprises at an academic medical center are strategically and tactically aligned, they enter a “virtuous cycle,” described by Stephen Wartman, president of the Association of Academic Health Centers. Clinician-researchers embody it: They know their patients’ needs, what treatments are available and the frustration when those options are insufficient. They take clinical problems back to the lab to figure out solutions, and are highly motivated to develop products because their goal begins and ends with helping the patient.

The academic medical center is the epicenter of therapeutic development. It’s where potential drug targets are identified and mapped from animal models to human tissues, and where clinical trials will ultimately take place. Yet, in the past, pharmaceutical and biotech companies have driven drug development, while academia has been a piecemeal contributor

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Big pharma’s changing business model: Inviting academia to take the lead

(photo: kisforkate/Flickr)

In almost every conversation I have these days with potential industry partners, I hear what seems to be the new buzz phrase: “Clear path to clinic.” A molecule with a “clear path to clinic” can become a medicine quickly, by virtue of a well-defined patient population, clear endpoints for clinical trials and measurable biomarkers.

Industry is increasingly recognizing that investigators at academic research centers know this path to clinic best. This has brought a shift in how industry interacts with academia, and new and equitable partnership structures are poised to facilitate joint therapeutic development like never before.

Pharma companies have a strong incentive to change.

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From kittens to Fragile X: Do all autisms share a common thread?

(AmberStrocel/Flickr)

Mark Bear’s research interests have taken him from studying vision in kittens to learning and memory in mouse models, and more recently, to the study of Fragile X syndrome, one of the leading genetic causes of autism and intellectual disability in humans. Along the way, he has made several ground-breaking contributions to neuroscience – one of which he described as one of MIT’s presenters at this week’s inaugural CHB-MIT Research Enterprise Symposium, which kicked off an exciting new scientific collaboration between MIT and Children’s.

I have followed Mark Bear’s work since I was an undergraduate at Brown University, where he used to teach the Introduction to Neuroscience course. That’s where I first learned about the seminal experiments in kittens (see this PDF), showing that covering one eye at birth rewires their brains not to “see” out of that eye, work that Bear was continuing to refine. Our paths crossed again more recently due to our common interest in studying autism.

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Children’s partnership with Pfizer: A new way to speed therapeutic development

Over the past nine months, Pfizer has built collaborations with a number of premiere academic medical centers, including Children’s Hospital Boston. Wednesday marked the launch of the Boston branch of Pfizer’s Centers for Therapeutic Innovation (CTI), fostering independent collaborations with seven Boston institutions. The CTI aims to facilitate and support joint drug discovery and development — from the conception of an idea through early clinical trials.

So why is Children’s Hospital Boston, the #1 pediatric hospital in the country with an annual research base of $225 million, entering into a partnership with Pfizer? Simply, Pfizer has complementary knowledge, resources and infrastructure to support a number of our therapeutic projects. Pfizer, and other companies, can help us move early-stage discoveries out of the lab and safely into the clinic more quickly than we could on our own, ultimately supporting our mission.

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The graft that keeps on giving

A tissue engineered cartilage tube ready for implantation.

Tissue-engineered repairs and replacement parts aren’t just concepts out of science fiction – they promise to provide the ideal solution for thousands of children born each year with congenital anomalies or who suffer devastating injuries. A study released yesterday in The Lancet and covered on NPR reports on the latest tissue engineering advance.

Anthony Atala, a former Children’s Hospital Boston urologist and now director of the Institute for Regenerative Medicine at Wake Forest University School of Medicine, reports on five young boys in Mexico City whose damaged urethras he replaced with laboratory-grown urethras over five years ago. The patients had suffered damage to their urinary tracts from auto accidents, leaving them unable to urinate without a catheter.

In an approach he began at Children’s back in the late 1990s, Atala and his colleagues took a biopsy of bladder tissue from each boy, and expanded the cells in the laboratory until there were approximately 100 million cells

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