Stories about: TIDO

What’s trending in neurological drug development?

Advanced MRI scans of the brain showing neural network connections
Credit: Boston Children’s Hospital

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. So what are the key ways that researchers and drug industry collaborators are discovering new therapies for preventing or reversing neurological disease?

Experts weighed in recently to offer their insights.

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Making breastfeeding a breeze: Cleft lip/palate and beyond

Breast Breeze
Breast Breeze developers Olivia Oppel (left) and Janet Conneely (Photos: Katherine C. Cohen)

Janet Conneely, BSN, RN, CPN, was visiting a new mother in the hospital who had just delivered a baby with a cleft palate to let her know about Boston Children’s Hospital’s Cleft Lip and Palate Program. The mother was trying, without success, to breastfeed, but because of cleft palate, her baby didn’t have an intact hard surface on the roof of her mouth, so couldn’t create enough suction to draw milk.

“I was new to seeing these moms,” Conneely recalls. “This mother was in tears, pleading for ‘some way to be able to breastfeed my baby!’” She adamantly did not want to be shown the specialty bottle typically used for babies with cleft palate.

Conneely tapped her colleague, Olivia Oppel, BSN, RN, CPN, CLC, and together, they reviewed existing breastfeeding products. The few that were available — nipple shields, bottle attachments and a sling that holds the bottle against the breast — were either awkward to use or didn’t really allow for skin-to-skin contact.

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

Rare disease research: DNA helix pictured here
Genetic mutations underpin many rare diseases.

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.

Panelists:
David Williams, MD: President, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center; Senior VP, Chief Scientific Officer and Chief of Hematology/Oncology, Boston Children’s
Wayne Lencer, MD: Chief of Gastroenterology, Hematology and Nutrition, Boston Children’s
Phil Reilly, MD, JD: Venture Partner at Third Rock Ventures
Alvin Shih, MD, MBA: Chief Executive Officer at Enzyvant

Even at a place like Boston Children’s Hospital, where doctors regularly see children with rare diseases from all over the world, there are big challenges when it comes to drug discovery and treatment.

“Roughly 70 percent of drugs to treat children are used off-label,” says David Williams, Boston Children’s chief scientific officer. “That’s because these drugs were initially developed for adults and have not been tested formally in children.”

In order to cure rare diseases in children and adults, scientists must bridge the gap between research and industry. On May 25, Boston Children’s Technology and Innovation Development Office (TIDO) and MassBio held a candid panel discussion about what it will take to advance the development of rare disease therapies. Here are three of the biggest takeaways

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Getting academic diagnostic discoveries to market: 6 tips from industry

diagnosticsdiagnostics (008) Loading of QIAsymphony platform“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. So why hasn’t it happened?

Aro, senior director for new product planning at Myriad, outlined the business obstacles at a recent panel hosted by Boston Children’s Hospital’s Technology and Innovation Office (TIDO).

First, who are the target patients for a pancreatic cancer test? Skinny diabetics, patients with chronic pancreatitis, patients with hereditary cancer risk — or all three? “Those three patient types all sit in different doctor’s offices,” said Aro. Simultaneously reaching endocrinologists, gastroenterologists and high-risk patients would be an insurmountable challenge, Myriad concluded.

Second, the assay would likely need to be validated in all three patient populations, with confirmatory imaging. Could the test populations be large enough to make the results statistically significant?

Third, a new test wouldn’t change care, as there is no treatment for pancreatic cancer. In fact, no current data show that earlier diagnosis improves survival. So who would pay for it?

Aro’s story exemplifies just some of the challenges in developing a new diagnostic test.

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Accessing startup resources: 10 tips for physicians and scientists

accessing startup resources

How do you get companies or investors to support your project in a startup world where “many are called, few are chosen?”

Vector attended a panel last week on the subject, moderated by Ryan Dietz, Senior Licensing Manager at Boston Children’s Hospital’s Technology and Innovation Development Office (TIDO). The panelists were:

Below is their distilled advice for physicians and scientists seeking to commercialize a drug discovery, device or health app.

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Digital health, innovation and partnerships: A Q&A with Boston Children’s Chief Innovation Officer

Brownstein
Brownstein

During the last decade or so, health care has been rapidly transforming from a reactive, paper-based system to a responsive digital model.

Massachusetts, under Gov. Charlie Baker’s leadership, has launched a comprehensive public-private partnership to accelerate the state’s digital health care sector. The partnership has identified multiple ways to drive investment and growth in the state.

Technology transfer from universities to private companies is just one example. In the past, each transfer required completely new agreements. Three new standardized templates for licensing, technology transfer and sponsored research will help facilitate these processes. In 2016, the partnership will expand its Mentorship Speakers Series with a stronger focus on digital health care. Finally, the Digital Healthcare Innovation Hub and Accelerator will provide a space to support and grow new digital health companies in Boston.

Vector visited with John Brownstein, PhD, Boston Children’s Hospital’s Chief Innovation Officer, to better understand the background and potential impact of this new initiative.

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It’s just a hat: Simplicity in innovation

Clinical innovations don't have to be complex. Sometimes, as nurse Karen Sakakeeny has found, an innovation can be as simple as a hat (shown here on a doll). (Courtesy Karen Sakakeeny)

When we think about innovation, especially in health care, our thoughts often turn to the highly complex: new surgical procedures, new drugs, new devices or machines, etc.

But innovation in medicine and patient care doesn’t have to be complex. Sometimes it can be very simple. Like a hat.

Karen Sakakeeny has been a clinical nurse for more than 30 years, spending much of that time in the operating room. While doing a stint in cardiac surgery, she found herself thinking about ways to improve the rewarming process for infants undergoing open heart surgery.

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