Stories about: patient care

Putting patients first in the translational research pipeline

During a follow-up visit, pediatric hematologist/oncologist Sung-Yun Pai, MD, hugs a patient who received gene therapy for X-linked severe combined immunodeficiency.
During a follow-up visit at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, pediatric hematologist/oncologist Sung-Yun Pai, MD, hugs a patient who received gene therapy for X-linked severe combined immunodeficiency.

This is part II of a two-part blog series recapping the 2018 BIO International Convention. Read part I: Forecasting the convergence of artificial intelligence and precision medicine.

The hope to improve people’s lives is what drives many members of industry and academia to bring new products and therapies to market. At the BIO International Convention last week in Boston, there was lots of discussion about how translational science intersects with patients’ needs and why the best therapeutic developmental pipelines are consistently putting patients first.

As a case in point, Mustafa Sahin, MD, PhD, of Boston Children’s discussed his work to improve testing and translation of new therapies for autism spectrum disorder (ASD). As a member of PACT (Preclinical Autism Consortium for Therapeutics) and director of Boston Children’s Translational Neuroscience Program, Sahin aims to bridge the gap between drug discovery and clinical translation.

“Our mission is to de-risk entry of new therapies in the ASD drug discovery and development space,” said Sahin, who is also a professor of neurology at Harvard Medical School.

One big challenge, says Sahin, is knowing how well — or how poorly — autism therapies are actually affecting people with ASD. Externally, ASD is recognized by its core symptoms of repetitive behaviors and social deficits.

Read Full Story | Leave a Comment

Hacking our way to a new mobile app

Brian Rosman holds up a tablet app he and a team of Children's and MIT Media Lab staff developed over the past two weeks during the Health and Wellness Hackathon

At 10 a.m. he’s directing two actors on set, at 10:34 a.m. he’s filling up a catheter and at 11:01 a.m. he’s gushing about the importance of pediatric avatars. Brian Rosman, a Robotic Surgery Research Fellow in the Department of  Urology at Children’s Hospital Boston, has been working non-stop at the MIT Media Lab’s Health & Wellness hackathon to create a new app for post-operative care. His duties have included directing a video about the app, rounding up realistic props and explaining how the program works to judges and hackathon attendees.

Rosman and his team of coders, clinicians and industry professionals are competing against five other teams for a $10,000 prize awarded to the best open source healthcare application.

Read Full Story | Leave a Comment