The distance between a patient’s suffering and a researcher’s potential treatment is too often too great, particularly in rare disease. Closing that gap for children with rare congenital conditions is the mission of Shire’s partnership with Boston Children’s Hospital.
“The collaboration between industry such as Shire and academic institutions is very important to bringing drugs forward,” said Mark Puder, M.D., Ph.D., assistant program director of pediatric surgery at Boston Children’s Hospital. “We see the diseases, and we see the mortality. We can actually see what the true problems are that need to be solved.”
Researchers at Children’s found they have ideas on how to address conditions but may need industry support and expertise in drug formation and clinical trial design to take the next step. Shire and Boston Children’s Hospital embarked on a partnership to develop a medication for children with certain conditions, leveraging the core competencies of each organization to accelerate the development process and help bring therapies to children more quickly.
“As a physician and scientist, sometimes you hesitate to work with industry because you fear your loss of autonomy or control over the science,” explained Prathima Nandivada, M.D., research fellow at Boston Children’s Hospital. “I have to say that our collaboration with Shire has been really reassuring in that sense. The opportunity to work together to come up with a therapy for these families is really amazing.”
The collaboration between the physicians and researchers at Boston Children’s Hospital and the development team at Shire has offered a blueprint for a shorter bridge from the clinic to the lab and has illustrated the tremendous promise these kinds of collaborations hold to deliver not just hope but help for people managing rare diseases.