Archive for precision medicine
Precision chemo-immunotherapy for pancreatic cancer?
Pancreatic cancer is highly lethal and in great need of better treatments. Only about 10 percent of patients remain alive five years after diagnosis. In a new study, researchers in the lab of Marsha Moses, PhD, at Boston Children’s Hospital offer a glimmer of hope. Key takeaway An antibody-drug combination effectively targeted, penetrated, and shrank ... Read More about Precision chemo-immunotherapy for pancreatic cancer?
Against all odds: Mila’s unique mutation, and her own custom drug
Ed. note: Mila passed away in February 2021, at age 10. The Mila’s Miracle Foundation continues to work to pave a pathway for personalized treatments. The FDA recently released a draft guidance on testing custom drugs such as Mila’s in patients. As a baby and toddler, Mila was healthy, active, and — in some ways ... Read More about Against all odds: Mila’s unique mutation, and her own custom drug
Shooting for the moon: From diagnosis to custom drug, in one year
Ed. note: Mila passed away in February 2021, at age 10. The Mila’s Miracle Foundation continues to work to pave a pathway for personalized treatments. The FDA recently released a draft guidance on testing custom antisense oligonucleotides (ASOs) in patients. One weekend in January 2017, Timothy Yu, MD, PhD, was relaxing at home when his ... Read More about Shooting for the moon: From diagnosis to custom drug, in one year
Small samples, big data: A systems-biology look at a newborn’s first week of life
The first week of a baby’s life is a time of rapid biological change. The newborn must adapt to living outside the womb, suddenly exposed to new bacteria and viruses. Yet scientists know surprisingly little about these early changes. Reporting in Nature Communications, an international research team provides the most detailed accounting to date of the ... Read More about Small samples, big data: A systems-biology look at a newborn’s first week of life
Typing medulloblastoma: From RNA to proteomics and phospho-proteomics
Medulloblastoma is one of the most common pediatric brain tumors, accounting for nearly 10 percent of cases. It occurs in the cerebellum, a complex part of the brain that controls balance, coordination and motor function and regulates verbal expression and emotional modulation. While overall survival rates are high, current therapies can be toxic and cause secondary ... Read More about Typing medulloblastoma: From RNA to proteomics and phospho-proteomics
Families and data scientists build insights on Phelan-McDermid syndrome
This is the third year that Jacob Works has made the trip down to Boston Children’s Hospital from Maine. With research assistant Haley Medeiros, he looks at pictures, answers questions, manipulates blocks and mimes actions like knocking on a door. His father, Travis, and another research assistant look on through a window. “At first, we ... Read More about Families and data scientists build insights on Phelan-McDermid syndrome
Beyond appearances: Molecular genetics revises brain tumor classification and care
Pomeroy Scott Pomeroy, MD, PhD, is Neurologist-in-Chief at Boston Children’s Hospital. He practices in the Brain Tumor Center and is a member of the F.M. Kirby Neurobiology Center. For almost a century, brain tumors have been diagnosed based on their appearance under a microscope and classified by their resemblance to the brain cells from which ... Read More about Beyond appearances: Molecular genetics revises brain tumor classification and care
Pharmacogenomics: One size doesn’t fit all
In 2009, The New England Journal of Medicine reported the case of an otherwise healthy 2-year-old boy in Canada who died after surgery. He had received a codeine dose in the recommended range, but an autopsy revealed that morphine (a product of codeine metabolism) had built up to toxic levels in his blood and likely depressed his ... Read More about Pharmacogenomics: One size doesn’t fit all
Orphan diseases: Bringing academia, industry, and government into the game
“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 ... Read More about Orphan diseases: Bringing academia, industry, and government into the game
Childhood brain cancer: Learning to divide and conquer
Diversity is good in populations of people, but when it comes to cancer, it’s bad news. In the case of medulloblastoma—the most common malignant brain cancer in children—tumor diversity has been one of the greatest barriers to designing effective treatments. Now, in the largest genomic study of human medulloblastomas ever, Children’s researchers and their collaborators ... Read More about Childhood brain cancer: Learning to divide and conquer