Tag Archive: precision medicine

Jul 3 2019

Genetic Discovery Leads to New Treatment for Defective Lymphatic Circulation

The findings:

A study team at Children’s Hospital of Philadelphia discovered a gain-of-function mutation in the ARAF gene causing a life-threatening rare disease known as central conducting lymphatic anomaly that disrupts circulation of lymphatic fluid. They also identified an existing drug that acts on biological pathways affected by ARAF. The experimental treatment had dramatic results for a young boy with the disease who had worsening respiratory and swelling problems. The drug blocked the signals causing the dysregulated growth and abnormal lymphatic flow, prompting his lymphatic channels to reshape themselves into more normal anatomy and function.

Read the rest of this entry >>

Jan 30 2019

Borrowing a Weapon From Leukemia’s Arsenal to Develop New Immunotherapy Approaches

More than six years after Emily Whitehead became the first child to receive chimeric antigen receptor (CAR) T-cell therapy, doctors have had remarkable success in turning the immune systems of even more children with acute lymphoblastic leukemia (ALL) into top-notch fighters against the disease. For some patients, however, these superhero T-cells still fail in their mission to find and fight their cancer targets.

Read the rest of this entry >>

Mar 9 2018

Lung Stem Cells, 2021 Congress of the ISTH, Backseat Safety, Pan-Cancer Analysis, Gates Cambridge Scholarship

From the discovery of stem cells that multiply after a lung injury, to new data that advances how we think about (and treat) childhood cancer, our first roundup of March is packed with discovery. Read on to learn how our researchers stay at the forefront of pediatric science with a new study that expands what scientists know about the body’s extraordinary ability to regenerate, a pan-cancer project that distinguishes how cancer develops in children versus adults, and a handful of updates on what our investigators have in store for the near future.

Read the rest of this entry >>

Dec 29 2017

Gene Therapy Makes Headlines, Mitochondrial Medicine, Brain Injury Collaboration, Implicit Racial Bias Blog

After a year of huge headlines for children’s health, we’ve finally arrived at our last roundup of Children’s Hospital of Philadelphia research news for 2017. The latest developments cap off a benchmark year of discovery, with new advances in treating childhood blindness, novel initiatives to study traumatic brain injury, and wonderful stories from a variety of media outlets that highlight the efforts and accomplishments of our Mitochondrial Medicine Frontier Program and The Raymond G. Perelman Center for Cellular and Molecular Therapeutics. We think this edition’s collection of news items herald exciting things to come in 2018!

Read the rest of this entry >>

Jul 28 2017

Psychology in Media Award, Targeted Cancer Drugs, Dermatology App, CHOP ROP Model, Beckwith-Wiedemann Conference

CHOP Research In the NewsRead on for more exciting headlines from this week, including highlights from our inaugural “Deciphering Beckwith-Wiedemann Syndrome” conference.

Read the rest of this entry >>

May 9 2017

Scientific Symposium Emphasizes Future Directions of Research Institute

symposiumSynapses were firing throughout the conference room in the Colket Translational Research Building as attendees at the 2017 Research Institute Scientific Symposium held May 2 learned about their colleagues’ intriguing research endeavors.

Read the rest of this entry >>

Feb 8 2017

Philadelphia Coalition for a Cure: Re-defining Cancer Research Through Collaboration

PC4CThe newly launched Philadelphia Coalition for a Cure (PC4C) received a warm welcome at Children’s Hospital of Philadelphia in honor of World Cancer Day.

Read the rest of this entry >>

Sep 23 2016

CHOP Research In the News: Emmy Award, Kids and the Cancer Moonshot, Precision Approach to Epilepsy, Concussion Monitoring App

CHOP Research In the NewsThis week we’re all about getting smart in our highlights of research news from The Children’s Hospital of Philadelphia. Getting smart in the approach to tackling childhood cancer means identifying strategies that will make a decade’s progress in half the time.

Read the rest of this entry >>

Feb 25 2016

CHOP Big Data Center Visits White House for Precision Medicine Summit

precision medicineToday at the White House, President Obama welcomed guests, including Adam Resnick, PhD, representing The Children’s Hospital of Philadelphia, for a morning of remarks and discussions about what the Precision Medicine Initiative (PMI) has achieved to date, and how it can take the next steps into the new era of medicine that delivers the right treatment at the right time to the right person.

Read the rest of this entry >>

Jan 28 2016

Precision Medicine Initiatives, Medical Mysteries in Bench to Bedside

Bench to BedsideA blizzard of research happenings and news — from initiatives that are pushing precision medicine forward to a new way of thinking about how cancer progresses — appear in the January issue of Bench to Bedside.

Read the rest of this entry >>