Archive for clinical trials
Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
Manny Johnson of Boston, 21, previously required monthly blood transfusions to keep his severe sickle cell disease under control. After receiving a new gene therapy treatment, he’s been symptom-free for six months. Researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported Manny’s case Saturday at the American Society of Hematology meeting in San Diego. Manny ... Read More
In zebrafish, a way to find new cancer therapies, targeting tumor promoters
The lab of Leonard Zon, MD, has long been interested in making blood stem cells in quantity for therapeutic purposes. To test for their presence in zebrafish, their go-to research model, they turned to the MYB gene, a marker of blood stem cells. To spot the cells, Joseph Mandelbaum, a PhD candidate in the lab, attached a fluorescent ... Read More
Finally in the game: Patient in drug trial for PTEN mutation seems to benefit
From the time of Preston Hall’s birth at 30 weeks, his parents navigated multiple diagnoses, surgeries and sometimes life-threatening medical issues. At 11 months, Preston underwent skull revision surgery for trigonocephaly (a fusion of the skull bones causing a triangular-shaped forehead). After surgery, his doctors discovered serious airway and gastrointestinal issues that led to his ... Read More
Safety trial of algal anesthetic kicks off
Two years ago, we told the story of the quest of Charles Berde, MD, PhD, of Boston Children’s Division of Pain Medicine, to turn an algal toxin called neosaxitoxin into a long-lasting local anesthetic. At that time, Berde—together with Alberto Rodríguez-Navarro, MD, from Padre Hurtado Hospital in Santiago, Chile, and a Chilean company called Proteus SA—already knew that ... Read More
Failed cancer drug may extend life in children with progeria
Hutchinson-Gilford Progeria Syndrome, better known as progeria, is a highly rare genetic disease of premature aging. It takes a cruel toll: Children begin losing body fat and hair, develop the thin, tight skin typical of elderly people and suffer from hearing loss, bone problems, hardening of the arteries, stiff joints and failure to grow. They ... Read More
Can asthma be nipped in the bud?
Worldwide, asthma affects an estimated 300 million people, and is expected to surpass 400 million by 2025, according to the World Health Organization. About 1 in 10 U.S. children have asthma, and research shows that the vast majority of them also have allergy. Could that provide a clue to its prevention? Starting at 2 to ... Read More
Another use for mTOR inhibitors: Preserving vanishing bones in Gorham-Stout syndrome
The mTOR pathway is fundamental to nearly every cell in the body. It drives processes related to cell growth, protein production and metabolism, influencing everything from neurocognition to tumor growth. Because of this broad role, indications for drugs targeting the mTOR pathway are also remarkably broad. Alexander Malloy, 14, is one of the first patients ... Read More
Food allergies: Turning tolerance back on
Hans Oettgen, MD, PhD, is Associate Chief of the Division of Allergy and Immunology at Boston Children’s Hospital. He leads a research group investigating mechanisms of allergic diseases. Not long ago I received a wonderful email from “Sam,” an 18-year-old young man with peanut allergy. He was participating in a clinical trial of oral immunotherapy ... Read More
Autism clinical trials are ripe for improvement
Walter Kaufmann, MD, is co-director of the Fragile X Syndrome Program and a member of the department of Neurology at Boston Children’s Hospital. He was site principal investigator for three arbaclofen trials sponsored by Seaside Therapeutics and currently advises the company on data analyses. This post is second in a two-part series on clinical trials in ... Read More
Rett syndrome sees glimmer of hope in Phase I trial
Part 1 of a two-part series. (Read part 2.) In the world of neurodevelopmental disorders, an exciting trend is the emergence of specific molecular targets and treatments through genetic research. A case in point is IGF-1 therapy for Rett syndrome, a devastating disorder in girls that affects their ability to speak, walk, eat and breathe. It ... Read More