Archive for clinical trials
Leading the way: Ava’s experience with teduglutide
Ava Orsini visits Boston Children’s Hospital every two weeks. It’s more time than any kid wants to spend at a hospital, particularly when she lives a state away. But for this 9-year-old and her parents, the trips are worth it: For the past two years, she’s been participating in a clinical trial of a drug ... Read More about Leading the way: Ava’s experience with teduglutide
Children wait for new cancer drugs 6.5 years longer than adults
A 20-year analysis finds that FDA-approved cancer drugs took a median of 6.5 years to go from the first clinical trial in adults to the first trial in children. That’s not good enough for researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, who are calling for expanding children’s access to experimental cancer therapies. “It’s ... Read More about Children wait for new cancer drugs 6.5 years longer than adults
Nick: Overcoming his fears to make a difference for others
People decide to participate in a clinical trial for lots of reasons. For 14-year-old Nick Brown, it came down to wanting to make a difference in the world. Diagnosed with autism at age 5, Nick has had a lifelong aversion to hospitals, and especially to needles. Yet, when his parents told him about a new ... Read More about Nick: Overcoming his fears to make a difference for others
The space between heartache and happiness: Two sons with adrenoleukodystrophy
When Paul and Liliana Rojas talk about their life, they describe it in one of two ways — the way it was before their sons, 10-year-old Brandon and 7-year-old Brian, were diagnosed with ALD, and the way it is after. Their story is one of heartbreak — but also hope, in the form of a ... Read More about The space between heartache and happiness: Two sons with adrenoleukodystrophy
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 about Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
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 about In zebrafish, a way to find new cancer therapies, targeting tumor promoters
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 about Finally in the game: Patient in drug trial for PTEN mutation seems to benefit
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 about Safety trial of algal anesthetic kicks off
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 about Failed cancer drug may extend life in children with progeria
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 about Can asthma be nipped in the bud?