Boston Children's Hospital

Dupilumab Reduces Asthma Exacerbations in Children Regardless of Disease Duration

4 days ago

• Children with uncontrolled moderate to severe asthma experienced significantly fewer exacerbations when treated with dupilumab compared to placebo, regardless of how long they had been living with the disease. • The 52-week VOYAGE trial demonstrated that dupilumab improved asthma control in 6-11 year olds across all disease duration groups, with 62-79% of treated children achieving well-controlled status versus 35-56% on placebo. • Researchers noted that while longer asthma duration was associated with more frequent exacerbations overall, dupilumab provided consistent clinical benefits that could help reduce the impact of persistent symptoms on developing lungs.

FDA Expands Luminopia's Digital Therapeutic Approval for Amblyopia Treatment to Children Aged 8-12

a month ago

• Luminopia's VR-based digital therapeutic for amblyopia has received FDA clearance for children aged 8-12 years, expanding from its previous 4-7 year age indication based on robust real-world evidence. • The treatment offers a novel binocular approach using modified TV content through VR headsets, showing comparable efficacy in pre-teens as in younger children—the first such approval for this age group in over 20 years. • Patients previously treated with traditional therapies for an average of 1.8 years still gained an additional line of vision when switching to Luminopia, addressing a significant unmet need for approximately 400,000 older children with amblyopia.

FDA Approves VALTOCO Nasal Spray for Seizure Clusters in Children Ages 2-5

a month ago

• The FDA has approved Neurelis' VALTOCO (diazepam nasal spray) for treating seizure clusters in patients as young as 2 years old, expanding its previous indication to include younger children. • Clinical trials demonstrated VALTOCO's safety and efficacy in children aged 2-5, with 31 of 35 enrolled patients completing the 180-day safety period and no treatment-related serious adverse events reported. • The nasal spray formulation provides a significant alternative to rectal diazepam, addressing a major unmet need for caregivers managing seizures in young children.

Artis BioSolutions Launches with Landmark Bio Acquisition to Transform Advanced Therapy Manufacturing

2 months ago

• Artis BioSolutions has emerged from stealth mode with the strategic acquisition of Landmark Bio, positioning itself as a premier CDMO for advanced genetic medicine development. • Landmark Bio, founded in 2021 by leaders from academia and Boston's research hospitals, will continue to operate independently while expanding its capabilities in cell and gene therapy manufacturing. • The acquisition aims to address critical bottlenecks in the rapidly growing genetic medicines sector by providing integrated end-to-end solutions from preclinical development through commercialization.

Anuncia Medical's ReFlow EVD Receives FDA Breakthrough Device Designation for Intracranial Pressure Management

2 months ago

• Anuncia Medical has received FDA Breakthrough Device Designation for its ReFlow EVD, designed to manage brain swelling and elevated intracranial pressure through a novel noninvasive flushing mechanism. • The ReFlow EVD allows nurses to perform noninvasive flushing procedures without requiring a neurosurgeon, potentially reducing prolonged ICU stays, additional surgeries, and costly EVD replacements in over 100,000 annual procedures. • Beyond the ReFlow EVD, Anuncia is expanding its CSF management portfolio and partnering with organizations like NeuroKids.org to improve hydrocephalus care globally, including in underserved regions of Africa.

Aurion Biotech's AURN001 Shows Promise in Phase 1/2 Trial for Corneal Edema

5 months ago

• Aurion Biotech's AURN001, an allogeneic cell therapy, demonstrated statistically significant improvement in visual acuity in patients with corneal edema in a Phase 1/2 trial. • The high-dose AURN001 arm showed that 50% of patients achieved a ≥15-letter improvement in best-corrected visual acuity (BCVA) at 6 months, compared to 14.3% in the Y-27632-only arm. • Key secondary endpoints, including change in BCVA and central corneal thickness, also showed statistically significant improvements in the high-dose AURN001 arm. • AURN001 combines neltependocel (allogeneic human corneal endothelial cells) and Y-27632 (a rho kinase inhibitor) and is intended as a one-time anterior chamber injection.

First Sickle Cell Patient Cured with Base Editing Gene Therapy

5 months ago

• Branden Baptiste, a 20-year-old, is the first sickle cell disease patient cured using base editing, a precise form of gene therapy. • The BEACON trial, sponsored by Beam Therapeutics and led by Boston Children's Hospital, uses base editing to boost fetal hemoglobin production. • Base editing corrects single misspellings in a gene, offering a potentially safer alternative to traditional gene editing methods. • Following the treatment, Baptiste is off medication, experiences no pain, and is now able to exercise and work, marking a transformative improvement.

FDA Approves Ryoncil, First Mesenchymal Stromal Cell Therapy for Pediatric SR-aGVHD

6 months ago

• The FDA has approved Ryoncil (remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the United States. • Ryoncil is indicated for steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older. • Clinical trials showed a 70% overall response rate by Day 28 in children with SR-aGvHD treated with Ryoncil. • Ryoncil offers a new treatment option for a life-threatening condition with limited alternatives, improving outcomes for affected children.

Injectable Oxygen Microbubbles Show Promise in Treating Hypoxemia and Preventing Organ Damage

6 months ago

• Researchers have developed injectable oxygen microbubbles for rapid oxygen delivery during critical moments of cardiac or respiratory arrest, addressing the limitations of traditional methods. • In preclinical testing, pH-sensitive microbubbles delivered precise amounts of oxygen, significantly improving survival and preventing catastrophic organ damage in hypoxemic conditions. • The microbubbles are engineered with a solid-like polymer shell that dissolves into tiny soluble molecules when triggered by blood pH, ensuring rapid dissolution and preventing blood-flow obstruction. • This innovative approach represents a potential platform technology for delivering various gases in focused amounts, opening possibilities for treating other medical conditions beyond hypoxemia.

Antisense Oligonucleotide Therapies Show Promise in Treating Genetic Epilepsies

6 months ago

• Early trials of antisense oligonucleotide (ASO) therapies for rare genetic epilepsies demonstrate potential in reducing seizure frequency and increasing seizure-free days. • Stoke Therapeutics' Phase 1/2a trial of an ASO in Dravet syndrome showed significant reductions in convulsive seizure frequency in clinically evaluable patients. • Praxis Precision Medicine's PRAX-222 for SCN2A-related epilepsy led to a 43% median reduction in seizures and a 48% increase in seizure-free days in early-onset patients. • Despite promising results, the high cost of ASO treatments poses a significant challenge to accessibility, necessitating strategies like early genetic screening.

Gene Therapy for CALD Shows Long-Term Efficacy but Carries Hematologic Cancer Risk

7 months ago

• Elivaldogene autotemcel (eli-cel) demonstrates sustained efficacy in treating cerebral adrenoleukodystrophy (CALD) over several years. • A subset of patients treated with eli-cel developed hematologic cancers, including myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). • Researchers suggest that different conditioning regimens used in stem cell transplants may influence the risk of developing cancer. • The benefits of eli-cel gene therapy should be carefully weighed against the potential risk of oncogenesis, considering alternative treatments.

Xolair Evaluated for Asthma Prevention in High-Risk Young Children

8 months ago

• A clinical trial is evaluating whether omalizumab (Xolair) can prevent asthma development in young children with allergic conditions. • The study follows children who received Xolair or placebo for two years, assessing asthma incidence at ages 6 and 7. • Researchers are using molecular analyses to understand how early immune intervention affects the asthma march. • Biomarkers identified could help predict which children will respond to anti-IgE treatment, improving targeted therapy.

Liraglutide (Saxenda) Shows Promise for Weight Loss in Children Aged 6-11

8 months ago

• A Phase III trial of liraglutide (Saxenda) demonstrated a significant reduction in BMI in children aged 6-11 with obesity, compared to placebo. • The study, presented at EASD 2024, showed a 5.8% mean reduction in BMI in the liraglutide group versus a 1.6% increase in the placebo group. • Novo Nordisk plans to seek regulatory approval to expand the label for Saxenda to include children older than 6 years, potentially offering a new treatment option for this age group. • While generally safe, liraglutide was associated with gastrointestinal side effects, and BMI tended to increase again after stopping the medication.

Fentanyl Vaccine Poised for Clinical Trials, Offering Hope in Opioid Crisis

9 months ago

• A fentanyl vaccine developed at the University of Houston is set to enter clinical trials in mid-2025, aiming to prevent the opioid from reaching the brain. • The vaccine uses an adjuvant to stimulate the immune system to produce antibodies against fentanyl, preventing overdose by keeping the drug out of the brain. • Clinical trials will face challenges in recruiting participants with opioid use disorder and determining the vaccine's long-term effectiveness and optimal dosage. • Experts emphasize that the vaccine is not a standalone solution but a crucial addition to existing treatments like buprenorphine, potentially enhancing their efficacy.

NIH Launches Phase 1 Trial of Nasal COVID-19 Vaccine MPV/S-2P

a year ago

• The NIH has initiated a Phase 1 clinical trial for MPV/S-2P, a novel nasal COVID-19 vaccine, across three U.S. sites, aiming for improved protection against emerging variants. • MPV/S-2P utilizes a live-weakened murine pneumonia virus (MPV) to deliver a stabilized spike protein, potentially inducing a more robust mucosal immune response in the respiratory tract. • The trial will involve 60 healthy adults who have already received at least three mRNA COVID-19 vaccine doses, evaluating the vaccine's safety and immune response at varying dosages. • Researchers suggest nasal vaccines could offer advantages over traditional injections, including enhanced acceptance, easier storage, and potentially superior protection against the virus.