Boston Children's Hospital

AI medical devices are proliferating, but only 20% of those approved for children incorporate pediatric data. This underrepresentation in test cohorts raises concerns about potential harm when adult AI algorithms are used for children, as their anatomy and disease processes differ. Challenges include limited market size and increased costs for pediatric studies.

Novo Nordisk and Evotec select three projects for LAB eN², focusing on cardiometabolic diseases. LAB eN² expands to include Boston Children's Hospital, Johns Hopkins University, and others. The initiative leverages Evotec's R&D platform and Novo Nordisk's disease expertise for drug discovery.

Evotec SE and Novo Nordisk select first three projects for LAB eN² drug discovery accelerator, focusing on cardiometabolic diseases from Boston University, Harvard University, and Joslin Diabetes Center. The program expands to include five new academic institutions, offering initial Discovery Award funding and potential additional funding up to IND application stage, with Novo Nordisk maintaining the option to further develop and license specific programs.

Branden Baptiste, 20, underwent base editing gene therapy for sickle cell disease at Boston Children's Hospital, becoming the first person to receive this treatment. The therapy, aimed at boosting fetal hemoglobin production, has significantly improved his condition, allowing him to resume normal activities and plan for a career in civil engineering.

FDA grants RMAT designation to Mesoblast’s rexlemestrocel-L for treating children with hypoplastic left heart syndrome (HLHS). The therapy, an allogeneic mesenchymal precursor cell therapy, previously showed significant increases in left ventricular volumes in a phase 1/2 trial and improved survival in patients with heart failure in the DREAM-HF trial.

Boston Children’s cardiologist John Kheir, MD, and researcher Yifeng Peng, PhD, developed a safe and effective oxygen delivery method using injectable oxygen carried by gas microbubbles, significantly improving survival in pre-clinical testing. Their work paves the way for a future clinical trial, aiming to provide necessary oxygen during hypoxemia and cardiac arrest.

Early trials of antisense oligonucleotide (ASO) treatments for rare genetic epilepsies show promise in reducing seizures, but high costs pose challenges. ASOs have grown from a single N-of-1 study in 2019 to involve over two dozen patients, with some trials reporting significant seizure reductions. Despite promising results, the high cost of ASO treatments, potentially in the millions, raises concerns about accessibility. Neurologists emphasize the need for genetic screening at birth to identify patients early and streamline the development of these gene-based therapies.

Axonis, launched in 2020, aims to enhance KCC2 protein effectiveness for epilepsy and pain treatment, with clinical testing planned for 2023. The company secured funding to advance its lead program and explore psychiatric and neurodevelopmental disorder treatments. The epilepsy drug market is projected to grow from $10.15 billion in 2022 to $15.35 billion by 2030.

Most boys treated with eli-cel gene therapy for cerebral adrenoleukodystrophy (CALD) are faring well six years later, with 94% showing no neurological decline and 80% free of disability. However, six of 35 patients developed blood cancers linked to the therapy, highlighting the risks associated with gene therapies.

Researchers at Boston Children’s Hospital are studying if Xolair can prevent asthma in young children with allergic conditions by blocking IgE, potentially halting the 'allergic march' toward asthma.