Trehalose

Georg Schett presented data on CD19-directed CAR-T therapies in refractory autoimmune and rheumatic diseases at ACR Convergence 2024, highlighting low relapse rates and faster manufacturing processes.

The HEALEY ALS Platform Trial, launched in 2020, aimed to accelerate ALS treatment development. Despite challenges and mixed results, Clene Nanomedicine and Prilenia Therapeutics are advancing their treatments based on positive secondary endpoint data. The trial's innovative, patient-centric approach and efficiency in testing multiple drugs simultaneously mark a significant step in ALS research.

New imaging technologies and improved use of existing ones may advance precision medicine in inflammatory arthritis, according to Georg Schett, MD, at the 2024 Congress of Clinical Rheumatology West. Schett highlighted the potential of machine learning and MRI for diagnosing undifferentiated arthritis and differentiating between conditions like psoriatic arthritis and rheumatoid arthritis. He also discussed the use of high-resolution CT for bone mass determination and treatment efficacy checks, emphasizing the need for a 3D approach over conventional X-rays. Schett noted the infancy of functional imaging and introduced new technologies like multispectral optoacoustic tomography (MSOT) for entheseal tissue visualization in PsA and psoriasis patients. He concluded by suggesting that understanding tissue response in inflammation, such as with fibroblast activation protein inhibitors, could guide therapeutic decisions.

Despite challenges, the HEALEY ALS Platform Trial continues with optimism, as Clene Nanomedicine and Prilenia Therapeutics progress their ALS treatments based on positive secondary endpoint data. The trial, initiated by a $40 million donation from Sean Healey and AMG, aims to efficiently test multiple therapies simultaneously. While primary endpoints have not been met, the trial's design allows for quick identification of non-viable treatments, and positive outcomes in subgroups have supported further development for some candidates.

Autophagy, crucial for neuronal health, is impaired in ALS, making its regulation a potential therapeutic target. Various drugs, including Rapamycin, Trehalose, Lithium, and others, show promise in enhancing autophagy and improving ALS symptoms in animal models, though clinical efficacy varies. Gene therapies and stem cell transplantation also offer hope, with ongoing research into their safety and effectiveness for ALS treatment.