riluzole

Together, these findings reveal a novel mechanism where Riluzole promotes apoptosis through upregulation of EGR1 , which then cooperates with YAP/p73 to activate Bax expression. These insights establish Riluzole as a promising therapeutic intervention for OS treatment through modulation of the EGR1 /Yap/p73/Bax signaling axis.

P1, N=35, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Sep 2025 --> Sep 2026

Key findings include the following: (1) Epilepsy studies reveal more than 250 sodium voltage-gated channel alpha subunit 8 mutations with distinct genotype-phenotype correlations, where gain-of-function variants lead to severe epileptic encephalopathies, while loss-of-function variants are associated with generalized epilepsy, highlighting the potential of Nav1.6-selective blockers such as XEN901 and GS967...(4) Amyotrophic lateral sclerosis involves Nav1.6-dependent cortical hyperexcitability preceding motor neuron degeneration, with riluzole showing partial efficacy through sodium current modulation...Future directions emphasize the integration of advanced technologies-such as singlecell multiomics to map neuronal subtype-specific expression patterns, patient-derived organoids for personalized drug testing, and machine learning-assisted drug design-to accelerate translation. Large-scale collaborative efforts will be essential to validate therapeutic candidates and establish genotype-guided treatment protocols for Nav1.6-related disorders.

P3, N=495, Recruiting, AB Science | Trial completion date: Dec 2023 --> Dec 2027 | Trial primary completion date: Dec 2023 --> Dec 2027

P2/3, N=5, Not yet recruiting, University of Melbourne

The in vivo experiments demonstrated that LD-1 significantly suppressed tumor growth (TGI = 47.64 %) at a dosage of 40 mg/kg, coupled none obvious adverse reactions were observed. Collectively, LD-1, characterized by its novel structure, high potency, and selectivity as an MST3 inhibitor, showcases substantial potential for further investigation and therapeutic development.

P2, N=51, Recruiting, University of California, Irvine | N=34 --> 51 | Trial completion date: Dec 2026 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2026

Overall, our analysis revealed that blocking of adenosine A2A receptor downstream signaling pathway protein CK1δ with its inhibitor, riluzole, showed higher anti-GBM effect than its upstream signaling blocker, istradefylline. Thus, blocking adenosine A2A receptor downstream effector signaling protein through its antagonist and blocking its effector protein CK1δ could provide an opportunity to develop targeted therapy for GBM.

Additionally, riluzole suppresses the activity of matrix metalloprotease-2 (MMP2) in most of the osteosarcoma cell lines (but not the PDX cells). These results suggest that riluzole's inhibitory effects on osteosarcoma invasion may in part be attributable to the inhibition of MMP2 activity, and that riluzole is potentially an effective agent for inhibiting growth of primary and metastatic osteosarcomas with a wide range of genetic profiles.

Ril+Met co-treatment had a positive effect in terms of GBM cell death, decreased expression of genes involved in glucose metabolism and stemness, and reduced MMP2 activation. Disadvantage of Ril+Met treatment was increased cell migration. Taken together, these drug combinations may also allow the reduction of the concentration of Dex to minimize its side effects.

P2, N=80, Suspended, UNICANCER | Trial completion date: Mar 2025 --> Dec 2025 | Trial primary completion date: Mar 2025 --> Aug 2024

P1, N=10, Recruiting, Beijing Tiantan Hospital