(Formerly NU-9) Small Molecule
In July of 2021, Cure SPG4 Foundation initiated a collaboration with Hande Ozdinler, PhD at Northwestern University to explore the potential for AKV9 (formerly NU-9) to improve the health of upper motor neurons that become diseased due to SPG4. Dr. Ozdinler is an expert in upper motor neuron cellular biology. Her team has done extensive work on a small molecule, known as AKV9 (NU-9), which is showing great promise in the treatment of upper motor neuron diseases. The compound is thought to improve the structural stability of the cell through the endoplasmic reticulum. Because upper motor neurons display early degeneration in patients with the SPG4 mutation, it is of great interest to find out whether NU-9 could also improve the health of upper motor neurons that become diseased due to SPG4.
In Vitro Studies
March 2022 - In vitro studies of AKV9 for SPG4 have shown promising preliminary results; Project advances to SPG4 mouse model in vivo studies.
In Vivo Studies
August 2023 - testing in SPG4 mouse models continue as more mice are added to the project. Research team is focused on completing imaging, quantification, and cellular assessment.
Human Clinical Trials
June 2022 - AKAVA Therapeutics licensed NU-9 from Northwestern and renamed it AKV9
August 2023 - AKV9 receives FDA approval for Phase 1 human clinical trials in healthy patients.
ALS patient trials will follow.
Preclinical studies are ongoing for SPG4 with the goal of being included in future phases of the AKV9 human clinical trials for ALS.
Cure SPG4 Foundation is proud to partner with the Ozdinler Lab at Northwestern University, Feinberg School of Medicine, to advance targeted research for SPG4 Hereditary Spastic Paraplegia.