Safety and Dosimetry of a New Radiotracer to Detect Misfolded SOD1 Associated With Amyotrophic Lateral Sclerosis

Summary

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a rare neurodegenerative disease resulting in loss, primarily, of the motor neurons in the motor cortex, brainstem and spinal cord. It currently affects 3 of every 100,000 people in the US. Currently, there is no diagnostic tool for ALS, resulting in misdiagnosis and significant disease progression before formal diagnosis. An imaging test for early detection of ALS and for monitoring disease progression would have significant diagnostic and prognostic value. PET imaging with an appropriate radiotracer has great potential as a biomarker for ALS given that it would permit visualization of central nervous system (CNS) pathology in individuals living with the disease. To that extent, the primary goal of this phase I study is evaluating the safety and biodistribution of the new tracer \[89Zr\]Zr-DFO-AP-101 in healthy volunteers and ALS patients.

Timeline

Start

2023-11-23

Primary completion

2024-12-30

Completion

2026-03-31

Publications

• Croteau E, Rousseau E, Tremblay S, Rousseau JF, Espinosa-Betancourt E, Lavallee E, Dubreuil S, Ait-Mohand S, Lareau-Trudel E, Gosselin S, Carrier V, Cote-Bigras S, Allard C, Maier M, Salzmann M, Tetu A, Houde MP, Turcotte E, Guerin B. A phase I study to evaluate the dosimetry and safety of [89Zr]Zr-DFO-AP-101, a new antibody-based radiopharmaceutical to detect misfolded SOD1 in amyotrophic lateral sclerosis. Eur J Nucl Med Mol Imaging. 2026 Jun;53(7):4639-4651. doi: 10.1007/s00259-026-07853-y. Epub 2026 Mar 24.

Drugs

Indications