Effect of Cannabinoids on Pain in Fabry Disease Patients

Summary

Fabry Disease (FD) is a rare lysosomal storage disorder due to the absence or deficiency of hydrolase α-galactosidase A (α-Gal A) activity in lysosomes. This dysfunction results in progressive accumulation of glycosphingolipids in a wide variety of cells, resulting in major organ system damage. Patients with Fabry disease can suffer from neuropathic pain, since lysosomal accumulation affects small unmyelinated nerve fibers. Neuropathic pain is one of the prominent and debilitating symptoms significantly interfering with life quality in FD patients. Current treatment of Fabry patients with neuropathic pain is deficient, as they respond poorly to a conventional pain therapy, often require a high-dose opioids treatment and presentation to the Emergency Department. Sativex® has been shown to be a successful treatment option in neuropathic pain of different origin with minimal neuropsychological influence: in multiple sclerosis (MS), chemotherapy-induced neuropathic pain and other. It contains Δ-9-Tetrahydrocannabinol (THC) and Cannabidiol (CBD) and has recently been licensed in Switzerland for treatment of neuropathic chronic pain in MS. Sativex® is an oral spray.

Timeline

Start

2022-07-07

Primary completion

2023-12-29

Completion

2023-12-29

Publications

• Results Jensen TS, Baron R. Translation of symptoms and signs into mechanisms in neuropathic pain. Pain. 2003 Mar;102(1-2):1-8. doi: 10.1016/s0304-3959(03)00006-x. No abstract available.
• Results Rolke R, Baron R, Maier C, Tolle TR, Treede -DR, Beyer A, Binder A, Birbaumer N, Birklein F, Botefur IC, Braune S, Flor H, Huge V, Klug R, Landwehrmeyer GB, Magerl W, Maihofner C, Rolko C, Schaub C, Scherens A, Sprenger T, Valet M, Wasserka B. Quantitative sensory testing in the German Research Network on Neuropathic Pain (DFNS): standardized protocol and reference values. Pain. 2006 Aug;123(3):231-243. doi: 10.1016/j.pain.2006.01.041. Epub 2006 May 11.
• Results Serpell M, Ratcliffe S, Hovorka J, Schofield M, Taylor L, Lauder H, Ehler E. A double-blind, randomized, placebo-controlled, parallel group study of THC/CBD spray in peripheral neuropathic pain treatment. Eur J Pain. 2014 Aug;18(7):999-1012. doi: 10.1002/j.1532-2149.2013.00445.x. Epub 2014 Jan 13.

Drugs

Indications