Meclozine
What is Meclozine and what does it do?
Meclozine is a drug that has been on the market for 50 years as an over-the-counter anti-emetic (reduces nausea and vomiting) which has been shown to be effective in reducing some the effects of achondroplasia (explained below in more detail).
Although the exact mechanism of action this drug exerts in the chondrocytes is unknown, it has been shown by Prof. Matsushita, from Nagoya University, to increase the length of the radius, femur, ulna, tibia and vertebrae of mice, which suggests that it may have effects on both stature, proportionality and the spine length (and maybe avoid spinal cord compression) [1].
It has also been shown to postpone the closure of the epiphyseal growth plates in the occipital bone of mouse embryos when administered to pregnant mice, even though it showed no effect in the formation of bony bridges (ossification where there shouldn't be any) [2]. This means that it may have some effect in preventing foramen magnum stenosis, since the foramen magnum is located in the occipital bone.
Credits: Biopharm Pharmaceutical Industry. |
It has also been shown to postpone the closure of the epiphyseal growth plates in the occipital bone of mouse embryos when administered to pregnant mice, even though it showed no effect in the formation of bony bridges (ossification where there shouldn't be any) [2]. This means that it may have some effect in preventing foramen magnum stenosis, since the foramen magnum is located in the occipital bone.
What stage of development is it in?
Phase 1 clinical trial are to start in the next few months, but since this drug is already on the market and approved for adults, it will include children. The trial is going to be conducted at the Nagoya University, starting with one dose per day to assess toxicity in children.
Sources:
- Matsushita, M., et al., Meclozine promotes longitudinal skeletal growth in transgenic mice with achondroplasia carrying a gain-of-function mutation in the FGFR3 gene. Endocrinology, 2015. 156(2): p. 548-54.
- Matsushita, M., et al., Maternal administration of meclozine for the treatment of foramen magnum stenosis in transgenic mice with achondroplasia. Journal of Neurosurgery: Pediatrics, 2017. 19(1): p. 91-95.