Salbutamol is a medication authorized in France for asthma or chronic obstructive pulmonary disease (COPD). Research has shown that this molecule also has beneficial effects in several pathologies in which the neuromuscular junction (NMJ) is disrupted, as is the case with collagenopathies. Researchers have evaluated for the first time the effects of salbutamol in this group of diseases using mouse models carrying mutations in the COL6A1 gene, notably involved in congenital muscular dystrophy (CMD) of the Ullrich type and Bethlem myopathy.
A restructured JNM, stronger muscles
While untreated diseased mice exhibit fragmented NMJs and impaired nerve signaling, mice receiving daily salbutamol show restoration of NMJ structure and function.
The treatment also allows animals to regain muscle mass, in particular through better muscle regeneration, and a significant increase in their strength.
However, after one month on salbutamol, the treated animals show physiological changes indicative of potential desensitization to the molecule, and therefore of a drop in response to treatment.
As in previous research in SMA, this work confirms the interest in drug repositioning of salbutamol in neuromuscular diseases. The safety of this molecule, already proven in humans, would make it possible to accelerate possible therapeutic developments.
Source
Salbutamol repurposing ameliorates neuromuscular junction defects and muscle atrophy in Col6a1-/- mouse model of collagen VI-related myopathies.
Calabrò, S., Nogara, L., Jian, Y. et al.
Clin Transl Med 2024 14(7): e1688.
