A plant-derived compound called forskolin may be used to treat Duchenne muscular dystrophy (DMD) in the future, but only after more research is done to maximize its benefits and minimize its risks, according to a recent study published in the journal Skeletal Muscle.
In an earlier study using rats, forskolin improved muscle repair and movement in the short term. But it was unclear whether forskolin treatment is safe and effective when used for longer periods.
To explore this, researchers tested forskolin in a rat model of DMD over several months. A rat model of DMD carries genetic changes similar to those causing DMD in humans and develops muscle problems much like people with DMD do.
Short-term treatment with forskolin helped reduce scarring and improved muscle tissue in both injured and uninjured muscles. It also lowered harmful inflammation and increased the number and activity of muscle stem cells, which are crucial for muscle repair and regeneration.
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However, when forskolin was given over a longer period, its benefits were less clear. While there was an increase in muscle mass and grip strength, muscle force and fatigue didn’t improve significantly. Forskolin also had no effect on the diaphragm, which is the main muscle used for breathing.
More worryingly, treatment with forskolin had negative effects on heart muscle tissue, raising safety concerns for potential long-term use in patients.
“While these findings underscore the therapeutic potential of FSK [forskolin], they also reveal its limitations in long-term efficacy, particularly regarding muscle function and potential adverse effects on cardiac tissue,” the study’s authors said.
They emphasized the need for more research before forskolin can be considered a safe and effective treatment for DMD.
“This study represents a crucial step toward understanding the long-term benefits and potential limitations of FSK treatment, including the necessary optimization of dosing regimens to avoid adverse effects, which is essential for advancing FSK as a viable therapeutic strategy for DMD to the clinic,” they said.
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