Kennedy disease or, Spinal and Bulbar Muscular Atrophy (SBMA) is a form of Spinal Muscular Atrophy (SMA). SBMA causes muscle weakness throughout the limbs which results in impaired mobility (typical of SMA) but it also causes muscle weakness in the head and neck (the bulbar region). This causes SBMA patients to have trouble speaking and swallowing.
A recent study has initiated research into a potential therapy which could improve swallowing for SBMA patients
The Study
This study analyzed the safety and efficacy of leuprorelin in SBMA. It evaluated two different clinical trials of the therapy, both structured identically. They were titled JASMITT-06DB and JASMITT-11DB. In total, these trials included 283 patients who were all randomized to receive placebo (141) or leuprorelin (142). The trials both lasted 48 weeks with the treatment or placebo being given every 12 weeks throughout that time period.
The results from this study were published in the Journal of Neurology.
Safety Results
While leuprorelin was generally well-tolerated, some of the side effects were more severe in patients on the treatment than those who were taking placebo. That said, the incidence of side effects was almost equal between the two (81.7% and 80.1% respectively). Drug related side effects were also similar at 62.7% and 53.9% respectively. Side effects included weight gain, excessive sweating, decreased libido, skin reactions at injection site, and abnormal liver function.
Efficacy Results
To determine efficacy, researchers primarily investigated barium residue in the patient’s throats as they tried to swallow. Secondarily, they kept track of testosterone levels and patient’s quality of life as well as other characteristics.
Unfortunately, the primary objective was not met. At first, researchers evaluating the barium residue believed leuprorelin may be effective as there were differences between the treatment group and the placebo group after initial swallowing. However, from the initial analysis to the last patient visit, there were no significant differences between groups. On average, both groups of patients experienced a 4.12% decrease in barium after initial swallowing.
In Conclusion
This trial was able to conclude the overall safety of leuprorelin but researchers indicate more investigation is needed to examine its efficacy in SBMA. Still, there is hope for leuprorelin as a potential therapy for this condition.
You can read more about this study and leuprorelin as a potential therapy for SBMA here
