Efficacy

Efficacy of Ketas (Ibudilast) in ALS

Ketas, known generically as ibudilast, is a phosphodiesterase inhibitor that has been primarily used in the treatment of asthma and post-stroke complications, as well as in certain cases of multiple sclerosis in Japan. Its efficacy in Amyotrophic Lateral Sclerosis (ALS) has been a subject of interest due to its neuroprotective and anti-inflammatory properties. While ibudilast has not been approved specifically for the treatment of ALS, some clinical trials have suggested that it may slow the progression of the disease. However, the data on its efficacy is still limited, and more extensive clinical trials are needed to fully understand its potential benefits for ALS patients.

In a phase 2 trial (the SPRINT-ALS study), ibudilast was evaluated for its safety and tolerability in patients with ALS. While the primary focus was on safety, some efficacy signals were noted. Patients treated with ibudilast showed a slower rate of progression on the ALS Functional Rating Scale-Revised (ALSFRS-R), a measure of functional decline in ALS. Despite these promising results, the trial was not designed to conclusively determine efficacy, and further research is necessary to confirm these findings.

Efficacy of Qalsody (Tofersen) in ALS

Qalsody, also known as tofersen, is an investigational antisense oligonucleotide designed to reduce the production of superoxide dismutase 1 (SOD1), a protein with mutations known to cause a familial form of ALS. Tofersen aims to slow the progression of ALS by targeting the underlying genetic cause in SOD1-ALS patients. In clinical trials, tofersen has shown promise in reducing SOD1 protein levels, which is hypothesized to potentially slow the disease's progression.

The most significant data on tofersen's efficacy comes from a phase 1/2 and phase 3 clinical trials. In these studies, patients with SOD1-ALS who received tofersen experienced a slower decline in the ALSFRS-R score compared to those who received a placebo. Additionally, secondary outcomes suggested improvements in muscle strength and respiratory function. However, the results also indicated that the treatment might be more effective if started earlier in the disease course. While these findings are encouraging, tofersen is still under investigation, and further studies are required to confirm its long-term efficacy and safety in a broader ALS population.