Qalsody is the fourth drug approved by the FDA for the treatment of ALS and the first drug for the treatment of hereditary ALS. How should it be used?
How should Qalsody be used?
1. Recommended dose
The recommended dose is 100 mg per dose for intrathecal administration via lumbar puncture, with 3 loading doses every 14 days and a maintenance dose every 28 days thereafter.
2. Missed dose
(1) If you miss the second loading dose, take it as soon as possible and take the third loading dose 14 days later.
(2) If you miss the third loading dose, take it as soon as possible and take the next dose 28 days later.
3. Pre-administration instructions
(1) After pouring the solution into the syringe, it should be administered immediately at room temperature (within 4 hours after taking it out of the vial), otherwise any unused contents in the single-dose vial should be discarded.
(2) Before administration, use a lumbar puncture needle to obtain approximately 10 mL of cerebrospinal fluid, and use a needle to take out 15 mL of the required dose from the medicine bottle. Do not dilute. Use a lumbar puncture needle to administer the drug by intrathecal push injection for 1 to 3 minutes.
Therapeutic effect of Qalsody
In a 28-week clinical trial for patients with SOD1 mutation ALS, Qalsody significantly reduced the concentration of SOD1 protein in cerebrospinal fluid and neurofilament light chain (neural damage marker) in plasma, indicating that it effectively acts on the biological mechanism of the disease.
The primary clinical endpoint (ALSFRS-R functional score change) did not reach statistically significant differences in the subgroup with faster disease progression (treatment group -6.98 vs placebo group -8.14) and other secondary clinical endpoints. The researchers believe that neurofilament light chain levels can be used as a potential alternative marker for evaluating the efficacy of drugs for neurodegenerative diseases.
Qalsody needs to be administered by intrathecal injection, which can significantly reduce the levels of pathogenic SOD1 protein and neural damage markers, indicating its biological activity. Although the improvement in main functional scores did not reach a significant difference, continued treatment may delay functional decline and provide a targeted treatment option for SOD1-ALS patients.
