Efficacy

Lunasin and Amyotrophic Lateral Sclerosis (ALS)

Lunasin is a peptide that has been studied for its potential health benefits, including its role in the treatment of Amyotrophic Lateral Sclerosis (ALS). ALS, also known as Lou Gehrig's disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. While research on lunasin is still in the early stages, some preclinical studies have suggested that it may have neuroprotective effects that could be beneficial for patients with ALS. However, clinical data on the efficacy of lunasin for ALS is limited, and more research is needed to establish its potential therapeutic role in this condition.

Qalsody (Tofersen) and Amyotrophic Lateral Sclerosis (ALS)

Qalsody (tofersen) is an investigational drug specifically designed for the treatment of ALS caused by mutations in the superoxide dismutase 1 (SOD1) gene, which is one of the genetic causes of the disease. Tofersen is an antisense oligonucleotide that targets the SOD1 mRNA, aiming to reduce the production of the abnormal SOD1 protein that is toxic to motor neurons. The efficacy of Qalsody in slowing the progression of ALS has been evaluated in clinical trials.

In a Phase 1/2 clinical trial, tofersen demonstrated a reduction in SOD1 protein levels in the cerebrospinal fluid of patients with SOD1-ALS. Following these results, the Phase 3 clinical trial, known as VALOR, was conducted to further assess the efficacy and safety of tofersen. The trial aimed to evaluate whether tofersen could slow down the decline in motor function and improve survival rates in patients with SOD1-ALS.

While the results from the VALOR trial have shown promise, the full extent of Qalsody's efficacy in treating ALS remains under investigation. The U.S. Food and Drug Administration (FDA) has not yet approved tofersen for the treatment of ALS, and its use is currently limited to clinical trials. Patients with ALS who are interested in participating in clinical trials for tofersen or other investigational drugs should consult with their healthcare providers for more information and eligibility criteria.