Sailxen Bio's Autologous Polyclonal Regulatory T-cell Therapy Receives IND Acceptance from CDE for ALS Treatment

▲ Image Source: CDE Official Website

Amyotrophic Lateral Sclerosis, abbreviated as ALS and commonly known as Lou Gehrig's disease, is a rare and fatal neurodegenerative disorder. Most clinical manifestations include difficulties in speaking, swallowing, and degenerative conditions of limb muscles, posing a severe threat to patients' quality of life. As of 2015, statistics showed that 1.68 people per 100,000 are diagnosed annually worldwide, with over 220,000 ALS patients globally. It is projected that the global prevalence will increase by 69% by 2040. The high-risk age group for this disease is 60-79 years, with a significantly higher incidence in men than in women. The fatal aspects of this disease lie in two areas: on one hand, the survival period for patients is typically 2-5 years, with approximately 50% surviving less than three years, resulting in an extremely high overall mortality rate; on the other hand, the time of diagnosis is unpredictable, and many patients are already in the middle or late stages when diagnosed, making treatment decisions challenging.

The number of drugs currently reported or already on the market for the treatment of ALS is very limited, including Riluzole, Edaravone, Quinidine Sulfate/Sodium Phenylbutyrate, Tofersen, etc. These drugs mainly alleviate a specific pathophysiological symptom by inhibiting glutamate release (preventing neuronal overexcitation) or scavenging reactive oxygen species (preventing mitochondrial dysfunction). For most patients, these treatments cannot achieve multifaceted efficacy. Based on the unmet clinical needs of ALS, NOVABIOTX has developed human autologous polyclonal regulatory cells, hoping to delay the progression of ALS and improve the pathological state of ALS patients.

Studies have reported an imbalance between Teff and Treg cells in ALS patients, with excessive Teff promoting microglial activation, causing damage to astrocytes, and ultimately leading to demyelination and further neuronal injury. Changes in the activity and quantity of Teff cells show a certain positive correlation with disease status. NOVABIOTX alleviates neuronal damage, improves muscle function, and slows disease progression by intrathecally injecting Treg cells into patients, which suppresses Teff cells while recruiting oligodendrocytes.

Currently, the product is undergoing IIT research at the First Affiliated Hospital of Zhengzhou University, and the first ALS patient has completed two doses. According to feedback from the medical staff, the patient’s condition is currently stable, with a trend of slowed disease progression. We hope that more clinical data on the efficacy and safety of ALS treatment will be available in the future, bringing good news to patients with ALS.