The "Ice Bucket Challenge" once brought the rare disease of ALS into the public eye, and Cai Lei used his personal experience and unremitting efforts to let more people understand ALS and promote the whole society's attention to this disease. It is this indomitable spirit that inspires the scientific research team to continue exploring, and recently this rare disease treatment field has finally ushered in good news.

Recently, Shize Bio announced that its independently developed universal iPSC-derived subtype neural precursor cell injection product XS-228 injection has been officially approved by the US FDA to conduct registered clinical trials for the treatment of ALS (amyotrophic lateral sclerosis, ALS).

This breakthrough not only marks China's innovative breakthrough in the field of cell therapy, but also brings new hope to ALS patients.

one of the "four major

incurable diseases in the world", ALS is one of the "four major

incurable diseases in the world". Its complexity and harm cannot be ignored. ALS (amyotrophic lateral sclerosis, ALS) is a rare, progressive neurodegenerative disease that mainly affects motor neurons in the brain and spinal cord. These neurons are responsible for controlling muscle movement. When they gradually degenerate and die, patients will experience symptoms such as muscle weakness, atrophy, and stiffness, which will eventually lead to the loss of movement and even inability to swallow, speak, and breathe. Patients will feel as if they are gradually "frozen", so it is also called "ALS".

At present, the cause of ALS is not completely clear, but studies have shown that some patients have a family genetic background. The most common pathogenic genes are C9orf72 gene and copper/zinc superoxide dismutase (SOD1) gene. In addition, age, long-term exposure to toxic substances, smoking and high-intensity exercise may also be related to the onset of the disease.

In 2014, the "Ice Bucket Challenge" event brought ALS into the public eye and increased social attention to this rare disease. In addition, public figures like Cai Lei have inspired countless patients and researchers with their tenacious vitality and spirit.

In recent years, cell therapy, as an emerging treatment method, has brought new hope for the treatment of ALS. The core idea of cell therapy is to repair or replace damaged nerve cells by transplanting healthy cells. The iPSC-derived cell therapy program of Shize Bio is based on this concept. It differentiates induced pluripotent stem cells into specific neural progenitor cells and then transplants them into the patient's body to achieve the repair of the damaged nervous system.

From a technical point of view, Shize Bio's iPSC-derived subtype neural progenitor cell injection (XS-228 injection) uses advanced cell reprogramming and differentiation technology. By reprogramming the patient's own somatic cells into pluripotent stem cells and then differentiating them into specific neural progenitor cells, the therapy is expected to achieve the repair and regeneration of the nervous system of patients with ALS. In addition, this "off-the-shelf" cell therapy program also has the advantages of strong scalability and high cost-effectiveness.

It is worth mentioning that although it has not yet been launched, Shize Bio's XS-228 injection has won 1 "China's first" and 2 "world's firsts": China's first iPSC-derived cell drug certified by the US FDA and granted global orphan drug status, the world's first and so far the only iPSC-derived cell drug with orphan drug status for the treatment of ALS, and the world's first and so far the only regenerative neural cell therapy product for the treatment of ALS officially approved for registration clinical trials in the United States.

The FDA's approval of its clinical trials will provide further verification of the safety and effectiveness of the therapy. If the trial is successful, this will be a major breakthrough in the treatment of ALS and is expected to become an important means of treating ALS.

The emergence of innovative therapies

is gratifying that XS-228 injection is not the only "freeze" breaker. In recent years, a number of innovative therapies have emerged in the treatment of ALS, bringing new hope to this disease that lacks effective treatment methods.

These innovative therapies cover multiple directions such as ribonucleic acid (RNA) therapy and mesenchymal stem cell (MS) therapy.

RNA therapy

Tofersen is an antisense oligonucleotide (ASO) drug that inhibits the production of mutant proteins by targeting the mRNA of the pathogenic gene SOD1. Some ALS patients carry SOD1 gene mutations, which lead to the accumulation of toxic proteins and accelerate the death of motor neurons. Tofersen acts directly on the central nervous system through intrathecal injection, blocking the expression of pathogenic genes and delaying the progression of the disease from the root.

Tofersen's Phase III clinical trial showed that for patients with SOD1 mutations, it can significantly reduce the level of neurofilament light chain (NfL, a marker of neural damage) in cerebrospinal fluid and delay the decline of motor function. In 2024, Biogen announced that Tofersen injection was approved for marketing in China, becoming the first precision therapy for hereditary ALS.

Mesenchymal stem cell (MSC) therapy

Mesenchymal stem cell (MSC) therapy is an emerging cell therapy technology with broad clinical application prospects. MSCs are derived from a variety of tissues, such as bone marrow, umbilical cord, adipose tissue, etc., and have the ability of self-renewal, multidirectional differentiation and immune regulation. In recent years, MSC therapy has shown significant potential in the treatment of various diseases, especially in the field of amyotrophic lateral sclerosis (ALS).

In 2021, the article "A Randomized Placebo-Controlled Phase 3 Study on Inducing Mesenchymal Stem Cells to Secrete High Levels of Neurotrophic Factors for the Treatment of Amyotrophic Lateral Sclerosis" published in the international journal "Muscle&Nerve" showed that in the Phase 3 clinical study, MSC-NTF cell therapy was well tolerated and no safety issues occurred. Compared with placebo, MSC-NTF participants with milder disease retained more function at 28 weeks, and biomarkers in cerebrospinal fluid were significantly improved. This study provides important evidence for cell therapy for ALS, but larger-scale trials are still needed to confirm its efficacy.

The FDA approval of the universal cell therapy program of Shize Biotechnology marks an important step in the treatment of ALS. This innovative therapy not only brings new hope to patients with ALS, but also provides a new direction for the development of cell therapy technology. With the advancement of various innovative therapies for the treatment of ALS, we look forward to bringing substantial benefits to ALS patients as soon as possible.

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