This breakthrough clinical research milestone has not only garnered significant attention from the academic community both in China and internationally, but recently, many ALS patients from overseas have written letters seeking help, hoping to receive the domestically pioneered intrathecal Treg cell therapy. This innovative treatment brings an unprecedented ray of hope for hundreds of thousands of ALS patients worldwide.

▲ Dr. Lü Bo Delivers a Special Report at the IGC 2025 Conference

Significant Improvement in Patient Function: From Survival Struggles to a New Lease on Life. Maintaining objectivity in clinical research is crucial; all patients were recorded during treatment and follow-up visits.

In the special report "Research and Translational Advances in Immunocyte Therapy for Neurodegenerative and Autoimmune Diseases," Dr. Mingqi Lü systematically explained the scientific mechanisms of Treg cell therapy and the global R&D landscape, while also disclosing for the first time the interim results of NOVABIOTX's NP001 project:

1. Multidimensional Validation of Clinical Efficacy(1) Significant functional recovery: After treatment, the ALSFRS-R scale scores and grip strength significantly improved from baseline and remained stable (the scoring curve outperformed traditional drugs), and patients' quality of life markedly improved; (2) Breakthrough in slowing disease progression: The rate of lung function (FVC) deterioration decreased, providing crucial evidence for extending survival; (3) Safety confirmed: No immune rejection or serious adverse events occurred throughout the process, verifying the therapy's reliability and tolerability.

2. Animal Experiments Lay the Foundation: In the early studies, two motor function experiments in mice both suggested that Treg administration had a trend of improving motor function in SOD1 mice. After discontinuation of the treatment, motor function declined due to disease progression, confirming the scientific approach that continuous intervention is required for the therapy.

3. New Discoveries in Personalized TreatmentDr. Mingqi Lü summarized the findings of the research and development team: the correlation between Treg expansion and patient condition—the lower the ALSFRS-R score, the greater the difficulty in Treg cell expansion—providing a basis for personalized treatment and offering data anchor points for customized solutions for critically ill patients.

The follow-up video results of the first 52-year-old male patient who received Treg cell therapy (with a significant decline in ALSFRS-R score before treatment, gradually developing mobility issues, limb weakness, and other symptoms) were presented on-site, visually demonstrating the therapeutic efficacy through data:(1) Seven days after treatment, the patient's finger flexibility improved, and the frequency of spasms decreased; (2) Four months after treatment, the patient recovered from being unable to lift their head independently to being able to independently complete daily tasks such as eating and climbing stairs, demonstrating significant potential for neurological function recovery. Currently, the first patient has entered the 56th day after the final injection, with all indicators remaining stable, and the ALSFRS score has increased. The subsequent second and third patients also exhibited the same signs.

Innovative Therapies Take the Lead Globally: China's Solutions Break Through Medical Challenges

Amyotrophic Lateral Sclerosis (ALS), a neurodegenerative disease with a 100% fatality rate, can only be managed by traditional drugs to extend survival by 2-3 months. However, the intrathecal injection Treg cell therapy jointly developed by NOVABIOTX and the First Affiliated Hospital of Zhengzhou University has achieved a revolutionary leap from "slowing disease progression" to "functional repair" by modulating the neuroimmune microenvironment.

Currently, the NP001 projectIITClinical trials are proceeding steadily, with five patients successfully enrolled. The first patient has completed the entire treatment and remains in stable condition; the other patients' treatments are proceeding smoothly. NOVABIOTX is actively collaborating with the First Affiliated Hospital of Zhengzhou University to optimize efficacy evaluations and assessments of long-term survival benefits for patients, accelerating the implementation of this therapy to benefit patients worldwide. Dr. Mingqi Lü stated, “Treg cell therapy has pioneered a new paradigm for treating neurodegenerative diseases. NOVABIOTX will continue to advance clinical research and promote this 'Chinese solution' as the gold standard for ALS treatment globally.” One important issue currently under deep consideration is how to enable innovative outcomes born from Chinese efforts and ingenuity to more quickly benefit ALS patients around the world.

Regarding ALS and NP001-NDD02 IIT Clinical Research

Amyotrophic Lateral Sclerosis(ALS), commonly known as Lou Gehrig's disease, is a rare and fatal neurodegenerative disease with a globally increasing prevalence. Patients often succumb to respiratory failure within 2-5 years after diagnosis and there is a lack of effective treatment options. The NP001-NDD02 IIT clinical study (NCT06671236) conducted by NOVABIOTX in collaboration with the First Affiliated Hospital of Zhengzhou University aims to explore the safety and efficacy of intrathecal Treg cell injections in the treatment of neurodegenerative diseases, providing a Chinese solution to this medical challenge.