We developed the world’s first stem cell therapeutic product for ALS (Amyotrophic Lateral Sclerosis) name of NEURONATA-R®, received the permission of medicines by KFDS in July 2014 and began commercialized medication to patients in February 2015, for the fifth in the world.

NEURONATA-R® inj. (Autologous BM-MSC) is an autologous bone marrow mesenchymal stem cell (autologous BM-MSC) therapy that acts a neuroprotective effect and relieves progression of a disease through prevention of motor nerve cell, survival extension of motor neurons, releasing nerves inflammatory and immune regulation function.

The mechanism of lenzumestrocel on ALS is considered as an expression of anti-inflammatory factors, neuroprotective factors and immunoregulation. When lenzumestrocel was tested in the mouse ALS model, gene expression of Foxp3 was increased, which is the differentiation-inducing transcription factor of Tregs. In addition to the animal model, in an experiment using peripheral blood cells from healthy persons and ALS patients treated with lenzumestrocel, an increase in Tregs (CD4+, CD25+) was confirmed, along with increased expression of Foxp3 and IL-10. Based on these findings, lenzumestrocel is considered to show recovery effects on the disease by inhibiting the immune response-initiated apoptosis of neurons through secretion of anti-inflammatory factors that in turn induce an increase in the differentiation of Tregs.

As one of efficacy outcomes, the change in the ALSFRS-R® score (monthly decline rate) between the lead-in and follow-up period was significantly reduced in the MSC group in comparison to the control group.

Our most advanced pipeline, NEURONATA-R®, is at the stage of insurance approval of Korea to get a reasonable insurance drug price in Korea for enhancing brand awareness in the global market and others 3 pipelines such as CS20AT04 (Lupus), CS10BR05 (MSA) are at the stage of phase I.