CORESTEM launched the world’s first stem cell therapy for amyotrophic lateral sclerosis in South Korea in 2015. The company is now seeking out-licensing partners as it works toward product approvals in the US and Europe.

CORESTEM is a biotechnology company specializing in the research and development of personalized stem cell therapies for neurological and autoimmune diseases. Its lead product is NeuroNata-R (lenzumestrocel), the world’s first stem cell-based therapy for amyotrophic lateral sclerosis (ALS).

NeuroNata-R was approved as an orphan drug for the treatment of ALS by the Ministry of Food and Drug Safety (MFDS) in South Korea in 2014. “Back then there were no ground rules laid out by the MFDS for stem cell therapy, so CORESTEM has been paving the way in this regard, and the path we took was adopted by the MFDS as regulatory guidelines,” said KyungSuk Kim, CEO of CORESTEM.

ALS, also known as Lou Gehrig’s disease or motor neuron disease, is a progressive neurodegenerative disease that leads to muscle atrophy due to the death of motor neurons. The average life expectancy is three to five years after the onset of disease. The only approved treatments for ALS outside South Korea are the small-molecule drugs riluzole and edaravone.

NeuroNata-R is based on autologous bone marrow-derived mesenchymal stem cells (MSCs). Treatment involves extracting bone marrow from the patient, isolating and culturing MSCs, mixing with cerebro-spinal fluid collected from the patient and administering the final product by intrathecal injection. The first NeuroNata-R injection typically takes place four weeks after the first bone marrow extraction, followed by a second injection four weeks later.

Treatment with NeuroNata-R results in various effects that prevent motor neuron death and slow the progression of ALS. These include the release of immune-modulating factors, such as regulatory T lymphocytes and T helper 2 factors; a motor neuron protective effect through the expression of growth factors, such as brain-derived neurotrophic factor; and an anti-inflammatory effect due to microglial cells switching from an M1 to an M2 phenotype.