NEURONATA-R inj. (autologous BM-MSC)
1 Autologous BM-MSC (mainingredient) in Pre-filled syringe (4mL) 4 x 107ea
Autologous cerebrospinal fluid (suspending agent) ………… 4mL
(Dosage form) injection type
(Description) cell suspension with ivory color in a colorless transparent syringe
Amyotrophic lateral sclerosis (ALS: Lou Gehrig's Disease)
Releasing progress of the Amyotrophic lateral sclerosis; ALS (Lou Gehrig’s disease) by using in combination with Riluzole
Inject 0.1mL per 1kg of a patient's weight (1.0 × 106 cells) into cerebrospinal Fluid of an ALS patient who has taking Riluzole (100mg / day, orally)
-Phase I/II completed
-Phase III ongoing
-Route of Administration : Intrathecal Injection
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
4 months: 1.10, SE 0.35, 95% CI 0.40 to 1.79, p = 0.003
6 months: 0.92, SE 0.38, 95% CI 0.16 to 1.67, p=0.018).
The results revealed a markedly restrained disease progression in the MSC group that was maintained throughout 6 months.
The mechanism of lenzumestrocel on ALS is considered as 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.