Bio pharmaceutical Business Unit



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

  • Overview of NEURONATA-R

    Neuronata-R inj : Neuro “Nerve” + Nata “Birth”

  • Type

    Orphan drug

  • Approval

    2014. 07. 30 (MFDS)

  • Generic Name


  • Dosage

    1.0 x 106 cells/ kg of body weight concomitant with riluzole

  • Administration

    Intrathecal administration, two injections with 4 weeks interval

  • Effect & Efficacy

    Delaying of ALS progression

  • Characteristics

    White suspension fluid filled inside a transparent colorless plastic syringe

  • Amount

    4.0 x 107 cells /4mL, pre-filled syringe

  • Storage

    Cold storage (2~8℃)

  • Expiration Date

    48 hours after manufacturing

Introduction to Clinical Results

NeuroNata-R is an autologous bone marrow-derived stem cell therapy for the treatment of amyotrophic lateral sclerosis (ALS).

NeuroNata-R secretes various growth factors, immune modulators, and anti-inflammatory factors, which contribute to the alleviation of neuroinflammation,
prevention of motor neuron cell death, extension of survival, and neuroprotective effects.
As a result, it helps alleviate the progression rate of amyotrophic lateral sclerosis (ALS) as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R*).


Change of ALSFRS-R Score after NEURONATA-R® Treatment (Phase II)

  • Data analysis was conducted on a total of 59 ALS patients (MSCs group: 32 patients, CONTROL group: 27 patients) in a phase 2 clinical trial.
  • Four months after the procedure, there was a significant difference in the ALSFRS-R score between the MSCs and CONTROL groups, with a difference of 2.98 points (MSCs group score change: -1.69±2.51, CONTROL group score change: -4.67±3.25, P < 0.001). Additionally, at six months after the procedure, there was a difference of 3.38 points in the ALSFRS-R score (MSCs group score change: -3.10±3.51, CONTROL group score change: -6.48±4.53, P=0.003).
  • ALSFRS-R : Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised
  • MSCs : Mesenchymal Stem Cells


NEURONATA-R® derived from mesenchymal stem cell

in bone marrow produces anti-inflammatory cytokines dominant for neuroprotective effects on motor neurons.


  • Treatment with MSCs increases population ratio of Th2 and Treg cells by producing anti-inflammatory factors such as IL-4, IL-10 and TGF-β.
  • Treatment with MSCs increases M2 phenotype by suppressing TNF-α and IL-1β.
  • Treatment with MSCs has paracrine effects by secreting neurotropic factors such as BDNF, VEGF and IGF which have protective effects on motor neurons.
  • The mechanism of NeuroNata-R therapy in ALS is 1) T-regulatory cell induction, 2) Th2 cell activation, 3) Increased conversion of M2 population, 4) Increased anti-inflammatory cytokines, 5) Released neurotropic factor including VEGF, NGF, BNDF
  • Effectiveness of bone marrow stem cell was mediated by immune-inflammation modulation by elevating the T-regulatory cell population and converting microglial cells from M1 to M2 phenotype
  • Repeated intrathecal injections of autologous MSC was safe and effective for delaying the progression speed of ALS
  • Ref 1.Phase I trial of repeated intrathecal autologous bone marrow-derived Mes enchymal stromal cells in
    amyotrophic lateral sclerosis. Stem Cell Tansl Med, 2015 Jun;4(6):590-7
  • Ref 2.Endogenous regulatory T lymphocytes ameliorate amyotrophic lateral sclerosis in mice and correlate with disease
    progression in patients with amyotrophic lateral sclerosis. Brain 2011:134;1293-1314
  • Ref 3.Repeated intrathecal mesenchymal stem cells for amyotrophic lateral sclerosis. Annals of Neurology, 26 July 2018;
    Online published

Treatment Method

Neuronata-R consists of ingredients that are pharmaceuticals derived from
autologous bone marrow mesenchymal stem cells and autologous cerebrospinal fluid

To produce the pharmaceutical, collection of the patient's bone marrow and cerebrospinal fluid is necessary.
Neuronata-R is a pharmaceutical treatment accompanied by a spinal procedure for administration.

Category Content
Name of Product NEURONATA-R®
Amount 4.0×107cells / 4mL, pre-filled syringe
Characteristics White suspension fluid filled inside a transparent colorless plastic syringe.
Effect & Efficacy Delaying of ALS progression
Dosage 1.0 x 106 cells/ kg of body weight concomitant with riluzole
Approval 2014. 07. 30 (MFDS)
Specialized/General Specialty medicine, Orphan drugs
Developer/Manufacturer CORESTEMCHEMON Inc.