Scientific Evidence

What is Stemirac®?

An autologous mesenchymal stem cell product, intended as a future therapy for neurologic diseases

Approach
Stemirac is a regenerative medicine product produced from the patient’s own bone marrow and peripheral blood. Mesenchymal stem cells (MSCs) are isolated from the patient’s bone marrow fluid, cultured with serum derived from the patient’s own blood, and then cryopreserved for intravenous administration.

Regulatory Status
Stemirac holds a conditional approval from Japan’s Pharmaceutical and Medical Devices Agency (PDMA) for use in the treatment of acute spinal cord injury.

Mechanism of Action
Stemirac has been shown to (1) improve the integrity of the blood–spinal cord barrier, (2) protect motor neurons in the spinal cord, (3) reduce inflammation, and (4) secrete factors that support neurons.1,2,3

Clinical Research
Stemirac is currently undergoing clinical research for a range of conditions including acute and chronic spinal cord injury, stroke, dementia, traumatic brain injury and ALS.

Development Pathway

*STR04 is a multi-dose formulation of Stemirac

Clinical Results of Single Dose Infusion for Spinal Cord Injury (SCI)

A Phase II clinical study of a single IV infusion of Stemirac in 13 spinal cord injury patients demonstrated neurological improvement at 6 months post-infusion compared to pre-infusion.

Comparison of improvement in ASIA score for patients treated with Stemirac compared to no treatment historical controls.

Repeated Infusion of MSCs Reduces the Deterioration
of Motor Deficits in ALS in a Rodent Model

Slowed decline in motor function
  • A single MSC dose slowed decline in motor function measured by the Basso–Beattie–Bresnahan (BBB) locomotor rating scale vs control
  • Repeat MSC administration (4 doses) provided significantly slower decline in BBB scores vs single dose and control

Repeated MSC infusion prolonged survival in rodent model
Repeated MSC infusion maintained body weight in rodent model

References

  1. Sasaki M, Radtke C, Tan AM, Zhao P, Hamada H, Houkin K, Honmou O, Kocsis JD. BDNF-Hypersecreting Human Mesenchymal Stem Cells Promote Functional Recovery, Axonal Sprouting, and Protection of Corticospinal Neurons after Spinal Cord Injury. J. Neurosci. November 2009; 29(47):14932–14941.
  2. Magota H, Sasaki M, Kataoka-Sasaki Y, Oka S, Ukai R, Kiyose R, Onodera R, Kocsis JD, Honmou O. Intravenous infusion of mesenchymal stem cells delays disease progression in the SOD1G93A transgenic amyotrophic lateral sclerosis rat model. Brain Res. 2021a Apr 15;1757:147296.
  3. Magota H, Sasaki M, Kataoka-Sasaki Y, Oka S, Ukai R, Kiyose R, Onodera R, Kocsis JD, Honmou O. Repeated infusion of mesenchymal stem cells maintain the condition to inhibit deteriorated motor function, leading to an extended lifespan in the SOD1G93A rat model of amyotrophic lateral sclerosis. Mol Brain. 2021b May 7;14(1):76.
  4. Honmou O, Yamashita T, Morita T, Oshigiri T, Hirota R, Iyama S, Kato J, Sasaki Y, Ishiai S, Ito YM, Namioka A, Namioka T, Nakazaki M, Kataoka-Sasaki Y, Onodera R, Oka S, Sasaki M, Waxman SG, Kocsis JD. Intravenous infusion of auto serum-expanded autologous mesenchymal stem cells in spinal cord injury patients: 13 case series. Clin Neurol Neurosurg. 2021 Apr;203:106565.