Matched case-control: Autologous BM-MSC treating neurological sequelae post TBI

Cell & Gene Therapy Clinical Phase 1 Neurological diseases

Evaluation of the Safety and Efficacy of Autologous Bone Marrow Mononuclear Cell Infusion for the Treatment of Neurological Sequelae After Traumatic Brain Injury: Matched-Control Clinical Trial.

Principal Investigator (PI):

– Prof. Nguyen Thanh Liem

Objective:

To evaluate the safety and efficacy of intrathecal administration of autologous bone marrow mononuclear cells as an adjunctive therapy for neurological sequelae following traumatic brain injury.

Project Description (main page – 250 words):

Traumatic brain injury often leads to neurological sequelae that impair motor and cognitive functions, severely affecting quality of life. This study evaluated the safety and efficacy of intrathecal administration of bone marrow mononuclear cells in treating these sequelae.

A matched-control clinical trial was conducted on 50 patients. The intervention group received two intrathecal infusions of autologous bone marrow mononuclear cells, six months apart, combined with rehabilitation therapy, while the control group received rehabilitation only. Safety was assessed through adverse and serious adverse events; efficacy was evaluated using the Functional Independence Measure (FIM), Short Form-36 (SF-36), and Glasgow Outcome Scale–Extended (GOSE). MRI and PET-CT imaging monitored brain changes.

No serious adverse events were reported during 12 months of follow-up. Mild adverse events such as pain at the aspiration site and dizziness were self-limiting. The intervention group showed significant improvements in motor scores (+4.3; p=0.02), cognitive scores (+1.7; p=0.009), and quality of life in both physical and mental domains (p<0.05). MRI showed structural improvements, and PET-CT revealed increased brain metabolic activity.

Intrathecal administration of bone marrow mononuclear cells is a safe and effective therapy that improves motor function and quality of life in patients with neurological sequelae after traumatic brain injury. It is recommended as a complementary treatment alongside rehabilitation programs.