Ahead of Print: Intraspinal Stem Cell Transplantation in ALS
Background: No US-based clinical trials have attempted delivery of biologic therapies directly to the spinal cord for treatment of ALS, due to the lack of a meaningful FDA-authorized cell candidate and a validated delivery approach.
Objective: To assess safety of delivery of a neural stem cell-based treatment into the upper lumbar segments of the ALS spinal cord in the first FDA-authorized Phase I trial.
Methods: Each microinjection series was comprised of five injections (10[mu]l/inj) separated by 4mm. Each injection deposited 100,000 neural stem cells derived from a fetal spinal cord. Twelve patients were treated with either unilateral or bilateral injections. Group A, non-ambulatory patients, underwent unilateral (n=3) or bilateral (n=3) lumbar microinjections. Groups B and C were ambulatory (n=3 each) and respectively received unilateral or bilateral injections. Patients are followed clinically and radiologically to assess potential toxicity of the procedure.
Results: Twelve patients have been transplanted. There was one instance of transient intra-operative somatosensory-evoked potentials depression. In the immediate post-operative period there was one episode of urinary retention requiring foley catheter re-insertion. By discharge, none had a documented motor function decrement. Two required re-admission and re-operation for CSF leak or suprafascial wound dehiscence (n=1 each). Two deaths occurred at 8 and 13 months post surgery, neither related to the surgical transplant.
Conclusion: Our experience in 12 patients supports the procedural safety of unilateral and bilateral intraspinal lumbar microinjection. Completion of this Phase I safety trial is planned by proceeding to cervical and combined cervical + lumbar microinjections in ALS patients.
From: Intraspinal Stem Cell Transplantation in ALS: A Phase I Safety Trial, Technical Note & Lumbar Safety Outcomes by Riley et al.