Intraspinal Stem Cell Transplantation in Amyotrophic Lateral Sclerosis
| Autor: | Vita Kesner, Jonathan D. Glass, Jason Taub, Bethwel Raore, Jonathan Riley, Eva L. Feldman, Thais Federici, Meraida Polak, Nicholas M. Boulis, Crystal Kelly |
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| Rok vydání: | 2012 |
| Předmět: |
Adult
Male medicine.medical_specialty Foley catheter Lumbar vertebrae Lumbar Humans Medicine Injections Spinal Lumbar Vertebrae Cerebrospinal fluid leak business.industry Urinary retention Wound dehiscence Amyotrophic Lateral Sclerosis Middle Aged Spinal cord medicine.disease Surgery Transplantation Treatment Outcome medicine.anatomical_structure Female Neurology (clinical) Nervous System Diseases medicine.symptom business Stem Cell Transplantation |
| Zdroj: | Neurosurgery. 71:405-416 |
| ISSN: | 0148-396X |
| DOI: | 10.1227/neu.0b013e31825ca05f |
| Popis: | BACKGROUND: No United States-based clinical trials have attempted delivery of biological therapies directly to the spinal cord for treatment of amyotrophic lateral sclerosis (ALS) because of the lack of a meaningful US Food and Drug Administration-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 US Food and Drug Administration-authorized phase I trial. METHODS: Each microinjection series comprised 5 injections (10 μL/injection) separated by 4 mm. 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, nonambulatory 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 received a transplant. There was one instance of transient intraoperative somatosensory-evoked potentials depression. In the immediate postoperative period, there was 1 episode of urinary retention requiring Foley catheter reinsertion. By discharge, none had a documented motor function decrement. Two patients required readmission and reoperation for cerebrospinal fluid leak or suprafascial wound dehiscence (n = 1 each). Two deaths occurred at 8 and 13 months postsurgery; neither was 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. |
| Databáze: | OpenAIRE |
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