Abstract | OBJECT: METHODS: HuCNS-SC transplantation was tested in an open-label dose-escalation Phase I clinical trial as a potential treatment for infantile and late-infantile NCL. Study design included direct neurosurgical transplantation of allogeneic HuCNS-SCs into the cerebral hemispheres and lateral ventricles accompanied by 12 months of immunosuppression. RESULTS: Six children with either the infantile or late-infantile forms of NCL underwent low- (3 patients) and high- (3 patients) dose transplantation of HuCNS-SCs followed by immunosuppression. The surgery, immunosuppression, and cell transplantation were well tolerated. Adverse events following transplantation were consistent with the underlying disease, and none were directly attributed to the donor cells. Observations regarding efficacy of the intervention were limited by the enrollment criteria requiring that patients be in advanced stages of disease. CONCLUSIONS:
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Authors | Nathan R Selden, Amira Al-Uzri, Stephen L Huhn, Thomas K Koch, Darryn M Sikora, Mina D Nguyen-Driver, Daniel J Guillaume, Jeffrey L Koh, Sakir H Gultekin, James C Anderson, Hannes Vogel, Trenna L Sutcliffe, Yakop Jacobs, Robert D Steiner |
Journal | Journal of neurosurgery. Pediatrics
(J Neurosurg Pediatr)
Vol. 11
Issue 6
Pg. 643-52
(Jun 2013)
ISSN: 1933-0715 [Electronic] United States |
PMID | 23581634
(Publication Type: Clinical Trial, Phase I, Journal Article)
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Chemical References |
- Immunosuppressive Agents
- Tpp1 protein, mouse
- Tripeptidyl-Peptidase 1
- Dexamethasone
- TPP1 protein, human
- Mycophenolic Acid
- Tacrolimus
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Topics |
- Child
- Child, Preschool
- Dexamethasone
(administration & dosage)
- Drug Administration Schedule
- Feasibility Studies
- Female
- Humans
- Immunosuppressive Agents
(administration & dosage)
- Magnetic Resonance Imaging
- Male
- Mycophenolic Acid
(administration & dosage, analogs & derivatives)
- Neural Stem Cells
(transplantation)
- Neuronal Ceroid-Lipofuscinoses
(diagnosis, surgery)
- Neurosurgical Procedures
(methods)
- Stem Cell Transplantation
(methods)
- Stereotaxic Techniques
- Tacrolimus
(administration & dosage)
- Transplantation, Homologous
- Treatment Outcome
- Tripeptidyl-Peptidase 1
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