Safety and Tolerability Assessment of Intrastriatal Neural Allografts in Five Patients with Huntington's Disease
Overview
Authors
Affiliations
This study describes issues related to the safety and tolerability of fetal striatal neural allografts as assessed in five patients with Huntington's disease. Huntington's disease (HD) is characterized by motor, cognitive, and behavioral disturbances. The latter include psychological disturbances and, as a consequence, we took particular care to analyze behavioral changes, in addition to the usual "safety" follow-up. We conducted multidisciplinary follow-up at least 2 years before and 1 year after grafting. Psychological care extended to close relatives. The grafting procedure itself was altogether safe and uneventful, and there were no apparent clinical deleterious effects for 1 year. The immunosuppressive treatment, however, was complicated by various problems (irregular compliance, errors of handling, side effects). Direct psychological consequences of the transplantation procedure were rare and not worrisome, although mood alteration requiring treatment was observed in one patient. Indirectly, however, the procedure required patients and relatives to accept constraints that tended to complicate familial situations already marred by aggressivity and depression. All patients and close relatives expressed major expectations, in spite of our strong and repeated cautioning. It is clearly important to be aware of these particular conditions since they may eventually translate into psychological difficulties in coping with the long-term clinical outcome of the procedure, if not beneficial. Despite an overall good tolerance, therefore, this follow-up calls for caution regarding the involvement of HD patients in experimental surgical protocols.
Stem Cell Therapies in Movement Disorders: Lessons from Clinical Trials.
Marsili L, Sharma J, Outeiro T, Colosimo C Biomedicines. 2023; 11(2).
PMID: 36831041 PMC: 9953050. DOI: 10.3390/biomedicines11020505.
Therapeutic Strategies in Huntington's Disease: From Genetic Defect to Gene Therapy.
Jurcau A, Jurcau M Biomedicines. 2022; 10(8).
PMID: 36009443 PMC: 9405755. DOI: 10.3390/biomedicines10081895.
Current and Possible Future Therapeutic Options for Huntington's Disease.
Ferguson M, Kennedy C, Palpagama T, Waldvogel H, Faull R, Kwakowsky A J Cent Nerv Syst Dis. 2022; 14:11795735221092517.
PMID: 35615642 PMC: 9125092. DOI: 10.1177/11795735221092517.
Advances and Perspectives in Dental Pulp Stem Cell Based Neuroregeneration Therapies.
Luzuriaga J, Polo Y, Pastor-Alonso O, Pardo-Rodriguez B, Larranaga A, Unda F Int J Mol Sci. 2021; 22(7).
PMID: 33805573 PMC: 8036729. DOI: 10.3390/ijms22073546.
Cell therapy in Huntington's disease: Taking stock of past studies to move the field forward.
Bachoud-Levi A, Massart R, Rosser A Stem Cells. 2020; 39(2):144-155.
PMID: 33176057 PMC: 10234449. DOI: 10.1002/stem.3300.