Table 2.
Clinical transplantation in Huntington’s disease.
Study (year) | Clinical size | Type of cell | Clinical outcome | Negative effects | Ref. |
---|---|---|---|---|---|
Bachoud-Levi et al. (2006, 2009, 2000) | Five patients | Whole ganglionic eminence | Three of five patients showed stability of symptoms or clinical improvement for 4–6 years | One patient showed development of a putaminial cyst | [4,99,108] |
Capetian et al. (2009) | One patient | Whole ganglionic eminence | UHDRS score stability for 6 months. Survival and differentiation of grafted cells | None reported (patient died from unrelated causes) | [109] |
Cicchetti et al. (2009, 2014) | Three patients | Lateral ventricular eminence containing striatal primordia | Improvement of UHDRS in two of three patients for up to 18 months before returning to presurgical levels | Grafts underwent disease-like neuronal degeneration. Cortical hemorrhage, subdural hematoma following surgery | [5,100] |
Freeman et al. (2000) | One patient | Lateral ventricular eminence containing striatal primordia | Stability of UHDRS 15 months following transplantation. Transplants integrated into the host tissue | None reported | [110] |
Furtado et al. (2005) | Seven patients | Fetal striatal tissue | Transplants failed to restore fluorodeoxyglucose uptake and D1 and D2 receptor binding in subjects | Possible technical issues with regards to the ganglionic eminence and in targeting the striatum | [111] |
Hauser et al. (2002) | Seven patients | Fetal striata | Grafts developed striatal morphology, UHDRS improved significantly 12 months following surgery | Three subjects developed subdural hemorrhages, one patient died 18 months following surgery from probable cardiac arrhythmia | [112] |
Keene et al. (2007) | Two patients | Fetal lateral ganglionic eminence | Improved ambulation 3 months following transplant in one patient. In both patients, transplanted cells displayed morphology of neurons and astrocytes | One patient reported chronic headaches following surgery and was treated for bilateral subdural hematomas. Reported that transplants did not have an effect on the course of HD | [113] |
Keene et al. (2009) | One patient | Fetal neuronal tissue | Clinical improvement for UHDRS for 2 years. Patient died 121 months following surgery from complications of advanced HD | Three mass lesions and one large cyst were present on the left caudate and putamen. Five mass lesions and two cysts were present on the right caudate and putamen | [6] |
Kopyov et al. (1998) | Three patients | Lateral ganglionic eminence | Clinical improvement for UHDRS for all three patients 12 months following surgery. Graft survival and growth within the striatum without displacing host tissue | None reported | [114] |
Krystkowiak et al. (2007) | 13 patients | Fetal neuronal tissue | Pre- and post-UHDRS were not reported. Four of the 13 patients had grafts that did not display signs of rejection | Biological, radiological and clinical rejection of grafts in other subjects (reversible under immunosuppressive treatment) | [115] |
Reuter et al. (2008) | Two Patients | Whole ganglionic Eminence | Clinical improvement for UHDRS over 5-year period for one patient. Increased striatal D2 receptor binding, suggesting long-term survival and efficacy of grafts | None reported | [101] |
Rosser et al. (2002) | Four Patients | Whole ganglionic Eminence | Stability of UHDRS as well as cognitive ability up to 6 months following surgery. Graft survival without Overgrowth | None reported | [116] |
Philpott et al(1997) | Three Patients | Lateral ganglionic Eminence | Increased cognitive functioning 6 months following surgery | None reported | [117] |
Gallina et al. (2010) | Four Patients | Whole ganglionic Eminence | Stability or improvement in motor, behavioral and functional scores up to 24 months following surgery | None reported | [104] |
Madrazo et al. (1995) | Two patients | Whole ganglionic eminence | Stability or improvement on functional capacity for up to 25 months following surgery when a slow progression of HD was observed | None reported | [118] |
HD: Huntingdon’s disease; UHDRS: Unified Huntingdon’s disease rating scale.