TY - JOUR
T1 - Implantation of Spheramine® in advanced Parkinson's Disease (PD)
AU - Bakay, Roy A.E.
AU - Raiser, Cathy D.
AU - Stover, Natividad P.
AU - Subramanian, Thyagarajan
AU - Cornfeldt, Michael L.
AU - Schweikert, Alfred W.
AU - Allen, Richard C.
AU - Watts, Ray
PY - 2004/8/19
Y1 - 2004/8/19
N2 - Evaluation of the safety and efficacy of unilateral stereotactic implantation of cultured human retinal pigment epithelial (hRPE) cells attached to microcarriers (Spheramine®) in patients with advanced PD in an open label pilot study. Six patients with advanced PD (3 males; 3 females; mean age 52.2 years; mean duration of PD 10.2 years; mean Hoehn and Yahr stage "off" 3.75) were assessed at baseline and post-operatively using the modified CAPIT. Each patient underwent MRI-guided stereotactic transplantation of 325,000 hRPE cells attached to microcarriers in 5 tracts, 5 mm apart in the post-commissural putamen contralateral to the most affected side. Immunosuppression was not used. The UPDRS Motor (UPDR-M) score in the practically defined "off" state was the primary outcome measure. At 6 months post-op, the mean UPDRS-M (off) score improved to 35 (34%) from a pre-op baseline mean of 52 (p < .001). Secondary outcome measures improved including the total UPDRS (33%), Timed Motor Tests (on, 14%; off, 23%), PDQ39 QOL (30%), and Schwab and England score (on, 11%; off, 30%). Bilateral improvements have been observed in motor symptoms, with the greatest effect seen contralateral to the implants. Three of six patients currently have lower Dyskinesia Rating Scale scores than at baseline, while the scores of the other three are unchanged from baseline values. No "off-state" dyskinesias have been observed. Thus Spheramine® implantation therapy appears to be safe and well tolerated for 6 months post-implantation.
AB - Evaluation of the safety and efficacy of unilateral stereotactic implantation of cultured human retinal pigment epithelial (hRPE) cells attached to microcarriers (Spheramine®) in patients with advanced PD in an open label pilot study. Six patients with advanced PD (3 males; 3 females; mean age 52.2 years; mean duration of PD 10.2 years; mean Hoehn and Yahr stage "off" 3.75) were assessed at baseline and post-operatively using the modified CAPIT. Each patient underwent MRI-guided stereotactic transplantation of 325,000 hRPE cells attached to microcarriers in 5 tracts, 5 mm apart in the post-commissural putamen contralateral to the most affected side. Immunosuppression was not used. The UPDRS Motor (UPDR-M) score in the practically defined "off" state was the primary outcome measure. At 6 months post-op, the mean UPDRS-M (off) score improved to 35 (34%) from a pre-op baseline mean of 52 (p < .001). Secondary outcome measures improved including the total UPDRS (33%), Timed Motor Tests (on, 14%; off, 23%), PDQ39 QOL (30%), and Schwab and England score (on, 11%; off, 30%). Bilateral improvements have been observed in motor symptoms, with the greatest effect seen contralateral to the implants. Three of six patients currently have lower Dyskinesia Rating Scale scores than at baseline, while the scores of the other three are unchanged from baseline values. No "off-state" dyskinesias have been observed. Thus Spheramine® implantation therapy appears to be safe and well tolerated for 6 months post-implantation.
UR - http://www.scopus.com/inward/record.url?scp=2542478946&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=2542478946&partnerID=8YFLogxK
M3 - Article
C2 - 14766394
AN - SCOPUS:2542478946
VL - 9
SP - 592
EP - 602
JO - Frontiers in Bioscience - Landmark
JF - Frontiers in Bioscience - Landmark
SN - 1093-9946
ER -