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. Author manuscript; available in PMC: 2023 Oct 19.
Published in final edited form as: Nat Biomed Eng. 2023 Jan 12;7(4):370–386. doi: 10.1038/s41551-022-00987-y

Table 2. Ongoing and planned clinical trials of cell therapies for the treatment of PD. TBD, to be determined.

Trial Transeuro182 NCT01898390 ISCO183 NCT02452723 CiRA125 Chinese Academy of Sciences184 NCT03119636 American multicentre case report185 Summit for PD (in set up)59 NYSTEM-PD186 (in set up) European STEM-PD187(in set up)
Cell source Foetal ventral mesencephalona Parthenogenetic neural stem cellsb Allogenic iPSCsc Parthenogenetic embryonic stem cell derived neural precursorsd Autologous iPSCse Autologous iPSCsf H9 embryonic stem cellsg RC17 embryonic stem cellsh
Cryopreservation of the cell product No Yes No Data not available Yes Yes Yes Yes
Screening of cell product for genetic variance/quality control None Yes, flow cytometry and RT- PCR to assess markers of pluripotency. Yes, sequencing for genes of interest Data not available Whole genome sequencing Whole genome sequencing Yes, Karyotype analysis by G- banding. Viral testing. Genetic testing TBD. Yes, flow cytometry and RT- PCR to assess markers of pluripotency and midbrain dopaminergic progenitors. Genetic testing TBD
Functional testing Structural and functional MRI 18F-Dopa PET 11C-PE2I PET 11C-DASB PET MRI 18F-DOPA PET MRI 18F-FLT-PET MRI DAT-SPECT 18F-DOPA PET MRI CT scan 18F-DOPA PET DAT- SPECT 18F-FLT PET 18F-DOPA PET 11C-PE2I PET MRI MRI 18F-DOPA PET 11C-PE2I PET
Immunosuppression Cyclosporine, Azathioprine and Prednisolone Yes, but unclear Tacrolimus Data not available None None Tacrolimus, mycophenalate, basiliximab, prednisolone. TBD
MHC Matching? No No Yes Two groups - one HLA matched one mismatched Autologous transplant N/A No No
Patient cohort 30-68y/o early- stage PD patients 30-70y/o moderate to severe PD patients 50-70y/o moderate PD patients 50-80 y/o moderate PD patients One 69 y/o PD patient 45-70 y/o moderate PD patients. 40-70y/o moderate PD patients. Moderate stage PD patients 40- 70y/o
Date of first-in-human transplant 2015 2016 2018 2017 2018 TBD TBD TBD

aGrafting of foetal ventral mesencephalic tissue has previously demonstrated efficacy in PD animal models and patients27,55,57,188. bNSCs grafted into the brains of rodents and non-human primates was safe but with minimal clinical efficacy65,67,68.

cClinical grade iPSC-derived dopaminergic progenitors have demonstrated safety and efficacy in rodent and non-human primate PD models62.

dClinical grade ESC-derived neural precursors significantly improved motor dysfunction in some MPTP lesioned non-human primates, in the absence of tumour formation for up to 2 years184.

ePD patient-derived iPSC-derived dopaminergic precursors significantly reversed motor deficits in 6OHDA lesioned rats, without tumour formation185. Results were corroborated in one PD patient, who demonstrated clinical benefit up to 2 years after grafting, without the onset of serious adverse events185.

fData pertaining to the safety and efficacy of autologous iPSC-derived dopaminergic neurons is underway. 10 patient-derived iPSC lines have been successfully generated64.

gClinical grade H9 ESC-derived dopaminergic progenitors have demonstrated safety and efficacy in preclinical PD models135,150.

hClinical grade ESC-derived dopaminergic progenitors have demonstrated safety and efficacy in preclinical PD models84,88.