Table 1.

Population/schedule/administration differences

City of HopeBaylor CollegeUniversity of Pennsylvania/UCSF
Patient populationNCTNCT02208362NCT01109095NCT02209376
Patients1 (adult)17 (ped. and adult)10 (adult)
Molecular profileMGMT non met., IDH1 WTNot knownAll MGMT non met.
ConstructTargetIL13Ra2 (E13Y neoepitope) assessed by IHCHer2 (FRP5 exodomain) assessed by IHC, CMV pp65 antigenHumanized EGFRvIII (neoepitope) assessed by RNA-based NGS
Costimulatory molecules4-1BBzCD28z4-1BBz
VectorLentivirusRetrovirusLentivirus
Protocol/scheduleNumber of infusionsMultipleMultiple1
Dose2 to 5e61 to 100e6 cell/m2175 to 500e6 cells
RouteICV then IVTIVIV
ToxicityToxNo DLT, no CRSNo DLT, No cardiac tox., no CRSNo DLT, no skin or mucosal or lung, no CRS, 3 non–CAR-related neurologic events
EfficacyRadiologic responsesYesYes 1PR, 7SDNo 9SD, 1PD
ExpansionYes, 1 d (assessed in CSF)No expansionYes (assessed by flow and qPCR in blood)
Persistence7 d (CSF)12 mo in 6 pat. (blood tests by qPCR)14 d max 2 mo (at low level) (blood, qPCR, flow)
T-cell repertoire expansionYesNot knownYes
Antigen lossYesNoYes (IHC in 5/7 reoperated pat.)
Brain traffickingNot knownNot knownYes, max 2 mo after infusion