Safety and efficacy of first-in-man intrathecal injection of human astrocytes (AstroRx®) in ALS patients: phase I/IIa clinical trial results

Table 1 Cell information

Part 1: Cell number, Viability and Safety profile of formulated AstroRx cell product
Cohort	Patient Number	Cell count (X10⁶)	Cell viability (%)	Safety Profile
Cohort	Patient Number	Cell count (X10⁶)	Cell viability (%)	Bacteriology	Mycoplasma	Endotoxin level (EU/ml)	EPCAM (%)	SSEA4 (%)
A	1001	102	93.9	NC	NC	< 1.0	0.0	0.0
	1002	90	95.5	NC	NC	< 1.0	0.0	0.0
	1005	95	92.9	NC	NC	< 1.0	0.0	0.0
	1008	102	91.9	NC	NC	< 1.0	0.0	0.0
	1009	98.5	94.7	NC	NC	< 1.0	0.0	0.0
B	2010	245	96.6	NC	NC	< 1.0	0.0	0.0
	2012	270	97.6	NC	NC	< 1.0	0.0	0.0
	2015	258	96.6	NC	NC	< 1.0	0.0	0.0
	2016	261	96.4	NC	NC	< 1.0	0.0	0.0
	2017	265	95.6	NC	NC	< 1.0	0.0	0.0

Part 2: Cell characteristics of formulated AstroRx® product
Cell characteristics		Cohort (Average ± SEM)		Release Criteria
Cell characteristics		A	B	Release Criteria
Astrocytic identity	CD44 (%)	99.3 ± 0.2	99.8 ± 0.0	≥ 85%
	GLAST (%)	88.8 ± 7.6	69.9 ± 1.3	≥ 50%
	GFAP (%)	97.5 ± 0.9	92.2 ± 1.3	≥ 70%
Potency	TIMP-1 (ng/10⁶)	38.2 ± 7.7	43.9 ± 6.1	≥ 5 ng/106
Potency	MIDKINE (ng/10⁶)	14.3 ± 1.3	19.9 ± 2.0	≥ 0.5 ng/106

NC no contamination, EU endotoxin unit, EPCAM epithelial cell adhesion molecule, SSEA-4 stage-specific embryonic antigen-4, CD44 cluster of differentiation 44, GLAST glutamate aspartate transporter, GFAP Glial fibrillary acidic protein.
Table I: Cell Characteristics of formulated AstroRx cell product. Part 1: Cell number, viability, and safety profile of AstroRx® cells used for intrathecal injection for each ALS patient. Part 2: AstroRx cell characteristics used for each study cohort.

ISSN: 1479-5876