Table 5. GALS-001 and GALS-C CSF biomarker results.
GALS-C = Single compassionate patient treatment; GALS-T = GALS-001 treated group, and GALS-P = GALS-001 placebo group. ↑ = upregulation, ↓ = downregulation, DM* = disease modification, DP** = disease progression.
| CSF
Biomarkers |
SOD1
GALS-C (N=1) |
SOD1
GALS -T (N=8) |
SOD1
GALS-P (N=4) |
Cystatin
C GALS-C (N=1) |
Cystatin
C GALS-T (N=8) |
Cystatin C
GALS - P (N=4) |
total Tau
GALS-C (N=1) |
total Tau
GALS-T (N=8) |
total Tau
GALS -P (N=4) |
|---|---|---|---|---|---|---|---|---|---|
| Sample-ID | Con.ng/ml | Con.ng/ml | Con.ng/ml | Conc.µg/ml | Conc.µg/ml | Conc.µg/ml | Con.pg/ml | Con.pg/ml | Con.pg/ml |
|
healthy range -
CSF |
50–200 | 50–200 | 50–200 | 3.0–8.0 | 3.0–8.0 | 3.0–8.0 | 100–350 | 100–350 | 100–350 |
| Baseline-CSF | 27.228 | 186.6 | 137.94 | 1.97 | 3.11 | 3.23 | 60.55 | 305.03 | 386.85 |
| standard
deviation |
168.3 | 56.39 | 1.35 | 0.78 | 122.3 | 182.93 | |||
| Visit 6 (Week 2)
-CSF |
30.996 | 153.17 | 175.86 | 2.35 | 3.15 | 3.06 | 63.33 | 303.58 | 412.96 |
| standard
deviation |
76.14 | 84.56 | 1.41 | 0.76 | 139.37 | 196.62 | |||
| mean %
Change V6-BL |
13.84% | -3.75% | 30.45% | 19.29% | 1.57% | -4.57% | 4.59% | -1.16% | 6.43% |
| standard
deviation |
26.20% | 56.90% | 8.49% | 12.10% | 15.79% | 6.36% | |||
| Comments |
below
range, ↑=DM* |
high end
of range, ↓=DM* |
high end
of range, ↑=DP** |
below
range, ↑=DM* |
low end of
range, ↑=DM* |
low end of
range, ↓=DP** |
below
range, ↑=DM* |
high end
of range, ↓=DM* |
above
range, ↑=DP** |