Functional Status, Quality of Life and Survival After Repeated Intrathecal Administration of Autologous Lineage-Negative Cells in Amyotrophic Lateral Sclerosis
5 affiliations
- 1Department of Neurology, Pomeranian Medical University, 71-252 Szczecin, Poland
- 2Department of Hematology and Transplantology, Pomeranian Medical University, 71-252 Szczecin, Poland
- 3Department of General Pathology, Pomeranian Medical University, 70-111 Szczecin, Poland
- 4University Hospitals of Northamptonshire, Northampton General Hospital NHS Trust, Northampton NN1 5BD, UK
- 5Department of Pharmacology and Toxicology, University of Zielona Góra, 65-417 Zielona Góra, Poland
Abstract
Background/Objectives: Adjuvant cell-based strategies are still being explored in amyotrophic lateral sclerosis (ALS). Materials and Methods: In this open-label, single-arm study of intrathecal autologous bone marrow-derived lineage-negative (LIN−) cells, we report functional status, quality of life, and survival after three administrations at six-week intervals. A total of 45 patients with sporadic ALS were qualified: 42 received all three administrations and 23 completed the 24-week assessment schedule, while 19 stopped attending because of deterioration. Results: In the 23 completers, Norris scale scores were stable until week 13 and then declined (p = 0.00004), and the Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSAQ-40) followed the same pattern (p = 0.0019), whereas the ALS Functional Rating Scale—Revised (ALSFRS-R) declined at a constant 0.5 points per month. In all 42 treated patients the Norris score fell during treatment (0.95 points per month; 95% CI 0.52–1.38), so the plateau was confined to completers, who had milder baseline disease. Dose-related differences in quality of life were confounded by baseline imbalance; survival analyses were exploratory. Cell number did not correlate with cerebrospinal fluid brain-derived neurotrophic factor (BDNF) after the third administration in the 39 patients with paired samples (ρ = 0.14). No adverse event was recorded in 126 procedures, but harms were not graded systematically. Conclusions: These exploratory findings do not demonstrate efficacy; a sham-controlled trial with blinded raters is required.
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BibTeX
@article{golabjanowska2026functional,
title = {{Functional Status, Quality of Life and Survival After Repeated Intrathecal Administration of Autologous Lineage-Negative Cells in Amyotrophic Lateral Sclerosis}},
author = {Monika Gołąb-Janowska and Agnieszka Wełnicka-Wesołowska and Bartłomiej Baumert and Karolina Machowska-Sempruch and Agnieszka Meller and Anna Sobuś and Edyta Paczkowska and Dariusz Kotlęga and Przemysław Nowacki and Bogusław Machaliński},
journal = {Journal of Clinical Medicine},
year = {2026},
month = oct,
doi = {10.3390/jcm15207937},
url = {https://www.mdpi.com/2077-0383/15/20/7937},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Functional Status, Quality of Life and Survival After Repeated Intrathecal Administration of Autologous Lineage-Negative Cells in Amyotrophic Lateral Sclerosis
AU - Monika Gołąb-Janowska
AU - Agnieszka Wełnicka-Wesołowska
AU - Bartłomiej Baumert
AU - Karolina Machowska-Sempruch
AU - Agnieszka Meller
AU - Anna Sobuś
AU - Edyta Paczkowska
AU - Dariusz Kotlęga
AU - Przemysław Nowacki
AU - Bogusław Machaliński
PY - 2026
DA - 2026/10/01
JO - Journal of Clinical Medicine
DO - 10.3390/jcm15207937
UR - https://www.mdpi.com/2077-0383/15/20/7937
AB - Background/Objectives: Adjuvant cell-based strategies are still being explored in amyotrophic lateral sclerosis (ALS). Materials and Methods: In this open-label, single-arm study of intrathecal autologous bone marrow-derived lineage-negative (LIN−) cells, we report functional status, quality of life, and survival after three administrations at six-week intervals. A total of 45 patients with sporadic ALS were qualified: 42 received all three administrations and 23 completed the 24-week assessment schedule, while 19 stopped attending because of deterioration. Results: In the 23 completers, Norris scale scores were stable until week 13 and then declined (p = 0.00004), and the Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSAQ-40) followed the same pattern (p = 0.0019), whereas the ALS Functional Rating Scale—Revised (ALSFRS-R) declined at a constant 0.5 points per month. In all 42 treated patients the Norris score fell during treatment (0.95 points per month; 95% CI 0.52–1.38), so the plateau was confined to completers, who had milder baseline disease. Dose-related differences in quality of life were confounded by baseline imbalance; survival analyses were exploratory. Cell number did not correlate with cerebrospinal fluid brain-derived neurotrophic factor (BDNF) after the third administration in the 39 patients with paired samples (ρ = 0.14). No adverse event was recorded in 126 procedures, but harms were not graded systematically. Conclusions: These exploratory findings do not demonstrate efficacy; a sham-controlled trial with blinded raters is required.
SN - 2077-0383
ER -