In a small Polish study led from Pomeranian Medical University in Szczecin, researchers tested whether a patient's own bone marrow cells, delivered into the fluid surrounding the spinal cord, might steady a disease that has long resisted every intervention. Among the minority who completed the full schedule, motor and quality-of-life measures held or improved, yet the same study's wider view shows the illness still advancing for most who began it. The authors present the work as a first look that signals feasibility and invites rigorous testing, not as proof that the approach works.
Intrathecal Cell Therapy in ALS Shows Exploratory Gains in Function and Quality of Life, Pending Controlled Trials
The plateau was limited to patients with milder baseline disease.
Mark
What's the headline finding here?
Mimi
In the 23 patients who completed 24 weeks, motor scores on the Norris scale held steady until week 13, and quality of life on the ALSAQ-40 showed a similar pattern. No adverse events were recorded.
Luke
Before we get carried away: 23 of 45 is about half. Who dropped out, and why, isn't in what we have.
Mark
So the completer results may not represent everyone?
Mimi
Right, and the wider numbers show it. Across all 42 treated patients, Norris scores fell 0.95 points a month. The plateau was limited to those with milder disease at baseline.
Luke
And the standard ALS measure, the ALSFRS-R, still declined 0.5 points a month in completers. So the signal is mixed.
Mark
What about safety? No adverse events sounds reassuring.
Luke
It's weaker than it sounds. The authors say harms weren't systematically graded. Nothing recorded isn't the same as nothing happened.
Mimi
The dose question also stays open. Baseline imbalance meant they couldn't compare quality of life by dose.
Mark
Is there any comparison group?
Luke
Not that the reporting describes. Without a control and masking, we can't separate the treatment from the natural course of the disease. The authors say as much.
Mimi
Which is why the next step is a controlled, masked trial.
Der Puls
- Only 23 of 45 enrolled patients reached the end of the 24-week schedule, leaving the most encouraging results resting on a fragment of the original cohort.
- Norris motor scores held steady until week 13 and then shifted significantly, while ALSAQ-40 quality-of-life scores climbed, giving the team a hopeful signal to chase.
- Across all 42 treated patients, Norris scores fell nearly a point each month and ALSFRS-R declined, showing the plateau appeared only in milder cases.
- No adverse events were logged, but harms were never systematically graded, so the authors refuse to call this a safety verdict and call for a controlled, masked trial before any claim is made.
In a small Polish study led from Pomeranian Medical University in Szczecin, researchers tested whether a patient's own bone marrow cells, delivered into the fluid surrounding the spinal cord, might steady a disease that has long resisted every intervention. Among the minority who completed the full schedule, motor and quality-of-life measures held or improved, yet the same study's wider view shows the illness still advancing for most who began it. The authors present the work as a first look that signals feasibility and invites rigorous testing, not as proof that the approach works.
Researchers at Pomeranian Medical University in Szczecin, Poland, have reported an early look at an experimental cell therapy for amyotrophic lateral sclerosis. Led by Monika Gołąb-Janowska, MD, and published in the Journal of Clinical Medicine, the work followed 45 people with sporadic ALS. Each was to receive three injections of their own bone marrow-derived cells, called LIN− cells, into the fluid around the spinal cord, spaced six weeks apart. Forty-two received all three doses, but only 23 finished the full 24-week assessment.
Among those 23, motor scores on the Norris scale held steady until week 13 and then changed in a statistically significant way, and quality-of-life scores on the ALSAQ-40 improved. The team could not tell whether those gains depended on dose, since the groups were unevenly matched at the start.
The broader picture is more sober. Across all 42 treated patients, motor scores fell nearly a point a month, and the standard ALS efficacy measure still declined among completers. The plateau, in other words, seems to belong mainly to people whose disease was milder at the outset. No adverse events were recorded, though harms were not formally graded, and the study had no comparison group and no masking. The authors are clear that this is exploratory work. What it justifies is a controlled, masked trial to learn whether the approach truly helps.
A group of researchers at Pomeranian Medical University in Szczecin, Poland, has reported results from a small study of an experimental cell therapy for amyotrophic lateral sclerosis. The work, led by Monika Gołąb-Janowska, MD, appeared in the Journal of Clinical Medicine. It is an early, exploratory look, and the authors themselves say it cannot show that the treatment works.
The study enrolled 45 patients with sporadic ALS. Each was to receive three intrathecal administrations of autologous bone marrow-derived lineage-negative cells, called LIN− cells, spaced six weeks apart. Intrathecal means the cells were delivered into the fluid around the spinal cord. Autologous means they came from the patients' own bone marrow. Forty-two of the 45 received all three administrations, and 23 completed the full 24-week assessment schedule.
The researchers tracked functional status, quality of life, and survival. Among the 23 who finished, scores on the Norris scale, which measures resolution of motor deficit, stayed stable until week 13 and then changed at a statistically significant level (P<0.001). The source describes that change as indicating improved motor function. A similar pattern appeared on the ALSAQ-40, a disease-specific questionnaire covering physical mobility, daily activities and independence, eating and drinking, communication, and emotional reactions (P=0.0019). The team could not establish whether quality of life differed by dose, because the groups were unbalanced at baseline.
The picture is less encouraging when the lens widens. Across all 42 treated patients, Norris scores fell by 0.95 points per month (95% CI, 0.52–1.38). According to the report, that means the plateau seen in the completers was limited to patients whose disease was milder at the start. Among the 23 completers, the ALS Functional Rating Scale—Revised, the standard efficacy measure in ALS trials, still declined by 0.5 points per month across the study period.
No adverse events were recorded, which the authors take to suggest the approach is feasible in patients with sporadic ALS. They add a caveat: harms were not systematically graded, so the absence of recorded events is not the same as a formal safety assessment.
The design carries other limits worth keeping in view. Only about half of the original group, 23 of 45, reached the end of the assessment schedule, and the analysis of those completers describes a subset of patients. There was no comparison group and no masking. The authors state plainly that the findings are exploratory and cannot demonstrate efficacy.
What they say is needed next is a controlled, masked trial to determine whether LIN− cells can effectively treat ALS. Until such a trial reports, this study reads as a feasibility signal and a reason to test the question properly, not as evidence of a treatment.
Bemerkenswerte Zitate
The findings are exploratory and cannot demonstrate efficacy; a controlled, masked trial is still required.— Study authors, Journal of Clinical Medicine (paraphrased)
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