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Intrathecal administration of mesenchymal stem cells in patients with adrenomyeloneuropathy
Tomasz Siwek1,2, Beata Zwiernik1,2, Katarzyna Jezierska-Woźniak3
1Department of Neurology, University of Warmia and Mazury in Olsztyn, Olsztyn, Poland.
Background And Objectives:
X-linked adrenomyeloneuropathy (AMN) is an inherited neurodegenerative disorder associated with mutations in the ABCD1 gene and the accumulation of very long-chain fatty acids (VLFCAs) in plasma and tissues. Currently, there is no effective treatment for AMN. We have aimed to evaluate the therapeutic effects of mesenchymal stem cell (MSC) transplantation in patients with AMN.
Methods:
This is a small cohort open-label study with patients with AMN diagnosed and treated at the University Hospital in Olsztyn, Poland. All patients met clinical, biochemical, MRI, and neuropsychological criteria for AMN. MSCs derived from Wharton jelly, 20 × 106 cells, were administered intrathecally three times every 2 months, and patients were followed up for an additional 3 months. The primary outcome measures included a blinded assessment of lower limb muscle strength with the Medical Research Council Manual Muscle Testing scale at baseline and on every month visits until the end of the study. Additional outcomes included measurements of the timed 25-feet walk (T25FW) and VLFCA serum ratio.
Results:
Three male patients with AMN with an age range of 26-37 years participated in this study. All patients experienced increased muscle strength in the lower limbs at the end of the study versus baseline. The power grade increased by 25-43% at the baseline. In addition, all patients showed an improvement trend in walking speed measured with the T25FW test. Treatment with MSCs in patients with AMN appeared to be safe and well tolerated.
Discussion:
The results of this study demonstrated that intrathecal administration of WJ-MSC improves motor symptoms in patients with AMN. The current findings lend support to the safety and feasibility of MSC therapy as a potentially viable treatment option for patients with AMN.
Insights
Mesenchymal stem cell (MSC) therapy shows promise for X-linked adrenomyeloneuropathy (AMN). Intrathecal MSC treatment improved muscle strength and walking speed in AMN patients, indicating a safe and feasible therapeutic option.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Genetics
Background:
- X-linked adrenomyeloneuropathy (AMN) is a genetic neurodegenerative disorder caused by ABCD1 gene mutations.
- It leads to very long-chain fatty acids (VLFCA) accumulation, causing progressive neurological damage.
- Currently, no effective treatments exist for AMN.
Purpose of the Study:
- To evaluate the therapeutic potential of mesenchymal stem cell (MSC) transplantation in AMN patients.
- To assess the safety and efficacy of intrathecal Wharton's jelly-derived MSC (WJ-MSC) administration.
Main Methods:
- A small cohort, open-label study involving three male AMN patients (ages 26-37).
- Intrathecal administration of 20 × 10^6 WJ-MSCs three times over 4 months.
- Primary outcome: blinded assessment of lower limb muscle strength (MRC scale); secondary outcomes: timed 25-foot walk (T25FW) and VLFCA serum ratio.
Main Results:
- All patients demonstrated improved lower limb muscle strength, with a 25-43% increase in power grade.
- A positive trend in walking speed improvement was observed via the T25FW test.
- MSC treatment was found to be safe and well-tolerated in all participants.
Conclusions:
- Intrathecal WJ-MSC administration significantly improved motor symptoms in AMN patients.
- MSC therapy presents a potentially viable and feasible treatment option for AMN.
- Further research is warranted to confirm these promising findings.
