Open-labeled study of unilateral autologous bone-marrow-derived mesenchymal stem cell transplantation in Parkinson's
Neelam K Venkataramana1, Satish K V Kumar, Sudheer Balaraju
1Advanced Neuroscience Institute, BGS-Global Hospital, Bangalore, India. drnkvr@gmail.com
Abstract:
Parkinson's disease (PD) is a progressive neurodegenerative disease for which stem cell research has created hope in the last few years. Seven PD patients aged 22 to 62 years with a mean duration of disease 14.7+/-7.56 years were enrolled to participate in the prospective, uncontrolled, pilot study of single-dose, unilateral transplantation of autologous bone-marrow-derived mesenchymal stem cells (BM-MSCs). The BM-MSCs were transplanted into the sublateral ventricular zone by stereotaxic surgery. Patients were followed up for a period that ranged from 10 to 36 months. The mean baseline "off" score was 65+/-22.06, and the mean baseline "on" score was 50.6+/-15.85. Three of 7 patients have shown a steady improvement in their "off"/"on" Unified Parkinson's Disease Rating Scale (UPDRS). The mean "off" score at their last follow-up was 43.3 with an improvement of 22.9% from the baseline. The mean "on" score at their last follow-up was 31.7, with an improvement of 38%. Hoehn and Yahr (H&Y) and Schwab and England (S&E) scores showed similar improvements from 2.7 and 2.5 in H&Y and 14% improvement in S&E scores, respectively. A subjective improvement was found in symptoms like facial expression, gait, and freezing episodes; 2 patients have significantly reduced the dosages of PD medicine. These results indicate that our protocol seems to be safe, and no serious adverse events occurred after stem-cell transplantation in PD patients. The number of patients recruited and the uncontrolled nature of the trial did not permit demonstration of effectiveness of the treatment involved. However, the results encourage future trials with more patients to demonstrate efficacy.
Insights
This pilot study explored autologous bone-marrow-derived mesenchymal stem cells (BM-MSCs) for Parkinson's disease (PD). While safe, the study showed promising but inconclusive results for PD symptom improvement, warranting further investigation.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Clinical Neurology
Background:
- Parkinson's disease (PD) is a progressive neurodegenerative disorder with limited treatment options.
- Stem cell research, particularly using autologous bone-marrow-derived mesenchymal stem cells (BM-MSCs), offers potential therapeutic avenues for PD.
- Current treatments for PD primarily manage symptoms but do not halt disease progression.
Purpose of the Study:
- To evaluate the safety and potential efficacy of unilateral transplantation of autologous BM-MSCs into the sublateral ventricular zone in PD patients.
- To assess the impact of BM-MSC transplantation on motor and non-motor symptoms of Parkinson's disease.
- To explore changes in medication requirements and overall quality of life post-transplantation.
Main Methods:
- A prospective, uncontrolled, pilot study involving 7 PD patients (aged 22-62) with a mean disease duration of 14.7 years.
- Stereotaxic surgery was used for single-dose, unilateral transplantation of autologous BM-MSCs into the sublateral ventricular zone.
- Patients were followed for 10-36 months, with assessments including Unified Parkinson's Disease Rating Scale (UPDRS), Hoehn and Yahr (H&Y), and Schwab and England (S&E) scores.
Main Results:
- Three out of seven patients demonstrated steady improvement in "off" and "on" UPDRS scores, with mean reductions of 22.9% and 38%, respectively.
- Improvements were also observed in H&Y and S&E scores, alongside subjective symptom relief (facial expression, gait, freezing).
- Two patients were able to significantly reduce their PD medication dosages; no serious adverse events were reported.
Conclusions:
- Autologous BM-MSC transplantation appears to be a safe procedure in PD patients, with no serious adverse events observed.
- The pilot study suggests potential therapeutic benefits, including symptom improvement and reduced medication needs, although effectiveness could not be definitively demonstrated.
- Larger, controlled clinical trials are warranted to establish the efficacy of BM-MSC transplantation as a treatment for Parkinson's disease.
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