Hope on the Horizon: Stem Cell Therapy for Parkinson's Disease and it's atypical variant

Parkinson’s Disease (PD) is the second largest affecting neurodegenerative disease in the world and there are around eight million people living with Parkinson’s disease globally. This is? a disease which involves loss of dopamine producing dopaminergic (DA) neurons in the substantia nigra pars compacta,? a region in the ventral midbrain due to which the patients with PD exhibit severe symptoms that are associated with decreased motor function, resting tremors, bradykinesia, rigidity, postural instability, and gradual decline of autonomic, cognitive functions.

Bone marrow stem cells have the characteristic ability to differentiate into different types of cells and one among that is the ability to differentiate into dopaminergic neuron cells. Recent Researches have confirmed Mesenchymal Stem Cells (MSCs) as a potential treatment for PD. MSCs, found in bone marrow and fat tissue, have shown promising results in early clinical trials.

In a phase I controlled, randomized clinical study (NCT01824121 Phase I & II), patients? suffering from progressive PD were administered autologous BMSCs via intra-arterial injection at 1.2 - 2 Million stem cells? per kg dosage. The results demonstrated that autologous BMSCs are safe and reduce disease progression. After a year, these patients showed no worsening of symptoms and some even experienced improvement in their symptoms. All the evaluable patients had a stable Unified Parkinson's Disease Rating Scale (UPDRS) score at the 12 months follow-up. Apart from this, patients with Progressive Supranuclear Palsy, a sporadic atypical Parkinsonian syndrome, the Progressive Supranuclear Palsy Rating scale (PSP-RS) also demonstrated disease stabilization at 6 and 12 months in all the evaluable patients, except one. Another important positive effect was recorded by ?Hoehn and Yahr (H&Y) staging, which remained stable over time.

In another clinical study by Venkataramana NK, et al seven patients aged between? 22 to 62 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). 3 out of 7 patients have shown a steady improvement in their UPDRS score with an improvement of 38% from the baseline and similar improvements from 2.7 and 2.5 in H&Y along with 14% improvement in? Schwab and England (S&E) scores, respectively. A subjective improvement was found in symptoms like facial expression, gait, and freezing episodes. Two patients have significantly reduced the dosages of Parkinsonian drugs.

Though the exact mechanism of action is still undergoing research, MSCs are believed to either develop into dopamine-producing cells or create an environment that protects remaining brain cells. These findings suggest MSCs could be a safe and effective therapy for Parkinson's disease, offering hope for improved quality of life for patients.

References

1)? Giordano R, et al. Autologous mesenchymal stem cell therapy for progressive supranuclear palsy: translation into a phase I controlled, randomized clinical study. J Transl Med. 2014;12:14.

2)? Canesi M, et al. Finding a new therapeutic approach for no-option Parkinsonism: mesenchymal stromal cells for progressive supranuclear palsy. J Transl Med. 2016;14(1):127.

3) ? Venkataramana NK, et al. Open-labeled study of unilateral autologous bone-marrow-derived mesenchymal stem cell transplantation in Parkinson’s disease. Transl Res. 2010;155(2):62–70.