What is a Parkinson’s disease?
Parkinson’s disease is a progressive neurodegenerative disorder characterized by the gradual loss of dopamine-producing neurons in the brain. This leads to a decline in motor control and other symptoms that worsen over time. The disease affects approximately 1 million people in the United States and 6 million globally.
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How does Parkinson’s disease develop?
The human brain contains approximately 100 billion neurons, but only about half a million of these are dopamine-producing cells. These dopaminergic neurons are among the largest cells in the body, with some extending several meters. Each of these cells can form two to ten million synapses with other neurons, creating an extensive network.
Due to their size and the vast number of connections they form, dopaminergic neurons require substantial energy to function. However, the process of energy production generates waste products, such as oxygen free radicals, which can damage cells.
Additionally, waste accumulation within these cells can impair protein function, ultimately affecting the cells’ ability to operate efficiently. These sick dopaminergic neurons give rise to symptoms of Parkinson’s disease.
Recent therapies to treat Parkinson‘s Disease
The drug used to treat Parkinson’s disease is called Levodopa. While it is not a cure, it serves as a maintenance therapy that eventually loses its effectiveness over time.
Leveodopa works by converting into dopamine in the brain, which helps replenish the diminished levels of this neurotransmitter in individuals with Parkinson’s disease. This conversion process is crucial because dopamine itself cannot cross the blood-brain barrier, but levodopa can.
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A new stem-cell therapy to treat Parkinson’s disease
Parkinson’s disease is particularly suitable for stem-cell therapy because it involves the degeneration of a specific type of neuron (dopaminergic neurons) located in a distinct region of the brain known as the putamen.
Dr. Viviane Tabar and Dr. Lorenz Struder, physician-scientists at Memorial Sloan Kettering Cancer Center (MSK), have developed a treatment for Parkinson’s disease that involves transplanting nerve cells developed from embryonic stem cells into the patients. This study has successfully completed a phase 1 clinical trial and has gotten approval for the phase 3 trial.
This study involved transplanting dopamine-producing neurons, derived from embryonic stem cells, into 12 Parkinson’s patients.
In Parkinson’s patients, the loss of dopamine-producing neurons leads to symptoms such as tremors, slowness, stiffness, and imbalance in gait. Dopamine is crucial for coordinating movement and maintaining balance. The goal of this therapy is to increase the number of dopamine-producing cells in the brain.
Over the course of 18 months, follow-up observations revealed that the transplanted cells successfully integrated into the brain without any significant side effects. The patients showed significant improvement in Parkinson’s-related symptoms and appeared more stable.
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The FDA has approved the progression to phase 3 clinical trials, which will involve 100 patients divided into two groups: a study group and a placebo group. This trial is expected to begin in the first half of 2025.
Improvement in patients motor skills after the treatment
This study focused on the improvement of motor symptoms in patients. Typically, these symptoms worsen each year, leading to an increase in scores by a few points. However, in this study, not only did the scores not increase, but they actually dropped by 20 points in the highest dose group.
Additionally, patients experienced an improvement in daily functioning, gaining an extra 2.7 hours of effective functioning each day after the therapy, which is a significant enhancement.
While there are still many unknowns, the results are quite remarkable.
The future of this stem-cell research
Dr. Tabar, the lead researcher in the clinical trial, emphasized that this is a critical step towards regenerative brain repair. The treatment is the result of two decades of rigorous collaborative work. While it may seem unusual for a cancer center to pursue regenerative medicine for a neurodegenerative disease, the ability to replenish cells lost to disease and rebuild brain circuitry could extend to other treatments, ultimately benefiting cancer patients as well.
Dr. Tabar and their team have developed a method to transform embryonic stem cells into dopamine-producing neurons in large batches. These identical cells, called bemdaneprocel, can be frozen until they can be transplanted to the patient.
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