RACTHERA Co., Ltd. Dopaminergic(CT1-DAP001/DSP-1083)

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Parkinson’s disease is a disorder that mainly causes motor symptoms, including resting tremor (shaking while at rest), bradykinesia (slowness of movement), muscle rigidity (stiffness), and postural instability (difficulty maintaining balance, leading to falls). It often begins after the age of 50, and the number of patients increases with age.
The main cause of this disease is the progressive degeneration (loss) of dopaminergic neurons in the substantia nigra, a region of the midbrain. Dopamine supplied by these neurons to the striatum is essential for regulating movement. When dopamine levels drop, various movement problems appear. Furthermore, Parkinson’s disease also affects other neural systems, leading to non-motor symptoms such as autonomic dysfunction (constipation, dizziness), depression, sleep disturbances, and cognitive decline. These symptoms occur frequently and can significantly affect daily life.

About Parkinson’s Disease

Current Treatments

The main approach to treating movement symptoms is medication that compensates for the lack of dopamine. The most effective medication is levodopa, a precursor of dopamine, which is widely used. For the first 3-5 years after onset, patients often experience what is called the “honeymoon period,” during which medications work well and daily life is almost unaffected.

However, as the disease progresses, the effect of medication becomes less stable. Patients may experience “wearing-off” (when the drug’s effect fades before the next dose) or dyskinesia (involuntary twisting or writhing movements when medication is active). These motor complications make it harder to keep symptoms under control.
When this happens, device aided therapies that require surgery may be considered. A typical example is deep brain stimulation (DBS), in which electrodes are implanted in specific brain regions involved in movement control, and electrical stimulation is used to improve symptoms. Unfortunately, these treatments only relieve symptoms. They cannot stop the loss of dopaminergic neurons or restore lost function. As a result, the disease inevitably continues to progress.

Our Goal with Regenerative
and Cellular Therapy

By implanting dopaminergic progenitor cells derived from iPS cells into the striatum, we aim to replace lost dopaminergic neurons and restore their function. Once these implanted cells integrate into the brain, they can produce and supply dopamine and also synthesize dopamine from levodopa, enhancing the effect of existing medications.
Through this cell implantation, we hope not only to improve motor function but also to partially reverse disease progression, extending the period during which medications remain effective and helping patients maintain a better quality of life for longer.

Improvement of Motor Symptoms