Parkinson’s Potential Treatment Enters in Phase I of Clinical Trials

Recently, researchers at Kyoto University in Japan published their findings of Phase I of clinical trials launched in 2018 on a small group of human subjects. Patients received bilateral transplants of dopaminergic (DA) progenitors derived from induced pluripotent stem (iPS) cells. The investigator initiated an open-label, single-center trial to determine the safety and efficacy of striatal transplantation.

Using a neurosurgical navigation system, the product containing the DA progenitors is transplanted into the putamen. RT-qPCR analysis confirmed that iPS cells differentiated into a final product containing 60% DA progenitors and 40% DA neurons, while no serotonergic neuron cell markers were detected.

After 24 or 32 weeks, no tumour-like overgrowth or adverse effects were recorded. Side effects were very mild in nature, and based on the MRI scans, the treatment seems comparatively safe. There was a noteworthy overall improvement in motor function. The researchers state that further research is necessary to substantiate the safety and efficacy of the treatment, but so far, it shows promising results. Results demonstrate an overall beneficial effect on the MDS—UPDRS, which is a Unified Parkinson’s Disease Rating Scale.

The road and journey ahead…

Therefore, based on the results, the clinical trial can be given a green light for Phase II and ahead. The overall efficacy and safety of that will ultimately seal the fate of the treatment in the long run. Whether the treatment ever makes it into the market as an effective and cost-efficient option will be of particular concern.

Parkinson’s disease is a neurodegenerative disorder that affects the central nervous system. The loss of dopaminergic neurons in substantia nigra causes Parkinson’s disease. These neurons in the mid-brain and areas like the hypothalamus and the olfactory bulb play a crucial role in various brain functions. The human body uses dopamine as the primary neurotransmitter for motor and non-motor functions like reward, motivation, and cognition. Hence, treatment and diagnosis for Parkinson’s disease are widely studied across the world.

Finally reprieve from the slow, silent killer?

Symptoms in Parkinson’s disease start slowly, then gradually progress over time. Currently, there’s no cure for the disease, only management and treatment to slow down the symptoms. Once healthy individuals face symptoms such as tremors, bradykinesia, rigidity/stiffness, speech and posture changes, poor balance, loss of automatic movements, and other non-motor symptoms. Even though medical treatment alleviates symptoms of the early stages, continued use leads to further complications. Other alternative treatment options lack efficacy, have added side effects, and ethical issues render them unusable.

If the treatment proves viable, it will help about a staggering 1% of the population over 60 years. In India, Parkinson’s disease has the highest absolute prevalence of 15 to 43 per 1 lakh population. Whereas, in the USA alone, about 1 million people live with this disorder, most after Alzheimer’s disease. This number is projected to be a staggering 1.2 million by 2030. In a similar study published the same day, scientists in the USA reached similar conclusions. According to the team, “Although Phase I studies are rarely designed to determine the efficacy, there is indeed room for improvement”. The finish line is still far in the distance, meanwhile race towards it continues. The faster a treatment is found, all those suffering can breathe a sigh of relief.