Stem cell therapy to start human trials after reversing Parkinson’s disease in mice
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Stem cell therapy to start human trials after reversing Parkinson’s disease in mice.
Stem cell therapy shows great promise in many fields, but there is one application that scientists are particularly excited about: next-generation treatments for Parkinson’s disease.
An experimental group in the field has shown that implanting carefully cultivated stem cells into mice can lead to a dramatic return to the disease’s typical motor symptoms, and now they are setting their sights on upcoming human trials.
Parkinson’s disease is considered a prime target for innovative stem cell therapies because the disease can be traced to the degeneration of a specific type of cell in a specific area of the brain.
Neurons in the substantia nigra, a structure in the midbrain, are responsible for producing dopamine, which helps control activities such as movement.
The loss of these neurons is responsible for the motor symptoms in Parkinson’s disease patients, so using stem cell therapy to replace them is a very attractive idea, and one that has started to migrate from animal testing to humans.
In a world-first trial in Japan in 2018, Parkinson’s disease patients had stem cell-derived precursor cells implanted in their brains, where they developed into dopamine-producing neurons, and some subjects were said to do well .
Similar early-stage trials are currently underway in the United States, where scientists are working to determine the safety and efficacy of using induced pluripotent stem cells (iPS) to restore normal movement in Parkinson’s disease patients.
These iPS cells started out as harvested adult cells, treated with reprogramming factors, and returned to their embryonic state. The second stage involves exposing them to more growth factors, which in turn turn them into dopamine-producing neurons.
Scientists at Arizona State University have been experimenting with this process and testing their engineered neurons in mice in a proof-of-concept study.
The idea is to optimize the method of producing these cells, tweak them, and then see which version best improves Parkinson’s symptoms in rodents, and ideally brings the technology closer to clinical use.
This means that the stem cells were placed in different time periods during the second stage of reprogramming, with different cell populations being cultured for 17, 24 or 37 days.
As a result, the team found that the 17-day stem cells were significantly better than other types of stem cells, and when the stem cells were transplanted into the brains of mice, their motor symptoms were significantly restored.
In addition, dosage was found to be key, with a small number of stem cells having negligible effects, while a large number of stem cells entered the brain and bound to neural tissue, forming synapses and producing dopamine.
Ultimately, this allowed the treated mice to completely reverse the symptoms of Parkinson’s disease.
Following these promising results, the technology will now join a handful of similar technologies being studied in humans in what is considered a first trial.
It will focus on a specific group of people with Parkinson’s disease, who are known to have mutations in the Parkinson’s gene.
These patients experienced a decline in motor symptoms but no cognitive decline or dementia, which scientists say makes them ideal candidates for testing.
Jeffrey Kordower, author of the paper on the study, said: “We are very excited about the opportunity to help people with this inherited form of Parkinson’s disease, but the lessons learned from this trial will also directly impact people living with the disease. of patients with sporadic or non-genetic forms.”
Stem cell therapy to start human trials after reversing Parkinson’s disease in mice
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