A new stem cell therapy for Parkinson’s disease is one step closer to human trials after scientists were able to successfully rebuild neural damage in an animal model.
Researchers from Scripps Research and Cardiff University used induced pluripotent stem cells (iPSCs) from skin cells of two people with Parkinson’s disease to grow young neurons, which were then transplanted into rats with the degenerative condition. By timing the growth of the new cells just right, they’re able to replace the damaged neurons and potentially reverse the physical impairments that come with the neural damage.
While there are current stem cell clinical studies underway, this is the first one to make use of autologous therapy, which means the cells are harvested from the person who will ultimately receive the transplant.
“This paper reports important progress toward development of an autologous cell replacement therapy for Parkinson’s disease,” said senior author Jeanne Loring, professor at the Center for Regenerative Medicine at Scripps Research. “These results give us confidence that personalized therapy is feasible for Parkinson’s disease.”
Cells taken from foreign bodies require additional treatment, which can be very difficult on the patient.
“When you transplant neurons derived from someone else’s cells, those cells will be rejected by the immune system, requiring the use of immunosuppressive drugs that are often not well tolerated,” Loring added.
The researchers were also able to determine at which stage in cell growth the transplant needed to occur, pinpointing the goldilocks moment in the iPSCs’ genetic development.
“At this earlier time point, the cells are poised to become neurons, and when they are put into the brain, they receive the signals to turn on those genes and finish their development,” said co-senior author Mariah Lelos. “This allows them to make connections with the host. “If they’re farther along in development, they no longer respond to those initial developmental signals.”
While there’s no cure yet for Parkinson’s disease, effective neural therapy would be able to intervene in its progression and even reverse existing damage. Worldwide, around 10 million people are living with the disease and there are very limited options for treating the symptoms that can rob sufferers of their independence.
“Knowledge of which genes are turned on in neuronal precursors that are in the optimal developmental state to treat Parkinson’s can help researchers screen cells before transplanting them into patients,” Loring said. “The gene expression analysis should greatly improve the probability of successful transplants.”
Using the versatile ‘universal’ iPSCs, which have a broad range of cell differentiation potential, researchers believe this therapy could also inform new treatments for Huntington’s disease, heart failure and age-related macular degeneration.
The research was published in the journal Stem Cells and Development.
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