A new stem cell therapy for Parkinson’s disease is one step closer to human trials after scientists successfully recreated nerve damage in animal models.
Researchers at Scripps Research and Cardiff University used induced pluripotent stem cells (iPSCs) from the skin cells of two Parkinson’s patients to grow young neurons, which were then transplanted into mice with the degenerative disease. By just timing the growth of new cells, they are able to replace damaged neurons and potentially reverse the physical damage from nerve damage.
Although clinical research on stem cells is currently underway, this is the first clinical study using stem cells autologous therapywhich means the cells are taken from the person who eventually receives the transplant.
“This paper reports an important advance in the development of autologous cell replacement therapy for Parkinson’s disease,” said senior author Jeanne Loring, a professor in the Center for Regenerative Medicine at Scripps Research. “These results give us confidence that personalized treatments for Parkinson’s disease are feasible.”
Cells removed from the foreign body require additional treatment, which can be very difficult for the patient.
“When you transplant neurons derived from other people’s cells, those cells will be rejected by the immune system, requiring immunosuppressive drugs that are often poorly tolerated,” Lorraine added.
The researchers were also able to determine at which stage of cell growth the transplant needed to occur, thereby pinpointing the optimal moment in the genetic development of the iPSCs.
“At this earlier time point, cells are ready to become neurons, and when they’re placed in the brain, they receive signals to turn on these genes and complete their development,” said co-senior author Maria Lelos. “This allows them to bond with their host.” If they develop further, they no longer respond to those initial developmental signals.
While there is no cure for Parkinson’s disease, effective neurotherapies will be able to intervene in its progression and even reverse existing damage. Worldwide, approximately 10 million people suffer from the disorder, and options for treating symptoms that can rob patients of independence are very limited.
“Knowing which genes are activated in neuronal precursors that are at their optimal developmental state for treating Parkinson’s disease could help researchers screen cells before transplanting them into patients,” Lorraine said. “Gene expression analysis should greatly improve the likelihood of successful transplantation.”
The researchers believe this approach could also inform new treatments for Huntington’s disease, heart failure and age-related macular degeneration, using multifunctional “universal” iPSCs with broad cell differentiation potential.
The study was published in the journal Stem Cells and Development.