A radical new therapy for Parkinson’s disease will use stem cell transplants | Parkinson’s disease

Early next year, a radical new treatment for Parkinson’s disease involving tissue transplants will receive its first trial with patients – including a group from the UK.

Stem cells grown in the laboratory and transformed into nerve cells will be used to replace those destroyed by the disease. It is hoped that these will stop the spread of the debilitating symptoms.

“It has taken a long time to get to this point, but I hope the results of these trials will mean that in a few years we may be able to offer tissue grafts as standard treatments for Parkinson’s disease,” said Professor Roger Barker, from the University of Cambridge. . “It’s certainly a promising approach.”

In the UK, around 145,000 people live with Parkinson’s disease and around 18,000 new cases are diagnosed each year. The disease is triggered when nerve cells that supply dopamine to the brain begin to die due to a combination of genetic and environmental factors.

Dopamine helps a person control their movements. When supplies run low, it results in tremors, stiffness, depression, and other symptoms that can lead to wheelchair use or bed rest. The progression of the disease can be slowed by the drug L-dopa, which replaces some of the lost function of dopaminergic cells. Treatments become less effective over the years. Scientists have been searching for new approaches for years.

One idea has been to replace dying dopamine cells with unaffected versions, which has been tried by several centers around the world. This initially involved using tissue from aborted fetuses that had been donated for medical research.

Fetal tissue contains dopamine-producing cells that can supply the missing chemical, although at least six or seven fetuses are needed to supply enough material for a patient. In trials in Europe, these cells have been injected into the brains of patients with encouraging results. However, other trials in the United States found these treatments to be much less effective.

The use of tissue from aborted fetuses has been opposed by many people on religious grounds. It was also difficult to find sufficient supplies for widely used treatments. However, Barker and his Cambridge team – working in conjunction with scientists led by Professor Malin Parmar of Lund University in Sweden – have developed technology that avoids these problems.

The new approach uses stem cells, from which all cells with specialized functions are generated in the human body. These stem cells can be grown in laboratory cultures. Even better, scientists have learned how to turn them into dopaminergic cells. These will form the core of the transplants that will be performed next month.

“We now know that introducing dopaminergic cells into the brain will work and that the procedure is safe,” Barker said. “There is no longer a problem of supplying enough tissue because we can manufacture these cells in large numbers in the laboratory. The cost is relatively low. A supply of dopaminergic cells – made from stem cells – has become a standardized product and we don’t have contaminating cells, which you can get from fetal tissue.

“This means that we are now at a point where we can use stem cell transplants as treatments for patients with Parkinson’s disease, although it will take many years before we know these transplants work and can be used. as standard treatments for Parkinson’s disease.”

Trials will begin in the coming months and continue over the next year. There will be four Swedish participants and four British participants. “Cells are in a freezer and ready to go,” Barker said. “The transplants will be done in Sweden because they have the instruments to do it. This will be followed during the year with further trials.

Scientists expect their trials to take at least two years. They will be followed by a careful examination of the results and possible side effects. Provided these proceed satisfactorily, tissue grafts could be ready for wider use in about five years.

“Younger patients will benefit the most from this therapy,” Barker said. “It will be a one-time treatment, so the complications you get with chronic medications won’t occur, while these advanced therapies involving deep brain stimulation won’t be needed so often.”

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