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Personalised treatments for Parkinson’s disease

16 January 2019

Cells

Scientists at Cardiff University are helping to bring personalised treatments for Parkinson’s disease closer to the clinic, thanks to a major investment of over £50,000 from American charity, The Summit for Stem Cell Foundation.

Working in partnership with Professor Jeanne Loring of the Scripps Research Institute, Cardiff University’s Dr Mariah Lelos is testing the potential of alternative dopamine cell therapies to reduce the symptoms of Parkinson’s disease.

“Parkinson’s disease causes dopaminergic cells in the brain to die, which in turn can cause a wide range of motor and non-motor symptoms in patients. One strategy to reduce these symptoms is to replace the missing dopamine in the brain, using cell replacement therapies,” explained Dr Lelos.

“However, there is a limited supply of the gold-standard foetal tissue needed for this treatment. What’s more, while both foetal tissue and new stem cell-derived therapies work well, they require the patient to take immunosuppression drugs, which can be detrimental to their health.”

One potential solution is to use cells taken directly from the patient (known as ‘induced-pluripotent stem cells’, or iPSCs), and transform them into dopamine cell therapy. This iPSC-derived treatment is tailored to each patient and avoids the use of immunosuppressive drugs, which means that the therapy should be safe with no side effects.

The team hope to determine whether two potential iPSC-derived dopamine cell therapy products can survive in a rodent model of Parkinson’s disease and improve motor impairments.

Initial results show potential, with cells kept in culture for 18 days being able to survive in rodents and successfully alleviating the motor impairments associated with Parkinson’s disease.

Professor Jeanne Loring of the Scripps Research Institute, praised the results of this collaborative research project: “Further analysis is ongoing, but these results indicate that cells from Parkinson’s patients could potentially be used to develop an effective dopamine cell therapy that alleviates the symptoms of the illness. We are excited that our collaboration with Dr Lelos’ team has given us a great step forward in our development of patient-matched dopamine neuron replacement therapy for Parkinson’s disease.”

Dr Lelos added: “Stem cell-derived therapies hold real promise for people with Parkinson’s disease. Professor Jeanne Loring’s lab is developing an exciting new cell therapy and, by working together, we are delighted to be able to demonstrate how incredibly well this treatment works in our model of Parkinson’s disease. These important results move personalised medicine for Parkinson’s patients one step closer to the clinic.”

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