Taking a new cell therapy to a trial in patients with Parkinson’s disease
Parkinson’s disease is a common disease in which nerve cells in different parts of the brain die prematurely, causing tremors, progressive weakness and difficulty walking and moving. It affects approximately 20-30,000 people in the UK.
This loss of nerve cells (‘neurons’) includes some that produce a chemical called ‘dopamine’. Medications that replace dopamine help treat the symptoms of Parkinson’s disease.
However, these drugs create side effects in the long term and do not treat the underlying disease, meaning it continues to progress despite these medications.
We wanted to see if we could make dopamine neurons from human stem cells, which in theory able to develop into all cell types, including brain cells.
Working with a team in Sweden, we have developed a product called STEM-PD, in which stem cells have been engineered to make brain cells (dopamine neurons) of the type lost in Parkinson’s disease.
This new product has now been tested in the laboratory and shown to be safe, with signs that it can help with features of Parkinson’s disease. Furthermore, we can manufacture these cells very efficiently, making this attractive for companies to develop should early human trials, including our own, show that transplanting these dopamine neurons works and is safe.
If successful, this treatment could remove the need for drugs and target the underlying cause of Parkinson’s disease, not just the symptoms.
We are now undertaking a first-in-human trial of STEM-PD in Cambridge and Lund, Sweden.
Eight people with Parkinson’s disease will receive transplants of STEM-PD into the brain and be followed up for 36 months to check that it is safe and assess whether it helps their symptoms.