STEM-PD

A first-in-human trial to test the safety and feasibility of using a stem cell product in people with Parkinson’s disease.

Research summary

Parkinson’s disease (PD) occurs when an area of the brain begins to lose nerve cells that produce a chemical called dopamine. Dopamine is an important chemical, and one of its functions is that it helps to regulate body movement. The loss of these nerve cells leads to a reduction of dopamine in the brain. Medications used to treat PD temporarily replace this lost dopamine, but they do not repair the underlying disease.

One of the most promising PD therapies to date has been the transplantation of dopamine producing cells into the brain. Unlike current treatments, these therapies may be able to repair the damage caused in PD. In this trial, we will transplant a new stem cell therapy, called the STEM-PD product, into the area of the brain affected in people with PD. These stem cells can develop into many different cell types, including dopamine-producing nerve cells. We will transplant the stem cells using a device that has been previously used for similar transplants in Lund. This is the first time that the STEM-PD product will be given to humans.

The trial aims to assess whether the STEM-PD product is safe to use in people with PD. We will also be looking for preliminary signs of efficacy.

The trial will recruit participants with PD from the UK and Sweden. Eight participants will undergo the STEM-PD product transplant. Participants will receive a single dose of the STEM-PD product. Participants will attend for 23 visits primarily at their local recruiting hospital. For participants from the UK, some of the imaging will be performed locally in Cambridge and some imaging will be performed in Lund. All participants will be followed up for 36 months following surgery.

More information about the STEM-PD trial can be found at https://stem-pd.org/.

Main inclusion criteria

  • Have given written informed consent to participate in the trial
  • Diagnosed with Parkinson’s disease (PD) as defined using Queens Square Brain Bank criteria
  • Moderate disease as defined by having Hoehn and Yahr stage 2-3 in OFF state
  • Disease duration > 10 years
  • Male or female, aged between 50 and 75 years (inclusive)
  • Have a significant response to dopamine (DA) therapies
  • Have symptoms that are not appropriately controlled by existing oral anti-PD medications
  • Ability to travel to Lund for surgery
  • Followed up for at least 12 months prior to inclusion in this trial in the TransEUro observational study
  • Be fluent in English/Swedish to enable completion of questionnaires
  • Be approved by the Trial Management Group (TMG) clinical sub-group for trial participation

Main exclusion criteria

  • Tremor dominant disease
  • Significant drug induced dyskinesias as defined by a score of > 2 in the Abnormal Involuntary Movement Scale (AIMS) dyskinesias rating scale, in any body part in the ON state
  • Ongoing major medical or psychiatric disorders, including depression (Montgomery-Åsberg Depression Rating Scale [MADRS] > 20) and psychosis, that make participation unsuitable
  • Any contraindication to neurosurgery
  • Unable to be imaged using MRI
  • Extensive ventral striatal loss or normal findings on fluorodopa positron emission tomography (PET) at screening
  • Significant cognitive impairment indicative of an incipient dementia/established dementia or values consistent with Montreal Cognitive Assessment (MoCA) score of ≤ 24
  • Unable to perform normal copying of interlocking pentagons and/or a semantic fluency score for naming animals of less than 20 over 90 seconds
  • Other concomitant treatment with neuroleptics (including atypical neuroleptics) and/or cholinesterase inhibitors
  • Previous neurosurgery to the brain, or cell or organ transplantation, or recipient of repeated blood transfusions
  • Any contraindication to immunosuppressive therapy, prophylactic antibiotics and/or osteoporosis prophylaxis
  • High levels of pre-formed specific anti-human leukocyte antigen (HLA) antibodies to the cell product
  • Severely reduced thiopurine methyltransferase (TPMT) activity (less than half of the lower normal TPMT activity level)
  • History of documented severe/significant allergy requiring treatment
  • Female who is pregnant or breastfeeding
  • Received an investigational drug (including investigational vaccines) or used an invasive investigational medical device within 4 weeks of the screening visit, or is currently enrolled in an interventional investigational trial
  • Female of childbearing potential or male unwilling to follow contraception requirements
  • Any other condition which, in the opinion of the investigator, makes the patient inappropriate for entry into the trial

Funders and sponsors

Chief investigator

Professor Roger Barker

Contact details

Clinical Trials Coordinator: Bronwen Harry

Email:[email protected]