Active not recruitingPHASE1INTERVENTIONAL

A Trial to Determine the Safety and Tolerability of Transplanted Stem Cell Derived Dopamine Neurons to the Brains of Individuals With Parkinson's Disease

This trial is looking at a new way to treat Parkinson's disease using special cells called stem cells. In Parkinson's, the brain loses cells that produce a chemical called dopamine, which helps control movement. Current medicines replace dopamine temporarily, but don't fix the underlying problem. This new treatment, called STEM-PD, involves putting stem cells into the brain. These stem cells are designed to grow into dopamine-producing cells, potentially repairing the damage. This is the first time this specific stem cell treatment will be used in humans. Researchers want to check if it's safe and if it shows early signs of being effective in people with Parkinson's, aiming to improve their movement and quality of life.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

Region Skane

Enrolment target

Start

30 Nov 2022

Estimated completion

01 Nov 2027

Parkinson Disease

What is this study about?

Parkinson's disease happens when certain nerve cells in your brain, which make a chemical called dopamine, start to die. Dopamine is really important because it helps your brain tell your body how to move smoothly. When you lose these cells, you have less dopamine, and that's what causes the movement problems seen in Parkinson's.

At the moment, medicines for Parkinson's help by temporarily boosting your dopamine levels. However, they don’t fix the broken cells or stop more from dying. This new study is exploring a different approach: transplanting special cells, called stem cells, directly into the part of the brain affected by Parkinson's. These stem cells have the potential to grow into new, healthy dopamine-producing nerve cells, which could help repair the damage and potentially improve your symptoms.

This is the very first time this particular stem cell treatment, called STEM-PD, will be used in people. The main goals are to find out if it's safe to use and to see if there are any early hints that it could help people with Parkinson's. If successful, this kind of treatment could offer a more lasting solution than current medications by actually replacing the cells that have been lost.

Key takeaways

This is a new stem cell treatment for Parkinson's aimed at repairing brain damage.
It's the first time this specific treatment will be used in humans, focusing on safety.
The study involves brain surgery to implant the stem cells.
Participants will be closely checked for three years after the surgery.
It's not a cure but aims to improve movement and reduce symptoms.
Only a small number of participants will be included in this first stage of the trial.

Who may be eligible?

To be considered for this study, you would need to be between 50 and 75 years old and have been diagnosed with Parkinson's disease using specific UK criteria. Your Parkinson's should be at a moderate stage, and your symptoms should respond well to your current Parkinson's medication. However, your current medications shouldn't be fully controlling your symptoms, meaning there's still room for improvement.

You also need to have been part of another study called TransEUro for at least 12 months before this trial. You must be able to travel to Lund in Sweden for the surgery, and you should be able to speak and understand English or Swedish well enough to fill out questionnaires.

However, you wouldn't be able to join if you have a lot of shaking (tremor-dominant Parkinson's), experience severe uncontrolled movements (dyskinesia) from your medication, or have other serious long-term health problems, including mental health conditions like severe depression or psychosis. Other reasons for not being able to join include if you've previously had brain surgery, can't have an MRI scan, or have significant memory problems.

Quick self-check

Are you between 50 and 75 years old?
Do your Parkinson's symptoms improve with your current medication, but aren't fully controlled?
Can you travel to Sweden for surgery and some appointments?
Can you understand and speak English or Swedish well?
Have you been part of the TransEUro study for at least 12 months?
Do you feel you have good overall health, without other major medical or mental health issues?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you're one of the eight people chosen for this study, you will receive a single transplant of the STEM-PD cells into your brain. The surgery will happen in Lund, Sweden. You’ll have appointments before and after the surgery in Lund, and some special scans in London if you're from the UK.

Most of your follow-up appointments, which total 25 visits, will be at your local hospital in the UK or Sweden. You will be closely monitored for 36 months (that's three years) after your surgery to check your health and how you are responding to the new treatment.

Potential risks and benefits

This is an early-stage study, so the potential benefits are currently unknown, but researchers hope it could lead to new ways to treat Parkinson's by replacing damaged brain cells. As with any new treatment, there are potential risks, including those linked to brain surgery. These will be fully explained by the study team. You have the right to withdraw from the study at any time without giving a reason, and your medical care will not be affected.

Locations (2)

Region Skåne - Skåne University Hospital
Lund, Sweden
Cambridge University Hospitals NHS Foundation Trust
Cambridge, United Kingdom

Common questions

What is Parkinson's disease?

Parkinson's disease is a condition where specific cells in the brain that produce dopamine, a chemical that helps control movement, stop working properly and die. This leads to symptoms like tremors, stiffness, and slow movement.

What are stem cells?

Stem cells are special cells in the body that have the ability to turn into many different types of cells. In this study, they are hoping to use them to create new dopamine-producing cells for the brain.

Will this cure Parkinson's disease?

This is a very early study (Phase 1) focused on checking if the treatment is safe. It's too soon to say if it will cure Parkinson's, but researchers hope it could lead to significant improvements in symptoms.

Where does the surgery take place?

The surgery to implant the stem cells will take place in Lund, Sweden.

How long will I be followed up after the treatment?

You will be followed up by the study team for three years (36 months) after the surgery.