Behind the scenes of a novel stem cell therapy for Parkinson’s Disease

Parkinson’s Disease is a neurodegenerative disease characterized by the loss of normal motor control, leading to rigidity, slow movements, tremor, and difficulties walking. The disease affects approximately 1% of people above the age of 60 and, with an aging population, its prevalence is increasing. Parkinson’s Disease, and many of the symptoms associated with it, is caused by the loss of a particular type of dopamine-producing nerve cell in the brain, located in a brain region called the substantia nigra.

Today, Parkinson’s is treated symptomatically with daily doses of medications, such as levodopa, which, with time, lose efficacy and can cause severe side effects. However, some researchers have proposed that instead of medications, the disease may be treatable through stem cell transplantation.

In October 2022, following 13 years of preparatory work, a novel stem cell transplantation product, STEM-PD, was approved by the Swedish regulatory authorities for a Phase I/IIa clinical trial in Parkinson’s Disease, and the first patient in this trial was dosed with stem cells in February 2023. This is the first time in Europe a stem cell therapy of this type is being tested in Parkinson’s Disease, representing a significant leap forward for the clinical implementation of stem cell therapies.

This is an important step towards bringing stem cell therapies for Parkinson’s Disease to patients. Such therapies have the potential to reform the way we treat the disease,” says Associate Professor, Agnete Kirkeby, who led the preclinical development of the product.

Now, the group behind the development of the stem cell product has released a behind-the-scenes article describing the in vitro and in vivo tests conducted on the product to achieve regulatory approval for transplantation to the first patients. In the case of trials conducted by pharmaceutical companies, such data is often hidden to the public, however, for the STEM-PD researchers, it was important that their data was made publicly available.

Releasing such data provides an unusual window into the comprehensive and rigorous testing needed to bring novel stem cell therapies into clinical trial. This is highly valuable information for other scientists in the field and may hopefully help to speed up clinical development of stem cell therapies also for other diseases,” says Dr. Kirkeby.

If the outcome of this initial academic clinical trial demonstrates safety of the treatment after 12 months, research partner Novo Nordisk A/S is planning to conduct further trials and product development, with the potential of bringing the STEM-PD product to the market.

The full paper for the preclinical work on STEM-PD was published on October 5th 2023 in the journal Cell Stem Cell and is entitled: “Preclinical Quality, Safety and Efficacy of a Human Embryonic Stem Cell Derived Product for the Treatment of Parkinson’s Disease, STEM-PD.

More information on the STEM-PD trial and STEM-PD team can be found here:

www.stem-pd.org

https://www.lunduniversity.lu.se/article/first-patient-receives-milestone-stem-cell-based-transplant-parkinsons-disease

https://www.lunduniversity.lu.se/article/nerve-cells-could-transform-treatment-parkinsons

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