Bayer (BAYGn.DE) subsidiary BlueRock has become the first company to report initial success treating Parkinson’s disease in humans using an experimental stem cell therapy, the drugmaker said on Wednesday.

The hunt for Parkinson’s treatments has seen many setbacks over decades.

Bayer said that one year into a Phase I trial with 12 volunteers, BlueRock’s therapy was shown to be well-tolerated and that transplanted cells grew as intended in patients’ brains.
This encouraged it to advance testing on humans to the second of three stages, with patient enrolment seen in the first half of 2024.

A slew of research projects around the globe have recently honed in on the approach to transplant modified cells to restore an area of the brain that normally produces dopamine.

Some of this work is done by Britain’s’ Cambridge University, South Korea’s Bundang CHA Hospital, International Stem Cell Corp’s (ISCO.PK) Cyto Therapeutics in Australia, the Chinese Academy of Sciences, Harvard University and Japan’s Kyoto University Hospital.

For BlueRock’s experimental therapy, researchers took induced pluripotent stem cells, which are modified to regain the ability to form any type of specialised tissue, and transformed them into dopamine-producing nerve cells.
When surgically implanted into the brain of a person with Parkinson’s disease, the therapeutic cells are designed to restore neural networks destroyed by the disease.

Initial trial results showed the cells multiplied and started making dopamine, an important brain signalling molecule which is lacking in Parkinson’s patients.

Parkinson’s, for which there is no cure and which affects more than 10 million people worldwide, causes progressive brain damage. Common symptoms are loss of muscle control, tremors, muscle rigidity and slowness of movement while dementia is seen in some patients.

Bayer acquired BlueRock Therapeutics and Asklepios Biopharmaceutical in 2019 and 2020, respectively, in a push to build a cell and gene therapy business,

Bayer last year struck a partnership deal with Mammoth Biosciences in the San Francisco Bay area, co-founded by Nobel laureate Jennifer Doudna, to develop therapeutic tools based on CRISPR/CAS9 gene editing.

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