#New treatment for Parkinson’s may be found in a brain protein called GDF5

#New treatment for Parkinson’s may be found in a brain protein called GDF5

Parkinson’s disease, a brain disorder that affects over 10 million people worldwide, is caused by the gradual loss of dopamine neurons. The loss of these neurons leads to involuntary tremors, stiffness, and balance problems. While there are drugs to treat these symptoms, no drugs exist to slow the progression of the disease. However, we found a brain protein that may be able to prevent the loss of dopamine neurons. This discovery could be important for developing treatments.

For many years, scientists have been investigating the use of neurotrophic factors to slow the progression of Parkinson’s disease. These proteins are normally found in the brain and play an important role in protecting and nurturing different types of neurons, including dopamine neurons, which are critical for controlling movement.

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In 1993, one neurotrophic factor, called glial cell line-derived neurotrophic factor (GDNF), was found to protect dopamine neurons in laboratory tests. Following extensive laboratory studies in which GDNF displayed many benefits, clinical trials were started in the early 2000s.

In these trials, GDNF was administered directly into the brains of Parkinson’s patients. Promising results were reported from the early trials, in which small numbers of patients all received GDNF treatment. Researchers became excited about the potential of using neurotrophic factors to treat Parkinson’s disease.

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But to prove that a treatment is effective, it must be tested in clinical trials in which patients are randomly allocated to receive the experimental drug or a placebo. A GDNF clinical trial was established, but unfortunately, it showed that treating the brain with GDNF did not significantly improve movement symptoms in patients with Parkinson’s when compared with patients who received the placebo.

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Despite attempts to improve the delivery of GDNF to the brain, a 2019 placebo-controlled clinical trial of GDNF still produced disappointing results. This was a huge blow to the Parkinson’s community and has led to researchers questioning the potential benefit of neurotrophic factors.

An illustration of a brain-derive neurotrophic factor molecule.