Pharmaceutical Business review

Adult stem cell breakthrough in search for Parkinson’s therapy

GDNF has been shown to protect the neurons that produce dopamine that becomes depleted in Parkinson’s patients. GDNF has also been shown to have a beneficial role in protecting neurons in animal models of Amyotrophic Lateral Sclerosis (ALS) and Spinal Cord Injury (SCI).

Most of the current treatments of neurodegenerative diseases provide limited benefit to patients, and drugs for Parkinson’s disease, which focus on dopamine supplementation, often cause prohibitive side effects.

The same scientific team at Tel Aviv University, led by professor Eldad Melamed and Dr Daniel Offen, has previously developed patent-pending technology to differentiate human bone marrow into dopamine producing neuron-like cells, which showed functional benefit in animal models of Parkinson’s disease.

“Neurologists have long thought that GDNF can be used to preserve and maintain the integrity of dopaminergic neurons in Parkinson’s disease. However, delivery to the appropriate location is a major challenge. Direct delivery of the protein has failed and there are current ongoing attempts at gene therapy. We believe that our approach, based on neural transplantation of stem cell derived GDNF producing cells, without any genetic manipulation, is preferable,” said professor Melamed.