Bayer is working on a double approach to offer transformative therapies for Parkinson’s disease, one cellular and the other gene, BlueRock and AskBio, both candidates in the clinical development phase. These therapies could open a door to the possibility of stopping and reversing the effects of this degenerative disease.
Bayer recently announced that BlueRock Therapeutics (BlueRock)a clinical development and biopharmaceutical company 100% subsidiary of Bayer AGhas successfully administered the first dose of dopaminergic neurons derivated of pluripotent stem cells (called DA01) to a patient with the Parkinson’s disease in the context of an open phase I clinical trial.
Real dopaminergic neurons to restore motor function
Through the use of real dopaminergic neurons, tries to reinnervate the affected areas of the human brain and reverse the degenerative process, which could potentially restore motor function. The clinical trial of BlueRock will recruit 10 patients in the United States and Canadawhich will undergo a surgical transplantation of dopamine-producing cells in the putamen, a deep brain structure affected in Parkinson’s disease.
The main objective of phase 1 of this clinical trial is to evaluate the safety and tolerability of cell transplantation one year after the intervention. The secondary objectives of the study are to assess the evidence of survival of transplanted cells and its effect on motor ability at one year and two years post-transplant, continually assess safety and tolerability at two years, and assess transplant feasibility.
Bayer is working on a double approach to Parkinson’s to offer transformative therapies, one cellular and the other gene, both candidates in the clinical development phase
Promote regeneration of midbrain neurons for motor recovery
In parallel to the gene therapy programme, Bayer is carrying out a phase Ib clinical trial in which they are currently recruiting and evaluating patients powered by Asklepios BioPharmaceutical Inc. (), company of adeno-associated virus (AAV) gene therapy, also aimed at providing advanced therapies for Parkinson’s disease.
The AskBio approach consists of use of an adeno-associated virus vector (AAV) which introduces the glial cell-derived neurotrophic factor gene (GDNF) in neurons in the putamen, causing the expression and secretion of GDNF proteins in areas of the brain affected by Parkinson’s disease.
The AAV-GDNF combination has shown in long-term experiments that sustained expression of GDNF can promote regeneration of midbrain neurons and significant motor recovery in rodent and primate animal models. The AskBio clinical trial is currently recruiting and evaluating patients in the Phase Ib study in the United States to assess the safety and preliminary efficacy of this treatment.
Possibility of being able to stop and reverse the effects of Parkinson’s
As pointed out Wolfram CariusHead of Cell and Gene Therapy at Bayer, “the potential of these candidate therapies from BlueRock and AskBio for the treatment of Parkinson’s disease can be immense”. In fact, “for the first time in history, we would be facing the possibility of stopping and reversing the effects of this degenerative disease and really help patients with a clear unmet medical need. We are still in early stages of development, but we are fully committed to developing innovations with the potential to dramatically improve the lives of patients who have been waiting for innovative treatments for too longsays Wolfram Carius.
Parkinson’s disease is the and affects more than 10 million people worldwide. It appears due to the deterioration of nerve cells in the brain, which causes a decreased dopamine levels (a neurotransmitter involved in processes such as memory or movement). This disease usually begins with tremors in one handalthough it includes other symptoms such as stiffness, cramps and the dyskinesia (involuntary movements in the face, arms, legs or trunk). Today there is no therapy available that restores motor function. The goal of cell and gene therapy is act directly on the disease and its originand go beyond the treatment of symptoms.