June 15, 2021 – by Victoria Johnson
Bayer provided an update on their phase 1 and 1b studies of dopaminergic neurons and GDNF gene therapy, respectively, for the treatment of PD.
Wolfram Carius
This content originally appeared on our sister site, GeneTherapyLive. Read it here.
The first patient has been dosed in BlueRock Therapeutics’ phase 1 study (NCT04802733) of DA01 for the treatment of Parkinson disease (PD), Bayer, the holding company of BlueRock, recently announced. Asklepios BioPharmaceutical (AskBio), another subsidiary of Bayer, is also enrolling patients in its phase 1b clinical study (NCT04167540) of glial cell line-derived neurotrophic factor (GDNF) gene therapy for PD.1
“The potential of BlueRock and AskBio’s clinical candidates to treat PD could be immense,” said Wolfram Carius, head, cell and gene therapy, Bayer, in a statement.1 “For the first time, it might be possible to stop and reverse this degenerative disease and truly help patients with their high unmet medical need. The start of clinical trials represents the beginning towards a truly breakthrough treatment option to dramatically improve the lives of patients.”
Stem Cell-Derived Dopaminergic Neurons
The DA01 candidate consists of pluripotent stem cell-derived dopaminergic neurons designed to innervate the putamen to reverse the regenerative process and potentially restore motor function in people with PD. Unlike standard-of-care symptomatic treatments like levodopa, whose effects diminish over time, BlueRock aims to target PD at its root cause with DA01.
The trial will enroll 10 patients at sites in the US and Canada who will undergo surgical implantation of the DA01 cells. The primary end points of the study are safety and tolerability at 1 year post-transplant. Secondary end points are transplanted cell survival and motor effects at 1- and 2-years post-transplant, as well as continued safety and tolerability and feasibility of transplantation.
Glial Cell Line-derived Neurotrophic Factor
The GDNF study, supported by the California Institute for Regenerative Medicine, dosed its first patient with an infusion of GDNF in August 2020. Ten patients have been enrolled since then into the study to assess safety and preliminary efficacy. The one-time treatment provides a continuous production of GDNF, an advantage over intermittent protein infusions. The therapy is designed to promote the survival and functioning of vulnerable brain cells that degenerate in PD.
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