Cell therapy weekly: gene therapy trial for high-grade glioma
This week: The first patients have been enrolled into a Phase I/IIa study of a gene therapy for high-grade gliomas, a cell therapy manufacturing platform that combines a bioreactor and centrifuge into one device has been launched, and the FDA has approved the launch of a clinical trial investigating an MSC-based therapy for juvenile idiopathic arthritis (JIA).
The news highlights:
- Gene therapy trial for high-grade glioma
- Automating cell therapy manufacturing
- MSCs move to clinical trial for juvenile idiopathic arthritis
Gene therapy trial for high-grade glioma
The first two patients have been enrolled in the study of an investigational gene therapy for the treatment of high-grade gliomas. The therapy, DB107, combines a retroviral vector that selectively infects and modifies cancer cells, and an oral prodrug that converts into a chemotherapeutic agent within the tumor. This approach aims to destroy tumor cells while triggering a lasting immune response with minimal toxicity.
Chris Beardmore, co-founder and CEO of Anova Enterprises (IL, USA) said, “This is revolutionary science where the treatment infects cancer cells and genetically changes them to make chemotherapy inside of the infected cells. This is known to reduce systemic toxicity commonly seen with systemic treatments. It also has a chance to infect cells that cannot be removed by surgery and other interventions to improve outcomes in newly diagnosed brain tumor patients.”
The Phase I/IIa trial, funded with a California Institute for Regenerative Medicine grant, is a multicenter, open-label study assessing whether DB107, when combined with standard treatments, improves outcomes for newly diagnosed high-grade glioma patients.
Automating cell therapy manufacturing
The life science platform technology company Limula (Lusanne, Switzerland) has launched LimONE, an automated platform designed to streamline cell therapy manufacturing by integrating a bioreactor and centrifuge into a single device.
The LimONE also uses the same single consumables throughout all stages of therapeutic development, removing the need for lengthy technology transfer and accelerating the transition to the clinic. LimONE offers a flexible solution for both hospitals and biopharma companies, enabling the production of various cell therapies, including CAR-T and gene-edited hematopoietic stem cells.
“Bringing together end users from both academic and industry for these workshops was invaluable in shaping LimONE’s design. It will help us meet the needs of the variety of target customers working in cell therapy manufacturing, ensuring that LimONE is not just a technological innovation, but a practical, user-centred solution,” said Caroline Boudousquié, Limula’s Head of Solutions.
MSCs move to clinical trial for juvenile idiopathic arthritis
Hope Biosciences (TX, USA) has received FDA approval to launch the first pediatric clinical trial investigating its adipose-derived mesenchymal stem cells (HB-adMSCs) for JIA. This Phase II study will explore whether intravenous HB-adMSC infusions can reduce symptoms and enhance the quality of life for children aged 2–16 with oligoarticular or polyarticular JIA. Previously, HB-adMSCs have been tested in related conditions such as rheumatoid arthritis, osteoarthritis and lupus, showing promising safety and pain relief outcomes.
JIA is a chronic autoimmune disorder affecting around 300,000 children in the US and 3 million worldwide, often causing joint pain, fatigue, fever and other complications.
“Our technology has safely treated children as young as 18 months with conditions ranging from congenital muscular dystrophy to cerebral palsy, epilepsy and autoimmune disorders,” said Donna Chang, CEO of Hope Biosciences. “We are confident that the same technological advancements that have delivered consistent, high-volume and repeatable cell therapy treatments in adult trials will pave the way for effective treatments in pediatric patients. Encouraging results from our adult rheumatoid arthritis trials, where participants showed significant and lasting improvements in joint pain and function, give us great hope for similar success in children with juvenile arthritis.”
