This week: Cancer Research UK (CRUK; London) has invested in an institute outside of London to accelerate advancement in personalized medicine and early detection, The University of Cambridge (UK) has announced it will open a new facility to deliver CAR-T cell therapies, enabling more treatments for patients. Plus, an investigational gene therapy for Parkinson’s disease has demonstrated promising results in a randomized, double-blind study.
The news highlights:
US$225 million investment towards world-class research institute
CRUK has announced a major investment of US$225 million in the CRUK Cambridge Institute to enhance cancer research and treatment. This funding will allow researchers to better understand how cancers develop, grow and spread, while also exploring new ways to utilize the immune system to fight the disease. With resources for the latest technology and efforts to attract skilled professionals, the institute aims to accelerate advancements in personalized medicine and early detection techniques. This significant investment, the largest by the charity outside of London, will support research initiatives over the next seven years.
“We are delighted to confirm this incredible investment which is a reflection of the world-leading research community at the [CRUK] Cambridge Institute,” commented Michelle Mitchell, Chief Executive at CRUK. “The funding will underpin long-term cutting-edge discovery research, as well as supporting researchers to find new ways to improve cancer prevention and treatment, while creating innovative solutions to diagnose the disease earlier.”
New facility to deliver CAR-T cells
The University of Cambridge is to open a new state-of-the-art laboratory, which will be operated by Cambridge University Hospitals NHS Foundation Trust (UK), to manufacture and develop CAR-T cell therapies. The Trust recently received a US$1.8 million grant from the National Institute of Health Research (Newcastle, UK) to equip the laboratory and support the delivery of more clinical trials for patients across the East of England.
The new cleanrooms, expected to open within the next 12 to 18 months, will expand the current Cambridge Cellular Therapy Laboratory at Addenbrooke’s Hospital (Cambridge, UK) enabling more treatments for more patients. This development also paves the way for the future Cambridge Cancer Research Hospital, scheduled for completion by 2029.
“At the moment, there are a number of novel cell therapy products available commercially, but as an NHS Trust we have to buy them in for our patients,” explained Sarah Albon, Director of the Cambridge Cellular Therapy Laboratory. “Having this state-of-the-art space is the missing part of the puzzle for bringing cell therapies from the research bench to bedside.”
Albon also highlighted that the new laboratory will be able to, “translate research into high-quality medicine, readily available for [patients] and the new Cambridge Cancer Research Hospital, planned to be built here in Cambridge.”
Encouraging results from Parkinson’s disease trial
MeiraGTx Holdings plc (NY, USA) has announced promising data from its MGT-GAD-025 study assessing AAV-GAD, a gene therapy designed to reprogram dysfunctional brain circuits through the local production of a neurotransmitter, which may help restore normal activity in critical cells affected by Parkinson’s disease.
The study involved 14 patients with idiopathic Parkinson’s disease and tested two dose levels of AAV-GAD, showing significant motor improvements and enhanced quality of life, particularly in the higher dose group dose. In the 6-month, three-arm, randomized, double-blind, sham-controlled trial, AAV-GAD was well tolerated, with significant and clinically meaningful improvements from baseline observed at the 26-week endpoint. Long-term follow-up studies are currently underway.
“The significant, substantial and clinically meaningful changes observed in this small, sham-controlled study provide us with a clear path forward in our clinical development strategy and underpin our discussions with regulators in the US, Europe and Japan with the goal of initiating a Phase III study to support approval of this disease-modifying treatment globally,” explained Alexandria Forbes President and CEO of MeiraGTx.
