The Medicines and Healthcare products Regulatory Agency (MHRA) has announced that it is trialling a pioneering technique for growing stem cells that could dramatically boost their supply to enable treatment of a wide range of serious diseases. The Agency hailed the technique as potentially transformational.
Although stem cells have a unique ability to differentiate into different types of cells with specialised functions, so potentially are able to replace cells that have been damaged or lost from disease, therapeutic use has been limited by availability, as stem cell-based therapeutics are difficult to manufacture.
The new technique, the CellQualiaTM Intelligent Cell Processing System – dubbed a 'robot' stem cell producer – could offer a stable supply of cells at reduced cost and make cell therapies much easier to produce. The technique, developed in Japan, uses an automated instrument for cell expansion, with intelligent process analytical technologies within a closed system, to maintain aseptic operations and sterilisation steps.
Potential for Increased Production with Uniform Quality at Lower Cost
Whilst skilled staff must still make decisions about cellular morphology and confluency, using a robot for the manual steps in manufacture both increases output and reduces labour costs. The technique allows for parallel manufacturing with multiple systems to scale up cell manufacturing, either for a single facility or at multiple sites – potentially including at the bedside – with uniform product quality.
The MHRA said that the robotic system "has the potential to bring safer and more cost-effective treatments to people with a wide range of diseases". The MHRA's UK stem cell bank is one of only two places in the world to test this technology, the other being in Japan.
The trial is part of a UK-based international research programme, launched in 2021, which features a partnership between the MHRA; Sakarta Ltd, a Scottish regenerative medicine start-up company that builds automated solutions for stem cell manufacture and cell therapy, and Sinfonia Technology, the Tokyo-based electrical equipment manufacturer that developed the CellQualiaTM Intelligent Cell Processing System. The collaboration is supported by the Foundation for Biomedical Research and Innovation at Kobe, a Japanese biomedical innovation foundation.
'New Hope' for People with Degenerative Diseases
The UK stem cell bank aims to test the robot over a 12-month period to see whether the cells produced by the fully automated system meet the standards needed for them to be used in the manufacture of cell therapies for degenerative conditions such as Parkinson’s disease. The MHRA said this could offer "new hope to patients with serious diseases".
Marc Bailey, MHRA chief scientific officer, said: "Cell-based therapeutics have the potential to treat, and even cure, a vast number of diseases, but their availability has been limited because they are often very difficult to manufacture.
"The new intelligent cell processing system being tested at the MHRA, of which there are only two machines in use in the world, could make this manufacturing process much easier and therefore transform the availability of these treatments. It also has the potential to reduce human error in this process and produce a more consistent final product, which will result in safer and more effective treatments. We look forward to communicating the results of our testing."
Health and Social Care Secretary Steve Barclay hailed the technology as "a partnership between the MHRA and industry" that was an example of clinical research enabling patients to benefit from "the very latest scientific breakthroughs".
He said: "This pioneering new robot has the potential to speed up access to more cost-effective, safer stem cell therapies. By replacing cells that have been damaged or lost, this new technology could transform the lives of tens of thousands of people with Parkinson’s and other devastating diseases."
Technique Could Offer a 'Viable Therapy' for Parkinson's Disease
Professor David Dexter, associate director of research at Parkinson's UK, told Medscape News UK: "Parkinson's is the fastest growing neurological condition in the world. The movement problems associated with Parkinson's are due to the loss of nerve cells within the brain that produce a chemical messenger called dopamine.
"For many years, replacing the lost dopamine producing cells with stem cells has been considered a potential treatment for Parkinson's. This research is finally translating into stem cell transplant clinical trials in several countries.
"If these trials are successful, neurosurgeons will require a readily available source of stem cells to make this a viable therapy for Parkinson's. This is exciting news today that the MHRA stem cell bank will be trialling one of the new CellQualia robotic systems for the automated production of stem cells, which could help pave the way for a new treatment for Parkinson's."
'Huge' Range of Conditions That Might be Helped
Asked by Medscape News UK what other conditions the prospective cell therapy treatments might help, an MHRA spokesperson told us: "The range is huge – there are many cell therapies currently in clinical trials that offer potential for the treatment of a wide range of conditions. These include neurons for Parkinson's disease and spinal injury, mesenchymal stromal cells for bone and cartilage repair, cardiomyocytes for cardiac repair, and pancreatic islets for Type 1 diabetes.
"In addition, CAR-T therapies, although classed as a gene therapy, are hugely promising autologous T-cell based therapies for the treatment of some cancers. The stem cell machine has the potential to accelerate the development of all of these."