Gene therapies are an exciting technology that could be hugely beneficial for people with blood disorders. But they come with a risk: the process can sometimes create potentially cancerous cells. Our Curestarter researchers in Italy have developed an innovative way to test cells after gene therapy to make sure they are safe. Developing this tool further could give researchers better ways to study and monitor cells for cancer – making starting new cures faster and easier.
While this work may seem very technical, it represents an important step toward making revolutionary treatments like gene therapy safer and more accessible to patients who need them...This kind of careful, methodical research is essential for advancing cancer treatment, and we couldn't do it without supporters who believe in the power of scientific discovery.
Your genes store all of the information that your body needs to function.
But sometimes, genes don’t work properly – maybe we inherit a faulty gene, or it changes over time. These gene mutations can cause diseases, and gene therapy has the potential to treat them by modifying or replacing faulty genes – extracting a patient’s bone marrow cells, treating them and then returning the healthy cells back to the patient.
Unfortunately, there is currently a risk that the changes that gene therapy makes could accidentally cause cells to become cancerous. Thanks to the support of our Curestarters, researcher Dr Stefano Biffo and his team have been able to investigate new, better ways to detect if cells might develop into cancer in the future.
This could make gene therapies safer, helping to prevent cancer cases from developing after treatment and ultimately, saving lives.
The team used a special device called a MOAB, which creates a miniature environment for cells. This allowed them to observe cells growing over much longer timescales than traditional lab methods.
What did the researchers discover?
Testing and improving this device provided a better chance of spotting any cells that might cause cancer. The MOAB mimics the conditions of a real body, including blood flow – something other methods often fail to simulate. It can spot as few as 100 potentially cancerous cells among 100,000 healthy cells, giving the team a better chance to monitor those potential problem cells, and learn how they would behave in real patients’ bodies.
Why is this finding exciting, and what could it mean for patients?
Because of the current risks of gene editing, gene therapy is usually only used when patients have no other options. The conventional method for testing if gene therapy treated cells are safe is to implant them into mice in the lab and monitor them for tumour development – but this can take a long time, and if there are only a few potentially cancerous cells they can be easily missed.
Dr Biffo’s work is a proof-of-concept that this device can detect even very small numbers of cancer cells, creating a tool that can be used in the lab to make research more reliable, as well as in the clinic – giving doctors, patients and their families confidence in safer treatment options.
This research is the first step in making technology like this available to help treat patients - but none of it would be possible without our Curestarters.
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