In a project funded by JDRF, scientists have turned on a gene in cells from a person with type 1 diabetes that allows them to produce insulin again.
In a world-first, JDRF-funded Australian researchers have successfully turned an insulin gene back on by injecting a drug called GSK126 into pancreatic cells donated from one person with type 1 and two people without.
How do we make insulin?
Insulin is made by beta cells in the pancreas. Our bodies stop being able to make beta cells when we are babies whether we have type 1 diabetes or not. This is because the insulin gene that makes them gets turned off by a molecule called EZH2.
What is GSK126?
GSK126 is an existing drug that stops EZH2 from working, which enables the genes for producing insulin to start working again – essentially turning them back on. This allows other cells in the pancreas to develop into insulin-producing beta cells. GSK126 is currently licensed by the FDA – the organisation that approves drugs in the US – so we know it is safe for humans.
When type 1 diabetes happens, the immune system attacks the beta cells, which means the body can’t produce its own insulin. The study’s lead researcher, Professor Sam El-Osta, said: ‘By the time someone is diagnosed with type 1 diabetes, much of their pancreatic beta cells have been totally destroyed by the immune system.’ So, manipulating the insulin gene could allow people with type 1 to make their own beta cells again to replace those they have lost.
Our other beta cell research
You may be familiar with our research that aims to produce beta cells from stem cells, which are a type of cell that can transform into many other cell types. Although this research is exciting, we still have a little way to go before it is a viable treatment for people with type 1.
A future treatment for type 1 diabetes
So, how does this new drug compare to these beta cell therapies? Professor El-Osta said: ‘Our research shows vital insulin production after just two days of drug treatment when compared to three to four months with alternative approaches using stem cells. This non-surgical method has the added benefit that treatments are less vulnerable to the risks associated with transplants.’
A small study, so more research is needed
It’s important to remember that the study only used cells from three people, so much larger projects are needed to confirm their results. But Professor El-Osta, said: ‘The discovery of the genetic barrier to insulin which can be unlocked by a drug is an important milestone and a step in the right direction for the path to accelerate cures.’