The outcome of a recent clinical trial conducted by pharmaceutical company Vertex indicates huge promise for the development of a stem cell cure for type 1 diabetes.
The phase 1/2 study, whose results were presented at the American Diabetes Association 84th Scientific Sessions this month, found that the infusion of VX-880 – a new stem cell derived therapy – occasioned significant improvements in diabetic patients’ wellbeing. [1]Â
What is type 1 diabetes?Â
Type 1 diabetes is an autoimmune condition that stops the pancreas producing insulin. Insulin is the hormone the body produces to move glucose (sugar) from the bloodstream into cells to make energy. Without insulin, glucose builds up in the bloodstream and leads to hyperglycemia. [2]
Complications from hyperglycemia can be severe, affecting major organs like the heart and kidneys. Glucose build up can also damage nerves, as well as the blood vessels in the eyes which can result in blindness. [3]
Those who have type 1 diabetes are required to take insulin everyday, either with meals or at regular intervals, a process which can drastically alter lifestyle habits as well as quality of life. [4]
There is currently no known cure for type 1 diabetes and its root causes are unknown, although many believe the condition to be the result of either genetics or environmental factors like exposure to viruses.
According to Diabetes UK, around 1 in 10 people have type 1 diabetes. [5]
What were the results of the trial?
12 patients volunteered for the trial, all with type 1 diabetes and average haemoglobin A1C levels of 7.8%, a level that verges on risks from diabetic complications.
A1C levels measure the amount of haemoglobin with attached glucose in the bloodstream. A higher percentage equates to a higher level of glucose. The average A1C level for a non-diabetic is below 5.7%. [6]
The 12 patients underwent an infusion of VX-880, a new therapy containing islet cells – cell clusters that produce insulin in the pancreas – derived from stem cells.
Following the infusion, all patients demonstrated engraftment of the islet cells. By day 90, all patients also demonstrated glucose-responsive insulin production. All patients’ A1C level also dropped below 7%.
Out of 12 patients who received a full dose of VX-880, seven no longer needed daily insulin injections. Two more patients required about 70% less insulin to manage their blood sugar levels.
These results are incredibly promising and the trial has since been expanded to include 37 patients as research into the effectiveness of VX-880 continues. [7]
What is VX-880 stem cell therapy and how does it work?
VX-880 therapy involves introducing fresh islet cells derived from stem cells into the patient’s body.Â
Because stem cells have the unique ability to differentiate into other specialised cells in the body, they can be reprogrammed to become a specific cell type that can then be used to replace or restore certain cells that may be missing through disease.Â
The stem cells used in the trial were allogeneic, meaning that they were sourced from a donor and required both a good match in addition to immunosuppressant drugs to prevent them from being rejected by the patients’ immune systems.Â
The goal is for these new islet cells to restore the ability of the patients’ pancreases to produce insulin, effectively curing the disease and drastically reducing the need for them to have to administer insulin exogenously. [8]
Cord Blood Banking and Stem Cells
Breakthroughs like this underscore the incredible potential stem cells have in treating severe conditions like type 1 diabetes.Â
Though not yet an approved treatment, clinical trials like this one show that stem cells are at the forefront of developing regenerative therapies for diseases that are currently incurable.
By banking your baby’s umbilical cord stem cells, you can ensure that they have them ready and waiting should they ever need to access one of these regenerative therapies in the future.Â
To find out more about saving stem cells for your baby, fill out the form below for a free Welcome Pack.Â
References
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