Stem cell-derived therapy may be an avenue for treating Type 1 diabetes, according to a clinical trial by Vertex Pharmaceuticals.
The trial was centered on one patient, who was diagnosed with Type 1 diabetes 40 years ago. For decades, he had been dependent on insulin injections to manage his blood sugar.
Approximately 1.6 million Americans live with the disease, including about 200,000 young people under 20 years old, according to Juvenile Diabetes Research Foundation (JDRF), the leading global organization funding Type 1 diabetes research.
Type 1 diabetes is a disease that requires constant monitoring and management. People with Type 1 diabetes need to continually balance their insulin intake and measure blood-sugar levels by pricking their fingers at least six times a day or by wearing a glucose monitor, according to JDRF.
If their glucose levels are not within the right range, people with Type 1 diabetes can be in life-threatening situations.
In the year prior to treatment, the patient for this clinical trial experienced five severe, potentially life-threatening episodes where his blood sugar was too low. He injected 34 units of insulin per day into his bloodstream, and tests showed his body was unable to create its own insulin.
As part of the clinical trial, the patient received an infusion of a stem cell-derived treatment called VX-880. The researchers only injected half the amount of the target dose as part of an initial test.
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Vertex published positive results in October, 90 days after the infusion, stating that the treatment was well-tolerated by the patient. It resulted in a 91% decrease in the patient's daily insulin requirement and a restoration of insulin production.
“These results from the first patient treated with VX-880 are unprecedented. What makes these results truly remarkable is that they were achieved with treatment at half the target dose,” Dr. Bastiano Sanna, executive vice president and chief of cell and genetic therapies at Vertex, said in a statement.
There were a few mild to moderate health events in the 90-day period, none of which were serious, according to Vertex. The patient experienced a few severe low blood sugar events and developed a rash, but those incidents were not related to VX-880, Vertex says.
Vertex points out that data from one patient may not be indicative of final clinical trial results, and there's still much to be explored. However, the potential benefits of the treatment could be life-changing.
"This potentially obviates the lifelong need for patients with diabetes to self-inject insulin as the replacement cells 'provide the patient with the natural factory to make their own insulin,'” explained Harvard professor Doug Melton, co-director of Harvard Stem Cell Institute, in a statement following the trial.
Michelle Shen is a Money & Tech Digital Reporter for USA TODAY. You can reach her @michelle_shen10 on Twitter.
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