A study written by a doctor at the University of Pennsylvania has shown that new stem cell therapy may eliminate the need for insulin therapy in patients with type 1 diabetes, a disease in which the pancreas does not produce insulin.
Early clinical trial results restored the ability to produce insulin in 10 of the 12 people with type 1 diabetes treated with apex drug therapy, according to a study published last month in the Journal of New England Medicine. This means their bodies can regulate their blood sugar levels without external help.
Therapy is made from stem cells. Stem cells are cells that do not determine what type of cells they want to be. Like career fair recruiters chatting with impressive students, scientists are clusters of cells that cope with these cells to become islet cells, and regulate blood glucose levels by producing insulin and other hormones.
These cells are injected into the body, migrate to the liver, where they become active.
Zimilecel could be a “functional treatment,” said Marlon Pragnell, vice president of research and science at the American Diabetes Association, who was not involved in the study.
Treatment appears to release patients with daily symptoms of type 1 diabetes, but does not address the autoimmune dysfunction that defines the disease. As a result, patients become more vulnerable to becoming ill from infection, as they require lifelong immunosuppressive therapy.
“Even though it's still early on, if these results can be replicated successfully in a larger and more diverse population, dimisle cells can redefine the treatment paradigm for type 1 diabetes,” Pragnell said in an email.
The costs have not been announced by the company yet. If regulators determine that it is safe and effective, it is likely that the jimisle cells are still several years away from reaching patients widely.
What is type 1 diabetes?
Type 1 diabetes is an autoimmune disease in which the body accidentally destroys insulin-producing cells in the pancreas. Insulin is a hormone that acts like a key, unleashes the “door” to the cells and allows sugar to enter. Without it, sugar accumulates in the blood.
Over time, hyperglycemia can damage the heart, nerves, kidneys and blood vessels.
There is no cure for the disease. People can manage their symptoms by taking insulin injections. However, it is difficult to maintain the proper balance of insulin in the body.
People with this condition may experience severe or hypoglycemia if they are taking more insulin than necessary. This can lead to seizures, loss of consciousness, coma, and even death.
Half of people with type 1 diabetes also suffer from at least one severe hypoglycemia event each year.
The clinical trials studied the treatment of 12 people with type 1 diabetes who had repeated severe hypoglycemia and who did not generate warning signs of the coming episode of glucose.
Participants in the study ranged from 24 to 60 years of age. The third was a woman, all white.
How treatment works
Zimilecel aims to restore the body's ability to respond to blood glucose fluctuations in real time. When that gets high, insulin comes to the rescue. When it is low, the complementary hormone, glucagon, rushes.
Within 90 days of receiving treatment, none of the 12 study participants had experienced severe hypoglycemic events. Ten of them were not dependent on insulin a year later. The other two only needed a small amount of insulin.
Michael Rickels, the research author who directs Penn's islet cell transplant program, said two people died, but their deaths were deemed unrelated to treatment. One of the deaths was caused by pre-existing health problems, including severe dementia. The other died of meningitis after elective surgery, in which drugs banned by research protocols were used.
“To take away that burden and enable people to live their lives the way they want it can be a truly powerful and inspiring experience,” said Rickels, who reportedly serves as a consultant at Vertex Pharmaceuticals.
The catch is to ensure that patients need immunosuppressive therapy for life and not destroy new cells like their bodies are old. Immunosuppression can increase the risk of infectious diseases and other side effects. It is generally well tolerated in studies, with most side effects being mild or moderate.
Some labs are working on genetic modifications of cell lines used to avoid the need for immunosuppressant agents.
“It's amazing and could potentially broaden treatment to everyone with type 1 diabetes,” said Pragnell, an ADA expert who is not involved in the study.
Currently, clinical trials are at an important stage, in which treatments are widely tested in people for long-term safety and efficacy. The company is also starting trials in patients with type 1 diabetes with kidney transplants already using immunosuppressants.
If everything goes well, Vertex Pharmaceuticals hopes to file it in 2026 for regulatory approval.
The only equivalent treatment for ZimisleCel is DonisleCel, an FDA-approved therapy that involves isolating healthy islets from the pancreas of deceased donors. However, there are not enough donor islands to meet the demand.
Stem cells ensure unlimited supply.
“We hope this will provide a sufficient amount of high quality islands with a more reliable and consistent supply,” Riquels said.