An Illinois woman is one of three Americans who have been cured of their type 1 diabetes thanks to a “life-changing” clinical trial.
After taking insulin for 25 years, Marlaina Goedel, 30, no longer needs daily injections and can finally enjoy sugar again after receiving pioneering stem cell therapy.
She was one of three people to receive an islet cell transplant, a unique infusion that involved transplanting shapeshifter cells into her liver to help her body produce insulin on its own.
In just four weeks, Ms. Goedel no longer needed to take the sugar-controlling hormone, and her doctor called her to tell her, “Stop taking insulin.” You are cured.’
Another patient was left with only a third of his usual insulin dose and a third stopped taking the medication completely in just two weeks.
Mrs Goedel told DailyMail.com of the treatment: “The cure is available.”
The mother-of-one was just five years old when she was diagnosed and doctors told her she “should have been in a coma” because her blood sugar levels were so high.
Her condition was so extreme that she felt deprived of a normal childhood, telling DailyMail.com that she was in and out of hospital with life-threatening diabetic ketoacidosis, which causes toxic chemicals to build up in the blood due to a lack of insulin. .
Marlaina Goedel (pictured), 30, was just five years old when she was diagnosed with type 1 diabetes, constantly worrying about whether she would wake up the next day. He has now been cured with an islet cell transplant
Her “turning point”, she told DailyMail.com, was when her daughter (pictured with Ms Goedel and her partner) found her passed out on the floor in the middle of the night due to a sugar drop in the blood.
As an adult, Mrs. Goedel crashed her car into a brick building during an attack of diabetes.
The condition also robbed her of the opportunity to have more children, as blood sugar fluctuations made her prone to miscarriages.
But it was when her 12-year-old daughter found her passed out on the kitchen floor in the middle of the night after suffering a seizure that she said, “Something needed to change.”
“That was my turning point,” he told DailyMail.com.
After researching new therapies online, Ms. Goedel enrolled in the BESTOW trial, which is being conducted at the University of Chicago Medicine Transplant Institute.
The patients received an experimental drug called tegoprubart, which is made from laboratory-made antibodies that trick the immune system into believing that the body produced the cells on its own, preventing them from being rejected.
The next step was a stem cell transplant.
Stem cells are a type of blank cell that can become the different types of cells the body needs to function.
Under the right conditions, stem cells can become brain, muscle, kidney, or even pancreas tissue.
This treatment used a new chemical cocktail to convert the patient’s stem cells into pancreatic cells.
They were infused into small blood vessels in the recipient’s liver through a catheter.
Those cells then lodged in the blood vessels and began producing insulin.
Mrs. Goedel said the procedure was “back and forth” and lasted only an hour.
Ms. Goedel calls Dr. Witkowski (pictured), who led the trial, her “superhero” for pursuing her case.
Mrs. Goedel is now looking forward to making up for lost time, going back to school and riding horses without worrying about an accident.
He said the main side effect was “feeling like I’d been hit in the ribs.”
But four weeks later, the cells began to produce insulin.
On August 15, he received a call from Dr. Witkowski, who told him, “Today is the day.” Mark your calendar. Stop all insulin. You are cured.’
“He told me: ‘Go tell your family, your friends and enjoy life without insulin.’
After two and a half decades of wondering if she would wake up the next day, Goedel is ready to start a new chapter in her life and make up for lost time.
She plans to go back to school and ride horses without worrying about having a seizure and causing an accident.
Dr. Piotr Witkowski, principal investigator of the trial, said, “They are another step in our quest to achieve a functional cure for type 1 diabetes.”
Additional trials are planned to test the treatment. Similar therapies have been successful elsewhere, including a woman in China who has not received insulin for a year.
Dr David-Alexandre C Gros, chief executive of tegoprubart manufacturer Eledon Pharmaceuticals, told DailyMail.com that this treatment is intended for diabetics like Ms Goedel, whose blood sugar levels are very unstable and not well controlled with standard insulin.
These patients have what is known as brittle diabetes, which causes frequent episodes of high and low blood sugar levels.
About three in every 1,000 type 1 diabetics have brittle diabetes.
Dr Gros said: “For these patients, islet transplantation could help restore endogenous insulin production, allowing normalized glucose control and potentially freeing them from daily insulin dependence.”
The researchers also stated that the transplant function was three to five times greater than in three other patients who received a different type of immunosuppression.
They said this suggests that the new drug may be less toxic to transplanted islets than expected.
Dr Gros said: “We are very encouraged by the levels of interest in tegoprubart and are working intensively to advance this development program to bring this new option in immunosuppression to transplant patients as quickly as possible.”
Tegoprubart trials are in Phase 2 and the effect of tegoprubart on preventing organ rejection in patients undergoing kidney transplants is being studied.
It is unclear when the drug will be widely available to transplant patients, although the approval process typically takes at least five to 10 years.
General health experts have also noted that the technique of crafting personalized transplants using the recipient’s own cells is currently difficult to scale up profitably, meaning the price of this diabetes cure could be staggeringly high at first.
The team noted that because islet cell transplants are regulated by the FDA as a biologic drug rather than a transplant, it could prevent people from accessing them outside of a clinical trial.
Currently, islet cell transplants are estimated to cost around $100,000.
For now, Goedel is taking advantage of the time he’s back. For the first time in her life, she can ride horses and spend time with her daughter without worrying about a drop in blood sugar. She will also be returning to school to become an equine massage therapist.
She said: “It took me a while to get used to saying, ‘I’m cursed.’ I don’t have diabetes. “It’s been very liberating.”
‘No one should have to live with this disease. I know it now more than ever.