An Illinois woman is one of three Americans cured of their type 1 diabetes thanks to a “life-changing” clinical trial.
After 25 years of using insulin, 30-year-old Marlaina Goedel no longer needs daily injections and can finally enjoy sugar again after a groundbreaking stem cell therapy.
She was one of three to undergo an islet cell transplant, a one-time infusion that transplants islet cells into her liver to help her body produce insulin on its own.
Within just four weeks, Mrs. Goedel no longer had to take the sugar-regulating hormone. Her doctor called her to say, ‘Stop all insulin. You are healed.’
Another patient only needs a third of his normal insulin dose, and a third had stopped taking the medication completely in just two weeks.
Ms Goedel told DailyMail.com about the treatment: ‘The medicine 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. She told 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 here), 30, was just five years old when she was diagnosed with type 1 diabetes, which left her constantly worried about waking up the next day. She has now been cured with an islet cell transplant
Her “tipping point”, she told DailyMail.com, was when her daughter (pictured here with Ms Goedel and her partner) found her unconscious on the floor in the middle of the night from a blood sugar crash.
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As an adult, Ms. Goedel crashed her car into a brick building during a diabetes attack
The condition also robbed her of her chance to have more children as the fluctuations in blood sugar levels made her prone to miscarriages.
But when her 12-year-old daughter found her unconscious on the kitchen floor in the middle of the night after a seizure, she said, “Something had to change.”
“That was my turning point,” she told DailyMail.com.
After researching new therapies online, Ms. Goedel signed up for a lawsuit, which is being conducted by the University of Chicago’s Medicine Transplant Institute.
Patients were given an experimental drug called tegoprubart, which is made from lab-made antibodies that trick the immune system into thinking the body made the cells itself, preventing them from being rejected.
Patients then received islet cells from the pancreas of a deceased donor, which were then injected into their liver through a catheter into the recipient’s small blood vessels.
These cells then settled in the blood vessels and started producing insulin.
Ms Goedel said the procedure was ‘in and out’ and took just an hour.
Ms. Goedel calls Dr. Witkowski (pictured here), who led the trial, her “superhero” for taking her case
Mrs. Goedel is now looking forward to making up for lost time, going back to school and riding horses without having to worry about an accident
She said the main side effect was “feeling like I’d been punched in the ribs.”
But four weeks later the cells started producing insulin.
On August 15, she received a call from Dr. Witkowski, who said, “Today is the day. Mark your calendar. Stop all insulin. You are healed.’
“He said, ‘Tell your family, tell your friends and enjoy life without insulin.’
After twenty and a half decades of wondering if she would wake up the next day, Ms. 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 having to worry about a seizure or an accident.
Dr. Piotr Witkowski, principal investigator of the trial, said they are “another step in our search for a path to functional cure for type 1 diabetes.”
Additional studies are planned to test the treatment. Similar therapies have had success elsewhere, including in a woman in China who has been insulin-free for a year.
Dr. David-Alexandre C Gros, CEO of tegoprubart manufacturer Eledon Pharmaceuticals, told DailyMail.com that this treatment is intended for diabetics like Ms Goedel, whose blood sugars are severely unstable and not well controlled with standard insulin.
These patients have so-called brittle diabetes, which causes frequent episodes of high and low blood sugar levels.
About three in 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 found that transplant function was three to five times higher than in three other patients who received a different type of immunosuppression.
They said this suggests the new drug may be less toxic to transplanted islets than hoped.
Dr. Gros said, “We are very encouraged by the strong interest in tegoprubart and are working aggressively to advance this development program to bring this new immunosuppression option to transplant patients as quickly as possible.”
Testing of tegoprubart is in phase 2 and is investigating the effect of tegoprubart on preventing organ rejection in patients undergoing kidney transplants.
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.
Health experts have also generally noted that the technique of manufacturing personalized transplants using the recipient’s own cells is currently difficult to scale up cost-effectively – meaning the price tag for this diabetes treatment 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, this could prevent people from accessing them outside of a clinical trial.
Currently, islet cell transplants are estimated to cost approximately $100,000.
For the time being, Mrs. Goedel is taking advantage of the time she has back. For the first time in her life, she can ride horses and spend time with her daughter without having to worry about a blood sugar spike. She is also going back to school to become an equine massage therapist.
She said, “It took me a while to get used to saying, ‘I’m healed. I am diabetes free. It was very liberating.
‘No one should have to live with this disease. I know that now more than ever.’