by 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 a pioneering stem cell therapy.
She was one of three people to undergo an islet cell transplant, a unique infusion that involved transplanting shapeshifting 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, with her doctor calling to say: “Stop all insulin.” You are healed.
Another patient was left with only a third of their usual insulin dose, and a third stopped taking the drug completely in just two weeks.
Ms Goedel told DailyMail.com of the treatment: “The cure exists.”
The mother-of-one was just five 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 repeatedly hospitalized with life-threatening diabetic ketoacidosis, which causes a buildup of toxic chemicals in the blood due to 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 worrying about whether she would wake up the next day. She has now been cured thanks to an islet cell transplant
Her “tipping point”, she told DailyMail.com, came when her daughter (pictured here with Ms Goedel and her partner) found her passed out on the floor in the middle of the night from a drop in blood sugar.
As an adult, Ms. Goedel crashed her car into a brick building during a diabetic attack.
The disease also robbed her of her chance to have more children, as fluctuations in her blood sugar levels 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 had to change.”
“That was my tipping point,” she told DailyMail.com.
After researching new therapies online, Ms. Goedel enrolled in the BESTOW trial, conducted at the University of Chicago Institute of Medical Transplantation.
The patients received an experimental drug called tegoprubart, made from laboratory-made antibodies that trick the immune system into thinking the body is making the cells itself, preventing them from being rejected.
The next step was a stem cell transplant.
Stem cells are a kind of blank cell that can be transformed into many types of cells that the body needs to function.
Under the right conditions, stem cells can transform into brain, muscle, kidney or even pancreatic tissue.
This treatment used a new chemical cocktail to transform the patient’s stem cells into pancreatic cells.
They were infused into small blood vessels in the recipient’s liver via a catheter.
These cells then lodged in the blood vessels and began producing insulin.
Ms Goedel said the procedure was “in and out” and only lasted an hour.
Ms. Goedel calls Dr. Witkowski (pictured here), who led the trial, her “superhero” for championing her case.
Ms. Goedel now looks forward to making up for lost time, returning to school and riding horses without worrying about a fall.
She said the main side effect was “feeling like you’ve been punched in the ribs.”
But four weeks later, the cells began producing insulin.
On August 15, she received a call from Dr. Witkowski who told her, “It’s the big day. Mark your calendar. Stop all insulin. You are healed.
“He says, ‘Go tell your family, go tell your friends and go enjoy life without insulin.’
After two and a half decades of wondering if she would wake up the next day, Ms. Goedel is ready to open a new chapter in her life and make up for lost time.
She plans to return to school and ride horses without fear of suffering an attack and causing an accident.
Dr. Piotr Witkowski, principal investigator of the trial, said it was “another step in our quest to achieve functional cures for type 1 diabetes.”
Additional trials are planned to test the treatment. Similar therapies have had success elsewhere, including in a woman in China who has not received insulin for a year.
Dr David-Alexandre C Gros, chief executive of tegoprubart maker Eledon Pharmaceuticals, told DailyMail.com the treatment is aimed at diabetics like Ms Goedel whose blood sugar levels are severely unstable and poorly controlled with standard insulin.
These patients have what is called brittle diabetes, which causes frequent episodes of high and low blood sugar.
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, thereby allowing normalized glycemic control and potentially freeing them from daily insulin dependence.”
The researchers also said that transplant function was three to five times better 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 expected.
Dr. Gros said: “We are very encouraged by the levels of interest in tegoprubart and are actively working 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 studying the effect of tegoprubart on preventing organ rejection in patients undergoing kidney transplantation.
It’s 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 creating personalized grafts from the recipient’s own cells is currently difficult to develop cost-effectively, meaning the price of this diabetes treatment could be incredibly high at the moment. beginning.
The team noted that because islet cell transplants are regulated by the FDA as a biologic drug rather than a transplant, that could prevent people from accessing them outside of a clinical trial.
Currently, islet cell transplants cost around $100,000.
For now, Ms. Goedel is enjoying the time she has. For the first time in her life, she can ride a horse and spend time with her daughter without fear of a drop in blood sugar. She is also returning to school to become a horse massage therapist.
She said: “It took a while to get used to saying, ‘I’m cursed.’ I am no longer diabetic. It was very liberating.
“No one should have to live with this disease. I know this now more than ever.
