An Illinois woman is one of three Americans who have been cured of their type 1 diabetes thanks to a ‘lifechanging’ clinical trial.
After taking insulin for 25 years, Marlaina Goedel, 30, no longer needs daily shots and can finally enjoy sugar again after receiving a pioneering stem cell therapy.
She was one of three to have an islet cell transplant, a one-off infusion that involved transplanting islet cells into her liver to help her body produce insulin on its own.
Within just four weeks, Ms Goedel no longer needed to take the sugar-controlling hormone, with her doctor calling her to say: ‘Stop all insulin. You’re cured.’
Another patient is down to one third of his regular insulin dose, and a third was fully off the drug in just two weeks.
Ms Goedel told DailyMail.com of the treatment: ‘The cure is out there.’
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-sugars were so high.
So extreme was her condition she felt robbed of a normal childhood, telling DailyMail.com she was in and out of hospital with life-threatening diabetic ketoacidosis, which causes toxic chemicals to build up in the blood due to lack of insulin.
As an adult, Ms Goedel crashed her car into a brick building during a diabetic attack
The condition also robbed her of her chance to have more children, as the 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 fit that she said: ‘Something needed to change.’
‘That was my tipping point,’ she told DailyMail.com.
After researching novel therapies online, Ms Goedel signed herself up for trial, which is being run out of the University of Chicago 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 on its own, preventing them from being rejected.
Patients were then given islet cells from a deceased donor’s pancreas, which were then infused into the recipients’ small blood vessels in their liver through a catheter.
Those cells then lodged into the blood vessels and started producing insulin.
Ms Goedel said the procedure was ‘in and out,’ lasting just an hour.
She said the main side effect was ‘feeling like I got 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’s the day. Mark your calendar. Stop all insulin. You’re cured.’
‘He’s like, “Go tell your family, go tell your friends, and go enjoy life with no insulin.’
After two and a half decades wondering if she was going to 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 on going back to school and riding her horse without worrying about suffering an attack and causing an accident.
Dr Piotr Witkowski, lead researcher of the trial said they ‘are another step in our quest to achieve a path for functional cures in type 1 diabetes.’
Additional trials are planned to test the treatment. Similar therapies have shown success elsewhere, including a woman in China who has been insulin free for a year.
Dr David-Alexandre C Gros, chief executive of tegoprubart manufacturer Eledon Pharmaceuticals, told DailyMail.com that this treatment is meant for diabetics like Ms Goedel whose blood sugars are severely unstable and not well controlled with standard insulin.
These patients have what’s known as brittle diabetes, which causes frequent episodes of high and low blood sugar.
About three in every 1,000 type 1 diabetics has brittle diabetes.
Dr Gros said: ‘For these patients, islet transplantation could help to restore endogenous insulin production, allowing for normalized glucose control, and potentially freeing them from daily insulin dependence.’
The researchers also stated that the 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 levels of interest in tegoprubart and are working aggressively to advance this development program to bring this new option in immunosuppression 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 transplants.
It’s unclear when the drug will be available widely to transplant patients, though the approval process usually takes at least five to 10 years.
Health experts have generally also noted that the technique of crafting personalized transplants using the recipient’s own cells is currently difficult to scale-up cost effectively — meaning the price tag for this diabetes cure could be staggeringly high in the early going.
The team noted that because islet cell transplants are regulated through the FDA as a biologic drug instead of 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, Ms Goedel is taking advantage of the time she’s gotten back. For the first time in her life, she can ride her horse and spend time with her daughter without worrying about a blood sugar crash. She is also going back to school to become a horse massage therapist.
She said: ‘It took a while to get used to saying, “I am cured. I am diabetes free. It’s been very freeing.
‘No one should have to live with this disease. I know that now more than ever.’
