SymbioCellTech’s Stem Cell-Enabled Therapy Could Offer a Functional Cure for Type 1 Diabetes Without Requiring Anti-Rejection Drugs
Under the leadership of stem cell experts Christof Westenfelder, MD, and Anna Gooch, PhD, SymbioCellTech is showing promising results on a T1D therapy involving “Neo-Islets.” They’ve cured T1D in mice, have had good results in dogs, and will begin human trials as soon as 2025.
The Challenge
When most people think about Type 1 diabetes (T1D), they think about a friend or family member who has to take shots of insulin in order to stay within a healthy blood sugar range. Or maybe you know someone who wears an insulin monitor on their arm and gets their blood sugar numbers sent to their phone. T1D is a devastating disease that affects millions of people, but thanks to the advent of new patches and pumps and apps, the public sees Type 1 diabetes as challenging but manageable.
For Anna Gooch, PhD, Type 1 diabetes conjures a more urgent picture, one that has driven decades of work in the lab and what could be a once-in-a-generation scientific breakthrough.
When Gooch thinks about T1D, she thinks about the patients her father took care of as a nephrologist. When she was as young as 10 years old he would take her on rounds with him as he cared for people with kidney disease.
“I got very attached to two patients in particular,” Gooch recalls. “They were both blind. My job was to sit and read out loud to them while they got their dialysis. They were bored to death.” They were also, as it turned out, living with diabetes.
One day she and her father arrived in the clinic to discover that one of these patients – who had recently had the good fortune of receiving a kidney transplant – was very sick. They had to rush him to the emergency department because his body was rejecting the transplant, and the anti-rejection drugs weren’t working.
“Transplants are great, but these drugs suck,” says Gooch. “You need something different. These drugs cause serious infections, cancer, and they destroy cells that are placed in the liver.”
That experience – both of caring for chronic disease patients and of seeing the body’s rejection of transplants – embedded itself in Gooch’s mind. She went on to get a PhD and spent 15 years working on cell therapy and regenerative medicine, often focusing on treatments for the same patent population that her father dedicated his life to.
Origin Story
Along her research journey Gooch found a kindred spirit in Christof Westenfelder, MD, a clinical nephrologist and expert in stem cell research. Westenfelder is an expert in regenerative medicine and previously founded Allocure. Together they began focusing their work on the problems inherent in certain diabetes treatments. Namely, the field of islet transplants. For years scientists have been honing this idea of injecting cells from a healthy pancreas into the body of a person with Type 1 diabetes, thus allowing them to produce insulin.
One major problem with typical islet cell transplants is that they require the life-long use of immunosuppressants that are often associated with infections, cancers, and kidney disease.
In 2014, the two came up with a hypothesis that, if correct, would change the course of diabetes treatment forever. They hypothesized that if they could work with the body’s natural functions, rather than against them, they could reduce islet cell therapy rejection, and make donor islet cells more readily available. It went like this:
“Islet cells in the pancreas contain small numbers of stem cells that monitor the health of the micro-environment continuously and help to fix minor problems,” explains Drs. Westenfelder and Gooch. “In a diabetic state, these cells get damaged and can no longer protect cells that produce insulin and other important hormones for metabolic control.” They hypothesized that if they increased the number of these protective stem cells they could help the insulin and other hormone-producing cells to survive and function. “These stem cells have a fantastic capacity to block the destruction of the cells that make insulin, which is what T1D is all about. We don't manipulate any of the cells, we just regroup them and reorganize them to work with each other, i.e., to collaborate with each other.”
Under the Hood
Drs. Gooch and Westenfelder set about doing what they did best: testing their hypothesis in a lab and publishing their research. At first, they had a healthy dose of scientific skepticism.
“This can't possibly work. How are you going to overcome the challenges of such a complex immune system?” Gooch recalls thinking in the early days.
Then came a day when that skepticism turned to hope.
They were in their lab in Salt Lake City, feeling “fairly jaded and cynical,” when they began to read the results of their first major mouse study. One after another they saw that each mouse, once fully diabetic, had normalized blood sugar. They had cured these mice of their diabetes, and they’d done it without harmful anti-rejection drugs.
The team moved from mice to dogs, which meant leaving the lab and testing their theories in the real world, with “patients” who didn’t always behave perfectly. Again, their results were incredibly encouraging.
“We had one diabetic dog who lived to be 17 years old,” says Dr. Gooch. “That’s astounding for a diabetic dog. They usually live about one year past diagnosis.”
Around 2017, the team published a detailed study of their “Neo-Islet” technology, explaining how it works, step by step. They also had earlier demonstrated their success through a clinical trial that showed that these stem cells could help patients undergoing cardiac surgery and patients that are at very high risk of losing their kidney function. The stem cells had a strong protecting effect for these patients, and now, 10 years post study, there's been no loss of kidney function, no deaths, no progression to chronic kidney disease compared to controls.
Next Steps
All eyes now are on human trials.
The SymbioCellTech team has had a successful pre-IND meeting with the FDA, which resulted in a request to do one more study using human cells, the final clinical product, in a suitable mouse model. Once they have their IND in hand, they’ll set up their human clinical trial, which they hope to have up and running in the second half of 2025.
SymbioCellTech has raised about $17M to date in support of this work, largely from friends, family, and angel investors passionate about curing Type 1 diabetes. The team regularly receives emails from families who hear about their work and want their family member to be part of a future clinical trial. For Drs. Westenfelder and Gooch, that’s something they take very seriously.
“You end up feeling responsible,” says Gooch. “You want to make sure that this works, is safe, is effective, is affordable, and can help them.”
SymbioCellTech has plenty of work to do, and questions to answer, before their hypothesis becomes a breakthrough for humans, but they’re already thinking about global impact.
“Once we show that it works in the US, we will already be well established in Europe with colleagues, scientists, and members of our board. We want to take this to the European market, but also to markets where there is a very limited amount of funding for this type of work.”
As with any scientific breakthrough, the proof will be in the data. We’re proud to support SymbioCellTech because they’ve formed a team with deep cell therapy expertise and their methodical progress is building towards a true breakthrough. If their human trials are successful, we could see a functional cure to Type 1 diabetes.
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