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Ever since his own diagnosis with type 1 diabetes (T1D) during college, Dr. Gary Meininger has dreamed of a day when he won’t need to take insulin in order to survive. It’s taken three decades, but now as clinical lead for a pharmaceutical company researching a potential diabetes cure, Meininger believes he’s closer to seeing that day than ever before.

Meininger is senior vice president and head of clinical development at Vertex Pharmaceuticals in Boston, Massachusetts — the company that recently announced early trial results showing that a patient living with T1D for 40 years saw “cure-like results” after 90 days of receiving Vertex’s novel islet cell transplantation, the company has said.

Specifically, their first patient saw a 91 percent decrease in daily insulin requirements and a return to glucose-responsive insulin production in the body, to the extent of basically being without diabetes.

Although he doesn’t use the word “cure” lightly (and exaggerated media coverage can make many in the Diabetes Community cringe), Meininger is optimistic that they’re paving the way to something exciting.

“We did expect to see improvements… but the results were remarkable and better than we’d expected,” Meininger told DiabetesMine. “What this shows is a promise that we’re able to slow a person’s endogenous insulin needs, with unprecedented efficacy. Going forward, we now have reason to believe we have a functional cure for type 1 diabetes.”

While the concept of islet transplants isn’t new, this particular research by Vertex Pharmaceuticals is the first to use stem cell-derived treatment in this way.

What is a ‘functional cure’?

A functional cure is when a health disorder is not completely eliminated in the body, but can be controlled without medication.

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DiabetesMine readers may recall that Vertex moved into the T1D space back in 2019, acquiring stem cell startup Semma Therapeutics that had been founded by high-profile researcher Dr. Doug Melton of Harvard Stem Cell Institute in Cambridge, Massachusetts.

A D-Dad himself, Melton made big news in 2013 when his research was hailed as a breakthrough. Though a few years later, his original work was disappointingly retracted. Still, the essence of his approach was solid, and his 2014-founded startup Semma — a mashup of the names of his two T1D kids, Sam and Emma — went on to become one of the first funding projects of JDRF’s venture philanthropy T1D Fund in 2017.

Melton’s work has been a fascinating example of research aimed at using stem cells to create new insulin-producing cells in the body.

Vertex had been focused largely on cystic fibrosis up until then, but by buying Semma, the company entered the diabetes cure research game. At a price tag of $950 million, it was considered the largest T1D cure-focused transaction on record to date.

Specifically, Vertex bought and has pushed forward on Semma’s two-pronged approach to this research:

  • Working to create a new supply chain of beta cells from human-derived stem cells, with the aim of transplanting these new cells directly into the liver, where they can produce insulin to naturally regulate blood glucose levels.
  • Creating a device that can be implanted with the new insulin-producing cells housed inside, protecting them from the immune system attack. (Others working on this include ViaCyte, Eli Lilly with Sigilon Therapeutics, and the Diabetes Research Institute with its BioHub).

That first prong is what Vertex is focusing on first, with the implantable device coming later down the road.

In this early Phase 1/2 trial, Vertex is examining its investigational treatment dubbed VX-880. It uses beta cells derived from stem cells, which is different than the pancreatic islet cells used in existing islet cell replacement therapy for those with T1D.

Per a recent New York Times story, a 64-year-old man named Brian Shelton who’s lived with T1D for about 40 years is the first of two people to undergo this treatment and he’s the one the early research is based on.

In June 2021, Shelton received a single infusion of a half-dose of VX-880, administered in the hepatic portal vein along with immunosuppressive drugs to stop the body from rejecting the treatment.

The study data records that Shelton’s A1C improved from 8.6 percent before treatment to 7.2 percent, and daily insulin dose decreased from 34 units per day to an average dose of 2.9 units per day — a 91 percent decrease in insulin use.

The research further reports that he “achieved successful engraftment and demonstrated rapid and robust improvements in multiple measures, including increases in fasting and stimulated C-peptide, improvements in glycemic control, including HbA1c, and decreases in exogenous insulin requirement. VX-880 was generally well tolerated.”

With just one patient success story so far, it’s still too early to draw broad conclusions. But the Vertex team, and many across the Diabetes Community, have high hopes.

“This individual in my mind is really a hero,” Meininger said. “He’s put his trust and faith in science and Vertex, and through his bravery he’s been able to help not only himself but the broader type 1 and medical community. I think through his trust in us and these unprecedented results it’s generated… he is opening the door for many other patients to consider this therapy.”

Meininger tells DiabetesMine that in 2022, the company will continue the clinical trials with the second person also receiving a half-dose but future study participants receiving the full dose of VX-880.

That study’s first phase is expected to be completed in 2024 and the second phase in 2028.

Additionally, Vertex also plans to move forward in 2022 on the second prong of the Semma-acquired research: the encapsulation technology, in hopes of bypassing the need for immunosuppressant drugs.

Dr. Gary Meininger

For Meininger, this work at Vertex is ever-personal and brings hope for his whole family. His twin brother was also diagnosed with T1D years later as an adult, and his now-teenage daughter was diagnosed during childhood.

He recalls starting work at Vertex in 2019, just months before the company acquired the diabetes startup Semma and moved into the T1D space. The timing was coincidental, but he also laughs about a colleague one day calling him and asking if he wanted to help cure type 1 diabetes.

Meininger had been following Melton’s research for many years, long before Vertex took on that work internally. Now, he sees it all as part of his destiny working to improve lives impacted by T1D.

“The idea of getting into this area of research and helping with diabetes in general has been of particular importance to me,” he said.

The media hype around Vertex’s initial results is not without controversy. Those of us who’ve lived with this condition for years are naturally skeptical about any talk of a possible “cure” — since we’ve heard it so many times before.

Regarding Vertex, many in the Diabetes Community are pointing out on social media how immunosuppression drugs are still required at this time, which is a big drawback. Others note how the Vertex data here isn’t yet peer-reviewed and won’t even be published in a medical journal until after the second round of clinical trials end in 2028.

Others also pointed out the critical issue of affordability, noting that Vertex’s practices that are highly expensive and unaffordable for those who need them — along the lines of the cystic fibrosis drug Orkambi at a list price of $272,000 per year. For those who already struggle to afford healthcare or their insulin to survive, the idea of such a high-end “concierge” diabetes treatment only available to some is not appealing.

Nevertheless, the JDRF praised the research findings, noting their own support in this area of research dating back to Melton’s work in 2000.

“As early funders of beta cell replacement therapies, we are excited to see continual advancement around this area of research, which can ultimately lead to a cure for the type 1 diabetes community,” the JDRF said in a statement. “We look forward to additional results as the trial continues, and JDRF is committed to seeing beta cell replacement therapies in the hands of people with T1D and other insulin requiring diabetes.”

Melton, by the way, is now consulting with and owns stock at Vertex, so naturally he’s a big fan of this “foundational work.”

He says Vertex’s recent announcement led his family — particularly his two T1D children, Sam and Emma — to have tears in their eyes.

“The results from Vertex are significant and exciting from my perspective… The top line being that stem cell-derived islets work, and may be even better than expected,” Melton told DiabetesMine.

“For now, I accept it’s only one patient and only 90 days, but the results couldn’t be more promising in my opinion,” he added.