San Diego’s Biotechnology “Functional Treatment” for Diabetes

A century ago, type 1 diabetes was sentenced to death. However, San Diego’s biotechnology ViaCyte says two studies published this week show that the company is approaching what it calls a “functional cure” for the disease.

Two studies, published Thursday, are based on early-stage clinical trials in which the company transplanted a small device under the skin of patients with type 1 diabetes. These devices carry pancreatic cells, and researchers have shown that they can grow into cell types that naturally prevent blood sugar levels from becoming too low or suddenly spiked.

This approach, known as cell replacement therapy, is expected to allow patients with type 1 diabetes to abandon the needles, syringes, and pumps they use to constantly monitor their blood glucose levels. But first, the findings reported based on the first batch of patients participating in an ongoing study must withstand as the study expands and in follow-up studies. And ViaCyte is still tinkering with the device to more consistently and reliably induce insulin production.

Still, the latest findings are causing true optimism from companies that have encountered many twists and turns and turned to the quest to treat what remains a difficult and devastating illness.

“The cells we’re transplanting actually work — they secrete insulin,” said Dr. Howard Foyt, Chief Medical Officer at ViaCyte. “We have already done that, so it’s just a question of whether these results can be reproduced in more patients.”

A recent study came about a month after Massachusetts biotechnology company Vertex announced its progress towards cell-based treatment of type 1 diabetes.

According to the Juvenile Diabetes Research Foundation, it’s all part of an effort to help people suffering from illnesses that affect 1.6 million Americans. Patients with type 1 diabetes often suffer from constant fatigue, weight loss, thirst, and increased urination, among other symptoms.

Without treatment, things get worse and can lead to heart, kidney and nerve damage. Greek doctors in the second century once described diabetes as “meat and limbs dissolve in urine.”

The cause of these patients’ worries is a friendly fire from their own immune system that wipes out the cells of the pancreas that make insulin, the hormone that regulates blood sugar levels. People with type 1 diabetes can inject insulin and use a blood glucose monitor to track their blood glucose levels, but these corrections are costly and not always effective.

ViaCyte is convinced that replenishing these lost cells will be a more permanent solution. Researchers have known for about 20 years that transplanting pancreatic cells from an organ donor can cause insulin production, but there are not enough donor tissues to keep up with demand. That’s why biotechnology uses stem cells. Stem cells can grow in large numbers and induce them to form virtually any of the cells of the body.

One of the company’s implants, known as PEC-Direct, is about half the size of a credit card and contains millions of pancreatic cells derived from stem cells. These cells can also grow into beta cells that lower insulin and blood sugar, and alpha cells that make glucagon, a hormone that raises blood sugar.

Foyt believes that combining both cell types can avoid quiet and fatal problems for some people with type 1 diabetes. Dangerous hypoglycemia It starves glucose in the brain, leading to coma, seizures and even death.

A small, flexible device of biotechnology slides under the skin (usually under the side of a person’s forearm or back), where blood vessels meander inside and are covered with small pores that supply cells with oxygen and fresh nutrients. I have been. However, these pores also allow the patient’s immune cells to attack the transplanted cells, so recipients need immunosuppressive drugs to keep the new cells alive.

The company is also investigating whether cells can be genetically modified to eliminate the need for immunosuppressants, but that effort is still in its infancy.

Recent studies published in the journal Cell stem cell When Cell Reports MedicineParticipated in an early-stage clinical trial of ViaCyte implants launched in 2017, examining 15 and 17 patients, respectively. The latter study found that 6 of the 17 participants who obtained the implant began to create detectable levels of a molecule called C-. A peptide that is produced when insulin is produced.

Why didn’t the researchers measure insulin directly? Because the patient was still receiving a direct insulin injection. But the only way they have C-peptide is if they make their own insulin.

“We obviously wanted more,” Voith said of the fact that about one-third of the participants made their own insulin. “(But) so far no one has been able to do that, and that’s why we reported it.”

The amount of insulin produced by the study participants was not enough to no longer require the injection of hormones, but its production increased and eventually decreased after the patient ate, the familiar reassurance. I followed the patterns I could.

That’s exactly what happens in non-diabetic patients, suggesting that the transplanted cells normally sensed and responded to blood glucose. And when researchers removed the implants from the participants to find out more, they found that the device was filled with both alpha and beta pancreatic cells, as they wanted.

The company then fine-tuned the device’s design, adjusting the number and location of small holes, and the number of implants each participant received, enough glucose regulation to keep participants in a healthy range of blood glucose levels. Helped to make hormones. Foyt says biotechnology expects results from eight more patients by August.

Working with ViaCyte is similar to working with Vertex in mid-October. report Patients with type 1 diabetes from an independent ongoing clinical trial started producing insulin after receiving stem cell-derived pancreatic cells. In particular, participants needed about 90% less insulin injections to control their blood glucose levels three months after treatment than before the study.

However, these findings are based on a single patient and were published in a press release rather than in a scientific journal. And Vertex’s treatment is to inject the cells directly into the liver. By comparison, Foyt states that ViaCyte’s cell-retaining device allows the implant to be easily removed in the event of any problems, providing a convenient advantage.

Founded in 1999, Via Cyte employs approximately 125 people in Torrey Pines. At that time, it came up with many ways to deliver pancreatic cells to diabetes, none of which has yet been approved by the regulatory agency. But Foyt is confident that the company is moving in the right direction.

“For the past seven to eight years, we’ve only gone step by step, overcoming one obstacle and encountering another,” he said. “There is no doubt that cells work. The challenge was engineering to understand how to get cells into the body in a way that allows them to do what they do.”