Thousands of Americans are diagnosed with Type 1 diabetes every year, according to the American Diabetes Association. But a new medication now available worldwide has the potential to drive that number down, at least in the short term. Seventh-grader Mason Webb is the first young person in San Diego County to experience what’s possible. The 12-year-old from Pacific Beach found himself at Rady Children’s Hospital recently, receiving daily infusions for two weeks of Teplizumab, a new drug that the U.S. Food and Drug Administration approved in 2022 to delay the onset of Type 1 diabetes in children and adults. But there is a catch. In its early stages, diabetes causes no symptoms, but the drug, marketed as Tzield, is only effective during this quiet phase. This is the window when the drug can block the ongoing action of mistargeted white blood cells that, unchecked, cause the immune system to kill insulin-producing beta cells in the pancreas. Mason is an example of what can happen when developing diabetes is detected early, in what experts call “stage 2.” In his case, a blood test ordered as part of a routine physical detected that his A1C blood sugar score was higher than would be expected for a boy of his age. Had that test instead been ordered to diagnose the cause of symptoms such as extreme thirst and hunger, weight loss and fatigue, it would have been too late. He would have already progressed to stage 3, where beta cells are already damaged, destroying or severely reducing insulin production. Without this critical hormone, the body cannot regulate blood sugar and a person must spend the rest of their lives supplementing with insulin shots and careful diet management. “What’s so exciting is this is the first FDA-approved drug we’ve ever had that can delay onset,” said Dr. Carla Demeterco-Berggren, the pediatric endocrinologist heading up Rady’s Tzield effort. “It’s a big, big deal.” The decades of research it has taken to bring stem cell technology to the point where a drug like this can enter the nationwide standard of care, she said, is doubly exciting for what it portends. “This is just the foot in the door with the FDA, and there will be much more to come for Type 1,” Demeterco-Berggren said. Technically, Mason is not the first kid in San Diego County to receive Tzield, but he is the first to receive it in stage 2 when he can reap its full benefits. Rady participated in the clinical trials that led to the drug’s approval and, during that long process, a dozen kids already in stage 3 received infusions as part of ongoing work studying whether it can be effective in those whose beta cells have already stopped producing insulin. Two adults, one treated by UC San Diego Health and another reportedly infused by a local doctor in private practice, have also received Tzield recently. The sheer luck of picking up on this developing problem early is not lost on Valeria and Terrance Webb, though they know that skill was also involved. Their family doctor had to order the test in the first place and it certainly didn’t hurt that Rady endocrinologists have recently been reaching out to pediatric specialists, making sure they’re aware of Tzield’s availability. It was hard, on a recent afternoon, to see the big picture as Mason prepared to receive his 10th infusion. Living for two weeks with an IV port in one’s arm somewhat overshadowed the fact that, until very recently, nobody had the option of delaying Type 1. On Wednesday, Oct. 2, 2024, in San Diego, Rady Children’s Hospital, Mason Webb receives his 10th of 14 Tzield treatments intravenously. The drug is the first to delay the onset of Type 1 diabetes. (Nelvin C. Cepeda / The San Diego Union-Tribune) But learning more about the timing that allowed him to receive the treatment brought out his appreciation. “I kind of feel lucky to be born (at) this time, because I don’t know, if I was older, would I still be able to get it?” Mason said. He was actually diagnosed in late 2023, but it took some time for insurance clearances to come through. Officials said that Medi-Cal, the state’s health insurance program for low-income and disabled residents, has not yet listed Tzield as an approved medication, though that information could not be confirmed with the state Friday. Though insurance companies can and do negotiate lower prices, the drug is not cheap, with the FDA pegging its wholesale cost at “$13,850, which translates to $193,900 for a 14-vial continuous regimen.” Demeterco-Berggren, the Rady endocrinologist, said that screening efforts are starting with the relatives of patients known to have Type 1 diabetes. While family relations have a greater probability of developing the disease, environmental factors that are not yet understood also play a role, in addition to genetics, meaning that a person may be diagnosed without other cases in their family tree. That is Mason’s situation. Neither of his parents has been similarly affected. Tzield is the first opportunity to delay the onset of this disease in human history, and that fact delights those who diagnose it every day. The biggest personal pleasure, Demeterco-Berggren said, is to prevent a child from ending up in an emergency room in severe metabolic distress because the disease has progressed to stage 3 and started generating severe symptoms. “Over 50 percent of all the new cases of Type 1 diabetes, they have to come to the hospital because they’re really sick with this acute complication called diabetic ketoacidosis,” Demeterco-Berggren said. “If we can know ahead of time, we can prevent that.” Work is already underway, she added, to make Tzield available to those without private health insurance. While a candidate covered by Medi-Cal has not yet been found to qualify for the drug, Demeterco-Berggren said that Rady will request coverage on a case-by-case basis and has instituted around-the-clock staffing at its infusion clinic to make treatment easier to access if parents’ jobs don’t follow a 9-to-5 schedule. She and the small handful of centers nationwide that are already using the new drug are careful to note that it does not prevent the development of Type 1, only delaying its onset for an average of two years. That might not seem like much to the average person, but it is significant for families who face all of the extra work that comes along with this disease, notes Demeterco-Berggren. “If you tell a family that already has a kid with Type 1 diabetes that they can have just one day where they don’t need to worry about counting how many carbohydrates their child is eating, or how many shots they have to receive or worry about blood sugar in the middle of the night or when they’re in sports, for some families, just one day would be amazing, let alone two years,” Demeterco-Berggren said. And, with the speed of progress that the biotechnology industry has been delivering lately, there are good reasons to believe that yet another breakthrough might be delivered within the next two years. Dr. Athena Philis-Tsimikas, of the Whittier Diabetes Institute at Scripps Health, noted while it’s true that Tzield delays Type 1 by two years, that is a median. Some have gone much longer. And, she added, there are local examples of what’s to come in the next round of diabetes breakthroughs. Replacing damaged beta cells is one such promising possibility on the near horizon. In 2022, Vertex Pharmaceuticals in Boston purchased San Diego-based ViaCyte Inc. for $320 million. Both companies were working on trials of technology that can cause stem cells to become beta cells, allowing insulin production to be restored. “Vertex is now in their phase 2 trials and looking to start their phase 3,” Philis-Tsimikas said. “Someone that maybe gains two to five years with this intermediary agent, maybe by that time the cells are developed, and that could lead to a life free of insulin (shots).” “There are things being developed all the time and it’s happening now, not in 10 or 20 years from now.”