How close is it to the treatment of type 1 diabetes? No one should know better than Camilo Riccordi, Maryland, director of the Diabetes Institute in Miami.
The diabetic community is understandably skeptical of experts who promise treatments will be discovered in the near future. We’ve heard it for decades. But the truth is that researchers have made real (if slow) advances, and all the experiments happening today can provide the key to the holy grail of diabetes research.
Dr. Ricordi played much of the fundamental work to bring us where we are today. Currently, he has a unique perch at the Diabetes Institute (DRI), which helps guide some of the most exciting clinical trials in the world of diabetes. One such test will almost certainly become the most enthusiastically planned experiment in the diabetes world: a new test of Pharmaceutical, apex of lab-grown islet cells that are physically protected from the immune system.
Science is very exciting, but the reality of implementing treatments can be depressing. Ricordi believes we are only a few years away from experimental proof of actual Type 1 diabetes treatments, but even this most optimistic scenario is full of regulatory and economic challenges. I don’t know how many years it will take to get treatment for a person with diabetes.
Disorders aside, Ricordi and DRI are completely dedicated to finding treatments for type 1 diabetes. This is what he had to tell us.
What is the definition of type 1 diabetes treatment?
There are many competing definitions of “treatment” for type 1 diabetes.
For example, some may say that pancreatic transplantation cures T1D. After all, in some cases, healthy insulin production can be fully restored and patients can eat whatever they like without using insulin. However, it is severe costs, repressive regimens of immunosuppressive drugs with side effects, and the likely chance of ultimate failure. Meanwhile, some biotech companies are focusing on “functional treatments.” This is a combination of medicine and technology that allows T1D people to live a completely normal life without changing the physiology underlying the disease.
Ricordi wants something better, a true cure, something that wipes out Type 1 diabetes and ensures it never comes back.
“My definition of treatment is the ability to replace the biological endocrine function of the pancreas that has been selectively destroyed by an autoimmune response, and do so without toxic interventions that could lead to uncher drugs or other problems. Diabetes cannot be replaced with another disease.”
“But my definition goes beyond that because my motto is, “There is no cure without prevention.” The moment cell therapy is successful, we need to prevent disease recurrence and work to prevent disease-related accelerated aging and chronic complications. ”
“What we’re introducing to prevent disease recurrence is the possibility of preventing disease in the first place. This is a 360-degree intervention. Primary prevention, treatment of diagnosed people, prevention of disease recurrence, accelerated aging and other complications.”
How close are we approaching treatments?
Ricordi is well aware that people in the diabetic community are sick and get tired of hearing that the treatment is “five years away.” Back in 1984, his colleagues pushed him onto a plane from Milan to America, saying, “They found a treatment for islet transplantation and need to take it back to Europe.” But there was still Decades The work you need to do.
In 1988, Ricordi developed an automated method of islet isolation that would help make islet transplant feasible. In 1990 he helped show that islet transplantation can revive insulin production in people without pancreas. In 1999, Edmonton’s protocol did the same for patients with autoimmune munmune diabetes. In 2021, the apex was successful with laboratory-grown islet cells, offering new possibilities for an endless supply of healthy islets for transplantation.
“It’s been a long way, but now we’re working on the last part: tolerance induction and recurrence of illness. It makes me very hopeful… The development of proof of concept of treatment can be within five years or more than 15 depending on many factors.”
Ricordi warns that even if true treatment is confirmed under experimental conditions within the next few years, “it takes five years to follow up with the first group.” It will then take even longer to scale up and make treatments widely available.
“The timing of implementation for all people with type 1 diabetes is a different question. We don’t want to create false hope.”
But he offers defense in past times when his colleagues have probably over-announced how close we are to treatment.
“If you don’t think that either of these trials or protocols or strategies can make a difference, I think it’s important to keep the focus and strength of our team in treatment, as if it’s around the next corner, it’s important to be aware that it will take another recade or more.”
Immunosuppression issues
In June, we found that two early apex patients were completely dependent on insulin. “It was an incredible milestone to show the stem cell-derived islands. can Induces reverse diabetes and long-term insulin independence,” says Ricordi.
However, the first successful T1D therapy at the apex, transplantation of laboratory-grown islet cells requires the use of immunosuppressant drugs. These drugs pose serious risks, including cancer and life-threatening infections.
It gets even worse. Prograf (Tacrolimus), the main anti-pump drug used for islet transplantation, is toxic to the cells it actually protects. In some cases, this drug can even cause diabetes. This toxicity helps explain why most islet transplants ultimately fail in the long run.
“The ultimate goal is to have these transplants without immunosuppression,” says Ricordi.
Best chance for treatment: VX-264
The most exciting research currently taking place at the Diabetes Institute tries to do just that. Ricordi is in the process of helping to activate the first human trial of VX-264, the latest in the apex therapy that encapsulates implanted islet cells and protects them from the immune system with physical barriers. According to Ricordi, the device is the result of “amazing engineering and nanotechnology.” DRI will become one of several sites in multiple countries to carry out this potentially game-changing task.
The Diabetes Institute is currently enrolled in the trial for VX-264 and is looking to find a brave and potentially very fortunate patient who is willing to accept gambling on what could be a milestone in the diabetes treatment race.
If everything goes according to the plan, the first patient will be implanted immediately. They receive partial doses of islet cells, and initial results are primarily evaluated for safety rather than efficacy. The trial is expected to end in May 2026, but earlier results will be available.
Diabetes Institute
Other exciting trials
DRI never puts all the eggs in one basket. The vertex is only one approach and has not yet been successful. Even so, it may not be the right treatment for everyone with type 1 diabetes.
“It’s true that there’s a pole position at the top, but we don’t know who’ll win until the race is complete,” says Ricordi. “I believe in combination strategies. I don’t know if there’s a bullet that’s 100% successful for anyone.”
Ricordi highlighted two other particularly exciting trials that DRI helps. Both focus on immunosuppression, what Ricordi called the “final part” of the treatment puzzle.
The first is the dream of a biotechnology startup named Itolerance, working on a microgel that can be mixed with islet cells prior to implantation. This substance can attack new islets and cause self-destructing T cells. It can also retrain the immune system to privilege the area, allowing islet cells to not alleviate their work. As a result, local immune resistance “stumbles the immune system and accepts the transplanted organ as if it were cells of its own” and it may all function without general immunosuppression.
“We are very pleased to have a non-device-based approach to resistance induction as well,” says Ricordi. At this point, we don’t know if physical immune system barriers like the apex work first or long term. Viacete, the original vertex competitor, appears to have seen the end of the physical encapsulation experiment.
Ricordi is equally excited about the trial of anti-rejection drugs, which could be significantly better than Programaf, a “co-stimulatory blocking molecule” developed by Eledon Pharmaceuticals. Unlike Prograph, this drug is not a diabetic agent at all. Ricordi says early studies have shown that “three times the production of insulin from implanted islets.”
Finding improvements over Prograph doesn’t exactly constitute a treatment, but Ricordi says it’s a “incredible step.” Islet transplantation can be much safer and longer lasting. This could make the procedure a viable option for more patients. “Maybe this could become a critical component of the resistance induction protocol to ultimately complete drug-free islet transplantation.”
Of course, there are other trials happening around the world that do not involve the Diabetes Institute. Diogenx, for example, is working on synthetic proteins that the pancreas may grow new beta cells. Buyed by the apex, the Baiasite laid the foundation for gene-edited islet cells to avoid the apex.
Some of these different strategies may join together to form the final treatment. Ricordi is somewhat suspicious of islet cells that have been gene-edited for “complete stealth,” for example. But he believes gene editing is “a big part of the line” with a lighter touch. “It’s easier to induce immune resistance in those islets when it reduces the immunogenicity of cells.”
How much does it cost to treat type 1 diabetes?
“Everyone asks when we’ll get treatment, but I also ask who can afford it. We need to keep things as nonprofit as possible.”
In Europe, the government is simultaneously approving drugs and prices, Ricordi explains. “But in the US, when the FDA approves a drug, it’s in the wild west.”
“Advanced cell therapy can cost hundreds of thousands of dollars. I’m wondering if it’s economically sustainable.”
Ricordi works closely with the pic on test therapy, but is completely separate from the team determining how to price approved treatments. Vertex spends far more than $1 billion to acquire and develop its experimental therapy, and has a strong incentive to charge in bulk for treatments that it receives as a miracle.
The vertex is to charge your arms and legs for type 1 diabetes treatment? All I can say is, “I hope not, but I don’t know.”
Choosing DRI
The commitment to the cause led to Ricordi in Miami in the first place. In the early ’90s, Ricordi, which was heavily adopted by multiple institutions, refused large dogs like Harvard University in favor of the Diabetes Institute.
“I chose DRI because it provided something unique. It’s a mission where I can concentrate on finding treatments for type 1 diabetes. It’s been my lifetime professional mission since my little cousin was diagnosed with type 1 when he graduated from medical school.”
Dr. Ricordi recently resigned as head of science, but he is deeply involved in the continuing work. New manager Mattias von Hellas, Maryland, has been appointed. Dr. von Herat, a global expert in diabetes and immunology, will not change the organization’s mission.
“This building must remain committed to finding treatment for diabetes.”
Ricordi has shown this commitment throughout his career. When he invented a new process for islet cells, a technology currently used for islet transplantation around the world, he freed up all his intellectual property and allowed him to expand as quickly as possible. When scientists join the Diabetes Institute, they are asked to make similar commitments:
“If you work for DRI, you need to swear that you are not here to keep your secrets. You have to work together, share and help everyone else. We can help anyone in the world who can help you get treatment as quickly and in the most efficient way possible.”
(TagStoTRASSLATE) Beta Cell (T) Treatment (T) Diabetes Institute (DRI) (T) Insulin (T) Intensive Management (T) Islet Cell Transplantation (T) ITERANCE (T) US Food and Drug Administration (FDA) (T) Vertex (T) Viacytete
