“Our Trials are Totally Different.” The Denise Faustman Interview

There’s no question that Dr. Denise Faustman does things differently.

Dr. Faustman’s research has won her avid fans and raised millions from donors, but it has also provoked significant pushback from major diabetes organizations. Dr. Faustman wears that repudiation like a badge of honor, proof that her work is disruptive and important.

A Maverick Approach

Dr. Faustman takes an unconventional approach. Her work, which is focused on the underlying immune response responsible for type 1 diabetes, has led her to a very old and inexpensive drug, a tuberculosis vaccine used widely in the developing world. It’s a treatment that may have been hiding in plain sight for generations.

Dr. Faustman’s immune intervention trials also concentrate on adults with long-standing type 1 diabetes. By contrast, virtually all of her peers have targetted patients with new-onset diabetes, or even patients that have yet to develop diabetes. This approach is generally considered more likely to succeed, because the targets of the interventions have not yet entirely lost their ability to secrete insulin. But in Dr. Faustman’s telling, it hasn’t accomplished much yet:

“Over the last 30 years, immune intervention trials in type 1 diabetes haven’t had very good outcomes. The important diabetes advances have been different versions of insulin and new forms of blood testing, but the immune intervention trials have been a very sad point. Nothing’s worked very well, no new treatments have been approved.”

“Our trials are totally different. They’re all in people that have had diabetes for decades.”

Dr. Faustman told me that a who’s who of diabetes experts has told that her approach is crazy, a response that has clearly tickled her contrarian side.

“We thought the need is to treat the people that actually have the disease. It sounds like a no-brainer, but keep in mind that nobody else is doing it!”

The decision to study patients with long-established diabetes, which was originally made well over a decade ago, was inspired in part by financial constraints: “we could not afford to do new-onset diabetic trials.” And focusing on an incredibly inexpensive generic drug meant that Dr. Faustman had no way of interesting large pharmaceutical companies and their very large R&D budgets.

“It’s a terrible drug to make money off of.”

BCG

The drug is BCG, the Bacillus Calmette–Guérin vaccine, which was first used a century ago in order to protect patients from tuberculosis. It is considered extremely safe, and is still administered to millions of infants annually, most prominently in the developing world.

BCG first piqued Dr. Faustman’s interest because it was “the only drug in animal models that has worked in established diabetic mice.” Exactly why it worked, though, still requires some unraveling. The results of her Phase I trial – results that were both celebrated and questioned – showed that patients receiving the vaccine experienced impressive improvements in glucose control without restoring insulin production or insulin sensitivity.

Most immune intervention drug trials involve the suppression of immune cells that are harming the body – in the case of type 1 diabetes, the T-cells that attack the pancreatic Beta cells. But the BCG vaccine works the opposite way, not by depleting the immune system, but by putting something else back in.

“BCG adds to the microbiome. It’s actually the tuberculosis organism, inactivated. So you’re putting back in, effectively, tuberculosis. When you revaccinate with this vaccine, the reason it takes a little while is that it gets to your bone marrow and actually resets your stem cells in your bone marrow.”

Why would you want tuberculosis in your bone marrow? Dr. Faustman referred to the hygiene hypothesis, the idea that many autoimmune and allergic diseases are caused at least in part by our excessively sanitary modern world. Myobacterium tuberculosis may have co-evolved with humans for thousands of years, and its absence may therefore trigger immune system dysfunction.

“The absence of this organism has allowed these rogue immune systems to take off and create all these autoimmune diseases. We’re just reestablishing this synergistic relationship to get immune tolerance.”

Because of its many possible effects, BCG has lately become “fashionable” in the research community, Dr. Faustman told me. The old vaccine is being evaluated as a treatment for other serious autoimmune diseases, and it has also been theorized to provide protection against the novel coronavirus that causes COVID-19.

But how does all that help with your blood sugar levels? Dr. Faustman believes that when BCG vaccine “retrains” the immune system, it also causes the immune system itself to begin utilizing large amounts of glucose in the bloodstream.

“What really happens is that their white blood cells now become the regulator of sugar, and have restored sugar transport. So there are underlying defects in type 1 diabetes in the use of the lymphoid system as a sugar regulator, and BCG restores that.”

The Effect

“It’s definitely a game-changer. Our hope, based on the early results, is that it will lower your A1c 10-15%, stably. You can use less insulin, and have better blood sugar control.”

It is, however, a slow process:

“It takes a number of years to have its metabolic and immune effects. We know we need to follow these patients for 3-5 years to get the maximal effect. But it’s got good durability. It’s quite permanent.”

The nine patients that originally received the BCG vaccine for the Phase I trial are still checking in with Dr. Faustman’s lab every six months, and she says that they continue to enjoy dramatic improvements in glucose management.

“Everyone wants to know if we have any [trial participants] off insulin. We have one person off insulin. Normally when he’s on a fairly low dose of insulin his A1c is in the normal range. When he goes off insulin, he no longer goes into DKA, but he goes up to around 7.2%.

“Is it gonna last twenty years? Thirty years? We don’t know yet.”

Sample Size

One of the chief concerns that other experts have raised with Dr. Faustman’s work is the tiny sample used in her Phase I trial: a mere nine patients received the BCG vaccine.

Dr. Faustman is completely unphased by this criticism, arguing that the statistical significance of her results speaks for itself.

“A p-value of .05 is as statistically significant in a sample of 10 as it is in a sample of 200,000. The only difference is, in the sample of 10, everybody responded, and the magnitude of the response was large.

“If you have to design big trials, you have small effects and unresponsive groups. And that’s what you’re seeing in the [rest of the] diabetes community.”

The criticism of her work by major diabetes authorities, particularly ADA and JDRF, doesn’t seem to have phased her, although she admits that it has slowed down the pace of her trials. She quoted one of her most significant boosters, the late Lee Iococca, a rockstar among American executives as the CEO of Chrysler during the 1980s. Iacocca told her, “I love it when they’re shooting the cannons at me. It means I’ve got something good.”

Dr. Faustman continued: “If they didn’t think it was competitive, it wouldn’t matter to them. The fact that they shot their cannons, to issue statements that were not truthful, it shows that it mattered to them a lot, that it was threatening to them.” She believes that the criticism set forth by the ADA and JDRF in a remarkable joint letter was not only misguided but dishonest.

Don’t Do It Yourself

Referring to the BCG vaccine’s long history of safety, Dr. Faustman suggested that there was little downside to her proposed intervention.

I felt an uncomfortable comparison to some of the pandemic era’s health controversies, in particular to the arguments advanced by many in favor of poorly investigated COVID-19 treatments such as hydroxychloroquine and ivermectin. I asked her what she would say to readers that are ready to go get the BCG vaccine themselves, before it’s been evaluated by the FDA. It’s a question she deals with constantly.

About once a month, Dr. Faustman gets a call from a physician, asking confidentially where one might acquire BCG of the correct strain. Sometimes it’s pharmacies attempting to fulfill a prescription, sometimes it’s the patients (or parents) themselves.

“Everyone’s trying to do it in the closet because it’s a safe drug, right? People try to do it all the time. But you should really wait until we get the right strain and the dosing. What we try to say is that if you get vaccinated with the wrong strain of BCG, we’re not sure, when we give you the right strain, that it’s gonna work.”

The BCG vaccine may be easy to come by in countries where it is used frequently, but there’s no telling what strain you’d get and what the effects would be.

For patients that are very eager about BCG, Dr. Faustman recommends registering for one of her clinical trials.

Timeline to Treatment

There’s already a phase II trial underway, which will test the BCG vaccine in adults with long-standing type 1 diabetes. This expands the study size from 9 to over 200; Dr. Faustman expects that good results in a much larger cohort will finally resolve the criticism and controversy that has dogged her for years.

Meanwhile, her lab is beginning its first trial of children with type 1 diabetes.

In an ideal world, she told me, the phase II trial for adults would be followed by phase III, an even larger study which she would then present to the FDA for evaluation. In a best-case scenario, the approved drug could be administered to regular patients in “five to eight years.”

“We’re moving as fast as we can.”

Source: diabetesdaily.com

Back to School with Diabetes Amidst the COVID-19 Variants

This content originally appeared on Beyond Type 1. Republished with permission.

By Lala Jackson

This article was published on August 13, 2021. As of Monday, August 23, the FDA has granted the Pfizer and BioNTech COVID-19 vaccine full approval for ages 16 and up, with the Emergency Use Authorization (EUA) still in effect for ages 12-15 and for booster doses for immunocompromised individuals. Full approval for other COVID-19 vaccines currently under EUA is expected soon.

While hopes were high that we could head back to school for the 2021 school year as though we were closer to “normal,” the development of COVID-19 variants amidst low vaccination rates has thrown a wrench in plans. But when kids need to get back to in-person schooling for quality of life, quality of learning, and socialization, how can we best keep them safe?

To help answer this and other questions about going back to school safely, JDRF—in collaboration with American Diabetes Association and Sansum Diabetes Research Institute—hosted a conversation with doctors and experts from the CDC, ADA, and the Fairfax County Health Department (Virginia).

Moderator Dr. Kristin Castorino, senior research physician at Sansum Diabetes Research Institute, kicked off the event with the most pressing question—is it even safe for students and their teachers who have diabetes to return to in person schooling, particularly for those under 12 who cannot be vaccinated yet?

“I’d change the question from ‘is it safe?’ to ‘is it appropriate?’ and I think it is,” answered Dr. Fran Kaufman, pediatric endocrinologist and chief medical officer at Senseonics. “There aren’t known answers as things change… but we need to get our kids back to school, not only for learning but for socialization.”

Dr. Kaufman stressed that the best way to make school safe is for everyone who can get vaccinated to do so. Dr. Christa-Marie Singleton, MD, MPH, senior medical advisor at the CDC later elaborated, “Vaccines protect folks against serious symptoms, hospitalization, and death. The best way to protect ourselves, our families, and our youngest people is for the adults and kids over the age of 12 around them to get vaccinated.”

“We also know about the importance of masking,” continued Dr. Kaufman. “It’s important to follow the CDC’s recommendation that all children and adults should be masked in the indoor school environment.”

What About the Legal Rights of Kids With Diabetes?

Particularly as some states ban school districts from being able to require masks in indoor learning environments, what legal protections do kids with diabetes have to stay safe in school? Crystal Woodward, MPS, director of the ADA’s Safe at School campaign, stressed “the rights of students with diabetes do not go away during a pandemic. They have legal protections under federal and state laws. Those accommodations may look a little different, but they do not go away.”

Similarly to how the Americans with Disabilities Act protects people with diabetes in the workplace, section 504 of The Rehabilitation Act protects the education of children with disabilities like diabetes. This law allows children with diabetes and their families to create what are known as 504 plans, which clearly outline agreed upon accommodations for students with disabilities at school.

While parents cannot dictate the actions of other students, they can include directives for their own children to stay safer from COVID-19 in 504 Plans, like instructions that their student must always wear a mask or will need extra physical distance in a classroom setting.

“It’s imperative that [children with diabetes] have a section 504 plan,” Crystal explained. “Everyone needs to be clear on what accommodations will be provided and by whom, like the student having the ability to take an exam at an alternate time if blood glucose levels are out of range during the scheduled test time.” Ensuring the student also knows what is in their own 504 plan can help them feel more empowered and comfortable asking for what they need.

For distance learning, 504 plans can dictate that children with diabetes can take snack or meal breaks at times best for the student, or have an agreed upon communication method with the teacher if the student needs to take a break to attend to a low or high blood sugar.

“Bottom line: the rights of students do not go away,” Crystal reiterated. “Students with diabetes and their families should work with schools and everyone needs to understand their role and responsibilities, and the plan should be updated as needed. It’s always better to get it in writing. Put the 504 plan in place while everything is going well—you never know if a principal or a nurse or a teacher is going to be there throughout the year.” Panel members stressed that families who don’t speak English, particularly in public schools, have a legal right to translators who can help establish 504 plans.

Jacqueline McManemin, RN, BSN, certified diabetes education and care specialist (CDECS) and assistant nurse manager for the health services division of Fairfax County Health Department in Virginia, spoke about what they’re continuing to do in their school district (one of the 15 largest in the nation) to keep students safe. “Parents should expect to see much of the same precautions this year that were in place last year. Particularly when students are inside, they should be masked.”

School administrators across the country can work to make schools more safe for all children, particularly those with chronic illnesses like asthma and diabetes, by putting in protective measures like establishing two different health clinics—one for people exhibiting symptoms of COVID-19 or other communicable illnesses and a separate clinic for routine care and injury treatment.  Meals can be eaten outside as weather permits and student interaction in hallways can be minimized by teachers rotating between classrooms rather than groups of students switching classrooms every period. Protocol also needs to be clearly communicated with all staff and parents about what to do if a student starts showing symptoms of COVID-19 while at school.

Getting Kids Mentally Ready for in-Person School

Back to school doesn’t just mean a change of location, it’s a change in schedules, types of interaction and stimulation, and levels of distraction that can also impact diabetes care. Psychologist Cynthia E. Muñoz, PhD, MPH, assistant professor of clinical pediatrics at the University of Southern California’s Keck School of Medicine and president of healthcare and education for the American Diabetes Association, reminded the community that the impact of the pandemic on each individual has been unique and therefore approaches to regain a sense of normalcy must be unique too.

“For parents and guardians, be aware of how you’ve been impacted. Seek support, through family, through primary care, through a therapist. Find ways to talk about your fears or concerns,” encouraged Dr. Muñoz. She went on to suggest ways to get kids mentally and physically ready for school again.

“Now that schools are starting to open, it’s time to start looking at sleep schedules, screen time, and start shifting routines and schedules to get children ready for the new routine,” she noted. “Many people watch a lot of content on social media or television—not just kids, everyone—but it’s a passive interaction with others. Shifting to a more active form of communication with others can be another way to help people ease into the change of a lot more interaction than people have had in the last year or so.”

Helping Kids Who Feel Singled Out

Kids with diabetes often deal with feelings of being the odd kid out, having to visit the school nurse, having to deal with special routines. When COVID-19 is added, kids with diabetes may feel like they’re the only ones taking special precautions, which can be additionally isolating. How can parents help children dealing with these feelings?

“I like to approach this question around the concept of support, building layers of support around the student,” Dr. Muñoz explained. “One level should be ensuring that someone at the school should know that the child has diabetes and knows what kind of support they need. Another category is who could know [the student has diabetes], but doesn’t necessarily have to, like friends. For the student with diabetes, getting support from a friend or classmate they trust could go a long way. If the student feels like they’re going to be the only one wearing a mask, they can ask a friend to wear it with them.”

“I think it’s important for adults to be sensitive to this,” Dr. Muñoz continued. “Saying “everyone has something different” might minimize how a student feels. Acknowledging their feelings and taking the time to ask them what will help goes a long way.”

To get advice from other parents and guardians or to help your student with diabetes find other kids who understand, be sure to join the Beyond Type 1 community.

Learn more about the JDRF – Beyond Type 1 Alliance here.

You Can Watch the Entire Conversation Here:

Source: diabetesdaily.com

Tackling Type 1 Diabetes – Where Are We on Technology and Research?

This content originally appeared on diaTribe. Republished with permission.

By Andrew Briskin

Andrew Briskin joined the diaTribe Foundation in 2021 after graduating from the University of Pennsylvania with a degree in Health and Societies. Briskin is an Editor for diaTribe Learn.

At the Milken Institute 2021 Future of Health Summit, leading experts in type 1 diabetes research and innovation discussed the path toward a cure, the latest in glucose monitoring technology, and the importance of screening for type 1 diabetes.

A group of leading experts in type 1 diabetes research and innovation took part in the panel discussion, “Tackling Type 1 Diabetes: Where the Science is Heading” at the Milken Institute 2021 Future of Health Summit last month. They exchanged insights on the advantages of continuous glucose monitoring, automated insulin delivery (AID), Time in Range for better diabetes management, as well as tantalizing new possibilities for curing type 1 diabetes.

The discussion from June 22nd was moderated by diaTribe Founder Kelly Close and included:

  • Aaron Kowalski, Ph.D. – CEO, JDRF International
  • Shideh Majidi, M.D. – Assistant Professor, Pediatric Endocrinology, Barbara Davis Center for Diabetes
  • Felicia Pagliuca, Ph.D.  – Vice President and Disease Area Executive, Type 1 Diabetes, Vertex Pharmaceuticals
  • David A. Pearce, Ph.D. – President of Innovation, Research and World Clinic, Sanford Research

The panelists began by discussing how continuous glucose monitors (CGM) now provide people with type 1 diabetes even more information and the power to manage their glucose levels. CGM data provides people with crucial metrics such as Time in Range (TIR), which corresponds to the percent of time someone spends within their target glucose range – usually 70 to 180 mg/dL. This target glucose range may vary though, for example, if you are pregnant. You can learn more about the helpful metrics that CGM provides here. Alongside A1C, TIR allows more insight into your day-to-day diabetes management by showing fluctuations in glucose levels caused by factors like meals, exercise, illness, and more.

However, CGM is not perfect or widely accessible yet. The panelists touched on this issue of access to CGM and the existing disparities in care across race and type of insurance. Dr. Majidi emphasized that in populations with access to this technology, CGM use has increased from 20% to over 80% of patients over the last five years. However, some studies have shown that providers tend to prescribe technology only to certain patients due to unconscious biases about which patients may be able to handle using advanced technology.

Advocating for early and consistent training for healthcare providers on addressing these biases, as well as provider and patient education on CGM and other technologies for glucose management, Dr. Majidi said, “we need to look at these unconscious biases to start providing everyone with the opportunity to use and learn about new technology.”

The panel then explored the advantage of AID hybrid closed-loop systems. These systems combine a CGM, insulin pump, and an algorithm that allows the CGM and insulin pump to talk to each other. Dr. Kowalski said he was especially encouraged by the advancements in AID systems, emphasizing that it not only decreases the number of highs and lows, but it also removes much of the burden of diabetes management from patients and their families.

AID systems especially benefit families with children who have diabetes, reducing concerns from parents about the safety of their children during the night or at other times when the risk of hypoglycemia is high. The panelists said they were hopeful that these new innovations are bringing us closer to developing a fully closed-loop artificial pancreas, which could automatically respond to changes in glucose in real time without the need for a person to deliver manual boluses or calibrations.

Echoing their advice on how to address disparities in CGM use, the panelists noted the importance of education for healthcare providers to combat disparities in prescribing AID to ensure equal opportunity for all to achieve better health outcomes.

The discussion then shifted to the latest research towards a cure for type 1 diabetes, focusing on beta cell replacement therapies. Because type 1 diabetes occurs as a result of the body’s immune system attacking and destroying its own pancreatic beta cells (the cells that make insulin), scientists have been researching how to replenish the beta cell population from stem cells. Scientists believe that stem cells, not yet fully differentiated or mature cells, could potentially be directed to become functioning beta cells.

Dr. Pagliuca shared updates from her work at Vertex, studying stem cell-derived beta cell transplants in type 1 patients with impaired awareness of hypoglycemia. This initial study relies on systemic immunosuppressive drugs (these are drugs that “turn off” the body’s immune system so it won’t attack the implanted cells) to protect the implanted beta cells. The hope is that future studies will seek to use a different method called encapsulation, which protects beta cells from the immune system with a physical barrier, avoiding the need for immunosuppressant medications.

So far, with the successful conversion of stem cells into mature beta cells accomplished in controlled lab settings, the science has developed to the point of testing stem cell-derived beta cells in clinical trials, with Vertex first clinical trial now enrolling patients. This initiative will encompass the entire type 1 community, with Dr. Pagliuca stressing that “transitioning these breakthroughs into the clinical phase will require participation from all stakeholders, patients, researchers, and healthcare providers.”

Considering the latest research into the immunobiology of type 1 diabetes, the panelists advocated for significant increases in screening for type 1 across the general population.

Dr. Pearce advised that testing for the presence of specific autoantibodies (small molecules in the body that are the cause of the immune system attacking a person’s own beta cells) in the general population is essential for implementing prevention programs, given that the presence of at least two of these autoantibodies is a very predictive measure to assess the risk of developing type 1 diabetes.

According to him, the predictive power of these screenings make it is possible to classify an individual as having type 1 diabetes years in advance of any symptoms, even while they still have normal glycemic control. In this way, type 1 diabetes can be classified into 3 stages – stage 1 is when someone has two or more diabetes-associated autoantibodies, but normal glycemia and no symptoms. Stage 2 is when you have the autoantibodies, have begun to develop glucose intolerance or abnormal glycemia, but still no symptoms. Stage 3 is when symptoms begin and you are diagnosed with type 1 diabetes. Classifying diabetes in this way and identifying those in the early stages could increase patient involvement in clinical trials, and help connect individuals to new drugs such as teplizumab (not yet approved by the FDA), that aim to delay the onset of symptomatic type 1 diabetes or prevent it altogether.

Drs. Pearce and Kowalski agreed, recommending a screening strategy involving primary care providers and screening children during the toddler years. On the importance of this screening process for involvement in clinical trials, Dr. Kowalski noted, “Diabetes is a global problem. The voice of the patient is hugely important on new devices and therapies, and clinical trial pathways are delayed when there isn’t equal participation in the trials.”

You can watch the panel discussion and hear insights from the four incredible experts here.

Source: diabetesdaily.com

An Update from the Controversial Dr. Denise Faustman

Remember Dr. Denise Faustman? Dr. Faustman is about as close as the type 1 diabetes world comes to having a celebrity researcher. Her lab, based at Massachusetts General Hospital, has given people with diabetes a lot of hope—and also generated plenty of controversy.

The BCG Vaccine

In 2018, Dr. Faustman’s team released the first results from a Phase I trial of a novel but appealingly simple and non-invasive treatment that could make blood sugar management much easier for patients with type 1 diabetes. The treatment? The bacillus Calmette-Guérin (BCG) vaccine, which has been used to prevent tuberculosis for a century. The BCG vaccine is one the oldest, longest-studied and best-understood vaccines; it is inexpensive and considered very safe. It is administered to as many as 100 million newborns across the globe annually.

The early results were encouraging: patients with type 1 diabetes who received just two doses of the BCG vaccine, spaced four weeks apart, achieved markedly lower A1c levels when compared to a control group. This result was first observed three years after the vaccine administration and continued for the next five years of follow-up. Participants receiving the experimental treatment had an HbA1c of just 6.18%, significantly lower than participants who received a placebo (7.07%). These results understandably created a lot of enthusiasm in the diabetes community.

The Authorities Push Back

The good results, however, also attracted some pushback from important diabetes authorities. The American Diabetes Association and JDRF took the unusual and striking step of co-authoring a joint statement to highlight “limitations” to Dr. Faustman’s research. The study, according to the statement, was minuscule – “nine people at the five-year time point, and three people at the eight-year time point” – and did “not provide enough clinical evidence to support any recommended change in therapy at this time.” While couched in cautious and moderate language, the letter clearly hoped to smother the hype that the BCG study had kindled.

A 2021 profile in Healthline stated that Dr. Faustman has now been “effectively shunned by the research community.” She has had to seek funding outside of the usual sources – something she’s done with aplomb, raising millions from private donors.

How BCG Might Work

Dr. Faustman hasn’t been as much in the news too much lately, but rest assured, she’s still working. And at last month’s ADA Scientific Sessions conference, we received an update to her ongoing work on the BCG vaccine. The team is still learning how and why BCG appears to work the way that it does.

The BCG vaccine is old, but its proposed mechanism in treating type 1 diabetes is new and surprising. BCG does not appear to restore insulin production, nor does it improve insulin sensitivity. So, what could explain the purported improvements in glycemic control?

Many experts have theorized that our immune systems have become less robust because our society is so much cleaner than it used to be. This idea, the so-called ‘hygiene hypothesis,’ also seems like a pretty good explanation for why type 1 diabetes is more common than it used to be. Dr. Faustman believes that the vaccine may help kick parts of the immune system into action, a mechanism that used to happen naturally back when we had a messier society. In particular, BCG helps trigger the proper function of “Tregs,” immune cells that are known to be dysfunctional in people with type 1 diabetes. Over a period of three years, participants in the study saw their Treg function slowly return to normal. This change also causes the immune system itself to burn more blood glucose, a shift that creates big glycemic improvements.

Interestingly, the BCG vaccine seems effective in patients that developed T1D during childhood (< 21 years), but not those that developed the condition as adults.

More Trials Planned

Meanwhile, several other trials of BCG have begun. A Phase II study is happening right now, and will monitor the effects of the treatment in more detail and with more participants; if the good results are confirmed, it could help allay the concerns raised by the research community about Dr. Faustman’s work. In addition, the Faustman lab is targeting a larger pediatric trial, which will test the vaccine in 150 adolescents, some with well-established type 1 diabetes, some newly diagnosed. Perhaps younger patients have an even greater opportunity to benefit.

The appeal of the BCG vaccine is obvious—a simple jab or two that could confer years of significant blood sugar improvements? Who wouldn’t be excited? But it would be best to continue to evaluate Dr. Faustman’s results with caution, given the skepticism of other researchers. Even if she’s right that BCG is a kind of silver bullet that can deliver durable A1c improvements, it will be years before she’ll have the data to make the case.

Source: diabetesdaily.com

Insulin at 100, Part 2: Failed Promises, Bold Breakthroughs

This content originally appeared on diaTribe. Republished with permission.

By James S. Hirsch

Insulin

Image source: diaTribe

The discovery of insulin in 1921 was heralded as the cure for diabetes. The reality was different.

Insulin, to be sure, could temporarily lower blood sugars to near-normal ranges, but it could also cause hypoglycemia – blood sugars that are too low – that could lead to shakiness and confusion or, in extreme cases, seizures, loss of consciousness, or death. Insulin was a daily, self-administered drug, but if used incorrectly, it could kill a patient just as well as it could save a patient. No self-administered therapy, before or since, has quite those same attributes.

What’s more, insulin’s therapeutic powers were overestimated. Yes, insulin lowered blood sugars, but maintaining near-normal levels was still very difficult – and elevated blood glucose over time was still dangerous. As a result, by the middle of the 1930s, patients who were taking insulin began developing serious complications caused by elevated glucose levels, including damage to the eyes, kidneys, nerves, and heart. Insulin hadn’t cured anything but had turned diabetes from a deadly condition into a chronic condition, and a perilous one at that. At the dawn of the insulin age and for many decades thereafter, even those who understood the importance of maintaining near-normal blood sugars did not have the tools to do so. Blood sugar levels were measured by proxy through urine tests, in which samples had to be boiled for three minutes. Simpler methods were developed by the 1940s, but home glucose monitoring was not available until the late 1970s.

Until then, patients – unaware of their blood sugar levels – gave themselves insulin doses flying blind.

But few people outside the diabetes world knew about the daily rigors and risks of the disease – not only because it affected a relatively small percentage of people but also because the insulin narrative was too powerful.

Diabetes, after all, had been cured or at least resolved. That’s what all the pictures showed. That’s what the headlines blared. And that’s what the ads promoted.

Eli Lilly’s ads, for example, initially touted insulin as “An Epoch in the History of Medicine” and later featured a beautiful bride on her wedding day, kissing her beaming father, with the tagline, “Our favorite picture of insulin.”

Even that picture paled in comparison to the astonishing newspaper and magazine stories about insulin, and not just those about Elizabeth Evans Hughes. Insulin was a redemptive tale about science and survival.

Eva and Victor Saxl were Czech immigrants who fled to Shanghai during World War II. There, Eva was diagnosed with diabetes, and when her insulin supplies ran short, Victor, a textile engineer, found a book that described how to make insulin and, using the animal organs from a nearby slaughterhouse, brewed up enough insulin for his wife to survive. After the war, they immigrated to the United States, and when their story was discovered, they soon found themselves on numerous radio and television shows, including Edward R. Murrow’s, and a movie was also produced – about a husband’s devotion to his wife, expressed through the salvation of insulin.

Other life-saving medical breakthroughs occurred – antibiotics in the 1940s, the polio vaccine in the 1950s – and these would treat more people than insulin. But the unique circumstances of insulin’s discovery, with the young, untested scientists finding the potion that would bring children back from the brink of death, was too dramatic to ignore. In 1988, that story was the subject of a television movie on Masterpiece Theater called “Glory Enough for All,” based on Michael Bliss’s definitive book, “The Discovery of Insulin.”

I watched the movie on PBS when it was released, and it featured the brawling Toronto researchers – Banting and Collip literally came to blows over control of the experiments. But ultimately, the movie was about the triumph of medical science in saving dying children, and among the researchers, there was “glory enough for all.”

And then the movie ended.

There was nothing about living with diabetes – about the wildly fluctuating blood sugars, about the relentless demands, about the injections and the doctor visits and the complications, about the dietary restrictions, about the stigma and the isolation and the limitations of insulin.

“Glory Enough for All” was introduced by Alistair Cooke. An American-born Brit with a silver tongue, Cooke was enthralled not only by insulin’s inspirational story but also by the phrase “islets of Langerhans,” used to describe the island of pancreatic cells discovered by Paul Langerhans. “Islets of Langerhans” just rolled off Alistair Cooke’s tongue. To him, insulin was not just a miracle. It was poetry.

The lyrical beauty of insulin was lost on patients. Many of them, in fact, were frustrated that their own stories weren’t being heard. The parents of young patients were frustrated as well.

In 1970, a professional singer in Philadelphia, Lee Ducat, had a 10-year-old boy with type 1 diabetes, and she was miffed by the breezy disregard of his doctor, who told her that “insulin was the cure.” Ducat knew that wasn’t true, so with several other parents, she formed the first chapter of the Juvenile Diabetes Foundation (which is now the JDRF). Other parents soon opened chapters in New York, Washington, New Jersey, and Miami, and their mission was to educate the public about the stark challenges of diabetes in hopes of raising money and finding a cure.

They had no use for the American Diabetes Association, which was founded in 1940 and for many years was little more than a social club and referral service for physicians. As far as the parents were concerned, the ADA was complicit in perpetuating the jaunty insulin narrative that had hurt the cause for decades. Unless the truth about diabetes was known, how could lawmakers, regulators, philanthropists, and journalists – not to mention clinicians – do what had to be done to improve the lives of people with diabetes?

That question was driven home when the JDF chapter in Miami bought a full-page newspaper ad in 1972 to publicize its cause. The ad featured a little boy in a crib holding a glass syringe, and it described the many complications that could arise from diabetes, including blindness and amputations. The headline read, “The Quiet Killer.”

On the day the ad appeared, Marge Kleiman, whose son has type 1, was working in the JDF office, and the phone rang.

“I’m Charles Best,” the caller said, “and I discovered insulin.”

Now retired, Best had become an icon who, after Fred Banting died in 1941, carried the mantle for the Nobel-winning team that had discovered insulin. Best had been praised by the pope, the queen of England, and other heads of state, and he had given the keynote address at the ADA’s first meeting and later served as its president. He happened to be in Miami on the day the JDF ad appeared, and he was outraged.

“What kind of propaganda are you using?” he screamed. “You’re frightening people! This is not the way it is!”

Kleiman knew better. “Dr. Best, what you did was wonderful,” she said. “It allowed people to live longer. But we’re not trying to frighten people. If you tell the truth, maybe they can avoid these complications. Please don’t tell us to keep quiet.”

The JDRF, now a massive international organization focused primarily on type 1,  has continued to tell the truth about diabetes – and fund research – ever since, but changing the insulin narrative was not going to be easy.

Patients could at least take solace that the insulins kept getting better. The first extended-action insulins were introduced in 1936 and continued with widely used NPH insulin (1946) and the Lente insulins (1951). But the real improvement came in the 1970s, spurred by concerns about actual insulin supply. Meat consumption was declining, and slaughterhouses were cutting production, while the number of people with diabetes had been rising steadily (in 1976, there were about 5 million Americans with the disease). At some point, insulin demand could outstrip the animal-based supply.

As described in the book Invisible Frontiers: The Race to Synthesize a Human Gene, by Stephen S. Hall and James D. Watson, the specter of an insulin shortage triggered a race to develop genetically engineered insulin using recombinant DNA technology. Investigators succeeded by inserting the insulin gene into bacteria, which produced insulin that was chemically identical to its naturally produced counterpart.

The first human insulins, Humulin (made by Eli Lilly) and Novolin (made by Novo Nordisk), were introduced in the 1980s. Whether they were superior to animal-based insulins is a matter of debate, but they alleviated fears about an impending global insulin shortage.

Moreover, researchers soon discovered that changing the order of two amino acids in the human insulin molecule created a faster-acting formulation, and that led to the introduction of Humalog (1996) and Novolog (1999). Known as “insulin analogs” because they are more analogous to the body’s natural release of insulin, they were considered clear advancements. Another huge leap came with long-lasting basal insulin analogs, specifically Lantus (by Sanofi in 2000) and Levemir (by Novo Nordisk in 2005). These insulins keep blood sugar levels consistent during periods of fasting and, typically taken once a day, replicate the insulin release of a healthy pancreas. They were immensely popular and also used by many type 2 patients – Lantus was a $5 billion a year drug by 2011.

The improved insulins changed how patients cared for themselves, as the new formulations led to “basal-bolus” therapy – a 24-hour insulin complemented by a mealtime insulin – and that became the standard of care for type 1 diabetes. (Insulin pumps use the same basal-bolus framework.)

A new era of diabetes care, thanks to these insulin breakthroughs, appeared to beckon.

Stay tuned for part three of this riveting story next week!

I want to acknowledge the following people who helped me with this article: Dr. Mark Atkinson, Dr. David Harlan, Dr. Irl Hirsch, Dr. David Nathan, Dr. Jay Skyler, and Dr. Bernard Zinman. Some material in this article came from my book, “Cheating Destiny: Living with Diabetes.”

About James

James S. Hirsch, a former reporter for The New York Times and The Wall Street Journal, is a best-selling author who has written 10 nonfiction books. They include biographies of Willie Mays and Rubin “Hurricane” Carter; an investigation into the Tulsa race riot of 1921; and an examination of our diabetes epidemic. Hirsch has an undergraduate degree from the University of Missouri School of Journalism and a graduate degree from the LBJ School of Public Policy at the University of Texas. He lives in the Boston area with his wife, Sheryl, and they have two children, Amanda and Garrett. Jim has worked as a senior editor and columnist for diaTribe since 2006.

Source: diabetesdaily.com

Teplizumab: Can We Delay the Onset of Type 1?

This content originally appeared on diaTribe. Republished with permission.

By Dr. Francine Kaufman

Provention Bio’s new injectable drug was recommended by an FDA committee for approval on May 25, 2021. Studies show that teplizumab can delay, for people at risk for type 1, the onset of the condition for two years or more. Learn what happened at the FDA meeting and what might come next.

Rarely does one get to witness the potential for an entirely new therapeutic or preventive drug to come onto the medical horizon. But that is the threshold we have now reached with the possible approval by the U.S. Federal Drug Administration (FDA) of the first drug that attempts to delay the onset of type 1 diabetes (T1D) ­– teplizumab.

Some questions remain to be seen: whether there is enough data, whether the data is sufficiently compelling, and whether the risk-to-benefit ratio is favorable enough for the FDA to grant teplizumab’s approval. To address these issues, the FDA convened an Endocrinologic and Metabolic Drugs Advisory Committee Meeting (EMDAC) on May 27, 2021.

What is teplizumab?

Teplizumab is a drug (specifically, an anti-CD3 monoclonal antibody) that binds to cells in the immune system called T-cells (these are the body’s infection-fighting cells). Scientists hope that teplizumab will change the types of T-cells in a person’s body so that autoimmunity – or the destruction of body tissue by ones’ own immune system – is decreased. This would reduce the self-destruction of the insulin-producing cells of the pancreas, called beta-cells, that leads to T1D.

What’s next for teplizumab?

The FDA now has to determine if teplizumab can truly help beta-cells survive. This will show the “effectiveness” or “efficacy” of the drug. The FDA also must determine if teplizumab is safe or if there are too many side effects (the “safety” of the drug).

How did we get here?

The drug manufacturer, Provention Bio Inc., applied to the FDA to approve teplizumab as a way to delay the onset of T1D in high-risk individuals. Even before Provention Bio’s TN-10 clinical trial, other studies were conducted with different forms of this type of medication.

Macrogenics (the previous company to own teplizumab) evaluated whether anti-CD3 could be used at the onset of T1D to preserve some of the insulin producing beta-cells.  If people with T1D could still make some insulin on their own, in addition to the insulin they needed to take, diabetes management could be made easier, with less hyperglycemia, lower A1C levels, and less risk of diabetes complications. However, the data did not show sufficiently strong outcomes needed for the FDA to approve anti-CD3 for new-onset diabetes. Perhaps it was simply too late in the diabetes process for this type of drug to make a significant difference. So, the focus turned to prevention – and to “delaying” the onset of T1D.

The TN-10 trial compared teplizumab to a placebo in 79 people with type 1 diabetes ranging in age from eight to 49 years. Teplizumab was given by intravenous infusion (IV) for 30 minutes over 14 days. To assure that study participants and research staff did not know who received teplizumab or the placebo, the placebo was given through IV in the same manner.

After two years, 43% of those given teplizumab developed clinical diabetes compared to 72%  of participants in the placebo group. The health outcomes between the treatment and placebo groups were significant and in favor of teplizumab, but the number of people in the trial was small (only 79 people). Similarly, TN-10 showed teplizumab to be safe, but it is hard to be sure about both the short- and long-term safety profile of the drug. Provention Bio is asking the FDA to allow teplizumab to be approved for children and adults who are at risk for (but do not yet have) diabetes. Because of this, understanding the safety and effectiveness of the drug is even more important.

What happened at the FDA meeting?

The EMDAC panel was asked to analyze the data and provide a recommendation on teplizumab, but the final decision, as always, rests with FDA. The panel was composed of 17 highly regarded endocrinologists, rheumatologists, cardiologists, biostatisticians and mathematical statisticians, a consumer representative, and a patient representative. All panelists agreed that teplizumab showed benefit to the participants of the TN-10 study by delaying the onset of diabetes by two years. However, there was concern about some of the data from prior studies on teplizumab which did not reveal a significant effect on glucose management in people with new-onset T1D.

The panel focused on safety concerns, mainly in terms of long-term risks. The data from TN-10 and the five previous studies in those with newly diagnosed T1D showed higher rates of diabetic ketoacidosis, viral infections, and cytokine release syndrome (which causes flu-like symptoms) in the anti-CD3 groups than in the placebo groups. There were also three total deaths in the treatment groups and none in the placebo groups. The EMDAC panel agreed that long-term surveillance studies would be required to better understand how safe this treatment is, if approved by the FDA.

Although five previous studies were submitted as evidence about teplizumab, the manufacturing and the rate at which the drug was cleared from the blood stream are not the same as what has been seen with the teplizumab under FDA review. This brings up the concern that the prior studies don’t truly support the present submission. The panel also agreed that if the FDA approved teplizumab, it could only be indicated for use in people with the same characteristics as those evaluated in TN-10 – people over the age of eight, with a family history of T1D, and similar glucose profiles. Since about 80% of people who develop T1D have no affected relative, the target population for the drug would be smaller than the actual number of people it could help, at least to begin with, though this could be reconsidered once more safety data is available.

Of the 17 EMDAC panel members, 10 voted in favor and seven voted against recommending FDA approval of teplizumab for the delay of T1D.

What does this mean in the diabetes community?

During the Open Public Hearing that helped influence the EMDAC’s decision, diabetes community leaders and organizations provided input, shared perspectives, and advocated for approval. The experts talked compellingly about the benefit of a two-year delay in diabetes diagnosis and what that would mean with regards to health. Several endocrinologists described the burdens of managing diabetes despite improvements in medications and technology, and the unmet needs that are still present in type 1 treatment regimens. The speakers included:

  • JDRF CEO Dr. Aaron Kowalski
  • Dr. Mark Atkinson (University of Florida)
  • Dr. Louis Phillipson (University of Chicago)
  • Dr. Jeff Hitchcock (Children with Diabetes)
  • Dr. Jeremy Pettus (University of California San Diego)
  • Dr. Jennifer Sherr (Yale University)
  • Dr. Korey Hood (Stanford University)
  • diaTribe founder Kelly Close (Close Concerns) and Jackie Tait (dQ&A), who detailed input from a survey by dQ&A on 1,078 people with T1D.

These speakers were supported by 187 letters posted to the FDA, as well as a dQ&A survey of 1,078 adults with T1D. Survey participants reflected upon how a two-year delay before needing to start insulin would have affected their life with diabetes.

Now it is in the hands of the FDA. If approved, teplizumab would become the first disease-modifying, preventive drug for T1D, and it could have the potential to change the paradigm of how the condition is managed. Stay tuned as we aim to keep you updated on this drug’s journey through the FDA approval process.

Source: diabetesdaily.com

Community Table: Nutrition, Health + Wellness in the Black Diabetes Community

This content originally appeared on Beyond Type 1. Republished with permission.

By JDRF-Beyond Type 1 Alliance

During our second Community Table discussion, Beyond Type 1 sat down with a group of experts and community members to discuss nutrition, health, and wellness in the Black diabetes community, and share helpful resources and perspectives. Watch the discussion in full!



Speakers included:

  • T’ara Smith, who served as the moderator for this event, was originally diagnosed with type 2 diabetes in 2017 but was re-diagnosed with LADA diabetes in 2019 and is Senior Manager of Beyond Type 2.
  • Keith Crear, who has lived with type 2 diabetes since 2017, is a sports photographer and multimedia specialist.
  • Alexis Newman, who has had type 1 diabetes for 37 years and is a registered dietician.
  • Dex Geralds, who has had type 2 diabetes since 2016 and works as a personal trainer and CrossFit coach.
  • Joy Ashby Cornthwaite, a dietitian and a certifies diabetes care and education specialist.

Partial transcript of conversation below, edited for content + clarity.

What does wellness mean or look like for you, and has your diagnosis changed the way that you live?

Dex: It’s balance. I know a lot of times when I’m with people who don’t typically know about diabetes, there’s a lot of things surrounding food that they believe I cannot have at all. Once you find the balance, you’re able to still eat some of the things that you’re eating before your diagnosis, maybe not at the same amount that you were eating before. Just finding balance with the way you eat, balance in the way you exercise. Whether it’s like myself who exercise maybe two hours a day or just going out for a 15-minute brisk walk. Just finding balance in what comes with that. The last thing in balance is just your mental health and making sure you check in with yourself and your feelings and your emotions and figure out what brings you happiness and joy.

Keith: It is a balance. It’s knowing what to eat, what not to eat, what to consume, when to consume it. Exercise is very, very important.

Alexis: For me, what it looks like is making sure that I am well, not only with how I’m eating, but also exercise. Making sure I’m honest and checking in with my friends and family, my support system, too. Also, the spiritual aspect of it. I’m a Christian, so making sure I’m connected in the sources that I feel encouraged in. When one of those are kind of out of whack, I don’t feel well.

Joy: I think of health on a continuum, and I encourage that in every day. In my family life, in my personal life, but also for those who I help to balance their journey with diabetes because everyone has their own journey and their whole complete individual. When someone comes to a session with me, I find out where on the continuum you are and what can we work on today? It has to come from you. It has to be what you want and not necessarily what I want. But I’m going to use my skills to get you to where you need to be.

What misconceptions have you encountered pertaining specifically to Black people with diabetes when it comes to fitness? What is some of the ways that you’ve helped your Black clients move past those stigmas and misconceptions?

Dex: The biggest thing is support in our community and in the world in general. If you’re overweight you get looked down upon and that can add to depression and lead to worse things. I know a big term going on right now is “unlearning” and taking everything, looking at it through a different scope, and then figuring out what brought you to this point and then what can I do to change this. It’s not just a one-stop shop kind of thing. It’s going to take a while for you to start to see changes for the most part. You have to create these better habits and getting through that way.

Learning to love yourself is vital, and when you learn to love yourself you want to do the things that’s right for you and you’re going to quiet that outside noise and put your blinders on and work for yourself and not think about the negative things that you might be getting from someone else. My family has a history of diabetes and obesity. My oldest sister, she’s lost 150 pounds now, but still just over 400 pounds. I remember things people would say to her growing up. Now that I support her and my family’s supporting her, she’s been on this incredible journey and losing weight because now she feels confident enough to do the things that once scared her or felt ashamed about. Being able to conversate and talk to my sister, or just clients in general, I’ve been able to learn what’s going on and been able to apply that to them in their journey in fitness.

Alexis: The kind of things that I’ve seen is that they believe that people think they’re lazy so they don’t want to push through that because they’re afraid of what people are going to think. I’ve also seen the fear of not knowing what to do overtake them in my discussions with my patients. Another idea is that they have this perceived idea of what exercise looks like, so really breaking it down into bite-sized pieces of like, “Look, as long as you’re like being consistent with the movement, whatever you choose to do, you can add on time, you can add on intensity. But, as long as you’re out there trying to move.” Those are the things that I have conversations with my patients about in terms of fitness.

Another thing too, is that an ideal weight of someone who’s African American may not be similar to another culture or race. We need to keep that in mind as we are discussing food, nutrition, health, and weight that the ideal weight that a doctor may have for you may not be appropriate. I think that needs to be said and also needs to be addressed when we’re talking to these patients about weight and health and glucose numbers and things like that.

Joy: When it comes to fitness, one of the great disservices, especially for the Black community, is to tell people that they need to lose weight if they’re moving. For many people who are living with diabetes, movement doesn’t always equate to weight loss. It also doesn’t always equate to better blood glucose values.

There’s a lot of things that go into thinking about exercise. When you’re telling someone that it’s going to make them better to exercise and they’re saying that their blood glucose is either crashing or going up way too high with exercise there’s a disconnect. You haven’t heard what they’re saying to you. You need to find out what people are experiencing in their exercise journey and then address those things and say, “Look, you may not lose weight, but let’s check your blood glucose before and after.” Celebrate the win over either the hyperglycemia that you have been feeling or the hypoglycemia that you were feeling if you didn’t pre-exercise meal or whatever reason. Celebrate the win that is more than weight.

How can we celebrate our culture or different types of Black cultures throughout the year, not just during Black History Month?

Joy: Support each other. I know for years I felt like I was the sole person saying, “Black people aren’t making ourselves sick with diabetes.” We need help. We need medications. We need diabetes health care and management. We need to know how to take care of ourselves and we don’t have to do that alone. Whatever we need to do we need to support each other and do it together and then we’re louder.

Dex: Our culture is so vast and rich, and it needs to be shared every day and whatever way possible. Whether it’s through social media or just through conversation or the way you dress, the way you wear your hair. It just needs to be expressed and be out there for people to see and enjoy and learn. I love my culture so much.

Alexis: Buy Black. There are so many amazing Black companies, I’ve been focusing on and amplifying those businesses on social media and telling my friends. I’m in a friend group and we’re talking about face care. I’m like, “All right, I’m using this” and they’re using this Black company. We’re just sharing information of these amazing Black companies that are out there. If we can’t do that, then no one else is going to. It’s really important to invest in Black businesses because they’re everywhere.

T’ara: Yes, please support Black businesses that you see, especially in the diabetes community. There’s so many Black people in the diabetes community who have businesses and organizations who could use amplifying on your social platforms. That social sharing could do a whole lot because you never know who can see their products, so please amplify it.

Keith: Constant delivery of content from Black creators. Constantly delivering things from Black history that could be done every day. The way you wear your hair, the clothes. Just something that constantly keeps it in the public eye so it’s not condensed down to just one month. There are a lot of people out there that are doing it and it’s always great to see that. If more of us continue to do that it’ll shift the narrative and it will shift the visual aspect of how we’re seen in society as a whole.

Source: diabetesdaily.com

How to Advocate for Yourself: Making Employer-Sponsored Health Plans Work for Your Diabetes Care

This content originally appeared on diaTribe. Republished with permission.

By Julia Kenney

The therapies, devices, and care that people with diabetes need can be expensive without adequate insurance coverage. For those with employer-sponsored health insurance, there are steps you can take to improve your insurance options and advocate for yourself.

Over 45 percent of Americans have diabetes or prediabetes and roughly half of US adults receive health insurance through their employer. Unfortunately, not all employer-sponsored health plans meet the needs of people with diabetes. According to a study of 65,000 people with type 1 diabetes on employer-sponsored health insurance, the average annual out-of-pocket cost of diabetes care was $2,500. Eight percent of study participants had annual costs well over $5,000. Since diabetes is most prevalent in low- and middle-income households, these costs, in addition to paying for premiums and non-diabetes healthcare, are unaffordable for many people.

If your health insurance does not cover a component of your diabetes healthcare, or if your diabetes care is covered but still unaffordable, you can work with your employer to get better coverage. Here is an overview of the different types of health insurance, who to go to for help, and how to advocate for better diabetes health coverage.

What are the different types of health insurance?

You will have expenses no matter what health insurance you have, but some plans can be more affordable for diabetes care. These are the expenses you will typically encounter with your health insurance plan:

  • Premium – Similar to paying rent, a premium is a fixed amount that you pay every month to keep your health insurance active. It’s common for employers to pay about half of your monthly premium, and sometimes more. In 2019, people with employer-sponsored insurance paid an annual average of $1,242 for health insurance premiums.
  • Deductible – The deductible is the amount you pay out-of-pocket before your insurance provider covers expenses. For example, if you have a $1,000 deductible, your insurance coverage will not kick in until you’ve paid $1,000 in healthcare expenses for that year.
  • Copays – Copays are a fixed amount that you pay for a health service or medication, and your insurance provider covers the rest of the cost. Copays are a helpful way to pay for diabetes care because they are fixed, predictable costs that people can plan for.
  • Coinsurance – Unlike fixed-price copays, coinsurance costs are a percentage of the total price of a health service or medication. These expenses are less predictable because medication prices can fluctuate.

There are three main types of health insurance – health maintenance organizations (HMO), preferred provider organizations (PPO), and high deductible health plans (HDHP). Here is an overview of the different types of health plans and what they might cost:

  • HMO – Health maintenance organizations have high premiums and low deductibles. An HMO plan covers healthcare within a network of hospitals and healthcare professionals. Your providers must be in-network in order to get your diabetes care covered. If your diabetes care professionals are in-network, this is often the most cost-effective healthcare option for people with diabetes.
  • PPO – Preferred provider organizations also have high premiums and low deductibles than HDHPs. PPOs are more flexible than HMOs because you are able to see providers out-of-network and you can see specialists without a referral. Because of this, PPOs typically have higher premiums and out-of-pocket costs than HMO plans.
  • HDHP – High deductible health plans typically have low monthly premiums and high deductibles. In 2020, the IRS defined a HDHP as any plan with a deductible of at least $1,400 for an individual and $2,800 for a family. If you have a high deductible health plan, you can open a health savings account where you set aside money to pay for medical expenses tax-free. These health plans are good for people who don’t anticipate needing regular healthcare; paying for diabetes care can be difficult with this type of plan because you will have high out-of-pocket costs upfront before you meet your deductible.
Advocacy

Image source: iStock Photo

Keeping these different types of health insurance and related expenses in mind, here are some things people with diabetes should think about when reviewing an employer-sponsored health plan:

  • What are my diabetes-related costs? Make a list of your diabetes healthcare costs including medications, devices, supplies, healthcare visits, and lab tests.
  • What are the health insurance costs? Look at the premium, deductible, and whatever cost sharing method (copay or coinsurance) is used for the health plan.
  • Are my medications and devices covered? Refer to your health plan’s Summary of Benefits and Coverage to see what is included in your insurance coverage. If a therapy or device is not covered, you may have to switch to one that is or submit a request to get it covered. Getting a new medication or device covered under your health plan can be a challenging and time-consuming process.
  • Is insulin covered pre-deductible? Some health plans cover insulin before you reach your deductible because it is considered preventive medicine. This can make insulin considerably more affordable, especially for people on high deductible health plans.
  • Are my healthcare professionals in-network? Accessing in-network healthcare is more affordable than out-of-network care. You should choose a health plan where your current providers are in-network or one that has good in-network options.
  • Can I access a flexible spending account (FSA) or health savings account (HSA) to save money? FSAs and HSAs are used to put aside money that is not taxed to help pay for medical expenses. HSAs are paired with high deductible health plans. FSAs can be used for any kind of health insurance and all FSA funds must be used in the same calendar year. Learn more about FSAs and HSAs here.

If I have a problem with my insurance, who do I go to for help?

Your employer’s human resources (HR) department should be able to address many of your insurance-related questions, since it likely helped select the health plan(s) available to you. Your HR department is your first resource for health insurance questions. If you need help selecting an insurance plan, want to see if your diabetes care is covered, need to file a claim, or are having trouble navigating your plan and understanding the costs, the HR department will support you.

For further questions, your HR department can refer you to a representative with the health insurance company or to a third-party administrator. A third-party administrator will help you understand your health plan, file health insurance claims, and navigate the appeals process if your insurance company denies coverage for a diabetes treatment. You can also apply for an exception to get treatments, medications, and devices covered if recommended by your doctor. A third-party administrator will guide you through these steps for getting important diabetes treatments covered.

Advocacy

Image source: iStock Photo

How can I make my employer-sponsored health coverage better for people with diabetes?

People with diabetes typically require expensive medications, devices, and regular visits with healthcare professionals to stay healthy. Robust employer-sponsored health insurance plans should make these expenses affordable and predictable. If you are trying to make permanent changes to your employer-sponsored health plan, your HR department can help you advocate for future health plans that better support diabetes needs. Employers have the power to make changes to their health coverage options every year. Here are some changes you can advocate for:

  • Add insulin and other diabetes care to the preventive medicine list.

In 2019, the IRS ruled that expenses for chronic disease management can be covered before you meet your deductible under a high deductible health plan. HMOs and PPOs also have preventive medicine lists. Diabetes care such as insulin, A1C testing, blood glucose meters, and eye screening – which are all considered preventive medicine – can be added to the preventive medicine list to reduce the copay or coinsurance costs for diabetes care. This saves employees money instead of paying full price before meeting their deductible.

  • Request to get a medication or device covered under your health plan.

If a device or medication you currently use (or want to try) is not covered under your health plan, you can ask for coverage in next year’s health plan. Diabetes devices, such as continuous glucose monitors (CGM) and insulin pumps, can help people with diabetes manage their glucose levels and increase their Time in Range, but are expensive without insurance coverage. Employers can typically negotiate to cover essential diabetes care, so request coverage for your medications and devices. Your diabetes treatment should be determined by your healthcare professional, not by what’s included in your health plan.

  • Share discounts and rebates with employees.

While list prices for diabetes medications may be high, your employer’s pharmacy benefit manager (PBM) can negotiate discounts and rebates on drug prices on behalf of the insurance plan and employer. The list price minus the negotiated discounts is called the net price. Sometimes PBMs and employers will keep the money saved; however, employers can pass discounts on to their employees to lower their out-of-pocket costs.

  • Use copayments for cost sharing instead of unpredictable coinsurance.

Coinsurance costs are unpredictable because they fluctuate as a drug’s net price changes. You can advocate for your employer to choose health plans that use copayments for healthcare cost sharing, instead of coinsurance.

More resources for accessing diabetes healthcare with your employer-sponsored health plan:

Feel free to share this article with your employer or your HR department. All people with diabetes deserve access to affordable, high-quality care. To learn more about health insurance and affording diabetes treatment, visit diaTribe.org/access.

Diabetes Series

Image source: iStock Photo

This article is part of a series on access that was made possible by support from Insulet. The diaTribe Foundation retains strict editorial independence for all content. 

Source: diabetesdaily.com

Breakthroughs in Pancreatic Cell Replacement: The ViaCyte Interview

I recently had the opportunity to sit down with Manasi Sinha Jaiman, M.D., M.P.H., Vice President of Clinical Development, and Mark Daniels, Senior Director of Clinical Development, of ViaCyte, “a regenerative medicine company focused on delivering novel stem cell-derived cell replacement therapies as a functional cure for all type 1 diabetes and a next-generation treatment for insulin-requiring type 2 diabetes.”

They have amazing things coming down the pike, so I was super excited to speak with them:

Tell our readers about ViaCyte. What’s the company’s mission and story?

Dr. Jaiman: ViaCyte is at the forefront of regenerative medicine approaches to develop a functional cure for type 1 diabetes (T1D). We have cell replacement therapies for pancreatic islet cells contained in a small retrievable pouch implanted under a patient’s skin.

The therapy is designed to enable insulin and glucagon (the counter-regulatory hormone that treats low blood glucose) production with the implanted cells to effectively control blood glucose levels, decrease the risk of hypoglycemia, and mitigate short-term and long-term diabetes-related complications for patients.

What is exciting is that ViaCyte is the first company to advance human stem cell-derived islet cell replacement therapy capable of producing insulin in the clinic, backed by two decades of research and expertise.

We are further augmenting our therapies by optimizing both the delivery device and the cells through collaborations with industry leaders, including W. L. Gore & Associates (the makers of GORE-TEX) and CRISPR Therapeutics.

Our mission is to develop cell replacement therapies offering long-term treatment to decrease the burden of the constant management needed with T1D.

What led you to your work at ViaCyte?

Dr. Jaiman: A significant focus of my medical career has been the integration of technology and medicine to advance treatments that can change the paradigm of diabetes disease management.

My experiences both in research and actively seeing patients with T1D have given me first-hand experience in seeing the daily work required to achieve any form of glycemic control as well as the burden patients and families face from complications from hypoglycemia or DKA.

I have also been able to see the importance of innovative approaches in addressing their needs. When I looked at the ViaCyte technology, I immediately saw great potential in their regenerative medicine approach to lessen the burden of disease and improve quality of life.

Mr. Daniels: Throughout my time in the industry I have been extremely fortunate to be able to work with innovative companies advancing potential, game-changing therapies for difficult-to-treat diseases. I was attracted to ViaCyte because the bar is set high; ViaCyte is looking to develop functional cures for diseases and is not just treating symptoms.

I also believe in the potential of cell replacement therapies and that these will be a meaningful new chapter in the advancement of medical treatments.

In ViaCyte, I have also found a team of extremely gifted and dedicated scientist-coworkers who are all generous with their knowledge and completely aligned in the mission to deliver a functional cure to the type 1 diabetes community.

It is exciting to be the first company to evaluate human stem cell-derived islet cell replacement therapy for its potential to functionally cure type 1 diabetes in the clinic.

Manasi Sinha Jaiman, M.D., M.P.H., Vice President of Clinical Development and Mark Daniels, Senior Director of Clinical Development

Manasi Sinha Jaiman, M.D., M.P.H., Vice President of Clinical Development and Mark Daniels, Senior Director of Clinical Development

What exciting new developments is ViaCyte currently working on?

Dr. Jaiman: Currently, ViaCyte has two clinical cell replacement therapy candidates. First, VC‑02 PEC-Direct is a treatment comprised of pancreatic islet cells in a pouch designed to allow blood vessels to enter the device and directly interact with the implanted cells to produce insulin and glucagon.

This treatment candidate is targeted for those with high-risk type 1 diabetes (hypoglycemia unawareness) able to tolerate immunosuppression.

In contrast, our groundbreaking VC‑01 PEC-Encap device is an advanced treatment comprised of pancreatic islet cells in a pouch that fully encapsulates the cells preventing immune cells from interacting with the implanted cells, which eliminates the requirement for immunosuppressants.

We are collaborating with W. L. Gore & Associates to optimize their innovative membranes which encapsulate the cells in our implanted devices. We expect to share clinical data in the second half of 2021.

Anything new in the pipeline that people with diabetes should be especially excited about?

Dr. Jaiman: One of the challenges with cell replacement therapies is to protect against adverse reactions and rejection of implants by the body’s immune system, which serves as a defense mechanism against foreign bodies.

In collaboration with CRISPR Therapeutics, we are employing gene-editing technology to engineer cells to avoid recognition by the immune system. Our partnership is focused on advancing gene-edited allogeneic stem cell-derived therapies from discovery through commercialization with the goal of developing a potential next-generation functional cure for all insulin-requiring type 1 and type 2 diabetes.

Mr. Daniels: With our preclinical candidate, VCTX210 PEC-QT, pancreatic islet cells would be in the same pouch as PEC-Direct, allowing the implanted cells to interact directly with blood vessels, an approach intended to enable robust and consistent engraftment.

Yet by designing the cells to be immune-evasive through CRISPR Therapeutics’ gene editing we would expect to eliminate the need for immunosuppressants as are required with PEC-Direct. We look forward to sharing more about this unique program in the future.

Photo credit: ViaCyte

Where do you envision ViaCyte and people’s lives affected by diabetes in five years? Ten years?

Mr. Daniels: This year marks the 100th anniversary of the development of therapeutic insulin to regulate blood glucose, yet dependency on tedious insulin injections are still a common course of treatment for many living with type 1 diabetes.

Within the next five years, we envision delivering significant progress in later clinical-stage studies with increased time in range, reduction in hypoglycemic events, and reduction in (or elimination of) the need for insulin injections in patients following our cell replacement treatments as we move toward making these therapies more widely available.

Dr. Jaiman: Within five years, we expect to be moving through the final phases of our regulatory process for our human stem cell-derived islet cell replacement therapy enabling availability more broadly for patients with type 1 diabetes.

It is our hope that within a decade, cell replacement therapy will offer longer-term treatment, easing the burden of constantly monitoring blood glucose. A functional cure will no longer be a dream, rather, a reality.

Is the ever-elusive cure on the horizon? A functional cure?

Dr. Jaiman: Yes, we believe a functional cure is on the horizon!

ViaCyte is focused on advancing cell replacement therapies toward a functional cure with a combination of implanted cells and device engineering.

This cell replacement therapy could represent insulin production protected from the immune system in a way that totally mitigates the underlying disease. Our technology is designed to safely implant the missing cells that make insulin and glucagon – that’s the breakthrough that gets us to the functional cure.

How can people with diabetes get involved or learn more?

Mr. Daniels: As ViaCyte is advancing novel treatments for type 1 diabetes, our team has been very fortunate to collaborate with multiple incredible research and advocacy organizations, including the Juvenile Diabetes Research Foundation (JDRF), Beyond Type 1, and California Institute for Regenerative Medicine (CIRM).

These organizations are focused on education and support for finding a cure for diabetes with resources for both patients and researchers on their websites. A great resource is the website www.clinicaltrials.gov – by typing “ViaCyte” into the search window, you can find more details regarding our ongoing clinical trials.

This includes details about the entry criteria to participate as well as the geographical locations of the sites (to find the one closest to you) and contact details necessary to reach out and connect with the study site team to learn more about what is involved in the study participation.

Photo credit: ViaCyte

Anything else you’d like to share?

Dr. Jaiman: Insulin treatment has largely transformed type 1 diabetes from a fatal illness to a chronic one, yet it is not a cure. At ViaCyte we recognize the long journey in the evolution of diabetes management, and we are keen to deliver a solution that offers real hope for a functional cure for type 1 diabetes.

Every single member of our team is passionate and dedicated to this endeavor. Managing diabetes can be difficult at any time, however, this past year has highlighted the need for accelerating therapeutic advancements to help reduce COVID-associated morbidity and mortality in the vulnerable population with diabetes.

With the pandemic still ever-present, we believe our mission of realizing a functional cure is even more critical for patients as they navigate living with a chronic disease during this very trying time. Our leadership team is wholly focused on improving patient care with an eye to the future.

Mr. Daniels: I am very appreciative of the Diabetes Daily team for providing this forum to connect to their community. The stories of family member’s and loved one’s experiences (including those of some of our own coworkers) with T1D resonate clearly within us and fuel our motivation behind the work we do.

It is only through the support of the T1D community and especially the valued study participants that we are able to advance this important research.

We are proud to be part of the biotech and biopharma community advancing some of the world’s most promising medical devices and therapeutic treatments. We look forward to sharing more details regarding the value of these treatments in the clinic.

Source: diabetesdaily.com

Screening for Type 1 Diabetes

This content originally appeared on Beyond Type 1. Republished with permission.

If you have a relative with type 1 diabetes, you may be at higher risk of developing the disease, making it imperative that you get screened. There are now simple blood tests that can determine your risk of developing type 1 diabetes before symptoms occur. Not only are there significant benefits to early detection, but as undiagnosed type 1 can be fatal, screening saves lives. Benefits to getting screened for type 1 diabetes (T1D) include:

  • Detecting T1D early reduces the likelihood of Diabetic Ketoacidosis and Hospitalization at diagnosis.
  • Opportunities to participate in clinical trials to prevent or delay the onset of T1D for years after screening
  • Knowing your results gives you peace of mind and time to prepare

For more information on the benefits of screening check out this interview with JDRF Director of Research Frank Martin and read 5 Reasons to Get Screened for Type 1 Diabetes.

Learn More About Screening Options

T1Detect From JDRF

Mom

Image source: Beyond Type 1

T1Detect, JDRF’s screening education and awareness program, will arm you with the information you need before and after getting screened for type 1 diabetes autoantibodies. Until now, T1D symptoms and a diagnosis often come out of the blue. Today, families can use testing to detect T1D early so they can plan and prepare. With one blood test, anyone at any age can find out—before symptoms even occur—if they are at risk for developing T1D. The test is easy, simple and can help save lives. Everyone, regardless of relation to someone with confirmed T1D, is eligible.

TrialNet

TrialNet

Image source: Beyond Type 1

If you have a relative with T1D, you’re in a unique position to help us learn more about the disease and how to prevent it. Offered through the TrialNet Pathway to Prevention Study, risk screening uses a simple blood test that can detect your risk of T1D years before symptoms appear. If you are in the early stages of T1D, you may be eligible for a prevention study. Take the first step and get screened.

Read the Latest Screening News

T1Detect is a new screening initiative designed to make early detection of type 1 diabetes easier and more accessible to a broad population. More

New Evidence Shows Vital Importance of Screening For T1D in Children

Kid

Image source: Beyond Type 1

By screening for type 1 diabetes in children, Bavaria was able to decrease rates of diabetic ketoacidosis at diagnosis. More

Firsthand Screening Accounts

Knowledge is Power: Screening for T1D

Family

Image source: Beyond Type 1

After their first daughter was diagnosed with T1D, the Brzozowski family decided to have their youngest child screened, and discovered that she would eventually develop Type 1 diabetes. More

My Decision to Get Screened for T1D

Family

Image source: Beyond Type 1

Jorge Aguilar’s mom has had T1D for his entire life; he explains his decision to be screened for T1D antibodies and offers advice to others who are considering being screened. More

Source: diabetesdaily.com

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