Celebrating the Best of 2021

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2021 with a laboratory flask

We have many reasons to celebrate in the type 1 diabetes (T1D) community. First and foremost, we celebrate YOU. Your support of our efforts is inseparable from the tremendous progress we’ve seen in accelerating cures, improving lives, and advocating for people with T1D and their loved ones.

We celebrate the impact and influence that you have made in research and advocacy to make a difference for members of the T1D community every single day. Here are some examples of how far we’ve come toward creating a world without T1D.

1. Accelerating Cures with Stem Cell-Based Therapies

Ninety-one percent less insulin, at half the target dose? Check.
Clinical trial for a gene-edited stem cell replacement therapy? Check.
First reported evidence of implanted stem cells secreting insulin in response to meal consumption? Check.

We’ve got a lot to be thankful for in stem cell therapies.

This year, The New York Times and Good Morning America highlighted the story of Brian Shelton, the first person to receive Vertex’s stem cell-derived therapy, VX-880, showing he needs 91 percent less insulin than before the transplantation. While these are early results from just one person, and this therapy is only being tested in people with severe hypoglycemia, breakthroughs like this bring us a step closer to finding cures for T1D.

Starting in 2000, Breakthrough T1D played a key role in the development of VX-880 by awarding Douglas Melton, Ph.D., a grant to make insulin-producing beta cells from stem cells, which his team did in 2014. Melton founded Semma Therapeutics in 2015, and the Breakthrough T1D T1D Fund made a catalytic investment in 2017. Vertex acquired Semma for almost $1 billion in 2019, and, in March 2021, the FDA fast-tracked its trial of VX-880—producing these exciting results just six months later.

Another big player in the stem cell-derived beta cell space is ViaCyte, who is a longstanding Breakthrough T1D partner and had two announcements. One is for VCTX210, which combines CRISPR Therapeutics’ gene editing technology with ViaCyte’s proprietary stem cell therapy, which will launch a clinical trial in Canada—the first for a gene-edited cell replacement therapy in T1D. The second is preliminary results from its second technology, VC-02™, which yielded the first reported evidence of implanted stem cells secreting insulin in response to meal consumption.

2. Coxsackie Vaccine Has Positive Results

Breakthrough T1D has been funding research into the role viruses play in the development of T1D since the late 1970s and, in the 1990s, we funded a postdoctoral fellowship for Heikki Hyöty, M.D., Ph.D., who demonstrated that enteroviruses—specifically coxsackie B—are the main culprit for T1D. Coxsackie B is a common pathogen where, in most circumstances, the infection is asymptomatic or results in mild symptoms. (Coxsackie B, however, in rare cases, may also lead to viral meningitis, heart or brain infection, or hand, foot, and mouth disease.)

Hyöty co-founded Vactech in 2001, who developed PRV-01, a vaccine targeting coxsackie B, and licensed it to Provention Bio*, which got an investment from the Breakthrough T1D T1D Fund for a first-in-human clinical trial. The results: Not only was it well tolerated, but it induced high concentrations of anti-coxsackie B antibodies.

This was only the interim results; the final results will be revealed this year. But, if they are the same as the interim results, continued development of the vaccine and its use to reduce the burden of T1D will be under way.

*Provention also has teplizumab, the first drug to delay the onset of T1D for nearly three years, under review by the FDA. Breakthrough T1D had a hand in the development of teplizumab from almost the beginning. We gave a Career Development Award to Kevan Herold, M.D., who had just started his faculty-level career at The University of Chicago, in 1988-1990. He showed, in an early animal study, that he could prevent autoimmune diabetes with an anti-CD3 antibody (which, later, became a humanized version, teplizumab). He has gone on to receive more than 15 grants from Breakthrough T1D, and was the lead on the clinical trial that demonstrated that it could delay the onset of T1D for nearly three years.

3. COVID-19 Update

Many of you joined our efforts to raise awareness of the impact that COVID-19 has on the T1D community. This work resulted in the U.S. Centers for Disease Control and Prevention (CDC) updating its guidelines to include T1D on the list of medical conditions associated with higher risk of becoming severely ill from COVID-19. It also helped us secure for members of the T1D community prioritized access to the COVID-19 vaccines and boosters when they first became available.

4. On Medicare? Accessing CGMs Just Got Easier

A regulation by the Centers for Medicare and Medicaid Services (CMS) puts in place expanded access to continuous glucose monitors (CGMs) for people on Medicare. The final rule provides coverage for all FDA authorized CGMs. Previously, CMS only covered therapeutic CGMs, which are those devices approved by the FDA to make insulin dosing decisions (e.g., without use of fingersticks in addition to CGM). This expansion means that people on Medicare with diabetes will now have access to a broader choice of CGMs, similar to what is available to those with commercial insurance.

The positive impact of CGMs on the health and well-being of those with T1D is clear and Breakthrough T1D has long advocated for broader coverage and choice of CGMs, resulting in private plan coverage starting over a decade ago, and Medicare coverage on a partial basis in 2017 and expansion last year. The CMS decision marks an important milestone for Medicare’s coverage of therapies that will improve the lives of those with T1D.

5. Regulatory Approval of Several T1D Therapies and Technologies

We fund research to facilitate the development of new therapies and technologies to make day-to-day life with T1D easier, safer, and healthier, until we can find cures or prevent this disease. In 2021, we got a lot to be thankful for.

Apps

  • The FreeStyle® Libre 2 iOS app received approval from the FDA for adults and children ages 4 and up with diabetes. This app is used with the Abbott FreeStyle® Libre 2 CGM system, which was approved on June 15, 2020. Prior to this approval, FreeStyle® Libre 2 users had to wave a reader over the glucose sensor. Now, they can use their iPhone instead.
  • The Bigfoot Unity™—a Bluetooth-enabled insulin pen cap—received FDA clearance for individuals 12 and older. For the first time, people who use multiple daily injections (MDI) to manage their diabetes will be able to integrate with a continuous glucose monitor and get personalized insulin dosing recommendations, lessening the burden of T1D.

Insulin

Infusion Set

Hypoglycemia

6. Artificial Pancreas Systems: Three at the FDA

With your support, we have ushered in therapy innovations that have made managing T1D easier and have led to improved health outcomes.

The most noticeable impact is in the influx of diabetes devices that have made T1D easier to control and have freed people from the constant worry about their disease. A significant milestone—or three to be exact—is that three artificial pancreas systems—Tidepool Loop, Omnipod 5, and Medtronic MiniMed™ 780G—have been submitted for FDA review.

The Tidepool Loop is an iPhone app that brings together an insulin pump, CGM, and automated insulin dosing algorithm that adjusts to the user’s basal rates as often as every five minutes and allows the wearer to bolus from the phone. The Omnipod® 5 System is the first and only tubeless artificial pancreas system, which pairs its disposable insulin pump (which you can change every 3 days) with a Dexcom G6 CGM. And the Medtronic MiniMed™ 780G is an update on the 670G, which was approved in 2016, and has a time-in-range of around 75 percent.

7. A Potential First-in-Class Adjunct Therapy

The FDA granted Breakthrough Therapy designation for vTv Therapeutics’ TTP399 as an adjunct therapy to insulin for T1D. Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious and life-threatening conditions.

In a JDRF-funded phase II clinical trial called Simplici-T1, TTP399 significantly improved HbA1c in people with T1D. Additionally, trial participants who received TTP399 showed a reduction in insulin dose, reduced hypoglycemia, and no increase in diabetic ketoacidosis (DKA).

The next step: A phase III clinical trial.

8. Leading Immune-Mediated Organizations Unite

Did you know that people with T1D are more likely to have a coexisting autoimmune disease? And someone with T1D is at a more than 3-fold increased risk of developing multiple sclerosis, as compared to someone who does not have type 1? And, if you have T1D and lupus, you are more likely to develop kidney complications, as opposed to people with just one disease.

That’s why Breakthrough T1D partnered with the Lupus Research Alliance and the National Multiple Sclerosis Society to advance the understanding of autoimmunity and to obtain more specific insights into commonalities and differences of immune pathways that govern these disease processes. The researchers will get a maximum of $450,000 for up to 2 years to generate the first tranche of results from their projects.

9. Eli Lilly Acquires Protomer Technologies

The Breakthrough T1D T1D Fund portfolio company Protomer Technologies has been acquired by Eli Lilly and Company. Protomer is developing glucose-sensing, or “smart,” insulin, which is designed to sense sugar levels in the blood and automatically activate as needed throughout the day. Smart insulin has the potential to revolutionize the treatment and quality of life of people with diabetes by improving both the effectiveness and safety of insulin therapy and significantly reducing the risks of both dangerous high and low blood-glucose episodes and life-threatening complications, leading to healthier, longer lives.

Breakthrough T1D has been an early supporter of smart insulin (also called glucose-responsive insulin, or GRI), and Breakthrough T1D and the T1D Fund have supported Protomer from before the company’s inception through its sale to Lilly. In 2013, Alborz Mahdavi, Ph.D., was a winner of the Breakthrough T1D Agnes Varis GRI Grand Challenge Prize. Subsequently, he founded Protomer, which was initially funded by Breakthrough T1D’s Industry Discovery and Development Program.

Following these successful grants from Breakthrough T1D, the T1D Fund made a minority equity investment in Protomer’s Seed round, alongside Eli Lilly, to advance the company’s highly promising glucose-sensing insulin program.

The successful sale of Protomer to Eli Lilly is a great example of how Breakthrough T1D research, the T1D Fund, and key diabetes industry players collaborate as a part of a larger ecosystem to help advance meaningful therapies for the T1D community.

10. Breakthrough T1D Centers of Excellence

A notable addition in our progress toward improving lives and curing T1D is the establishment of the Breakthrough T1D Centers of Excellence. There are now a total of five Breakthrough T1D Centers of Excellence: New England, Northern California, University of Michigan, Australia, and the University of British Columbia. These Centers of Excellence represent concentrated investments toward our goals by tackling a myriad of scientific questions that a single lab cannot undertake. By bringing together world-class, interdisciplinary research talents and resources at and across partner institutions, we are changing how we approach T1D research.

Movers, Shakers, T1D Changemakers

Your partnership is inseparable from these advances and many more. On behalf of our community, thank you for shaking things up—for more progress, more advancements, and more access—for everyone impacted by T1D.

We are excited for the progress that awaits us in 2022!