Encapsulation JDRF is turning Type One into Type None Type 1 diabetes (T1D) is an and advocacy, we’re driving the significant progress on each. This extraordinarily complex disease. discovery, development, and paper focuses on the progress we Finding the cure—a short-term delivery of therapies and are making on replacement of clinical intervention, with minimal technologies that will progressively beta cells through encapsulation side effects, after which all reduce the day-to-day challenges therapy. Beta cell encapsulation aspects of T1D are gone forever— of managing T1D, improve involves putting insulin producing requires restoring beta cell glucose control, and ensure that beta cells in a protective barrier function to provide insulin, patients are as healthy as possible and implanting them beneath and blocking or reversing the until we reach our ultimate goals the skin. These encapsulated beta autoimmune attack so the newly of a cure and universal prevention cells will sense a person’s glucose restored beta cell function is of T1D. levels and produce insulin as protected. This will take time. As the world’s leading private needed, while the barrier We know there will not likely be funder of research to cure T1D, shields them from the body’s a single “eureka” moment that JDRF is pursuing multiple autoimmune attack that initially magically eradicates T1D. So strategies to restore beta cell triggered the onset of T1D. meanwhile, through research function, and we are making 800-533-CURE I [email protected] I jdrf.org I 2 WHAT IT IS foundation funded in part by JDRF the acknowledged leader in the is moving ahead with yet a third space. We have helped bring one Beta cell encapsulation is a non- encapsulation design and it too product to early human testing invasive procedure which involves expects to launch human trials with several others heading to putting insulin producing beta cells within 12-24 months. human trials in the next year. in a protective barrier and We have invested in multiple implanting them beneath the skin. WHY IT MATTERS approaches to encapsulation These encapsulated beta cells will to avoid going all-in with one sense a person’s glucose levels and Beta cell encapsulation therapy technology, a multiple shots-on- produce insulin as needed, while has the potential to virtually goal approach we believe increases the barrier shields them from the eliminate the relentless daily the likelihood of success, and has body’s autoimmune attack that management burden for those the opportunity to impact second initially triggered the onset of T1D. living with T1D. No need for and third generation approaches. Encapsulation keeps the newly multiple daily insulin injections or Finally, we have brought the implanted cells alive by hiding them pump therapy, no more constant world’s leading scientists from from the immune system, providing blood testing or wearing multiple disciplines into a a safe environment where they can continuous glucose monitors, and cooperative and focused consortium function normally. The cells can no more carb counting. In short, structured to resolve the critical constantly assess the amount of people with T1D would be liberated remaining challenges we face, glucose in the blood and release to go about their daily lives for an initiative we know will accelerate exactly the correct amount of extended periods of time as if the push to bring an encapsulation insulin. What’s especially exciting they didn’t even have the disease product to those with T1D. about encapsulation therapy at all. Technically, it isn’t a cure; is that it overcomes one of BACKGROUND the major obstacles that has slowed progress in the islet People with T1D would be Beta cell encapsulation transplant field—the need liberated to go about their daily attracted considerable for lifetime administration activity in the 1980s and of powerful and toxic lives for extended periods of time, 1990s. But faced with immunosuppressive drugs as if they didn’t even have significant scientific obstacles, to prevent the immune researchers gradually system from destroying the the disease at all. drifted away from the field. newly introduced islets. And so encapsulation research remained under- This therapy is not an the immune system still wants to funded for the next 20 years. abstract idea being nurtured in destroy beta cells—it just can’t a lab. Indeed, in just a few short find the ones inside the device. But things began to change years, there have been a series For people with T1D who are on in 1999 when JDRF-funded of exciting advances in the duty all day every day just to stay researchers in Canada harvested encapsulation field, almost of all alive, it will sure feel like a cure. islet cells from the pancreas of of them stemming directly from organ donors, transplanted them JDRF funding. Today, three JDRF’S IMPACT into T1D patients, and achieved companies, including two funded insulin independence in many JDRF has built the most by JDRF, are conducting, or are patients. However, islet transplants advanced encapsulation research moving towards human clinical had two drawbacks: first, patients and development program in the trials of encapsulation therapies. required massive amounts of toxic world. We catalyzed a sleepy field And another non-profit research drugs to prevent the immune just a few years ago and remain system from attacking the new 800-533-CURE I [email protected] I jdrf.org I 3 insulin cells (so-called immuno- “cage” is porous enough to allow T1D therapies require cooperation suppressive drugs); and second, the cells to survive, detect glucose and collaboration amongst the available supply of islet cells levels, and release insulin. researchers around the world. for transplant procedures was This is especially true in the case sharply constrained making it Since launchingEncapsulation our program, of encapsulation – 1:36 because it impossible to perform the JDRF has prioritized several areas crosses so many different procedure on large numbers of research required to accelerate disciplines. So, early in 2013, JDRF of T1D patients. The Edmonton Protocol, as it became known, while not without issues, proved that cell replacement therapies could work. Meanwhile, significant advances in the biomaterials engineering and cell supply fields,1 prompted JDRF to take another look at the feasibility of beta cell encapsulation, and in 2007 JDRF launched its Beta Cell Encapsulation Program. THE JDRF BETA CELL formed the JDRF Encapsulation ENCAPSULATION delivery of encapsulation therapy Consortium. The consortium, to patients. These include similar to what JDRF organized to PROGRAM continuing research into ways move artificial pancreas systems The goal of the JDRF Beta Cell to create a replenishable, scalable to market, is already fostering Encapsulation Program is to supply of beta cells (so that collaborations among bioengineers, develop encapsulated beta cell millions of T1D patients could chemists, immunologists, transplant products to safely provide normal have the therapy once it is proven researchers, and pancreatic beta glucose control to individuals safe and effective), identifying the cell biologists to incorporate living with T1D. optimal material to use for the engineering concepts and designs permeable barrier that will protect into current efforts toward To understand encapsulation the beta cells (so the autoimmune improving islet cell encapsulation, therapy it’s useful to imagine a attack is rebuffed), and testing and it will play a central role in deep-sea diver in a shark cage. different device designs (so we bringing this therapy to patients The diver can conduct experiments, can identify which one works as soon as possible. take photos, and carry out other best from a performance and tasks from inside the cage while safety standpoint). JDRF has WHERE WE being safely protected from also begun to engage the U.S. sharks. The encapsulation therapy Food and Drug Administration on ARE TODAY would work much the same way. this cutting edge field to lay the Over the last 5 years alone, However, in this case the “cage” groundwork for regulatory review JDRF has provided substantial would surround the beta cells so of these novel products. funding for scientific research in they are protected from the encapsulation and xenotrans- JDRF understands that successful immune system attack, yet the plantation (transplanting insulin- 1JDRF was a leader in the effort to advance the stem cell research field. We funded early science in the field and led advocacy efforts to eliminate restrictions on stem cell research. The ongoing research in beta cell encapsulation is one of the very first real-world applications of this science to humans. 800-533-CURE I [email protected] I jdrf.org I 4 producing islet cells from one producing various hormones like a seaweed that is known to be species, e.g., pigs, to another, e.g., normal pancreas would, including compatible for use in humans. humans). Today, JDRF is working insulin and glucagon. Testing in LCT has already conducted small with two biotechnology firms, diabetic mice has confirmed that human trials with this product as well as another leading T1D the ViaCyte device maintained in several countries around the research foundation, to move blood sugars at normal levels. world. The results of the studies ViaCyte has are still being compiled but announced it preliminary results show the JDRF has made exciting progress intends to test product has had a positive effect the device in on reducing dangerous low blood in the encapsulation field. human clinical sugar (hypoglycemia) events. trials in 2014. More results are expected soon. encapsulation to the human • JDRF has also supported a • JDRF is co-funding another clinical trial phase. Specifically: project conducted by Living promising encapsulation Cell Technologies, a New strategy being carried out by a • JDRF is partnering with ViaCyte, Zealand company, involving the team of investigators at the a California biotechnology encapsulation of pig-derived Diabetes Research Institute at company, to support the islets for implantation into the University of Miami.