Highlights from the Annual JDRF Mission Summit
By Varun Iyengar
Our team was very fortunate to attend JDRF's recent Mission Summit, an annual gathering where the organization shares its biggest updates from the past year. The meeting provided the opportunity to hear from leading JDRF physicians and researchers about progress on everything from the artificial pancreas to islet cell replacement therapy to efforts in prevention. All in all, the two-day event was a remarkable opportunity to see firsthand how JDRF is driving progress in all areas of type 1 diabetes and to reflect on the impact the organization is making and will make in the future. Below, we discuss our top three highlights from the Summit:
JDRF CEO Mr. Derek Rapp delivered a compelling presentation on the organization’s progress over the past several years in moving the field forward on the artificial pancreas, glucose-responsive insulin, and adjunctive therapies.
ViaCyte President and CEO Dr. Paul Laikind presented encouraging results from the company’s phase 1/2 trial of the VC-01 islet cell replacement therapy. He stressed that the team is very much encouraged but that: “We STILL have work to do.”
Dr. Bruce Buckingham’s broad overview of artificial pancreas technology included an update on the Bionic Pancreas “insulin-only” system – which may precede the dual-hormonal version! – and new commentary on the Medtronic 670G.
Read our full coverage of these highlights below!
Top Three Highlights
STATE OF THE FOUNDATION
Derek Rapp (CEO, JDRF)
Mr. Derek Rapp delivered an optimistic talk on the past, present, and future of JDRF’s advocacy efforts. This was among the most passionate and heartfelt presentations we heard at the Mission Summit, and it was fantastic to hear his honesty and perspective as someone who lives so close to type 1 (his son was diagnosed in 2004). We thought he aptly summed up the state of the field as “progressing steadily toward making living with this disease less frightening” though was also quick to acknowledge there are plenty of opportunities to have an even larger impact moving forward.
Mr. Rapp’s also addressed the sizeable funding gap that existed when he came into his position as CEO in July 2014. While this gap has been partially addressed, Mr. Rapp shared that there remains a $250 million chasm between what JDRF would like to fund and what the organization has the means to fund. He stressed that while JDRF cannot fund all of the promising research out there, the JDRF leadership has “worked hard to do the right things for JDRF over both the short and long haul.” We would agree wholeheartedly on both fronts, especially given the tremendous progress JDRF has made in areas such as the artificial pancreas and glucose-responsive insulin. It is clear that Mr. Rapp and the JDRF team still have much bigger aspirations for the organization, and we found it inspirational to hear his vision to do “even more good for the greatest number of people.”
Mr. Rapp also spoke to leadership changes at JDRF, including recent news that Chief Scientific Officer, Dr. Richard Insel, will be leaving the organization this June – to say we have been impressed throughout Dr. Insel’s tenure at JDRF would be an understatement. He will surely be missed! We salute him for his significant contributions to the field of type 1 and to JDRF.
Below are some of our favorite quotes from Mr. Rapp’s fantastic presentation:
“I can see now, five years down the line, a world in which there will be multiple hybrid closed loop systems available for people, a world where there will be type 2 drugs repurposed for type 1. We can have several more cards turned over like encapsulation and glucose-responsive insulins. We are on path to make living with this disease less frightening.”
“For every good idea that we fund, there’s another good idea that we don’t fund. We have to make tough choices. It’s about doing as much as we can and being fiscally prudent in how we’re doing things. When I sit down with someone who has a great idea, it usually is a great idea … but I have to think about how it fits into the overall context. But this is a good thing because when we have good ideas we’re turning down, it means that there are opportunities for us to have an even greater impact.”
“We have worked to ensure that we are doing the right things for this organization over the long haul. I’d like to think that we’re near the finish line. We are fully committed to seeing it through. We want a world without type 1 diabetes.”
Paul Laikind, PhD (CEO, ViaCyte)
ViaCyte President and CEO Dr. Paul Laikind presented encouraging results from the team’s phase 1/2 trial of its VC-01 islet cell replacement therapy. The approach takes pancreatic progenitor (parent) cells and encases them in ViaCyte’s “Encaptra” device (to protect them from immune attack – see our “stem cell FAQ” here!), which is roughly 1 x 3 inches in size. Encaptra is then implanted under the skin, allowing the cells to mature into insulin-producing beta cells. In theory, they will mature to regulate blood glucose in a similar (if not identical) manner to natural pancreatic cells.
Twelve patients have now received the therapy, and excitingly, there have been no severe episodes of hypoglycemia or serious adverse events related to the device. Most of the adverse events have been related to the surgical procedure itself (e.g., pain and swelling at the implantation site). Dr. Laikind also shared that there hasn’t been evidence that the participants’ immune systems are harming the cells, noting optimistically that “… if [the cells] were going to be killed off, this would have likely happened much earlier.”
At the same time, it’s clear the ViaCyte team knows that it’s a long way to the finish line: “We STILL have work to do.” Indeed, ViaCyte’s goal with this first group of study participants is only to demonstrate that the therapy is safe and to develop their procedure for implanting the cells in humans. Next, the company plans to use these initial results to optimize the therapy before beginning testing the therapy in more people; this second stage will evaluate efficacy in 36 patients. While there’s still a ways to go, this early data provides important proof of concept and sets the team up for exciting progress moving forward.
PROGRESS IN CLOSING THE LOOP
Bruce Buckingham, MD (Stanford University)
Dr. Bruce Buckingham gave a broad overview of artificial pancreas technology, covering his experience testing a slew of closed-loop systems in development (Bionic Pancreas, Cambridge, DiAs, MiniMed 670G). Most notably, he shared new news that the Bionic Pancreas group’s “insulin-only study” is now complete – for context, the Bionic Pancreas team is known for its dual-hormone system (insulin + glucagon) though has recently been testing an insulin-only version of the prototype.
Indeed, we learned at the ATTD Conference on Saturday that the Bionic Pancreas may actually be approved and launched first as an insulin-only system – with glucagon only to be added later. It sounds like this approach would allow the Bionic Pancreas team to commercialize their device more quickly, launching the “iLet dual chamber integrated pump” with the glucagon chamber closed off. Then, when a stable glucagon is eventually approved, people could choose to add glucagon if they wanted.
At ATTD, Dr. Buckingham also shared the first-ever insulin-only data on the Bionic Pancreas. The headline? The insulin-only system showed roughly similar efficacy in pilot studies to other published systems: an average glucose of ~154-161 mg/dl (depending on the target glucose), with just ~1-3% of the time spent <70 mg/dl.
Additionally, Dr. Buckingham and colleagues have been testing Medtronic’s MiniMed 670G system (24/7 hybrid closed-loop) that is in pivotal studies this year. At the JDRF Summit, he could not present data, but did share valuable commentary on his experience with the new Enlite 3 sensor thus far. “I have not had ANY complaints about the sensor.” Medtronic’s newest sensor has been getting strong reviews from many in the field, and Dr. Buckingham’s enthusiasm is certainly telling. The MiniMed 670G pivotal study should be completed in May, and a US launch is expected by the first half of 2017 (pending FDA approval).