Sometimes, all the latest headlines on steps towards curing diabetes can seem like a broken record -- just researchers crying wolf about how big and bad their science projects are.
Fellow type 1 Lisa Hepner in California gets that, and as someone living with diabetes for more than 20 years, she often agrees. But the progress in the past few years involving islet cell transplantation and encapsulation still gives her hopeful goose bumps, and that hopeful feeling is what's behind the feature documentary she's creating about the research race to find a cure, The Human Trial, which she's filming with her husband, Guy Mossman.
She knows these projects don't always produce the desired results, but hopes her film will make type 1 is better understood and recognized as a condition that needs more funding and public attention.
"As filmmakers, we realize we may also capture some disappointment and that the new protocols may not work as well as hoped," the Los Angeles film producer said.
"Yet regardless of outcome, our objective is to educate mainstream audiences about type 1 diabetes help put the autoimmune disease back on the fast track to funding and research. We firmly believe that type 1 deserves to be better understood and recognized as a condition that adversely affects millions of children, young adults, and adult survivors."
Lisa was diagnosed back in college and we introduced her story back in 2011, at a time when the documentary feature film was named Patient 13 and focused on one PWD scientist's personal research project to cure type 1 using a thin implantable sheet of islet cells. Fast forward almost two years later, and a lot has changed.
Not only has her film title changed, but its focus has broadened to three different research projects. The documentary has also gotten the artistic support of a world-renowned graphic artist who's also living with type 1, Shepard Fairey from South Carolina, most widely known for creating the Barack Obama "Hope" poster during the 2008 presidential election.
Against the backdrop of the expanded film project, there's the evolving story of encapsulation and stem cell research that's gotten a bolus of energy, attention and financing in recent years and is bringing a renewed hope in what may lie ahead in the ever-elusive search for a cure.
Or as Lisa and her film crew like to believe: it's no longer a "quest for a cure," but a race. And it's on, especially between the three research projects that the Human Trial will feature. Lisa says all three are working on different ways to encapsulate islet cells and the sources for those insulin-producing cells vary.
The Original Research
PWD biotech engineer Scott King in San Francisco is still one of the researchers Lisa is filming, and his project remains largely the same. He's creating what's called the Islet Sheet, an implantable encapsulation device (or sheet) about the size of a business card that's made of mesh and an absorbing material could hold thousands of islet cells.
King's company that was called Cerco Medical at the time has now been appropriately renamed Islet Sheet Medical, and is being and being funded by the type 1 cure research non-profit Hanuman Medical Foundation.
Originally, rodent studies done by Fall 2011 were leading into the expected canine trials in late 2011 and early 2012. But funding ran out for dog clinical trials and the pig studies didn't work, so King had to hit reset on his research. As Scott tells us, he thinks the FDA will require data from a large animal model like a monkey, dog or pig. He told us the JDRF doesn't support large animal model studies, he says, because the organization believes the rodent research is sufficient. For pig studies, they turned to Dr. Jon Lakey who co-pioneered the now famous Edmonton Protocol for islet cell transplantation.
But King says their bet on those pig studies didn't pay off because there are various problems with that model of research. King and Lakey are doing a rodent study at the University California Irvine in order to tweak the encapsulation device, while another research team member is heading to the Netherlands in September to work on the Islet Sheet with Dr. Paul DeVos at the University of Groningen.
King told us that the renewed focus is finding investors or fellow researchers who might be interested in partnering up to explore canine or other "innovative sources" for islet tissue. That could very well involve synthesizing beta cells from stem cells, which King says has reinvigorated the effort behind an old idea of encapsulation devices.
"It's a matter of the glamor," he said. "Stem cells are very exciting."
And that stem cell excitement has lured a handful of researchers, including another group that Lisa has broadened the film to include.
The film crew also plans to follow San-Diego-based company ViaCyte, which we last reported in 2012 is developing an encapsulation device of its own that weaves in fully-functioning beta cells from embryonic stem cells. The company's since trademarked its device called Encaptra and is planning for human clinical trials in 2014. Our intern, Amanda Cedrone, had a phone chat recently with ViaCyte CEO Paul Laikind about their recent developments.
She was told that Encaptra would be a flat device about the width of a credit card and would be implanted in a patient's skin (through a simple outpatient surgical procedure) to last at least a year, if not longer -- up to five years -- before it would need to be replaced. Still, the most novel thing they're developing is the company's VC-01 product, which combines that encapsulation device with PEC-01 cells — or pancreas endoderm cells, derived from embryonic stem cells. So far, Laikind says the animal studies have proven effective and they're working to get investor funding for the human clinical trials.
"The bottom line is we've pretty much done all the animal tests we can do that are going to give us new information and we have a pretty good handle on this product and how to use it to control diabetes. All our focus now is on moving into the clinic," Laikind said.
In February, the JDRF and the California Institute for Regenerative Medicine (CIRM) pledged to contribute $3 million each to advance the product, following up on the $10.1 million ViaCyte had received from the institute last fall. Those contributions were precursors to what materialized in April of this year — the Alpha Stem Cell Clinics Network that's made up of five clinics nationwide to help enroll patients, manage regulatory procedures and share clinical study data from these funded stem cell projects.
"These clinics have the potential to revolutionize how we deliver stem cell therapies to patients," the CIRM president, Dr. Alan Trounson, stated in a recent press release. "Stem cell therapies are a completely new way of treating diseases and disorders so we need a completely new way of delivering those in a safe and effective manner. These clinics will help us do just that and the clinical trials carried out in this network will fulfill the agency's promise of bringing new therapies to patients who need them."
Back to the BioHub
The third research project Lisa plans to feature in the film is the Diabetes Research Institute's work on what it calls the BioHub.You may remember our post from March that talked about this bionic mini organ that would be a venous sac or a porous sponge-like container about the size of a quarter that could be implanted and filled with regenerated islet cells. As Dr. Camillo Ricordi told us early in the year, it could be years before this develops if at all. But Lisa says Ricordi expects the first clinical trials will take place in 2014, focusing on the use of a "silicone scaffold" within the BioHub to house the islets.
While it's too early to say which project might get the farthest first, Lisa says she is hopeful that the research will pay off.
"Each of these approaches has potential advantages, but only animal and human trials will determine which is safe and most effective," she said.
Sure, there's other stem cell research happening as well. The JnJ/Janssen-owned group called BetaLogics has its own stem cell and encapsulation device in the works, and a group of Chinese researchers are also exploring that scientific area. But for The Human Trial film, Lisa opted to concentrate on the two above involving stem cells and the DRI, that's been making news this past year. The project's not about taking a comprehensive look at all cure research, but rather about educating the masses through the use of the film.
So Lisa says her goal is to follow the three research teams until one of them reaches the clinic. Researchers anticipate that will be in 2014, she says, and if that happens the film would be completed and ready for film festival roll-out by January 2015.
As mentioned, the film got a boost not long ago when world-famous artist Shepard Fairey started supporting the project. A type 1 himself, Shepard not only drew the Hope poster leading up to the 2008 presidential election but also created Time magazine Person of the Year covers. He agreed to create a movie poster that's now being sold to raise money for the film, Lisa said.
"He spent a lot of time and energy creating this poster. We couldn't be happier. He's had type 1 for much of his life, and obviously gets it," she said.
The limited edition print can be purchased as a tax-deductible donation to the film's co-producer that is a non-profit. See www.poster.thehumantrial.com.
Aside from Shepard's involvement, JDRF has come on board as an outreach partner, and is hoping to also team up with the Diabetes Hands Foundation and the International Diabetes Federation to help with further outreach.
"We believe that stories are the most powerful vehicle for social change," Lisa said. "Witness the impact the documentary, The Inconvenient Truth, had on climate change. Films expose, enlighten, and educate -- and have the potential to drive meaningful action."
Needless to say, we sure hope so.