Nearly 18 months into a stem cell trial to cure type 1 diabetes, there’s reason to be cautiously excited. ViaCyte, a leader in stem cell therapies for diabetes, is conducting the first human trial of its kind. Husband and wife filmmakers, Lisa Hepner and Guy Mossman, (featured this month in Diabetes Forecast) are making the first documentary of its kind. They’re covering the trial results as they happen in real time.
The film, The Human Trial, shows the formidable obstacles and grinding effort of cure research, the complexity of the disease and the science, what it takes to harvest cells, regulatory rules and the $5 billion plus it typically takes to bring a drug to market.
Almost everyone with type 1 diabetes heard when they were diagnosed, “There will be a cure in five to ten years.” For me that was forty-four years ago. Why do we still not have a cure...
● Nearly a century after the discovery of insulin?
● Given more than 1.25 million American children and adults have type 1 diabetes? With nearly 80,000 newly diagnosed young people every year?
● With type 1 and type 2 diabetes only rising? 30 million Americans and 415,000,000 people globally suffering with both types of diabetes
● Given diabetes will be a 561 billion dollar global health expenditure by 2030?
● When it’s the seventh leading cause of death?
Why does a cure take so long and should we still be hoping? Hepner and Mossman think we should. They also want us to understand cure research and why it’s such a laborious and intricate process.
The Human Trial is following one research team and a handful of trial patients as they move from the lab through the first two years of the Phase 1/2 clinical trial. For Hepner, who has had type 1 diabetes for twenty five years, making this film is personal.
“While I’m hoping this trial may be the breakthrough we need for a cure,” Hepner told me, ”I also want people with diabetes, particularly type 1 diabetes, not to give up. I’d like them to understand the Herculean efforts behind most cure research and to know that they have good reason to keep hoping.”
The Trial
At the center of The Human Trial is San-Diego based biotech company, ViaCyte Inc. In partnership with the University of California, San Diego and the University of Alberta, ViaCyte has received FDA approval to test only the fourth embryonic stem cell derived product in the world.
Part of the reason the directors are excited about this trial is ViaCyte is theoretically growing enough insulin-producing cells - from a single donated embryo leftover from in vitro fertilization - to treat every patient on the face of the earth with type 1 diabetes.
This could also benefit anyone who takes insulin, including those with type 2 diabetes.
The patients participating in the Phase 1/2 portion of the trial received a less than therapeutic dose of the stem-cell derived therapy. The cells were implanted in their body in two semi-permeable encapsulation pouches. The pouch is about half the size of a credit card as seen below.
The pouch allows the body’s nutrients to enter and feed the cells while it protects them from being destroyed by the patient’s immune system. Once matured, the cells are designed to secrete insulin and other hormones necessary to control blood glucose levels. Implantation of these cells in animals has already proved successful in controlling blood sugar. This is the first time it’s being tried in humans.
Once the product proves safe, researchers will seek recommendations to proceed to the therapeutic phase of the trail - again in people with type 1 diabetes.
Typically islet cell transplantation requires the recipient to take a lifetime of immune suppressant drugs. The goal of this trial is to eliminate the need for such drugs. Typically there are also not enough cells to harvest, and transplanted cells begin to die within a few years. ViaCyte’s trial, with just one stem cell line, has the capacity to yield an unending amount of insulin-producing beta cells.
The Film
The idea for the film came to the husband/wife team the morning Hepner woke from a dreadful hypoglycemic event during the night. “It was so clear to me. Unless you or a loved one has type 1 diabetes, you don’t know how tough and scary it is to live with it. We all look healthy on the outside.
“So Guy and I brainstormed on how best we could tell the story of diabetes and we landed on doing a film about the quest to cure it. We wanted to understand why there hasn’t been a cure.”
Hepner says, and I agree, funding for a cure for type 1 diabetes suffers from how hidden and misunderstood the condition is from and by the public. “People don’t understand the physical, emotional and financial costs of type 1 diabetes,” says Hepner. “Perhaps if we walked around with IV drips that give us insulin there’d be more concern about the disease. Instead, our essential diabetes devices are getting smaller and less visible.”
I too hope the film, like the trial, will help accelerate a cure. I also believe it will help alleviate what so many of us also bear. What I call “Hope-Fatigue.” The exhaustion from years and decades of wishing for, praying for, hoping for, and being promised, a cure.
Note: To donate to The Human Trial and keep the cameras rolling please visit the film’s web page. You can also purchase an OBEY® Awareness T-shirt featuring Shepard Fairey’s artwork. 100% of the profits support the making of the film. Photos courtesy of The Human Trial.