Nearly 18 months into a breakthrough stem cell trial for type 1 diabetes, significant positive results are emerging. ViaCyte, a leader in stem cell therapies for diabetes, is conducting this first human trial of its kind. And a husband/wife team is producing the first documentary of its kind. They are filming the trial results as they occur.
Almost everyone with type 1 diabetes heard upon their diagnosis, “There will be a cure in five to ten years.” For me that was forty-four years ago this month. Why do we still not have a cure for diabetes…
- Nearly a century after the discovery of insulin?
- Given approximately 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 diabetes cure take so long, and should we still be hoping?
Filmmakers Lisa Hepner and Guy Mossman think we should. They also want us to understand what’s involved in cure research and why it’s a laborious and intricate process.
Filming the Trial That May Lead to a Cure
Hepner and Mossman are the directors of the feature documentary, The Human Trial. Their crew has exclusive real-time access to a top lab conducting the first human cell encapsulation research trial of its kind.
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 who are waiting for a cure, 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.”
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 in patients.
Making this even more historic, ViaCyte recently acquired the assets of BetaLogics, a company that for more than a decade has also been working toward a stem cell-derived therapy for diabetes. This brings together the two leaders in cell replacement therapies for diabetes. This brain trust can only further strengthen ViaCyte’s cure-based research.
The Trial Science and Encapsulation Device
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.
It could also benefit anyone who takes insulin, including those with type 2 diabetes.
The first patient received the VC-01 cell implant on October 20, 2014. Thus far, in at least one trial participant, the implanted cells, as hoped for, appear to have developed into insulin-producing beta cells – after just 12 weeks.
Each of the patients participating in 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.
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.
(Typically islet cell transplantation requires the recipient to take a lifetime of immune suppressant drugs. One trial goal is to eliminate the need for such drugs.
Typically too, there are 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 primary aim of this first phase of the trial is to test the safety of the cells and their encapsulation device. If the product proves safe, researchers will then seek recommendations to proceed with the therapeutic phase of the trail – again in people with type 1 diabetes – to see whether the therapy really can control blood sugar levels.
Grounding Hope In Reality
This is the first clinical trial of its kind and The Human Trial is the first film to take us backstage into the world of cure research: the formidable obstacles and grinding effort, 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. Hepner doesn’t want people to stop hoping, but to see the reality.
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 yet.”
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 visible that give us insulin, instead of blood sugar meters and monitors that keep getting smaller and less visible, there’d be more concern about the disease.”
Among the film’s supporters is outreach partner Beyond Type 1. Currently the filmmakers are seeking donations to get The Human Trial completed. Once finished, it will be shown at film festivals, private screenings, broadcast and streamed, encouraging far more people to donate toward the cure. “If more people see what’s involved and how close we are, it will inspire more donations and we’ll get to the cure faster,” says Hepner. “The cure is in sight.”
Note: If you’d like to donate to The Human Trial please visit the film’s website. There you can also purchase an OBEY® Awareness T-shirt featuring Shepard Fairey’s artwork. 100% of the profits support the making of the film.