Diabetes patients have been waiting decades for the promise of a cure for the disease

In the three decades since she was first diagnosed with type 1 diabetes, Lisa Hepner has clung to a vague promise she often heard from doctors convinced that medical science was about to make her body whole again. “Be strong”, they said. “The cure is only five years away.”

But the cure has not yet arrived, and Hepner, 51, a filmmaker in Los Angeles, remains hampered by her body’s inability to produce insulin, the sugar-regulating hormone produced by the pancreas. “I may look good to you,” she said, “but I feel terrible 70 percent of the time.”

Staying healthy can be exhausting for many of the 37 million Americans with some form of diabetes. There’s 24-hour monitoring of blood sugar levels, constant injections of life-sustaining insulin, and the potential threats from the diabolical complications of diabetes: heart disease, blindness, kidney damage, and the possibility of losing a gangrenous limb through amputation.

“‘The cure is five years away’ has become a joke in the diabetes community,” said Hepner. “If it’s so close, why does it take so long? Meanwhile, millions of us died.”

That sense of dampened hope led Hepner to spend nearly a decade following the evolution of ViaCyte, a small San Diego, Calif., biotech company working to create what would essentially be an artificial pancreas. If successful, its stem cell-derived therapy would eliminate the life-limiting stings and injections of insulin for 1.5 million Americans with Type 1 diabetes. Vertex Pharmaceuticals, a Boston biotechnology company developing a similar therapy , has already made significant progress.

Since its theatrical release in June, “The Human Trial,” a documentary she produced with her husband Guy Mossman, has electrified the diabetes community, especially those with type 1, a disease that initiates often confuse it with the more common type 2.

Unlike type 2, which tends to come on slowly in adulthood and can sometimes be reversed early with exercise and dietary changes, type 1 is an autoimmune disease that usually occurs without warning, in childhood or adolescence.

Type 1 is also much less prevalent, affecting about 10% of people with diabetes. A pancreas transplant can cure the disease, but donated organs are in short supply and the surgery carries substantial risks.

In most years, only 1,000 transplants are performed in the United States. To ensure that the body does not reject the implanted pancreas, recipients must take immunosuppressive drugs for life, making them more susceptible to infections.

Therapies developed from human embryonic stem cells, say many experts, offer the best hope for a lasting cure. “The Human Trial” offers a rare glimpse into the complexities and challenges of developing new therapies – both for patients who have volunteered in the extensive FDA-mandated clinical trials and for ViaCyte executives. , constantly struggling to raise the money needed to bring a new drug to market. Today, the average cost, including the many failed attempts along the way, is $1 billion.

At a time when the steep rise in the price of insulin and other life-sustaining drugs has tarnished the public perception of the pharmaceutical industry, the film is also notable for its admiring portrayal of a biotech company whose executives and employees seem genuinely committed to help humanity.

“The Human Trial,” which can also be viewed online, has become a rallying cry for Type 1 patients, many of whom believe only greater visibility can free up the research dollars needed to find a cure.

Those who saw the film were also strengthened to see their own struggles and dashed hopes reflected in the journeys of the two protagonists, Greg Romero and Maren Badger, who became one of the first patients to have the bags of experimental cells implanted under their skin.

Stigma often leads people with type 1 to hide the disease. In his quest to feel “normal” in college, said Todd Boudreaux, he avoided telling friends about the illness, a decision that could have dangerous ramifications in the event of a seizure caused by low blood sugar.

Hepner also spent much of her life playing down the illness, even with her husband, Mossman. She remembered her confusion early in their relationship, when he woke up to find her dazed and drenched in sweat, a result of hypoglycemia or low blood sugar. The more Mossman, a cinematographer, learned about the disease, the more he pushed her to make the film.

For years, Hepner stood her ground, fearing that she would draw unwanted attention to her health. “It’s a competitive world, and I just didn’t want people to think, ‘Oh, she’s not thinking straight because her blood sugar is high,'” she said.

But over time, the ubiquity of breast cancer awareness campaigns and highly publicized efforts to cure Alzheimer’s disease made Hepner realize that his cinematic skills could change the public’s perception of type 1, a nearly invisible disease, partly because many people who have it don’t look sick.

She hopes to change other misperceptions, including the notion that diabetes is a relatively inconsequential and “manageable” disease that was popularized by television commercials for wellness drugs from big labs, as they feature confident patients playing tennis, basketball and driving. hot air balloons.

In fact, the industry spends a fraction of its dollars on research to find a cure. The rest goes towards developing drugs and devices that make life easier with the disease, according to the Alliance to Cure Juvenile Diabetes.

The return on investment is undeniable. For those who can afford it, continuous glucose monitoring devices can obviate the need for the finger prick test, and the machines can be combined with cellphone-sized insulin pumps that take most of the guesswork out of dosing.

Hepner deeply appreciates the wonders of insulin: at one point in the film, she pays tribute to its inventor, Frederick Banting, during a visit to his home in Canada. But she notes that insulin-dependent diabetics don’t live on a picnic. Many people without insurance cannot afford the thousands of dollars the drug costs annually, forcing some to save and ration. And a miscalculated or untimely dose can cause convulsions, unconsciousness and even death.

Even with all the advances in care, only about 20% of adults with type 1 are able to maintain healthy blood sugar levels, according to a 2019 study. On one occasion, Hepner woke up in the ICU. ) after your insulin pump has failed.

“We need to stop trying to normalize this disease because, let’s face it, having diabetes is not normal,” she said. “It’s the other pandemic that killed 6.7 million people last year worldwide.”

Despite their frustrations, it would be inaccurate to describe Hepner and his film as pessimistic. Without wanting to reveal too much, “The Human Trial” ends on a hopeful note. And despite a series of near-bankruptcy issues, ViaCyte managed to secure the funding to keep the lab’s lights on.

Then there’s more recent news that didn’t make it into the movie. Last month, ViaCyte was acquired by Vertex, a competing biotechnology company that has been developing its own stem cell treatment. This treatment showed early success and the company announced that a retired postal worker who participated in clinical trials was cured of type 1 diabetes.

After nearly a lifetime of hearing that a cure was coming, Aaron Kowalski, chief executive of the Juvenile Diabetes Research Foundation, the world’s biggest funder of type 1 research, says he is optimistic. A dozen more drug companies are looking for a cure than a decade ago, he said, and the organization plans to spend $100 million on cure research this year.

“It’s not a question of whether it will happen, it’s a question of when,” said Kowalski, who is a scientist and has had the disease since childhood, like his younger brother. “Our job is to make that happen faster.”

Until that day, he added, people with diabetes, both type 1 and type 2, could enjoy a little empathy and understanding.

Translation by Luis Roberto M. Gonçalves