This headline turned heads across the diabetes landscape:
“CITY OF HOPE SETS NEW GOAL FOR TYPE 1 DIABETES CURE
More than $50 million in private funding aims to cure Type 1 diabetes in six years”
A recent grouping of donations totaling $50 million is outstanding, those in the trenches with diabetes said. But only six years to a cure? Really? You lost us there, some claimed.
Dr. Bart Roep, Ph.D., director of the program, now known as the Wanek Family Project at City of Hope, understands that view.
“Some people give up [on hope for a cure], and I respect that,” he said. He admits, too, that six years is a goal, not a promise. “I cannot be confident in the six years,” he said. “If I knew what was needed to do that, I’d do it on one year.” But, he feels the pathway City of Hope has laid out thanks to the donations leads the diabetes world on a road to better outcomes, and a place that, in six years, will be significantly better than where we are today.
What the program plans to do is change the entire way we view a “cure,” shifting from a one-size-fits-all method of research and goals to a system of precision medicine; a way to offer individualized and personalized therapies for people with diabetes much in the same way cancer treatment does today.
The program will draw heavily from a biorepository, something Dr. Roep says will save millions of dollars and many years in helping them embrace the concept of diabetes being unique in almost every individual. Armed with that knowledge they will dig back into human clinical studies that may not have succeeded on a mass scale and look to see if they can help patients on a smaller scale.
For instance, if a study failed for 70 percent of the participants, it may have held answers for other 30 percent.
The focus at City of Hope will be threefold: to stop the progression of the disease (something Dr. Roep calls the “low hanging fruit” of the effort), get people off injections for good (something he admits is “much more of a challenge but not impossible,”), and preventing, stopping, and reversing complications.
For stopping progression, Dr. Roep is excited about a vaccination he brought through Phase 1 studies while in the Netherlands. The vaccine takes cells of the patient and mixes them with Vitamin D3, and is believed to be effective in those with diabetes who have the genetic background which occurs in about 70 percent of the diabetes patient population. Contending it can fight the onset and possibly even reverse diabetes, the program plans to launch a Phase 2 study.
They also want to dig deeper into the concept of beta cell replacement, looking for even more advanced therapies than the encapsulation models in human trials now. First, Dr. Roep stated that they want to focus more on the newer knowledge that many people with Type 1 (even those who’ve lived with Type 1 for many years) still have beta cells. “They’re hiding; they’re stealth in the body,” he said. “What we want to see is if we can get them back into action. If we can stop the immune attack, can we do this?”
He said this would not suppress the immune system (“I love the immune system!”), but rather, would “negotiate with it (as in the case of allergies) in patients and with a velvet glove.”
As far as beta cell replacement, they will be pursuing a way to protect new beta cells introduced into the body with biological materials; almost a natural wrapper around the cells to allow them to get oxygen and produce and excrete insulin. Instead of encapsulating them, he said, they aim to coat them in a kind of biological immune protective coating.
One novel way to approach this, Dr. Roep said, is accepting that diabetes is not just an immune disease, but also a beta cell disease. “Just looking at the immune system is not enough,” he said. “We must look at beta cell health, too. The two are in dialogue.”
On the complications front, the goal will be to study individuals and pinpoint why some are predisposed to complications, then take action from there. “Once we know who will be most inclined to develop those complications, we can take action for them,” he said.
They also want to push the concept of caring for those beta cells hiding in the body of the person with diabetes. “It shows it pays off to control your blood sugar,” he said. “I know it must be frustrating, but I ask (patients) to please stay chin up and keep working. Preserve those beta cells.”
Dr. Roep admits there is much to be done, but is champing at the bit to do it. The donation allows researchers to take on more, and move ahead projects they’ve had going on for years. Pointing out that the City of Hope has a stellar record of treating cancer patients with immunology, he feels confident they can do the same for diabetes.
“I used to be a pessimist, but I am no longer,” he said, adding that he has known so many diabetes patients and cares deeply about finding better outcomes for them. “Six years, however ambitious it may be, we should be able to offer a cure for at least one patient.”
And even if they suffer some set backs, he said, there will be progress. “We will also learn from our failures. It’s tragic but true,” he said. “We will learn from those and have successes, too. It would be great if there were a magic bullet, but there isn’t. You know, we’ve come a long way in treatment, but 80 percent of people with diabetes cannot be treated to target. We’ve got to do better. We’ve got to find these cures. Because ‘cure’ in diabetes is a word we are still defining.”