Every day, more than 40 children are diagnosed with Type 1 diabetes. The only approach to treat the disease has been the use of insulin, which was discovered almost 100 years ago. Finding an actual cure has been out of reach.
Eli Lewis, director of Ben-Gurion University’s Clinical Islet Laboratory, says this may soon change, and believes he and his fellow researchers have found a cure for Type 1 diabetes, which is usually diagnosed in children and young adults, and was previously known as juvenile diabetes.
Recent findings from two rounds of clinical trials showed that several patients injected with the anti-inflammatory molecule alpha 1 (antitrypsin) in the form of the FDA-approved drug AAT, were able to reduce their insulin or become insulin-free altogether.
“I’d call it the closest we have to a cure,” says Lewis, who’s been collaborating with researchers at the Barbara Davis Center for Childhood Diabetes at the University of Colorado for 10 years.
The first round of clinical trials took place more than two years ago at the University of Colorado, the Joslin Diabetes Center and at two children’s hospitals
“Together we’ve been developing this approach which is not focusing on the immune system,” says Lewis, “but focusing on actual cells that make insulin.”
Lewis was in the Washington-Baltimore area last week to talk about his findings with researchers and endocrinology departments at Children’s National Medical Center in the District and Johns Hopkins University in Baltimore. He also talked about his findings in the more intimate settings of people’s homes, with smaller groups of those familiar with Ben-Gurion University’s work and families affected
by Type 1.
“What I do now in these visits is present all the results that we have, and hopefully connect to help some of the few families that found this as an option to do outside trials at off-label use,” explains Lewis. “We’re meeting people that have been following my university and showing them all these advances so they can get a new, updated snapshot of what we’re doing.”
Lewis’ Clinical Islet Laboratory specializes in inflammation, diabetes, immunology and transplantation.
Knowing that inflammation plays a big role in the disease, he and other researchers focused on administering AAT once a week for eight weeks, after administering a transplant of healthy pancreatic islets — portions of tissues structurally different from surrounding tissues — to reduce inflammation. AAT has already been approved by the FDA for on-label use — that is, to use the drug for an approved indication, dosage or age group — to treat lung disease and reduce inflammation from smoking and respiratory infections.
In 2008, a Jewish boy from San Diego was the first patient to participate in Lewis’ clinical study. His diabetes is now under control and he doesn’t take insulin anymore, says Lewis.
Both the younger and older patients who participated in the first clinical trial were found to have stopped or reduced their insulin use with no negative side effects since they finished the trial over two years ago, according to Lewis. The study was published in The Journal of Clinical Endocrinology and Metabolism.
Lewis says those who participated in the trials were diagnosed with Type 1 less than a year before starting the treatment.
Lewis is hopeful that the FDA will approve AAT for on-label use as a Type 1 cure because of its proven safety and the positive reactions he’s been getting from fellow researchers.
“This molecule has no drastic side effects,” he says.
“It’s actually a feasible approach compared to [other treatments such as] chemotherapy that’s been tried on kids. For families this is a very good opportunity. Parents with kids with Type 1 see this as a rare opportunity to try and correct the disease.”
Lewis also says that one of his goals is to convince physicians that the molecule’s safety could allow them to administer it for off-label use in case a family
approaches them and it looks like a viable option.
At the moment, Lewis says his team at Ben-Gurion is trying to develop AAT — which is derived from a natural human blood protein — for mass production. The way it’s manufactured currently by big blood derivative companies will never be enough to treat the entire population of Type 1 patients, he says.
“[We’re trying to] generate what we already have, a prototype for this molecule, to be more effective and administered to a large scale of individuals,” he says.
According to the Ben-Gurion website, the next three clinical trials will focus on patients who are at-risk or recently diagnosed, and will try to find the favorable dosage amount necessary to achieve the highest insulin reduction.
If AAT is proven to be a legitimate cure for Type 1 diabetes, in that it truly reduces or eliminates the need for insulin treatment, Lewis says it could take one to two years before the FDA approves AAT for on-label use.
Lewis is not alone in his quest for a cure of Type 1 diabetes. Researchers at the Gladstone Institutes in California reported they reversed diabetes in mice through the transplant of stem cells.
And researchers at the University of Pittsburgh School of Medicine are ready to launch a trial in which the patient will be treated through modifying certain of his or her cells.