Gleevec, the daily pill that turned a killer type of leukemia into a manageable disease, may also help slow the worsening of diabetes, researchers reported Monday.
In a follow up to a 2008 study in which diabetic mice were cured by the drug, a team reports “modest” effects in adults with type-1 diabetes. This is the type of diabetes often called juvenile diabetes and it’s caused when the immune system mistakenly destroys insulin-producing cells called beta cells.
Tests done in 67 adults with type-1 diabetes showed the drug appeared to boost their body's own production of insulin, Dr. Stephen Gitelman of the University of California San Francisco School of Medicine told a meeting of the American Diabetes Association.
“On average the people that got the medicine used less insulin,” Gitelman told NBC News.
He stressed that it is a small trial meant to show the drug can safely do in people what it did in mice.
“We just wanted to get a sense if this showed some benefit in adults so we could get to the target population in kids,” Gitelman said.
“The conservative estimate is that beta cell function was maybe 19 percent better at one year. So it’s not a slam-dunk home run.”
The team will have to get Food and Drug Administration permission to test the drug in children.
About 5 percent of the 29 million Americans with diabetes have Type-1 diabetes.
It’s an autoimmune disease, caused when the body mistakenly destroys pancreatic cells that produce hormones like insulin and glucagon that control blood sugar. High glucose levels damage tiny blood vessels, which in turn can lead to blindness, heart disease, stroke and kidney failure. People can lose toes, feet or legs to amputation.
When levels fall too low, patients can pass out and sometimes die.
There's no cure and the only treatment is to keep blood sugar under tight control with diet and insulin.
Most people with type-1 diabetes must constantly check their blood sugar throughout the day, administering insulin according to what they are eating and how much they are exercising.
If those dying pancreatic cells could be saved, they might have to do this less often.
“That would be one potential pathway -- to use the drug to try to get in as early as possible when there are still as many beta cells remaining as possible and to slow down progression and potentially even keep people off insulin,” said Andy Rakeman, director of discovery research at JDRF, the diabetes research charity that funded the study.
“It’s estimated that people at the time they are diagnosed with type 1 diabetes that they have anywhere between 10 and 15 or maybe even 40 percent of their beta cells still remaining,” Rakeman added.
“Some people maintain beta cell function for years. We used to think all or nearly all of the beta cells are destroyed very rapidly.”
The organization is paying for research looking at several ways to preserve these cells. Gleevec would be a good candidate because it’s been around for nearly 20 years and while it causes side-effects such as a vomiting and rash, they are usually not severe in the diabetes patients.
“It’s taking an old drug and repurposing it for a new use,” Rakeman said.
Gleevec, known generically as imatinib, and Sutent, known generically as sunitinib, interfere with an enzyme called tyrosine kinase. In patients with cancers such as chronic myelogenous leukemia, cutting back on this enzyme stops the cancer.
Cancer patients who also had autoimmune diseases who took Gleevec and Sutent reported that the drugs also appeared to ease the symptoms of the other conditions. That’s when a team at UCSF started testing Gleevec in mice bred to develop diabetes.
Gitelman says his team believes Gleevec may be taking some of the pressure off the pancreatic beta cells.
He is a little worried his study may be misunderstood. The team just finished the research last week and they’ve rushed to put together a quick presentation to the Diabetes Association meeting. It will be weeks before they can analyze the data and put it into a form that can be reviewed by other experts in a medical journal.
“It’s early and the message could be misconstrued,” Gitelman said.
“This definitely does not show that Gleevec is curing type 1 diabetes,” Rakeman stressed.
Plus, Gleevec is expensive.
It costs more than $140,000 a year, according to Dr. Hagop Kantarjian of the University of Texas MD Anderson Cancer Center, one of the original Gleevec trial leaders. A generic version, however, costs $400 in India.