Anti-Cancer Drugs in Lab Mice Trials Prevent or Reverse Type 1

Researchers at the University of California, San Francisco, have found that two drugs used to treat cancer can prevent or cure type 1 diabetes in mice.

The drugs, sunitinib (marketed as Sutent) and imatinib (marketed as Gleevec), prevented the development of type 1 in mice specially bred to have a predisposition to acquiring the disease. 

Of the already diabetic mice that received imatinib, 80 percent went into remission. And 80 percent of those that went into remission experienced a total cure-a cure rate of 64 percent.

The findings suggest that the struggle to prevent and cure type 1 may have found two powerful weapons that will not require years of research or large outlays of money to produce. Even better, UCSF researchers say that they hope to begin using the drugs in human trials within a year.

The UCSF research team was looking into the cancer drugs because they inhibit some of the enzymes that are associated with cell growth and proliferation. Scientists think the enzymes, called tyrosine kinases, are involved in almost every aspect of the immune system, from benign signaling that summons T and B cells for defense to misfirings that create inflammation that often leads to tissue damage.

Because type 1 is an autoimmune disease, the team wondered if the subset of tyrosine kinases controlled by the cancer drugs might be the same enzymes that create pancreatic inflammation in people with diabetes. 

What they found surprised them. The drugs’ effectiveness seemed to come from their ability to block a tyrosine kinase that has no known connection to diabetes, an enzyme called “platelet-derived growth factor receptor” (PDGFR). 

In a statement UCSF released to the press, Arthur Weiss, MD, PhD, UCSF professor of rheumatology, said, “This study opens up a new area of research in the field of type 1 diabetes and, importantly, opens up exciting opportunities for developing new therapies to treat this disease and other autoimmune diseases.” 

In the same release, Jeffrey Bluestone, PhD, director of the Diabetes Center at UCSF and an expert in autoimmunity, said, “There are very few drugs to treat type 1 diabetes, especially after disease onset, so this benefit, with a drug already proven to be safe and effective in cancer patients, is very promising. 

“The fact that the treated mice maintained normal blood glucose levels for some time after the drug treatment was stopped suggests that imatinib and sunitinib may be ‘reprogramming’ their immune systems in a permanent way.

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