Can a cancer targeted therapy be used to delay the onset of type 1 diabetes?

This study looked at the use of imatinib (Gleevac) in recently diagnosed type 1 diabetes (T1D). It found that imatinib improved pancreatic function and delayed the onset of T1D.

T1D is caused by autoimmune damage to the pancreas. This happens when the body's immune system mistakenly attacks its own cells. When the pancreas is damaged it stops producing enough insulin. C-peptide is produced by the pancreas at the same rate as insulin. C-peptide is often used in research as a proxy for insulin because it is easier to measure. Low C-peptide levels are a sign of damage to the pancreas.

In patients with recent onset of T2D, commonly not all the insulin-producing cells are damaged at once. New therapies are being investigated to delay the complete destruction of these cells and therefore the onset of fully-blown T1D.

Imatinib is a medication used to treat certain types of blood cancers such as chronic lymphocytic leukemia (CLL). It targets certain proteins in the body and interferes with their growth and destruction. From animal studies, it has been suggested that imatinib may help to halt autoimmune damage to the pancreas. However, it is not clear if this is the case in humans with recently diagnosed T1D.

67 patients with recently diagnosed T1D were involved in this study.  All had T1D symptoms within the previous 100 days, with blood tests that showed immune system problems. 45 patients received imatinib 400 mg per day for 26 weeks and 22 patients received a placebo. C-peptide levels were measured to test for T1D progression. Patients were followed up for 2 years.

After 12 months, the imatinib group had significantly higher C-peptide levels (by 0.095) compared to the placebo group. This is an indication of improved pancreas function. However, this effect was not seen after 2 years. 

6 patients (13%) in the imatinib group had to stop treatment due to problems such as skin rashes and liver function impairment.

This study showed that imatinib may help preserve pancreas function in newly diagnosed patients with T1D and delay the progression of the disease.  

This study had a small number of participants. Some problems with imatinib treatment were noted in the study. Further studies are needed to give more information on the safety of this medication in these patients.