Type 1 diabetes is an autoimmune disease in which the body loses its
ability to produce insulin. During the development of the disease, the
body's own immune system attacks the insulin-producing beta cells in the
pancreas. This often gives rise to the presence of antibodies against
the body's own proteins in the beta cells. One of these proteins is
GAD65 (glutamic acid decarboxylase), and several clinical trials are
underway of a drug known as GAD-alum, based on GAD65.
A small pilot study in which researchers attempted to slow attacks
mounted by the immune system on insulin-producing cells in type 1
diabetes has given promising results. The study by researchers at
Linköping University in Sweden has been published in the scientific
journal New England Journal of Medicine
‘A small pilot study in which researchers attempted to slow attacks mounted by the immune system on insulin-producing cells in type 1 diabetes has given promising results.’
In the study reported here, DIAGNODE, researchers from Linköping
University have injected GAD-alum directly into lymph nodes in the
groin, rather than under the skin, in order to determine whether this
causes the immune response to become more tolerant towards the body's
own GAD protein. This method is similar to one known as "allergen
immunotherapy" used in certain treatments for allergy, where it induces
tolerance against an allergenic substance.
Six patients aged 20-22 years who had been diagnosed with type 1
diabetes up to six months previously were included in the study. They were
injected with a small dose of GAD-alum on three occasions, and took
vitamin D supplements during the period of the study. The latter can
reduce the inflammatory response of the immune system.
"The results for these six patients are very promising. Type 1
diabetes usually progresses gradually as the patient loses the ability
to produce insulin, but this has not happened in these patients. We must
follow them for a longer period and we must include more patients
before we can say anything about the effectiveness of the treatment, but
the results so far are extremely exciting," says Johnny Ludvigsson,
senior professor at Linköping University and principal investigator for
The pilot study does not contain a control group of patients who do
not receive the treatment being tested. The report instead compares the
results with those from other studies of untreated patients. The
long-term blood sugar level (HbA1c) and the need to inject extra insulin
both fell in the patients in the current study. Their natural
production of insulin remained at a stable level. The six patients were
followed for at least six months; four of them for more than 15 months.
The researchers are now planning to continue the study by increasing the
number of participants, and including younger patients.
"If these results are confirmed when we test more patients, it would
be an extremely important advance. The way in which type 1 diabetes
progresses differs between individuals for many reasons, and this means
that it is not necessary to find a treatment that has excellent effects
for everyone. Even if it helps only half of patients, this would be a
major step forward," says Johnny Ludvigsson.