LA Times Staff Writer
July 14, 2003
If Daniel Kaufman hadn't had car trouble, he might
have missed the biggest discovery of his career.
As a young neuroscience researcher at UC San Diego's
Salk Institute in the early '90s, Kaufman left for home
one day only to find that his car wouldn't start.
He tried to return to his lab while awaiting a ride
but found the floor was being waxed. He then wandered
into the institute's library and picked up a medical
journal. It fell open to an article on Type 1 diabetes.
Kaufman knew that a particular brain protein he had
been studying also was released in the
pancreas - in the cells that secrete insulin. He suddenly
realized that the protein might playa
role in the development of Type 1 diabetes. “The
light bulb went off, " he says.
In Type I diabetes, which typically starts in childhood,
the immune system attacks the pancreas' insulin-producing
cells. Over time, this reduces insulin, the hormone
that controls blood-sugar levels. Type I diabetics must
have daily insulin injections so glucose can be transported
to cells and produce energy.
Kaufman soon joined researchers Allan Tobin and Jide
Tian at UCLA to further study the protein, called GAD.
The team believed that the development of diabetes could
be caused by the immune system attacking the GAD protein
in insulin-producing cells. In a study published in
Nature in 1993, they showed that giving young diabetes-prone
mice small amounts of GAD taught the mice's immune systems
to recognize GAD and not attack the protein as a foreign
substance. The mice did not develop the autoimmune response
that leads to diabetes.
In 1996, the team showed that a vaccine could inhibit
the autoimmune response after insulin- producing cells
already were under attack.
"This vaccine is based on injecting the very protein
that is being attacked,” Kaufman says. But instead
of boosting the attack, it is meant to activate cells
that release calming substances.”
UCLA eventually licensed the technology to a Swedish
company, Diamyd Medical, to develop and test the vaccine
in humans. In a study presented last month at the American
Diabetes Assn. convention, researchers showed that the
vaccine was safe and prolonged the ability of the pancreas
to make insulin in adults recently diagnosed with late-onset
Type 1 diabetes.
Type 1 diabetes takes years to develop and symptoms
often emerge only after insulin-producing cells are
damaged. Researchers hope, however, that giving the
vaccine to children as early as possible in the onset
of the disease will help prolong their ability to make
insulin. Future studies will address that question.
Kaufman's research also has produced a diagnostic test
that indicates who is at risk for developing diabetes.
With early diagnosis and the vaccine, the ultimate goal
is to prevent the disorder from developing.
"Since it worked on people with very late stages
of the disease process, we're optimistic that even better
efficacy will be found in young people who don't have
the full onset of the disease," Kaufman says. And
it proves that sometimes car trouble isn't so bad.
Type 1 diabetes often is referred to as juvenile diabetes
because it most often occurs in childhood; the average
age of onset is 12. However, the disease can occur at
any age. Symptoms include increased thirst and urination,
hunger, unexplained weight loss and fatigue.
This form of diabetes results from the body fighting
itself in a so-called autoimmune attack that causes
the pancreas to produce little or no insulin. People
with Type 1 diabetes must monitor their blood sugar
levels and inject insulin daily. About 1.7 million Americans
have Type 1 diabetes. (In Type 2 diabetes, the pancreas
continues to manufacture insulin, but the body develops
resistance to its effects. Obesity is a major risk factor
for developing Type 2 diabetes, which usually begins
There is no cure for Type 1 diabetes. Controlling blood
sugar levels is important to avoid further complications,
including heart disease, kidney failure and damage to
Original source: LA