In an interview with Jack Woodfield from
Diabetes.co.uk, Dr Bart Roep of the California-based City Of Hope
says he is on track to cure diabetes in six
'The C-word is controversial within diabetes
circles, yet the City of Hope had no reticence about making the
claim.' - Jack Woodfield.
Dr Bart Roep is the director of the diabetes research facilityat
the City of Hope's Diabetes and Metabolism Research Institute. Born
in the Netherlands, he leads a team whose mission is to cure type 1
diabetes, and while their ambitions are lofty, so is their early
In March, Dr Roep's team published the results of a 14-year-boy
with type 1 diabetes who underwent stem cell transplantation. The
boy has since been
free from insulin without any side effects for eight
This, Dr Roep said, was the first definitive proof that type 1
diabetes can be cured. But there are still several critical
questions to be answered.
Dr Roep acknowledges that cure is "a dangerous word to use" in
regard to type 1 diabetes research.
"What we are trying to do is understand why people get type 1
diabetes and to translate this to find a cure," Roep said. "That
is, of course, a dangerous word to use. But we think that we are
onto a couple of leads."
One of these leads is islet
cell transplantation, a procedure that involves transplanting
islet (insulin-producing) cells into patients from donor
In some cases, the transplants can help a patient come off
insulin, but other times the cells are rejected or attacked by the
immune system unless immunosuppressant drugs are also given, which
can cause side effects.
Dr Roep's team made a significant discovery along the way: by
reading the immune signatures of patients they were able to predict
how successful transplantation would be. Dr Roep says this is the
first step towards personalising medicine in type 1 diabetes.
"It turns out we can predict before surgery who has a fantastic
chance of lasting benefit, by which I mean cured for three full
years from type 1 diabetes without any additional therapy.
"We found those patients with that immune signature could be
treated, however we found another signature that showed a third had
no benefit at all. In other words we can now get a complete
prediction of who will get the ultimate benefit - complete
remission - and those who won't."
"The problem with the medication we're giving now is that these
drugs are great at preventing something new but they're pretty
lousy at reversing something that was already there," said Dr
"So the problem is that we are using the wrong drugs. The drugs
we're using to prevent rejection of islets induce insulin
resistance and type 2 diabetes.
"There are alternatives - people have been working for decades
on encapsulation - but there is still a long way to go because the
islets need to be able to respond very quickly, so the treatment is
not ready yet for the big stage."
Dr Roep's team is also working on a separate approach: a
type 1 diabetes vaccine.
Last year the team administered a patient with a vaccine
consisting of the patient's own immune cells, taken from his blood,
that were treated with vitamin D3 and a beta-cell protein.
Another eight people have since received the vaccine, and an
18-month study period is underway.
Dr Roep is excited about the study because, he says, researchers
can now tackle the root cause of type 1 diabetes, whereas before
they were only able to treat symptoms.
In February, he proposed that type 1 diabetes results from
a mistake of beta cells, not the immune system, an alternative
theory to how the autoimmune disease develops.
With this in mind, Roep believes the findings from their new
vaccine study could prove significant in correcting the autoimmune
response from these beta cells.
"I want to take advantage of the new insight that we have into
type 1 diabetes, and that's the diagnosis," he said.
"We get told in textbook medicine that you get diagnosed with
90% of beta cells destroyed and the other 10% follow suit, but
that's not true. As a matter of fact, the majority of [type 1]
patients have beta cells so that increases the sense of urgency to
protect those beta cells, and also to get a better picture of
predicting when they will be destroyed."
Dr Roep is confident that a cure is indeed on the horizon.
"These are very important times and while I'm not afraid to say
cure, I'm a little more reluctant to use time frames."
He admits that there is still colossal work to be done, but the
tools are in place to achieve it.
There will be setbacks along the way, but he insists these can
help teach scientists more about how best to personalise medicine
and future treatments.
"Understanding why [a treatment] sometimes fails will allow us
to design new treatment strategies for less fortunate patients.