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Welcome, everybody, this is Matthias von Herrath.
I'm a professor at La Jolla Institute and also
Vice President in the Global Chief Medical Office at Novo Nordisk.
Today, we will be discussing strategies and
obstacles on the path for type 1 diabetes therapies.
As you can see on the first slide,
we have a strong commitment to bringing new therapies to the clinic and Novo Nordisk.
For me personally, it has been a life long obsession,
to try to find something to alleviate the life for the patients with type 1 diabetes
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and we will discuss also in this talk why that is important.
We will discuss initially the new insights into
the pathology of human diabetes which we study in La Jolla Institute,
and then we will discuss the therapies that
we have so far been developing. We will also mention obstacles in bringing them to the clinic,
and also any type of regulatory or other considerations that will be
important for bringing therapies in type 1 diabetes to the clinic.
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On the next slide, you will see the overall prevalence and
the burden of type 1 diabetes as it presents itself these days.
We think that type 1 diabetes is an autoimmune disease,
at least in the phase around diagnosis when most of the beta cells are being destroyed,
and there's also a strong genetic component;
although, there are many genes that contribute to this disease.
So, it's not as easy as if you would have a few genes contributing to
type 1 diabetes and you can then influence these pathways, therapeutically, very easily.
We know that the beta cells ultimately are being destroyed.
Ultimately, in most patients,
that results in a complete insulin deficiency.
Although, one has to say that often after diagnosis,
there's still a fair amount of potentially
functioning beta cells left in the pancreas that can make insulin.
Type 1 accounts for about five percent of
diabetes and affects about 20 million individuals worldwide.
Of course, the main issue with a disease like type 1 is that one wants to avoid
complications and those are mostly complications that concern the vascular system,
but also the nervous system-
they are related among other factors,
some of which are actually not fully known to the glucose control in the blood cell.
So, once one is diagnosed with type 1 diabetes,
it is very important to try to achieve as normal as
possible glucose control with external insulin injections,
basal insulin usually and then mealtime insulin,
and that is actually quite a bit more difficult
than it might seem when you just hear it spoken,
and it's a big struggle for many patients,
and it is certainly a tremendous burden on the life and a day-to-day hassle.
That's a big reason why we try to find new therapies that will make
that life easier for patients with type 1 diabetes and
ultimately to completely prevent or cure the disease;
of course, that's the pie in the sky, so to speak.
The estimated life expectancy for
type 1 diabetes patients can be at some years of life are being lost,
and that depends really on the glycemic control and individual abilities to do so,
which is not, I should add, only a matter of
personal will or ability to control the sugar,
adhere to strict regiments of exercise and food intake, and so forth.
But, in some patients,
maybe sometimes for reasons we don't fully understand yet,
the excursions of the blood glucose at night and also during the day are larger than in
other patients and it's much harder for them to
spend the time glucose-wise and optimal range,
as we also know, now, on
the new continuous glucose meters that can be implanted in the skin, for example.
We know that earlier recognition,
with that also an approved treatment of type 1 diabetes might
lead to a much-improved prognosis in terms of the complications.
That is, of course, something we want to try to achieve;
you could even argue that detecting the disease early
enough would result in avoiding a diagnosis in ketoacidosis,
which is an acute emergency clinically and still
today's date often doesn't have a very good outcome,
so it's a dangerous condition.
In that respect, knowing if a person would be prone to diabetes would be very, very helpful.
We will discuss again in my talk how that can be done,
maybe the population-wide as an ultimate vision.
Now, we go to the next slide.
