Björn Mellgård, MD, PhD, Vice President, Global Program Lead, Rare Genetics and Hematology, Takeda, discusses Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
Transcription:
My name is Björn Mellgård. As you may understand from that name, I’m from Scandinavia and more precisely, Sweden. I’ve been working in pharmaceutical research since the beginning of the ’90s with AstraZeneca, Novartis, Shire, and now latest with Takeda.
I have a medical background from Sweden. I’ve been working in various stages of pharmaceutical research drug discovery. I started with drug discovery, working in translation of medicine, and then also late-stage clinical trial, and more recently.
I’ve been leading the ADZYNMA program for seven years. I moved here to Boston in 2017. Then followed this very interesting drug, I would say, over the past seven years.
Quite interesting. It’s a very rare disease to begin with. TTP comes in two forms. One is an inherited form, what we call congenital TTP. The other form, which is more common, is than an autoimmune disease where antibodies to this enzyme, ADAMTS13, which is the focus of our research, and which is the product of ADZYNMA. These antibodies would then block the function of this enzyme, and that’s the autoimmune or immune-mediated form of TTP.
Now, I think people are quite aware of bleeding disorders. Most people have heard about hemophilia, for example. These are then deficiencies in the coagulation cascade that will lead to bleeding. Now, I think you can look at this as, in many cases, there is a balance that the body is trying to balance bleeding on the one hand and clotting on the other hand.
TTP is then the opposite of bleeding. It’s an exaggerated clotting that happens in these patients. The symptoms all derive from these small blood clots forming in the bloodstream, and then the symptoms arise as a cause of these, or as an effect of these blood clots.
Most prominent is, of course, I would say, blood clots in the central nervous system and the brain. Patients can present then with severe CNS symptoms or central nervous system symptoms. Quite common over time is also the occurrence of stroke as a quite severe form, of course. But also, myocardial infarction, clots in the heart or kidney symptoms.
Then, the underlying cause of this is then a deficiency in this enzyme we call ADAMTS13. The function of this enzyme is interesting because it regulates the activity of a coagulation factor, which is called von Willebrand factor.
Some people may have heard that there is a disease called von Willebrand disease, which is a deficiency of this protein called von Willebrand factor. So, if you lack that one, you bleed. If you have too much, you coagulate. ADAMTS13 is then regulating, or down-regulating, I would say, the effect of this von Willebrand factor. When that regulation is absent, then you have TTP.
For more information about Hematologic Disorders: https://checkrare.com/diseases/hematologic-disorders/
