Empowered Patient Podcast Developing Recombinant Protein to Treat Ultra-Rare Hematology Disease cTTP with Dr. Björn Mellgård Takeda
Dr. Björn Mellgård, VP and Global Program Lead of rare genetics and hematology at Takeda, is passionate about finding a cure for cTTP, congenital thrombotic thrombocytopenic purpura. This ultra-rare disease, caused by an enzyme deficiency, presents in early childhood and results in life-threatening blood clots. With their investigational drug TAK-755, a recombinant enzyme, the volume is very small, and the infusion takes four to five minutes and is a replacement therapy to allow patients to avoid daily symptoms and acute episodes.
Björn explains, "What happens then is that we have our coagulation system, and many people have heard about bleeding disorders, mainly probably hemophilia, where you lack certain factors which are important to make the blood clot. TTP is on the other side of the spectrum, and the deficiency we're talking about, this ADAMTS13 enzyme, is also importantly involved in blood coagulation."
"But the effect is when you don't have this enzyme present. The blood has a tendency to spontaneously form blood clots in the circulation. And these blood clots then tend to lodge in critical organs such as the brain, the heart, and the kidney, and the patients then suffer symptoms based on that."
"So, our drug then represents a recombinant enzyme. So, it's a recombinant protein that is produced in a laboratory. It's exactly the same as we have in our bodies. And this kind of replacement therapy has been used for a long time. And I mentioned hemophilia as an example where Takeda and also other companies have recombinant factor VIII in that case. So, the principle behind this treatment is to give the patient what they're missing. That's pretty straightforward in some sense."
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