Dr. Frank Jirik

Developing novel preclinical models of Thrombotic Thrombocytopenic Purpura

Dr. Frank Jirik, Professor of Biochemistry & Molecular Biology

Research Aims of this Study:

"Using mice that are devoid of Adamts13, a key molecule whose deficiency is involved in causing human TTP, Dr. Jirik and his team will search for factors that may trigger TTP attacks. Monitoring kidney function and clot formation, they will first investigate whether activation of the immune system, as would be seen in autoimmune diseases and in certain infections, is able to trigger TTP in mice lacking Adamts13. Similarly, they will determine whether the inability of blood vessel lining cells to produce the clot preventing gas, nitric oxide, leads to TTP and kidney damage in these animals. Lastly, they will develop a way to study clot formation non-invasively in live animals, since this will give us a way to monitor TTP development, and also the effectiveness of future treatments for TTP."

How Dr. Jirik believes this research will impact individuals living with or impacted by TTP:

"One of the primary goals of the proposed research will be to set up mouse models of TTP, which mimic human TTP. This will not only allow Dr. Jirik and his team to study the sorts of factors that can trigger TTP and how these might be blocked, but also, having animals that develop the types of organ pathology seen in human TTP will provide a means with which to carry out the (pre-clinical) evaluation of new experimental therapies that are aimed at either preventing TTP, or treating this condition once it has begun."

How Dr. Jirik became interested in TTP research:

"Stemming from his medical training, he has had a longstanding interest in various aspects autoimmunity, genetics, and hematology. During a recent work trip to Toronto for the MS Society Dr. Jirik learned from a friend about the Answering TTP Foundation and our strong interest in stimulating research into this devastating condition. He has always considered TTP as being a rather mysterious entity, however, in reviewing the current literature on the topic, he was surprised by how relatively little research was being done on the basic science aspects of this disease. He realized how many questions were yet to be answered, for example: Why some individuals with a genetic lack of Adamts13 only develop TTP in middle age, or never develop it at all?  In acquired (autoimmune) TTP, are their antibodies besides those responsible for depleting Adamts13, that actually trigger the disease? Are there environmental factors (e.g. infections, or dietary factors) that cause the first episode of TTP? Which are obviously important questions to study."

Comments from Dr. Jirik to donors:

"We are extremely grateful to the many individuals who have devoted their time and effort to fundraising activities, to the many donors, to the scientists that reviewed our proposal, and especially to Answering TTP Foundation for giving us the opportunity to jump-start this new Canadian TTP research initiative."