Platelets are small, short-lived, non-nucleated cells present in the blood and are of paramount importance to prevent blood loss following injury. Low concentrations of platelets (thrombocytopenia) can lead to excessive blood loss and inability to heal wounds. Thrombocytopenia is a significant medical problem with high incidence in the US and can result from diseases such as blood disorders and cancers, certain infections such as AIDS and common medical treatments such as chemotherapy. The typical treatment for patients suffering from thrombocytopenia is the administration of donor platelets. However, patients who receive repeated doses of donor platelets often become refractory to treatment and therefore suffer a severe risk of bleeding. To treat these alloimmunized thrombocytopenic patients, we propose to utilize our expertise and technology developed over the last decade to expand and culture hematopoietic stem and progenitor cells (HSPCs) to produce high numbers of platelets that are more appropriately matched to the affected patients. As part of these studies specifically, we will optimize platelet production processes and demonstrate safety and efficacy for eventual human use.
Statement of Benefit to California:
Over 2 million platelet units are transfused yearly in the US to treat thrombocytopenia, which frequently occurs in patients suffering from a variety of cancers. In CA alone, 171,330 new cancer diagnoses are estimated for 2013. Platelet transfusion can lead to rejection due to alloimmunization and subsequently lengthened hospital stay. An alternative to donor platelets is needed to meet demand for matched platelets in alloimmune refactory thrombocytopenia. The Genomics Institute of the Novartis Research Foundation (GNF; San Diego, CA) was founded in 1999 and since its inception has developed cutting-edge technology platforms and cell-based therapeutics to address of areas of unmet medical need. Specifically, GNF has brought together over 450 experts with a breadth of expertise across regenerative medicine, immunology, engineering and chemistry to pioneer the development of stem cell-based therapies to treat blood disorders. We propose to leverage our technology and experience to derive platelets from umbilical cord blood stem cells for patients refractory to receipt of unmatched donor platelets. We will work collaboratively with other companies, universities and clinicians within CA to optimize our platelet product. We firmly believe that our unique infrastructure and history of success with stem cell-based therapies combined with our collaborative and inventive culture will provide an incomparable environment to catalyze the development of therapeutics for thrombocytopenia.