2013 Research Annual Report

Cancer and Blood Diseases Institute

Division Details

Division Data Summary

Research and Training Details

Number of Faculty67
Number of Joint Appointment Faculty22
Number of Research Fellows46
Number of Research Students17
Number of Support Personnel317
Peer Reviewed Publications145

Clinical Activities and Training

Number of Clinical Staff18
Number of Clinical Fellows16
Number of Other Students7
Inpatient Encounters20,159
Outpatient Encounters19,039

Division Photo

CBDI
J Palumbo, J Perentesis, Y Zheng, S Davies


Significant Accomplishments

Significant Accomplishments

Experimental Hematology and Cancer Biology

New approach targeting NF1

Neurofibromatosis type 1 (NF1) patients develop benign neurofibromas and malignant peripheral nerve sheath tumors that remain incurable. A group led by Nancy Ratner, PhD, found that NF1 mutation causes sustained ERK activation in these tumors. They showed that an MEK inhibitor reduces aberrantly proliferating tumor cells, shrinks neurofibromas in mice, and prolongs survival of mice implanted with human cancer cells. This study, reported in the Journal of Clinical Investigation, demonstrates that deregulated ERK signaling is critical for the growth of NF1 peripheral nerve tumors and provides a strong rationale for future clinical trials.

Passing through the blood-brain barrier

Dao Pan, PhD, and colleagues discovered a non-invasive procedure to deliver large-molecule therapeutic agents across the blood-brain barrier to treat neurological disorders. Findings were published online in February 2013 in PNAS. To realize the potential of large molecular weight substances to treat neurological disorders, they fused a receptor-binding peptide from apolipoprotein E with a potentially therapeutic protein that can be transcytosed into the central nervous system in a mouse model of a lysosomal storage disorder with central nervous system defects. They demonstrated the therapeutic potential of this approach by correcting brain glycosaminoglycan and β-hexosaminidase in the mice.

Stem cell regeneration mechanism

Krüppel-like factor 5 regulates pluripotent stem cell self-renewal. In a work published in Nature Communication, Jose Cancelas Perez, MD, PhD, and colleagues reported that Krüppel-like factor 5-deficient hematopoietic stem cells and progenitors fail to engraft after transplantation. They supplied evidence that Krüppel-like factor 5 is indispensable for adhesion, homing, lodging and retention of hematopoietic stem cells and progenitors in the bone marrow through Rab5-dependent regulation of β1/β2 integrins.

Chemicals targeting G-protein coupled receptor signaling

The G-protein-mediated Rho GTPase signaling axis has been implicated in human pathophysiology and is a potential therapeutic target. Yi Zheng, PhD, and colleagues reported in PNAS, the rational design and identification of a family of small molecule inhibitors specifically targeting Rho activators to benefit cancer stem cell therapy. Their work presents a new drug design strategy for dual inhibition of an enzyme-substrate pair that leads to enhanced efficacy and specificity of a targeted therapy.

Hematology

Ware named Division Director

After a thorough search process, the Hematology Faculty and Search Committee selected Russell Ware, MD, PhD, as the new Division Director. Ware comes to Cincinnati Children’s from Baylor College of Medicine in Houston. He obtained his MD and PhD degrees from Duke University, and completed his fellowship in Pediatric Hematology/Oncology at Duke. He has been involved in clinical and translational hematology research projects for more than 25 years, with a primary interest in sickle cell disease. Ware has substantial personal experience with directing patient-oriented research, and currently runs an NIH-funded project to investigate genetic modifiers of sickle cell disease. Ware also is the national principal investigator for several NIH-funded multi-center sickle cell clinical trials, including the Stroke With Transfusions Changing to Hydroxyurea (SWiTCH), TCD With Transfusions Changing to Hydroxyurea (TWiTCH) and Sparing Conversion to Abnormal TCD Elevations (SCATE) studies. Most recently he has moved his research efforts into the international arena, starting SCD pilot screening programs in Angola, Uganda and other regions of Africa. He has published more than 225 peer-reviewed articles and has personally trained dozens of students and fellows. Ware will lead the Division of Hematology with overall goals of expanding its research portfolio, growing clinical expertise, and developing national and international prominence.

Oncology

No. 1 pediatric cancer program in the nation

The Oncology Division has had a very successful year, culminated by being named the top pediatric cancer program in the country by US News and World Report. This honor reflects successful team science, team process improvement and team clinical care initiatives that are fundamental to the Division. Key associated advances included successful bench-to-bedside implementation of faculty-led, investigator-initiated clinical trials using targeted small molecules for relapsed leukemia, brain tumors, advanced sarcomas and liver/renal tumors. These initiatives all involved close integration of translational oncology efforts between Oncology, Experimental Hematology, Blood/Marrow Transplant, Pathology, Clinical Pharmacology, and Biomedical Informatics.


Division Publications

Cancer and Blood Diseases Institute Publications

  1. Abruzzo T, Patino M, Leach J, Rahme R, Geller J. Cerebral vasoconstriction triggered by sympathomimetic drugs during intra-atrerial chemotherapy. Pediatr Neurol. 2013; 48:139-42.
  2. Acosta MT, Bearden CE, Castellanos FX, Cutting L, Elgersma Y, Gioia G, Gutmann DH, Lee YS, Legius E, Muenke M, North K, Parada LF, Ratner N, Hunter-Schaedle K, Silva AJ. The Learning Disabilities Network (LeaDNet): using neurofibromatosis type 1 (NF1) as a paradigm for translational research. Am J Med Genet A. 2012; 158A:2225-32.
  3. Akunuru S, James Zhai Q, Zheng Y. Non-small cell lung cancer stem/progenitor cells are enriched in multiple distinct phenotypic subpopulations and exhibit plasticity. Cell Death Dis. 2012; 3:e352.
  4. Al Hawas R, Ren Q, Ye S, Karim ZA, Filipovich AH, Whiteheart SW. Munc18b/STXBP2 is required for platelet secretion. Blood. 2012; 120:2493-500.
  5. Ayas M, Saber W, Davies SM, Harris RE, Hale GA, Socie G, LeRademacher J, Thakar M, Deeg HJ, Al-Seraihy A, Battiwalla M, Camitta BM, Olsson R, Bajwa RS, Bonfim CM, Pasquini R, Macmillan ML, George B, Copelan EA, Wirk B, Al Jefri A, Fasth AL, Guinan EC, Horn BN, Lewis VA, Slavin S, Stepensky P, Bierings M, Gale RP. Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia. J Clin Oncol. 2013; 31:1669-76.
  6. Azam M. An in vitro screening to identify drug-resistant mutations for target-directed chemotherapeutic agents. In: y zheng, ed. Rational Drug Design - Methods Mol Biol. New York: Springer; 2012:175-84.
  7. Ballas SK, Kesen MR, Goldberg MF, Lutty GA, Dampier C, Osunkwo I, Wang WC, Hoppe C, Hagar W, Darbari DS, Malik P. Beyond the definitions of the phenotypic complications of sickle cell disease: an update on management. ScientificWorldJournal. 2012; 2012:949535.
  8. Barger JF, Gallo CA, Tandon P, Liu H, Sullivan A, Grimes HL, Plas DR. S6K1 determines the metabolic requirements for BCR-ABL survival. Oncogene. 2013; 32:453-61.
  9. Bell-Horwath TR, Vadukoot AK, Thowfeik FS, Li G, Wunderlich M, Mulloy JC, Merino EJ. Novel ROS-activated agents utilize a tethered amine to selectively target acute myeloid leukemia. Bioorg Med Chem Lett. 2013; 23:2951-4.
  10. Borges M, Barreira-Silva P, Florido M, Jordan MB, Correia-Neves M, Appelberg R. Molecular and Cellular Mechanisms of Mycobacterium avium-Induced Thymic Atrophy. J Immunol. 2012; 189:3600-8.
  11. Borissoff JI, Otten JJ, Heeneman S, Leenders P, van Oerle R, Soehnlein O, Loubele ST, Hamulyak K, Hackeng TM, Daemen MJ, Degen JL, Weiler H, Esmon CT, van Ryn J, Biessen EA, Spronk HM, ten Cate H. Genetic and pharmacological modifications of thrombin formation in apolipoprotein e-deficient mice determine atherosclerosis severity and atherothrombosis onset in a neutrophil-dependent manner. PLoS One. 2013; 8:e55784.
  12. Buchbinder D, Nugent DJ, Brazauskas R, Wang Z, Aljurf MD, Cairo MS, Chow R, Duncan C, Eldjerou LK, Gupta V, Hale GA, Halter J, Hayes-Lattin BM, Hsu JW, Jacobsohn DA, Kamble RT, Kasow KA, Lazarus HM, Mehta P, Myers KC, Parsons SK, Passweg JR, Pidala J, Reddy V, Sales-Bonfim CM, Savani BN, Seber A, Sorror ML, Steinberg A, Wood WA, Wall DA, Winiarski JH, Yu LC, Majhail NS. Late effects in hematopoietic cell transplant recipients with acquired severe aplastic anemia: a report from the late effects working committee of the center for international blood and marrow transplant research. Biol Blood Marrow Transplant. 2012; 18:1776-84.
  13. Bucuvalas J, Filipovich L, Yazigi N, Narkewicz MR, Ng V, Belle SH, Zhang S, Squires RH. Immunophenotype Predicts Outcome in Pediatric Acute Liver Failure. J Pediatr Gastroenterol Nutr. 2013; 56:311-315.
  14. Busaidy NL, Farooki A, Dowlati A, Perentesis JP, Dancey JE, Doyle LA, Brell JM, Siu LL. Management of metabolic effects associated with anticancer agents targeting the PI3K-Akt-mTOR pathway. J Clin Oncol. 2012; 30:2919-28.
  15. Chang KH, Sanchez-Aguilera A, Shen S, Sengupta A, Madhu MN, Ficker AM, Dunn SK, Kuenzi AM, Arnett JL, Santho RA, Agirre X, Perentesis JP, Deininger MW, Zheng Y, Bustelo XR, Williams DA, Cancelas JA. Vav3 collaborates with p190-BCR-ABL in lymphoid progenitor leukemogenesis, proliferation, and survival. Blood. 2012; 120:800-11.
  16. Chapman H, Waclaw RR, Pei Z, Nakafuku M, Campbell K. The homeobox gene Gsx2 controls the timing of oligodendroglial fate specification in mouse lateral ganglionic eminence progenitors. Development. 2013; 140:2289-98.
  17. Chen L, Acciani T, Le Cras T, Lutzko C, Perl AK. Dynamic regulation of platelet-derived growth factor receptor alpha expression in alveolar fibroblasts during realveolarization. Am J Respir Cell Mol Biol. 2012; 47:517-27.
  18. Chen W, Adams D, Patel M, Gupta A, Dasgupta R. Generalized lymphatic malformation with chylothorax: long-term management of a highly morbid condition in a pediatric patient. J Pediatr Surg. 2013; 48:e9-12.
  19. Chen X, Skutt-Kakaria K, Davison J, Ou YL, Choi E, Malik P, Loeb K, Wood B, Georges G, Torok-Storb B, Paddison PJ. G9a/GLP-dependent histone H3K9me2 patterning during human hematopoietic stem cell lineage commitment. Genes Dev. 2012; 26:2499-511.
  20. Chima RS, Abulebda K, Jodele S. Advances in critical care of the pediatric hematopoietic stem cell transplant patient. Pediatr Clin North Am. 2013; 60:689-707.
  21. Chima RS, Daniels RC, Kim MO, Li D, Wheeler DS, Davies SM, Jodele S. Improved outcomes for stem cell transplant recipients requiring pediatric intensive care. Pediatr Crit Care Med. 2012; 13:e336-42.
  22. Chou FS, Griesinger A, Wunderlich M, Lin S, Link KA, Shrestha M, Goyama S, Mizukawa B, Shen S, Marcucci G, Mulloy JC. The thrombopoietin/MPL/Bcl-xL pathway is essential for survival and self-renewal in human preleukemia induced by AML1-ETO. Blood. 2012; 120:709-19.
  23. Cost CR, Stegner MM, Leonard D, Leavey P. IL-8 predicts pediatric oncology patients with febrile neutropenia at low risk for bacteremia. J Pediatr Hematol Oncol. 2013; 35:206-11.
  24. Cost NG, Geller JI, DeFoor WR, Jr., Wagner LM, Noh PH. A robotic-assisted laparoscopic approach for pediatric renal cell carcinoma allows for both nephron-sparing surgery and extended lymph node dissection. J Pediatr Surg. 2012; 47:1946-50.
  25. Crosby LE, Barach I, McGrady ME, Kalinyak KA, Eastin AR, Mitchell MJ. Integrating interactive web-based technology to assess adherence and clinical outcomes in pediatric sickle cell disease. Anemia. 2012; 2012:492428.
  26. Dasgupta B, Ju JS, Sasaki Y, Liu X, Jung SR, Higashida K, Lindquist D, Milbrandt J. The AMPK beta2 subunit is required for energy homeostasis during metabolic stress. Mol Cell Biol. 2012; 32:2837-48.
  27. Davalos D, Ryu JK, Merlini M, Baeten KM, Le Moan N, Petersen MA, Deerinck TJ, Smirnoff DS, Bedard C, Hakozaki H, Gonias Murray S, Ling JB, Lassmann H, Degen JL, Ellisman MH, Akassoglou K. Fibrinogen-induced perivascular microglial clustering is required for the development of axonal damage in neuroinflammation. Nat Commun. 2012; 3:1227.
  28. Delaney MK, Liu J, Zheng Y, Berndt MC, Du X. The role of Rac1 in glycoprotein Ib-IX-mediated signal transduction and integrin activation. Arterioscler Thromb Vasc Biol. 2012; 32:2761-8.
  29. Dome JS, Fernandez CV, Mullen EA, Kalapurakal JA, Geller JI, Huff V, Gratias EJ, Dix DB, Ehrlich PF, Khanna G, Malogolowkin MH, Anderson JR, Naranjo A, Perlman EJ, Committee COGRT. Children's Oncology Group's 2013 blueprint for research: renal tumors. Pediatr Blood Cancer. 2013; 60:994-1000.
  30. Dowling MM, Quinn CT, Plumb P, Rogers ZR, Rollins NK, Koral K, Buchanan GR. Acute silent cerebral ischemia and infarction during acute anemia in children with and without sickle cell disease. Blood. 2012; 120:3891-7.
  31. Duan W, Gao L, Zhao W, Leon M, Sadee W, Webb A, Resnick K, Wu X, Ramaswamy B, Cohn DE, Shapiro C, Andreassen PR, Otterson GA, Villalona-Calero MA. Assessment of FANCD2 nuclear foci formation in paraffin-embedded tumors: a potential patient-enrichment strategy for treatment with DNA interstrand crosslinking agents. Transl Res. 2013; 161:156-64.
  32. Dumont LJ, Cancelas JA, Dumont DF, Siegel AH, Szczepiorkowski ZM, Rugg N, Pratt PG, Worsham DN, Hartman EL, Dunn SK, O'Leary M, Ransom JH, Michael RA, Macdonald VW. A randomized controlled trial evaluating recovery and survival of 6% dimethyl sulfoxide-frozen autologous platelets in healthy volunteers. Transfusion. 2013; 53:128-37.
  33. Dumont LJ, Cancelas JA, Graminske S, Friedman KD, Vassallo RR, Whitley PH, Rugg N, Dumont DF, Herschel L, Siegal AH, Szczepiorkowski ZM, Fender L, Razatos A. In vitro and in vivo quality of leukoreduced apheresis platelets stored in a new platelet additive solution. Transfusion. 2013; 53:972-80.
  34. Eapen M, Ahn KW, Orchard PJ, Cowan MJ, Davies SM, Fasth A, Hassebroek A, Ayas M, Bonfim C, O'Brien TA, Gross TG, Horwitz M, Horwitz E, Kapoor N, Kurtzberg J, Majhail N, Ringden O, Szabolcs P, Veys P, Baker KS. Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism. Biol Blood Marrow Transplant. 2012; 18:1438-45.
  35. El-Amouri SS, Cao P, Miao C, Pan D. Secreted luciferase for in vivo evaluation of systemic protein delivery in mice. Mol Biotechnol. 2013; 53:63-73.
  36. Fang J, Rhyasen G, Bolanos L, Rasch C, Varney M, Wunderlich M, Goyama S, Jansen G, Cloos J, Rigolino C, Cortelezzi A, Mulloy JC, Oliva EN, Cuzzola M, Starczynowski DT. Cytotoxic effects of bortezomib in myelodysplastic syndrome/acute myeloid leukemia depend on autophagy-mediated lysosomal degradation of TRAF6 and repression of PSMA1. Blood. 2012; 120:858-67.
  37. Filipovich AH. Gene therapy targets XLP. Blood. 2013; 121:1066-7.
  38. Flick MJ, Du X, Prasad JM, Raghu H, Palumbo JS, Smeds E, Hook M, Degen JL. Genetic elimination of the binding motif on fibrinogen for the S. aureus virulence factor ClfA improves host survival in septicemia. Blood. 2013; 121:1783-94.
  39. Freyer DR, Felgenhauer J, Perentesis J, Adolescent COG, Young Adult Oncology Discipline C. Children's Oncology Group's 2013 blueprint for research: adolescent and young adult oncology. Pediatr Blood Cancer. 2013; 60:1055-8.
  40. Gamis AS, Alonzo TA, Perentesis JP, Meshinchi S, Committee COGAML. Children's Oncology Group's 2013 blueprint for research: acute myeloid leukemia. Pediatr Blood Cancer. 2013; 60:964-71.
  41. Gazda HT, Preti M, Sheen MR, O'Donohue MF, Vlachos A, Davies SM, Kattamis A, Doherty L, Landowski M, Buros C, Ghazvinian R, Sieff CA, Newburger PE, Niewiadomska E, Matysiak M, Glader B, Atsidaftos E, Lipton JM, Gleizes PE, Beggs AH. Frameshift mutation in p53 regulator RPL26 is associated with multiple physical abnormalities and a specific pre-ribosomal RNA processing defect in diamond-blackfan anemia. Hum Mutat. 2012; 33:1037-44.
  42. Geiger H, Pawar SA, Kerschen EJ, Nattamai KJ, Hernandez I, Liang HP, Fernandez JA, Cancelas JA, Ryan MA, Kustikova O, Schambach A, Fu Q, Wang J, Fink LM, Petersen KU, Zhou D, Griffin JH, Baum C, Weiler H, Hauer-Jensen M. Pharmacological targeting of the thrombomodulin-activated protein C pathway mitigates radiation toxicity. Nat Med. 2012; 18:1123-9.
  43. George A, Pushkaran S, Konstantinidis DG, Koochaki S, Malik P, Mohandas N, Zheng Y, Joiner CH, Kalfa TA. Erythrocyte NADPH oxidase activity modulated by Rac GTPases, PKC, and plasma cytokines contributes to oxidative stress in sickle cell disease. Blood. 2013; 121:2099-107.
  44. Gow KW, Barnhart DC, Hamilton TE, Kandel JJ, Chen MK, Ferrer FA, Price MR, Mullen EA, Geller JI, Gratias EJ, Rosen N, Khanna G, Naranjo A, Ritchey ML, Grundy PE, Dome JS, Ehrlich PF. Primary nephrectomy and intraoperative tumor spill: report from the Children's Oncology Group (COG) renal tumors committee. J Pediatr Surg. 2013; 48:34-8.
  45. Goyama S, Mulloy JC. Making healthy stem cells: the new role of TPO. Cell Stem Cell. 2013; 12:8-9.
  46. Guo L, Moon C, Niehaus K, Zheng Y, Ratner N. Rac1 controls Schwann cell myelination through cAMP and NF2/merlin. J Neurosci. 2012; 32:17251-61.
  47. Gutmann DH, Parada LF, Silva AJ, Ratner N. Neurofibromatosis type 1: modeling CNS dysfunction. J Neurosci. 2012; 32:14087-93.
  48. Hassan A, Booth C, Brightwell A, Allwood Z, Veys P, Rao K, Honig M, Friedrich W, Gennery A, Slatter M, Bredius R, Finocchi A, Cancrini C, Aiuti A, Porta F, Lanfranchi A, Ridella M, Steward C, Filipovich A, Marsh R, Bordon V, Al-Muhsen S, Al-Mousa H, Alsum Z, Al-Dhekri H, Al Ghonaium A, Speckmann C, Fischer A, Mahlaoui N, Nichols KE, Grunebaum E, Al Zahrani D, Roifman CM, Boelens J, Davies EG, Cavazzana-Calvo M, Notarangelo L, Gaspar HB. Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency. Blood. 2012; 120:3615-24; quiz 3626.
  49. Hennigan RF, Moon CA, Parysek LM, Monk KR, Morfini G, Berth S, Brady S, Ratner N. The NF2 tumor suppressor regulates microtubule-based vesicle trafficking via a novel Rac, MLK and p38(SAPK) pathway. Oncogene. 2013; 32:1135-43.
  50. Hirai H, Kamio N, Huang G, Matsusue A, Ogino S, Kimura N, Satake S, Ashihara E, Imanishi J, Tenen DG, Maekawa T. Cyclic AMP responsive element binding proteins are involved in 'emergency' granulopoiesis through the upregulation of CCAAT/enhancer binding protein beta. PLoS One. 2013; 8:e54862.
  51. Horwitz MS, Corey SJ, Grimes HL, Tidwell T. ELANE mutations in cyclic and severe congenital neutropenia: genetics and pathophysiology. Hematol Oncol Clin North Am. 2013; 27:19-41, vii.
  52. Hoskins EE, Morreale RJ, Werner SP, Higginbotham JM, Laimins LA, Lambert PF, Brown DR, Gillison ML, Nuovo GJ, Witte DP, Kim MO, Davies SM, Mehta PA, Butsch Kovacic M, Wikenheiser-Brokamp KA, Wells SI. The fanconi anemia pathway limits human papillomavirus replication. J Virol. 2012; 86:8131-8.
  53. Hummel TR, Chow LM, Fouladi M, Franz D. Pharmacotherapeutic management of pediatric gliomas : current and upcoming strategies. Paediatr Drugs. 2013; 15:29-42.
  54. Hummel TR, Miles L, Mangano FT, Jones BV, Geller JI. Clinical heterogeneity of desmoplastic infantile ganglioglioma: a case series and literature review. J Pediatr Hematol Oncol. 2012; 34:e232-6.
  55. Ishikawa ET, Cancelas JA. Lack of communication rusts and ages stem cells. Cell Cycle. 2012; 11:3149-50.
  56. Jessen WJ, Miller SJ, Jousma E, Wu J, Rizvi TA, Brundage ME, Eaves D, Widemann B, Kim MO, Dombi E, Sabo J, Hardiman Dudley A, Niwa-Kawakita M, Page GP, Giovannini M, Aronow BJ, Cripe TP, Ratner N. MEK inhibition exhibits efficacy in human and mouse neurofibromatosis tumors. J Clin Invest. 2013; 123:340-7.
  57. Jiang X, Huang H, Li Z, He C, Li Y, Chen P, Gurbuxani S, Arnovitz S, Hong GM, Price C, Ren H, Kunjamma RB, Neilly MB, Salat J, Wunderlich M, Slany RK, Zhang Y, Larson RA, Le Beau MM, Mulloy JC, Rowley JD, Chen J. MiR-495 is a tumor-suppressor microRNA down-regulated in MLL-rearranged leukemia. Proc Natl Acad Sci U S A. 2012; 109:19397-402.
  58. Jodele S, Hirsch R, Laskin B, Davies S, Witte D, Chima R. Pulmonary arterial hypertension in pediatric patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy. Biol Blood Marrow Transplant. 2013; 19:202-7.
  59. Jodele S, Laskin BL, Goebel J, Khoury JC, Pinkard SL, Carey PM, Davies SM. Does early initiation of therapeutic plasma exchange improve outcome in pediatric stem cell transplant-associated thrombotic microangiopathy?. Transfusion. 2012; 53:661-7.
  60. Joshi K, Banasavadi-Siddegowda Y, Mo X, Kim SH, Mao P, Kig C, Nardini D, Sobol RW, Chow LM, Kornblum HI, Waclaw R, Beullens M, Nakano I. MELK-dependent FOXM1 phosphorylation is essential for proliferation of glioma stem cells. Stem Cells. 2013; 31:1051-63.
  61. Kamani NR, Walters MC, Carter S, Aquino V, Brochstein JA, Chaudhury S, Eapen M, Freed BM, Grimley M, Levine JE, Logan B, Moore T, Panepinto J, Parikh S, Pulsipher MA, Sande J, Schultz KR, Spellman S, Shenoy S. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biol Blood Marrow Transplant. 2012; 18:1265-72.
  62. Katayama K, Leslie JR, Lang RA, Zheng Y, Yoshida Y. Left-right locomotor circuitry depends on RhoA-driven organization of the neuroepithelium in the developing spinal cord. J Neurosci. 2012; 32:10396-407.
  63. Keng VW, Rahrmann EP, Watson AL, Tschida BR, Moertel CL, Jessen WJ, Rizvi TA, Collins MH, Ratner N, Largaespada DA. PTEN and NF1 inactivation in Schwann cells produces a severe phenotype in the peripheral nervous system that promotes the development and malignant progression of peripheral nerve sheath tumors. Cancer Res. 2012; 72:3405-13.
  64. Keng VW, Watson AL, Rahrmann EP, Li H, Tschida BR, Moriarity BS, Choi K, Rizvi TA, Collins MH, Wallace MR, Ratner N, Largaespada DA. Conditional Inactivation of Pten with EGFR Overexpression in Schwann Cells Models Sporadic MPNST. Sarcoma. 2012; 2012:620834.
  65. Khandanpour C, Krongold J, Schutte J, Bouwman F, Vassen L, Gaudreau MC, Chen R, Calero-Nieto FJ, Diamanti E, Hannah R, Meyer SE, Grimes HL, van der Reijden BA, Jansen JH, Patel CV, Peeters JK, Lowenberg B, Duhrsen U, Gottgens B, Moroy T. The human GFI136N variant induces epigenetic changes at the Hoxa9 locus and accelerates K-RAS driven myeloproliferative disorder in mice. Blood. 2012; 120:4006-17.
  66. Khandanpour C, Phelan JD, Vassen L, Schutte J, Chen R, Horman SR, Gaudreau MC, Krongold J, Zhu J, Paul WE, Duhrsen U, Gottgens B, Grimes HL, Moroy T. Growth factor independence 1 antagonizes a p53-induced DNA damage response pathway in lymphoblastic leukemia. Cancer Cell. 2013; 23:200-14.
  67. Kumar S, Filippi M. Molecular Regulation of Granulopoiesis. In: D Gabrilovich, ed. The Neutrophils: New Outlook for Old Cells. London: Imperial College Press; 2013:1-41.
  68. Kumar S, Xu J, Perkins C, Guo F, Snapper S, Finkelman FD, Zheng Y, Filippi MD. Cdc42 regulates neutrophil migration via crosstalk between WASp, CD11b, and microtubules. Blood. 2012; 120:3563-74.
  69. Largaespada D, Ratner N. Interweaving the strands: beta-catenin, an HIV co-receptor, and Schwann cell tumors. Cancer Cell. 2013; 23:269-71.
  70. Laskin BL, Nehus E, Goebel J, Khoury JC, Davies SM, Jodele S. Cystatin C-estimated glomerular filtration rate in pediatric autologous hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2012; 18:1745-52.
  71. Li J, Sipple J, Maynard S, Mehta PA, Rose SR, Davies SM, Pang Q. Fanconi anemia links reactive oxygen species to insulin resistance and obesity. Antioxid Redox Signal. 2012; 17:1083-98.
  72. Liu WM, Zhang F, Moshiach S, Zhou B, Huang C, Srinivasan K, Khurana S, Zheng Y, Lahti JM, Zhang XA. Tetraspanin CD82 inhibits protrusion and retraction in cell movement by attenuating the plasma membrane-dependent actin organization. PLoS One. 2012; 7:e51797.
  73. Lopez-Juarez A, Howard J, Ullom K, Howard L, Grande A, Pardo A, Waclaw R, Sun YY, Yang D, Kuan CY, Campbell K, Nakafuku M. Gsx2 controls region-specific activation of neural stem cells and injury-induced neurogenesis in the adult subventricular zone. Genes Dev. 2013; 27:1272-87.
  74. Marsh RA, Allen CE, McClain KL, Weinstein JL, Kanter J, Skiles J, Lee ND, Khan SP, Lawrence J, Mo JQ, Bleesing JJ, Filipovich AH, Jordan MB. Salvage therapy of refractory hemophagocytic lymphohistiocytosis with alemtuzumab. Pediatr Blood Cancer. 2013; 60:101-9.
  75. Marsh RA, Bleesing JJ, Filipovich AH. Flow cytometric measurement of SLAM-associated protein and X-linked inhibitor of apoptosis. Methods Mol Biol. 2013; 979:189-97.
  76. Marsh RA, Rao K, Satwani P, Lehmberg K, Muller I, Li D, Kim MO, Fischer A, Latour S, Sedlacek P, Barlogis V, Hamamoto K, Kanegane H, Milanovich S, Margolis DA, Dimmock D, Casper J, Douglas DN, Amrolia PJ, Veys P, Kumar AR, Jordan MB, Bleesing JJ, Filipovich AH. Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes. Blood. 2013; 121:877-83.
  77. Martin-Ibanez R, Crespo E, Esgleas M, Urban N, Wang B, Waclaw R, Georgopoulos K, Martinez S, Campbell K, Vicario-Abejon C, Alberch J, Chan S, Kastner P, Rubenstein JL, Canals JM. Helios transcription factor expression depends on Gsx2 and Dlx1&2 function in developing striatal matrix neurons. Stem Cells Dev. 2012; 21:2239-51.
  78. McCavit TL, Xuan L, Zhang S, Flores G, Quinn CT. National trends in incidence rates of hospitalization for stroke in children with sickle cell disease. Pediatr Blood Cancer. 2013; 60:823-7.
  79. Mehta PA, Faulkner LB. Hematopoietic cell transplantation for thalassemia: a global perspective BMT tandem meeting 2013. Biol Blood Marrow Transplant. 2013; 19:S70-3.
  80. Mehta PA, Svahn J, Davies SM, Pang Q, Harris R, Ghezzi P, Lanza T, Ferretti E, Barabino P, Mueller R, Dufour C. Etanercept treatment in Fanconi anaemia; combined US and Italian experience. Br J Haematol. 2012; 158:809-11.
  81. Mellor-Heineke S, Villanueva J, Jordan MB, Marsh R, Zhang K, Bleesing JJ, Filipovich AH, Risma KA. Elevated Granzyme B in Cytotoxic Lymphocytes is a Signature of Immune Activation in Hemophagocytic Lymphohistiocytosis. Front Immunol. 2013; 4:72.
  82. Merchant MS, Geller JI, Baird K, Chou AJ, Galli S, Charles A, Amaoko M, Rhee EH, Price A, Wexler LH, Meyers PA, Widemann BC, Tsokos M, Mackall CL. Phase I trial and pharmacokinetic study of lexatumumab in pediatric patients with solid tumors. J Clin Oncol. 2012; 30:4141-7.
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Grants, Contracts, and Industry Agreements

Experimental Hematology Grants

Experimental Hematology

Grant and Contract AwardsAnnual Direct

Akunuru, S

Training Programs in Cancer Therapeutics
T32 CA 11784609/14/12-08/31/14$48,435

Andreassen, P

14th Annual Midwest DNA Repair Symposium
R13 CA 16831704/01/12-03/31/13$6,000
FANCD2 Monoubiquitination in DNA Damage Responses
R01 HL 08558707/08/08-06/30/13$222,750

Cancelas-Perez, J

Rational Design of a Vav/Rac Inhibitor as a New Therapy
10/01/12-09/30/15$180,018

Choi, K

Regulation of Cellular Growth and Differentiation
T32 CA02062409/30/12-08/31/13$21,122

Degen, J

Analysis of Staphylococcus Aureus Host Interactions
R01 AI 02062409/30/10-08/31/13$49,971

Degen, J / Malik P (MPI)

Hemostatic Factors and Sickle Cell Disease
R01 HL 11260301/01/12-11/30/16$238,000

Feng, Y

Targeting Bone Marrow Niche to Eradicate Childhood Acute Myeloid Leukemia
06/01/13-05/31/14$25,000

Filippi, M

Regulation of Neutrophil Migration and Polarity
R01 HL 09067603/01/10-02/28/15$235,620

Flick, M

Mechanisms Linking the Hemostatic Protease Thrombin to Arthritic Disease
R01 AR 05699008/10/09-07/31/14$171,072
Cincinnati Rheumatic Disease Core Center (Core 2)
P30 AR 04736308/25/11-06/30/16$52,222

Geiger, H

Activated Protein C for Treatment of Radiation Combined Injury
R33 AI 08055709/13/10-08/31/13$77,488
Molecular Mechanisms and Therapies for Radiation-Induced Myelodysplastic Syndrome
04/01/12-03/31/17$181,818

Goyama, S

Suppressing a Tumor Suppressor RUNX1: A Novel Approach to Treat Pediatric Acute Leukemia
07/01/12-06/30/13$40,000

Hennigan, R

Regulation of Intracellular Trafficking in NF2
W81XWH131013606/01/13-05/31/16$129,421

Komurov, K

Targeting Metabolic Vulnerabilities of ERBB2-Driven Breast Cancer
04/01/13-03/31/14$40,000

Lin, S

Identification of Drug Development Pathway for Improving Prognosis of Pediatric Leukemia
05/01/12-04/30/13$30,000

Lutzko, C

NHLBI Progenitor Cell Biology Institute - Pilot Study
U01 HL 09999703/01/13-02/28/14$58,905

Malik, P

Ameliorating Sickle Nephropathy and Pulmonary Hypertension
R34 HL 10875208/18/11-06/30/14$150,000
Development of Safe and Efficient Gene Therapy Strategies
R01 HL 09848901/21/10-12/31/14$43,333
PLGF-H1F1a-mIRNA Axis in Sickle Pulmonary Hypertension
R01 HL 11137201/01/12-12/31/16$161,480
Cincinnati Cell Characterization Core
U01 HL 09999709/30/10-04/30/14$316,697
Cincinnati Cell Characterization Core - per assay
U01 HL 09999709/30/10-04/30/14$247,368

Mulloy, J

Genotype and Phenotype of Chemoresistant AML
R21 CA 16836903/01/13-02/28/15$130,500
Rac Signaling in MLL Leukemia
07/01/10-06/30/15$104,702

Olshavsky, N

Regulation of Cellular Growth and Differentiation
T32 CA 05926812/6/12-04/30/13$21,122

Pan, D

Genetic Therapy for CNS Manifestations in MPS I via BBB-Targeted Protein Delivery
R01 NS 06433009/30/08-08/31/13$214,375

Pang, Q

Role of FA Proteins in Hematopoiesis
R01 HL 07671204/01/10-03/31/15$235,620
Role of Tumor Necrosis Factor in Leukemogenesis
07/01/08-06/30/13$103,115
Targeted Improvement in Stem Cell Therapy for Leukemia and Bone Marrow Failure Syndromes
R01 CA 15753702/01/11-12/31/15$195,050

Patel, A

Neurofibromatosis Postdoctoral Traineeship
06/01/13-05/31/14$46,092

Ratner, N

Can Targeting Fat Cure MPNST?
06/01/13-05/31/14$30,000
Cincinnati Center for Neurofibromatosis Research
P50 NS 05753109/15/08-06/30/13$1,068,273
Ratner, NProject A$48,069
Cripe, TProject B$106,147
Rizvi, TProject C$81,328
Perentesis, JProject 1$297,056
Ratner, NProject 2$225,064
Ratner, NProject 3$276,814
Identification of Molecular and Cellular Contributors to Neurofibroma Formation and Growth
W81XWH121013307/01/12-06/30/15$221,965
In Vivo Testing of Anti-Oxidants in Nf1 CNS
07/01/12-06/30/13$45,455
Mitogenic Activities in Neurofibromatosis
R01 NS 02884009/15/11-07/31/16$231,250

Sampson, L

mTOR Signaling in Murine Intestinal Stem Cell and Progenitor Homeostasis
F32 DK 09787912/01/12-11/30/14$52,605

Starczynowski, D

Defining the Role and Therapeutic Potential of TNF Recep
06/01/13-05/31/16$68,182
Deregulation of TIFAB in Myelodysplastic Syndrome
07/01/11-06/30/14$50,000
Identification and Characterization of Genes in del(5q) Myelodysplastic Syndrome
R01 HL 11110312/15/11-11/30/16$238,000
Regulation and Function of TIFAB in Myelodysplastic Synd
W81XWH111046806/01/11-05/31/14$132,295

Tandon, P

Training Programs in Cancer Therapeutics
T32 CA 11784609/14/12-08/31/14$48,435

Varney, M

Environmental Carcinogenesis and Mutagenesis
T32 ES 00725005/01/12-04/30/14$6,894

Wu, J

STAT3 in Neurofibroma Tumorigenesis and Therapy
W81XWH111025907/01/11-06/30/14$132,862
Exploring the Plexiform Neurofibroma Interactone
05/15/13-05/14/15$292,910

Zheng, Y

Cincinnati Center for Excellence in Molecular Hematology
P30 DK 09097107/01/10-06/30/13$505,315
Zheng, YAdmin Core$91,497
Grabowski, GGenomics and Genetic Core$63,001
Cancelas, JCell Analysis and Sorting Core$65,112
Malik, PTranslational Core$163,824
Mulloy, JXenotransplant and Transgenic Core$68,765
Zheng, YSummer Students$30,370
Pharmacological Rejuvenation of Aged Hematopoietic Stem Cells
R34 AG 04298608/15/12-07/31/13$84,870
Rac GTPase-Specific Small Molecular Inhibitors
R01 CA 14134103/24/09-01/31/14$151,943
Training Program in Pediatric Hematologic and Oncologic Diseases
T32 HL 09180509/01/08-08/31/13$155,456
Digestive Health Center: Bench to Bedside Pilot Study
P30 DK 07839206/01/13-05/31/14$50,000

Zheng, Y / Mulloy, J

Targeting Cdc42 in Leukemia Stem Cells
R01 CA 15054703/10/10-01/31/15$189,199

Zheng,Y/ Geiger, H

Lineage Determination and Tissue HomeOstasis in the Aged Hematopoietic System
R01 AG 04011808/01/11-07/31/16$250,000
Current Year Direct$7,783,205
Industry Contracts

Mulloy, J

$164,254
$8,916
$37,668

Grassman, E

$63,176
Current Year Direct Receipts$274,014
Total$8,057,219

Hematology Grants

Hematology

Grant and Contract AwardsAnnual Direct

Gruppo, R

Hemophilia And Thrombosis Center
06/01/03-05/31/14$110,000
Hemophilia Comprehensive Care
H30 MC 0001510/01/97-05/31/13$22,000
Public Health Surveillance for the Prevention of Complications of Bleeding and Clotting Disorders
U27 DD 00086210/01/97-09/29/13$18,900

Kalfa, T

Rho GTPases in Terminal Erythroid Maturation
R01 HL 11635209/26/12-06/30/16$250,000
TCD with Transfusions Changing to Hydroxyurea (TWiTCH)
R01 HL 09564703/28/11-05/15/13$22,035
TCD with Transfusions Changing to Hydroxyurea (TWiTCH) - Per Patient
R01 HL 09564703/28/11-05/15/13$12,733

Kalinyak, K

Prevention of Stroke after STOP, A Retrospective Chart Review
01/01/13-12/31/13$4,312

Mullins, E

Mechanisms Linking Hemostatic Factors to Neuroinflammatory Disease
K08 HL 10567208/22/11-07/31/16$121,375

Palumbo, J

Hemostatic Factors and Inflammation-Driven Colon
04/01/13-03/31/14$100,000
Targeting the Clotting to Prevent Metastasis
06/01/13-05/31/14$50,000
Digestive Health Center - Pilot and Feasibility Study
U01 DK 06249709/10/09-05/31/14$47,983

Quinn, C

Hydroxyurea to Prevent CNS Complications of Sickle Cell
R34 HL 10875608/01/12-07/31/13$65,000

Shook, L

Cincinnati Sickle Cell Newborn Screening Network
U38 MC 2221806/01/11-05/31/15$232,407
Sickle Cell treatment Demonstration Program
U1EMC075509/01/11-08/31/14$7,400
Cincinnati Sickle Cell Project
07/01/98-06/30/13$123,469
Current Year Direct$1,187,614
Industry Contracts

Gruppo, R

$4,716
$52,527
$8,800
$6,205
$19,283
$4,501
$18,982

Kalfa, T

$2,245

Kalinyak, K

$2,425

Malik, P

$1,080

Palumbo, J

$43,187

Quinn, C

$12,405
$13,264
$1,500
Current Year Direct Receipts$191,120
Total$1,378,734

Oncology Grants

Oncology

Grant and Contract AwardsAnnual Direct

Adams, D

Phase II Study of Rapamycin for Complicated Vascular Anomalies
R01 FD 00371209/22/09-07/31/13$258,707

Chhipa, R

Identifying survival factors for Diffuse Intrinsic Pontine Glioma (DIPG) Stem Cells
06/01/13-05/31/14$25,000

Chlon, T

Pelotonia Fellowship Program
03/01/13-02/28/15$39,264

Chow, L

Micro-RNA Expression in Pediatric High-Grade Glioma
-09/01/11-08/31/13$22,727
Molecular Targeting of High-Grade Astrocytoma
-10/01/11-09/30/15$130,435
Molecular Targeting of Pediatric High-Grade Glioma
07/01/11-06/30/14$110,000

Cripe, T

Neurofibromatosis Preclinical Consortium Center Award
2011-05-00307/01/11-06/30/13$204,545

DasGupta, B

Regulation of Forebrain Neurogenesis by the Energy Sensor AMP Kinase
R01 NS 072591-01A107/01/12-06/30/17$230,208

Dorris, K

Molecular Epidemiology in Children's Environmental Health Training Program
T32 ES 01095710/01/12-06/30/13$40,635

Drissi, R

Telomerase as a Marker and Therapeutic Target for Pediatric High-Grade Glioma
TCCCF01/01/13-12/31/13$60,000

Fouladi, M

Children's Oncology Group Chair
U10 CA 09854304/13/12-02/28/14$11,550
The Pediatric Brain Tumor Consortium
U01 CA 0815504/01/11-03/31/14$44,115
Establishment of an International Diffuse Intrinsic Pontine Glioma (DIPG) Registry
01/01/12-05/31/14$231,092
The Pediatric Brain Tumor Consortium - Per Patient
U01 CA 0815504/01/11-03/31/14$9,259

Geller, J

Epigenetic and Clinical Impact of SMARCB1 Loss in Cancer
R21 CA 16679007/01/2012-06/30/2014$9,273

Moreira Ridsdale,D

The Role of CDC42 in MLL-AF9 Induced AML Xenograft Model
06/01/13-05/31/14$5,000

O'Brien, M

Molecular Targeting of Chemotherapy-Resistant Pediatric Acute Myeloid Leukemia
O'Brien-HOW12/01/12-11/30/13$75,000

Perentesis, J

Children's Oncology Group Chair - Millennium
03/01/12-02/28/17$10,395
Children's Oncology Group Chair - Workload Intensity
U10 CA 09854303/01/12-02/28/17$27,791
Children's Oncology Group Phase I / Pilot Consortium
U01 CA 09745209/01/06-07/31/15$22,525
COG Supplemental Reimbursement
03/01/12-02/18/17$39,193
Pediatric Phase I - Pilot Consortium (per case)
UM1CA09745207/24/12-05/31/15$46,010
Pediatric Phase I Scientific Leadership
UM1CA09745209/26/12-07/31/15$11,501
Children's Oncology Group Phase I / Pilot Consortium - per patient
U01 CA09745209/01/06-07/31/12$125,705

Phillips, C

Genetic Model of Cytarabine Sensitivity in Children with AML
07/01/12-06/30/14$50,000

Privette, L

Cincinnati Interdisciplinary Women's Health Research Career Training Grant
K12 HD 05195307/01/12-06/30/14$79,933
Defining the Role of the DEK Oncogene in Breast Cancer Stem Cell Tumorigenicity Ad Pre-Clinical Testing of Therapeutic DEK Targeting Strategies
RCF04/01/13-03/31/14$40,000

Romick-Rosendale, L

Environmental Carcinogenesis and Mutagenesis
T32 ES 00725009/01/12-06/30/13$41,170

Weiss, B

Phase I Trial of the MEK 1 Inhibitor AZX6244 with Plexiform
02/01/12-01/31/14$37,182
Children's Oncology Group Chair - Workload Intensity
U10 CA 09854303/01/12-02/28/17$11,550

Wells, S

Fanconi Anemia and HPV Transformation
R01 CA 10235709/28/09-08/31/14$191,834
Role and Regulation of the Human DEK Proto-Oncogene
R01 CA 11631609/05/12-06/30/17$174,318
Targeting the Ron-DEK Signaling Axis in Breast Cancer
W81XWH-12-1-019409/01/12-08/31/14$125,000

Williams, J

ETV2 Role in Tumor-Induced Lymphangiogenesis, A Putative Therapeutic Target
07/01/12-06/30/14$69,659
Current Year Direct$2,610,576
Industry Contracts

Fouladi, M

$19,250

Geller, J

$73,615

Wagner, L

$2,887

Weiss, B

$12,890
Current Year Direct Receipts$108,642
Total$2,719,218