Faculty Contacts

	Randy Brown, MD (352)273-5100
	Christopher R. Cogle, MD (352) 273-7493
	Nam Dang, MD, PhD (352) 273-9168
	John Hiemenz, MD (352) 273-9168
	Jack Hsu, MD (352) 273-7823
	James Lynch, MD (352) 273-9168
	Merry-Jennifer Markham, MD (352) 273-8699
	Stratford May, MD, PhD (352) 273-7760
	Jan Moreb, MD (352) 273-7823
	Maxim Norkin, MD, PhD (352) 273-5100
	John Wingard, MD (352) 273-7760
	Mona Wirk (352) 273-5100

Useful Links

	UF Department of Medicine Division of Hematology and Oncology
	Shands at UF
	UF Shands Cancer Center
	UF&Shands Clinical Trials

Hematologic Malignancies Update: Vol. 1, Issue 2

This newsletter reports on new therapies for acute myeloid leukemia (AML), the myelodysplastic syndromes (MDS) and myelofibrosis at the University of Florida Shands Cancer Center. If you would like additional information about any of these trials, please contact the trial's principal investigator.

If you would like to refer a patient with a hematologic malignancy or bone marrow failure syndrome, contact the UF&Shands Hematologic Malignancies and BMT Program patient coordinator, Atha Ellerker, at (352) 265-0111, ext. 2-9452.

In This Issue:

	Phase I Study of Oral Vidaza in Newly Diagnosed LOW & INT-1 MDS
	Phase III Study of Pomalidomide for Transfusion Dependent Myelofibrosis
	Phase III Study of Standard Induction Chemotherapy +/- Oral Midostaurin (FLT3 Inhibitor) For Newly Diagnosed AML
	Phase I Study of OXi4503 in Relapsed and Refractory AML and MDS
	A Phase III Study of Elacytarabine in Relapsed and Refractory AML
	Transplant For MDS Patients on Medicare

Phase I Study of Oral Vidaza in Newly Diagnosed LOW & INT-1 MDS

Azacitidine (Vidaza) offers improved outcomes in patients with MDS. Research indicates that exposure to prolonged and low doses of Vidaza may result in better responses. Moreover, an oral formulation would maximize convenience to the patient. Thus, oral Vidaza is being tested in a 14-day schedule. Data from the trial show safety, feasibility and early evidence of improved responses (JCO 2011). Eligible patients include those with transfusion dependent, Low and Intermediate-1 IPSS Risk MDS (ClinicalTrials.Gov Identifier NCT00528983). Study PI: Chris Cogle, M.D.

Phase III Study of Pomalidomide for Transfusion Dependent Myelofibrosis

Patients with myelofibrosis often have multiple sequela of disease and can develop anemia, which is difficult to treat with conventional oral chemotherapies like hydroxyurea. In early phase clinical trials, immunomodulatory drugs (IMiDs) such as pomalidomide have produced hematologic improvements and transfusion independence. Therefore, UF investigators are conducting a phase III clinical study of oral pomalidomide for patients with transfusion dependent myelofibrosis (ClinicalTrials.Gov Identifier NCT01178281). Study PI: Randy Brown, M.D.

Phase III Study of Standard Induction Chemotherapy +/- Oral Midostaurin (FLT3 Inhibitor) for Newly Diagnosed AML

The presence of a FLT3 mutation in AML patients is a poor prognostic factor with significantly increased relapse rates and diseased overall survival. In early phase clinical trials, the oral FLT3 inhibitor midostaurin (PKC 412) improved outcomes in patients with FLT3 mutant AML receiving standard induction chemotherapy. Therefore, a phase III study of standard induction chemotherapy (7+3) +/- midostaurin is being conducted in newly diagnosed AML patients (ClinicalTrials.Gov Identifier NCT00651261). Study PI: Jack Hsu, M.D.

Phase I Study of OXi4503 in Relapsed and Refractory AML and MDS

For patients with relapsed and refractory AML and MDS, new strategies are needed for re-induction. Researchers at the University of Florida Shands Cancer Center have recently discovered that the naturally occurring combretastatin, OXi4503, eliminates AML (Blood 2010). The investigators are now conducting an ongoing phase I clinical trial of OXi4503 in patients with relapsed and refractory AML and MDS (ClinicalTrials.Gov Identifier NCT01085656). Study PI: Chris Cogle, M.D.

Phase III Study of Elacytarabine in Relapsed and Refractory AML

For patients with relapsed AML, new strategies are needed for re-induction. One mechanism of leukemia resistance is lack of transport proteins to carry cytotoxic drugs across the leukemia cell membrane and into the cytoplasm. Thus a lipid formulation of cytarabine has been developed to bypass the need for active transport molecules. Instead, this lipid-conjugated agent passively flows into the leukemia cell and greater intracellular concentrations can be achieved. In early phase clinical trials, survival times were three times longer in patients receiving elacytarabine compared with historical controls. A phase III trial is now being conducted at the University of Florida and Shands Hospital to compare elacytarabine against conventional care regimens in patients with relapsed and refractory AML (ClinicalTrials.Gov Identifier NCT01147939). Study PI: Jack Hsu, M.D.

Transplant For MDS Patients on Medicare

Allogeneic hematopoietic cell transplant is the only curative treatment option for patients with MDS; however, the procedure can be risky and traditionally Medicare has not covered transplant for MDS patients. In response to new data that shows improved transplant outcomes in older individuals, the Centers for Medicare & Medicaid Services (CMS) decided to cover transplant for patients with MDS only for beneficiaries who are enrolled in an approved clinical study. An approved study is now open at the University of Florida and Shands Cancer Hospital. Eligibility includes Medicare beneficiaries of any age and with a diagnosis of MDS or CMML. Study PI: John Wingard, M.D.

Contact Info:

To refer a patient, please contact Atha Ellerker at (352) 265-0111, extension 2-9452, or ELLERA@shands.ufl.edu