APPLICANT’S BIOGRAPHICAL SKETCH

OMB No. 0925-0001 and 0925-0002 (Rev. 10/15 Approved Through 10/31/2018)

BIOGRAPHICAL SKETCH

Provide the following information for the Senior/key personnel and other significant contributors.

Follow this format for each person. DO NOT EXCEED FIVE PAGES.

NAME: Huynh Cao

eRA COMMONS USER NAME (credential, e.g., agency login): hcao01

POSITION TITLE: Assistant Professor

EDUCATION/TRAINING (Begin with baccalaureate or other initial professional education, such as

FIELD OF STUDY

A. Personal Statement

B. Positions and Honors

Positions

2014-2017 Hematology & Oncology Fellow, Loma Linda University Medical Center, Loma Linda, CA

2017-current Assistant Professor at Loma Linda University Medical Center, Loma Linda, CA

Honors and Awards

1999

Other Experience and Professional Memberships

2011-current Member, American College of Physicians

Induction chemotherapy with cytotoxic agents remains the initial standard of care for acute myeloid

leukemia (AML). The current treatment strategy is exceedingly toxic for older adult patients who

frequently present with multiple co-morbidities. Active vitamin D3 (1,25-(OH)2-D3) has shown promising

preclinical success in vitro. The concentrations of active VD3 required to effectively induce differentiation

(70-80% of blast cells) in vitro are typically in the range of 100-1000 nanomolar (nM); however, a serum

level of such concentration would certainly result in hypercalcemia in humans (the normal concentration

of active VD3 is around 0.1 nM in humans). To overcome this limitation, we used genetically engineered

MOLM-14 AML cells (designated as CYP27B1-GFP-MOLM14) as vehicle cells to deliver the VD3

activating enzyme (CYP27B1, 1-a-hydroxylase-25-OH-VD3) to the bone marrow where endogenous

inactive VD3 will be converted to active VD3. We used this gene therapy in combination with low dose 5-

Hematol 50: 1-12 (2017).

Cao H, Xu Y, Payne KJ, Necochea-Campion R, Chen CS, Baylink D, Tang X, Marcucci G. CYP27B1

gene therapy for acute myeloid leukemia. Presented abstract at the

Development Retreat 2018.

Manuscript in preparation. To be submitted to Leukemia. November 2019.

2. Vitamin D’s biological functions in immune regulation and tissue regeneration, and their

potentials in the prevention of malignant formation of cancer stem cells.

calcitriol specifically into the immune system like bone marrow, the gut, and the brain. To fulfill this goal, I

have been working on the projects of tissue-specific targeting and calcitriol’s role in Regulatory T cell’s

generation and transgene cell therapy for the AML, experimental colitis [an animal disease that is a well-

established model for human inflammatory bowel disease (IBD)] for the past two years. I am currently

writing two papers about the novel therapies for IBD. The goal is to further our understanding of

clinical applications of our innovative strategies.

Tang X. In vivo

3. Biological effects of anti-granulocyte-macrophage colony-stimulating factor (GM-CSF)

antibody formation. GM-CSF is a hematopoietic growth factor, which stimulates proliferation and

differentiation of hematopoietic progenitor cells. In a single arm study, we investigated the development

of binding and neutralizing antibodies to GM-CSF in patients receiving prolonged therapy with GM-CSF

as adjuvant therapy for melanoma. We found that 93% of 43 patients developed binding antibodies and

42% developed both binding and neutralizing antibodies. In those with neutralizing antibodies, we

observed a diminution of the biological effects of GM-CSF (increase in white blood cell count, percent

eosinophils, or neopterin levels). We concluded that the development of neutralizing antibodies to GM-

CSF might abrogate the potential benefit of this adjuvant treatment for melanoma.

enzyme-linked immunosorbent assay (ELISA) by using a combination of monoclonal antibodies against 2

different leukocyte antigens. Since CD81 is one of established surface markers of exosomes, CD117

CD81

double positive ELISA signals indicate the presence of CD117

exosomes and its quantity in the

plasma. Since plasma does not contain any cells, such double positive signals are considered to be

derived from exosomes. We used this assay to test plasma samples of 6 AML patients before and after

chemotherapy induction. The exosomes quantities correlated with the disease status in the bone

contact person between the Northern California Melanoma Center and the UCSF Melanoma Center. At