Hematology and Oncology

Stem cell therapy for Sanfilippo Syndrome

Faculty Mentor’s Name: Dr. Coy Heldermon
Email: coy.heldermon@medicine.ufl.edu
Phone Number: (352) 273-7497
Project Category: Translational
International Component or Travel: No

Research Project Description:
Stem cell sources will be transduced with a gene therapy vector and injected into mice with the disease. Effects on behavior, biochemistry and histology will be compared.

Is Hypomagnesemia Associated With Pertuzumab?

Faculty Mentor’s Name: Dr. Karen Daily
Email: karen.daily@medicine.ufl.edu
Phone Number: (352) 222-1423
Project Category: Clinical
International Component or Travel: No

Research Project Description:
As a breast medical oncologist, I – and others in the field – have observed in clinical practice hypomagnesemia with Pertuzumab, which is a HER2-targeted intravenous medication used in the treatment of HER2+ breast cancer. There is no reporting in the published trials of Pertuzumab regarding magnesium nor does the manufacturer Genentech have data on this topic; therefore, there is no information on the package insert about this or recommendations on checking magnesium levels in patients receiving Pertuzumab. Magnesium is not included on standard Complete Metabolic Panels. Hypomagnesemia is a known side effect of treatment with other EGFR-directed antibodies, specifically Cetuximab and Panitumumab. I have obtained IRB approval to prospectively check magnesium at each infusion of Pertuzumab for patients newly receiving this medication as part of their standard of care treatment. The primary purpose is to determine the frequency, severity, and timeline of hypomagnesemia associated with Pertuzumab if it exists. An informed consent form has been designed and IRB approved and a first patient enrolled. I sought statistical input for study design and sample size calculation. RedCap database will be used. This is an unfunded project. The primary endpoint of this study is the proportion of hypomagnesemia at 3 months Subsequent to this project, if significant hypomagnesemia is appreciated, this work could lead to a recommendation to routinely check magnesium for patients receiving Pertuzumab so hypomagnesemia can be identified and corrected with oral and/or IV supplementation. There was an ASCO abstract in 2015 from Memorial Sloan Kettering looking retrospectively at their experience, and I had an ASCO abstract in 2018 on the same. I reached out to the MSK team, and they haven’t followed up on this work.



The role of the medical student would be (1) data collection from EPIC on enrolled patients and entry of that data into RedCap, (2) meeting with myself and UFHCC statistician for data review and interpretation, and (3) writing an abstract and manuscript from the data. The majority of this work can be done remotely by computer, telephone, Zoom. This is my second time participating as a faculty mentor in MSRP (summer 2020 Luderve Rosier).