Palak Patel

Palak Patel
Mentor: Dr. Peter Stacpoole
College of Medicine
 
"Our world advances, mobilizes, and changes because of research. I knew I needed to be a part of that. UF offered me an opportunity to join in on research that prolongs the lifespan of children with a rare disease. This is the most meaningful thing I could do with my time, since it not only benefits these patients, it benefits me. I have been educated with advanced knowledge and enhanced with an appreciation of those who dedicate their lives in service to others."

Major

Microbiology & Cell Science

Minor

Theatre, Chemistry

Research Interests

  • Rare Disease
  • Pediatrics
  • Oncology

Academic Awards

  • Treasure Coast Gator Club Scholarship
  • Life Happens Scholarship
  • Bright Futures
  • University Scholars Program 2016

Organizations

  • Honors Ambassadors
  • Gators of Tomorrow
  • She's the First

Volunteer

  • Mobile Outreach Clinic
  • Baby Gator
  • Relay for Life

Hobbies and Interests

  • Tennis
  • Piano
  • Traveling
  • Poetry

Research Description

Phase 3 Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency
The objective of this project is to conduct a pivotal phase 3 trial of treatment leading to the FDA approval of the investigational drug dichloroacetate (DCA) in young children with deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency (PDCD) is the most common cause of congenital lactic acidosis and is a frequently fatal metabolic disease of childhood for which no proven treatment exists. It can cause neurological symptoms such as developmental delay, growth retardation, poor acquisition or loss of motor milestones, hypotonia, seizures, ataxia and dystonia. We predict that DCA represents targeted potential therapy for PDCD because of its ability to increase both the catalytic activity and stability of the enzyme complex. I will assist in a randomized, placebo-controlled, double-blind trial of 24 evaluable children, aged 1 month through 17 years, with proven deficiency of the PDC. The study will begin with a 1-month lead-in period for baseline clinical data collection and subjects will then be entered in a crossover design in which each subject receives DCA or placebo by mouth or feeding tube for 4 months. This period will be followed by a 1-month washout period, during which all subjects will receive placebo, followed by crossover to the alternate treatment for 4 months. Following the conclusion of this crossover period, the patient will be offered the opportunity to receive open label DCA at the same dose he or she was receiving during the blinded portion of the trial, in order to monitor chronic safety of the drug. Patients will be stratified according to their predicted rate of DCA metabolism and clearance, based on genotyping prior to randomization.