Doctors at Nationwide Children’s Hospital in Columbus, Ohio have conducted research that leads them to believe that a cure for MPS II has been discovered. Dr. Fu and Dr. McCarty have conducted years of research and are relying on parent-led organizations to raise the funds needed to carry the drug development process through clinical trials. Human clinical trials involve administering the drug in a controlled environment to gather data on efficacy. This is usually done before making the drug available to all affected patients.
These funds will cover expenses such as securing the facility where the clinical trials will be held, along with building of vectors that will be used to introduce the cure to our boys. The remaining balance of the $2.5 million dollars will be used to cover medical costs, room and boarding costs, and miscellaneous costs for the patients who will be participating in the clinical trial. If all deadlines are met, Dr. Fu and Dr. McCarty hope to start clinical trials by end of Summer 2018.
The Research Institute at Nationwide Children’s Hospital
700 Childrens Drive
Columbus, OH 43205
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Concept Phase
The first step in standard project management models is the concept phase, in which a project team is appointed to design the new device in accordance with design control requirements. The design control steps are to create the project control plan and to obtain design input. Project team members meet to identify the design requirements of the product, including requirements for product configuration, formulation, processing and manufacture, packaging, labeling, performance, storage, handling, distribution, regulatory status, bench and animal testing, and human clinical trials. This process is called design input. The team also develops a project control plan describing in general terms who will do what by when. The concept phase of product development culminates when a general plan for building, testing, and manufacturing the product is completed.
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Prototype Phase
$200,000During the prototype phase—called the feasibility phase by some companies— various departments will begin evaluating the feasibility of the concept. The manufacturing group will look at production issues, whereas the marketing department may conduct focus panels to evaluate design concepts. If test outcomes are unsuccessful, the prototype is redesigned. If test outcomes are successful, the product design is frozen, signifying the culmination of the prototype phase. Hereafter, all design changes are captured through design change control.
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Development Phase
$500,000In the prepilot phase—sometimes called the development phase—the manufacturing department works to develop the production process, while other departments develop the processes necessary to carry out functions such as packaging, distribution, or marketing. In design control language, this is the phase of design verification—the period of testing and proving the design features of the device. The design is changed if study outcomes are unfavorable, and design changes are captured through the design change control system. The prepilot phase culminates when the manufacturing process has been finalized.
Postmarket surveillance studies and marketing studies are typically performed during the production phase. Postmarket surveillance studies are usually required by FDA in exchange for early introduction of a device. The studies are designed to assess the incidence of patient complications.
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Pilot Phase
$500,000Pilot plants are small manufacturing sites in which processes or techniques planned for full-scale production are tested in advance. Scale-up refers to the activity of moving from limited production to full production, and typically takes place during the pilot phase of product development. The pilot or scale-up phase is that in which design and process verification are completed and transferred into device specifications. The pilot phase culminates when the manufacturing process is confirmed and transfer to production facilities begins.
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Commercialization Phase
Postmarket surveillance studies and marketing studies are typically performed during the production phase. Postmarket surveillance studies are usually required by FDA in exchange for early introduction of a device. The studies are designed to assess the incidence of patient complications.
During the production phase the clinical research group also handles medical complaints, supports regulatory submissions, prepares publications and presentations, and assists with customer preference evaluations.
Meet the Doctors working to save the lives of those affected by Hunter Syndrome (MPS II)
Douglas McCarty, PhD
Principal Investigator, Center for Gene Therapy / Associate Professor, Department of Pediatrics
The Research Institute at Nationwide Children’s Hospital / The Ohio State University College of Medicine
Doulas McCarty, PhD, is a researcher in the Center for Gene Therapy at the Research Institute at Nationwide Children’s Hospital, and associate professor in the Dept of Pediatrics at The Ohio State University in Columbus. After graduating from West Virginia University, he spent several years in the coal industry before returning to academics and earning a PhD from University of Florida in Immunology and Medical Microbiology. Dr McCarty continued his research at SUNY, Stony Brook, working with adeno-associated virus (AAV) which is now a leading vector for use in gene therapy applications. He continued with the development of AAV vectors as Director of the Vector Core Facility at University of North Carolina. Together with his wife and long-time collaborator, Dr Haiyan Fu, Dr McCarty moved to The Research Institute at Nationwide Children’s Hospital to take advantage of the strong clinical and translational gene therapy program, and to advance their research in gene therapy for Sanfilippo syndrome to clinical trials. In addition to his gene therapy projects, Dr McCarty continues to pursue basic science in DNA recombination factors that determine the fate of the AAV genome after it gets into its target cell. The Sanfilippo gene therapy project has led to IND (investigational new drug) approvals for clinical trials for MPS IIIA and MPS IIIB, and a licensing agreement with Abeona Therapeutics for commercial development.
Haiyan Fu, Phd
Principal Investigator, Center for Gene Therapy
The Research Institute at Nationwide Children’s Hospital
Haiyan Fu, PhD, is a principal investigator in the Center for Gene Therapy at the Research Institute at Nationwide Children’s Hospital. Dr. Fu’s NIH and privately funded research program focuses on gene therapy, mechanisms of neuropathology and biomarker development targeting MPS III/lysosomal storage diseases and neurodegenerative diseases.