Research

Dr. David Wilcox

Dr. David Wilcox is an Associate Professor of Pediatric Hematology, Oncology and Bone Marrow Transplant at the Medical College of Wisconsin, Adjunct Investigator in the Blood Research Institute of the Versiti Blood Center of Wisconsin, and Investigator, Children’s Research Institute at the Children’s Hospital of Wisconsin. He is also President of Platelet Targeted Therapeutics, a company dedicated to developing and bringing unique platelet-based therapies to patients. For the last two decades, Dr. Wilcox and his team have focused their efforts to understand platelets and the function of platelets in treatment and possible cure of serious diseases. These efforts have led to significant advances in our current understanding of platelets and of specific proteins and their role in addressing several genetic disorders. Additionally, Dr. Wilcox has been investigating the potential for utilizing the unique properties of platelets. As the recipient of numerous grants and awards, both public and private, these efforts have led to three platforms for megakaryocyte-specific gene therapy, addressing diseases including Glanzmann’s thrombasthenia and Hemophilia A, as well as several types of cancer. With the ever-expanding understanding of the complex function of platelets, Dr. Wilcox has specifically focused on developing novel strategies to genetically modify bone marrow to permit platelets to manufacture, store, and deliver therapeutic proteins at the site of vascular injuries to permanently cure rare and common inherited bleeding disorders. Thus, Dr. Wilcox and his team remain committed to exploring new directions and opportunities, by continuing to translate their basic research studies into first-in-human clinical gene therapy trials with an emphasis on maximizing benefit vs. risk to the patients.

research

From Dr. Wilcox

“My project is novel to gene therapy because it is specifically concerned with replacing the affected protein in Glanzmann’s thrombasthenia by targeting synthesis of a normal protein to platelets, which is the cell-type most affected by GT.

Prior to last year, two patients with GT donated bone marrow derived cells for our studies and we were able to demonstrate correction of their cells grown in the laboratory following treatment of the cells with our gene therapy protocol.

Contributions to the Glanzmann’s Research Foundation have greatly furthered this effort by providing support for research supplies, housing and caring for animals.

“I hope that you continue to share in my enthusiasm for this work and thank you once again for your support.”

Because of the efforts of the GRF, we have restored platelet function in mice affected with GT now at 6 months past transplantation of gene therapy corrected cells into the animals. This is a very encouraging result and was accepted for presentation at the annual meeting of the American Society of Hematology in Philadelphia in December of 2002.

Following further tests, we will present this data for formal review by my peers in the scientific community and publication in a scientific journal. While results in the mice are very exciting, there are several steps that still need to be taken before proposing to correct GT in humans.

Beginning this year we will try to correct larger animals that have been found to have GT. This is a very challenging goal but also very necessary to ensure safety and clinical efficacy of our gene therapy protocol. I hope that you continue to share in my enthusiasm for this work and thank you once again for your support.”

UPDATE 2020

"All the funding (received from GRF) from 2001 onward was used to support research to test if gene therapy could correct human cells, mice and dogs affected with GT. The GRF was and is acknowledged for financial support in all publications. The current state of our research is: We have developed a potential cure for GT that works in mice, dogs, and cells from human patients affected with GT. The current goal is to test stem cell gene therapy in humans with a phase I first-in-human clinical trial to see if it is safe and effective."