Updates on HoG-Funded Research, Gene Therapy for a Cure

By Jeff Cornett RN MSN, Director of Training, Research, & Advocacy

In July 2012, Hemophilia of Georgia began funding research at St. Jude Children’s Research Hospital in Memphis, Tennessee.  This research seeks to find a cure for hemophilia that is, in the words of the researchers, “safe, effective, and widely available.” You can read about the basis for the research in this newsletter article from December 2012: https://www.hog.org/publications/detail/update-on-clinical-trials-to-cure-hemophilia.

While the seven patients treated with gene therapy had an increase in their factor level, some of them also had temporary inflammation of the liver.  In an effort to make the gene therapy potentially safer and more effective, the researchers worked on improving the gene-carrying virus that is used.  The improved “viral vector” passed all safety testing except one: it failed toxicity testing in mice at an independent lab.  The researchers did additional experiments and determined that the toxicity was not due to the virus but to the very high levels of human factor IX that were produced in the mice.  Since levels that high will not be generated in human patients, the researchers feel that the viral vector is safe for clinical trials.  In late 2013, they received approval to re-open the clinical trials to patients.  The first patient will receive the improved gene therapy treatment at St. Jude in January 2014.  He will be monitored for several months and, if all goes well, the next patient will be treated.  Watch your HoG newsletter for updates on these treatments.

Hemophilia Clinical Scientist Development Grants

Since 2003, Hemophilia of Georgia has funded bleeding disorder physicians who are the early stages of their research careers.  The purpose of the five-year grants is to allow them to advance to the status of an independent investigator. Through this program, Hemophilia of Georgia hopes to help create the next generation of hemophilia researchers. Seven physician researchers have completed the grant funding cycle.  Two more are currently being funded and more are expected to be added this year.

By all measures, the program has been a success.  These bleeding disorder researchers are recognized as some of the brightest young minds in the field.  They have continued their roles as both researchers and clinicians in Hemophilia Treatment Centers.  Two are now the directors at their HTCs.  One is the Regional Medical Director for hemophilia.  All have kept bleeding disorders as the primary focus of their research and patient care.  A full listing of physician researchers who have completed the grant program is available on the HoG website: https://www.hog.org/programs/page/hemophilia-clinical-scientist-development-grantees.