Hooray for the Home Team

A group of researchers at The University of Texas Health Science Center at Houston have developed a modified Factor VIII protein that will help treat inhibitor patients.

From the press release:

With a shortage of the blood-clotting protein Factor VIII (FVIII), people with Hemophilia A typically receive injections of FVIII derived from plasma or produced synthetically to control potentially life-threatening episodes of bleeding. Unfortunately as many as 1 in 3 people with Hemophilia A produce inhibitor antibodies, which attack the externally-administered FVIII and negate its blood-clotting benefits.

To combat this problem, scientists in the laboratory of Sudhir Paul, Ph.D., at The University of Texas Medical School at Houston, developed a chemically modified version of FVIII which during laboratory tests neutralized these inhibitor antibodies, thus paving the way for the correction of the blood-coagulating process. The modification is called electrophilic FVIII analog (E-FVIII).

“It’s a two-step process,’’ said Paul, the senior author. “The E-FVIII permanently inactivates the antibodies that inhibit blood clotting in 20 to 30 percent of patients receiving Factor VIII replacement therapy. Once the antibodies are cleared, additional FVIII can be injected.” The study involved blood donated by eight people with FVIII-resistant Hemophilia A.

Miguel Escobar, M.D., of the department of pediatrics and internal medicine at the UT Medical School at Houston and the Gulf States Hemophilia and Thrombophilia Center, led the clinical aspects of the work.(emphasis mine)

This is great news from just around the corner. Congratulations to Dr. Escobar and the rest of our HTC team. We are so very fortunate that Houston has one of the premier Medical Centers in the nation, if not the world. We are doubly blessed for Jack to be under the care of Dr. Keith Hoots and the whole Gulf States HTC staff (the finest in the nation, and yes we’re biased).