Bone marrow treatment used to combat inhibitors

According to a Children's Hospital of Wisconsin press release researchers there have shown success using patients' bone marrow to treat inhibitors.

The findings were published in the 1 October 2008 issue of Blood Journal.

From the press release:

"...investigators describe how a gene-modified bone marrow transplant can be used to initiate clotting in hemophilia. This type of approach may work in the 30 to 35 percent of hemophilia patients that have developed inhibitory antibodies against the missing clotting protein.

The bone marrow is removed from the patient and stem cells are treated with Factor VIII, a clotting factor, which is placed in the platelets. The marrow is given back to the patient, who then retains the essential clotting mechanisms to stop bleeding that otherwise would lead to complications."

Inhibitors help combat AIDS?

The lead from the article:

Antibodies found in lupus and some hemophilia patients may prove to be a potent preventative against HIV transmission and progression, according to researchers who presented two new studies yesterday at the 17th International AIDS Conference in Mexico City.

Antibodies in some hemophilia patients? Could these be inhibitors? If not what are they? What else could be unique to hemophilia patients?

Link to the article.

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).