Breakthrough research could improve quality of life for hemophiliacs

Engineers in the Cockrell School of Engineering at The University of Texas at Austin have developed the first-ever capsule to treat hemophilia (Courtesy/Cockrell School of Engineering)
Engineers in the Cockrell School of Engineering at The University of Texas at Austin have developed the first-ever capsule to treat hemophilia (Courtesy/Cockrell School of Engineering)

AUSTIN (KXAN) — While hemophilia can be managed, those who live with it must look at the world differently. A simple bump, bruise or cut could become life-threatening.

The blood disorder is passed down from parents and about 1 in 5,000 children have it. Treatment to protect them comes in the form of injections and infusions multiple times per week, which can be especially tough on young ones.

“It was tragic, it was scary,” remembers Shelley Clawson, whose son was diagnosed at 7 days old. “And then once the surgeon was able to get the bleeding to stop and once we knew what we were dealing with, it was a shock of how different our lives were going to be.”

(Courtesy/Lone Star Chapter of the National Hemophilia Foundation)
(Courtesy/Lone Star Chapter of the National Hemophilia Foundation)

Her son Jace got an infusion port at 10 months old, so Shelley could give him treatments. Early on, the infusions were especially difficult, and she needed someone else to be there to help hold and comfort him during the process. Jace only gets infusions three days a week to help ensure his body won’t reject the medicine.

Researchers have been working to improve the treatment method for years, and recently made a breakthrough. Engineers in the Cockrell School of Engineering at The University of Texas at Austin have developed the first-ever capsule to treat the disease, a less painful and less expensive treatment option.

“My most pressing concern was the treatment of younger patients who suffer from hemophilia and who have to apply injections every two days,” said Nicholas A. Peppas, the director of UT Austin’s Institute of Biomaterials, Drug Delivery and Regenerative Medicine.

While putting the medicine in a capsule may seem like an easy solution, it’s extremely complicated to make it work successfully.

Dr. Linda Govea Shaffer helps explain why. “These molecules are fairly large and when they get into the stomach they get digested, and then by the time they get into the bloodstream they are no longer active,” said Dr. Shaffer, a Pediatric Hematologist at Dell Children’s Medical Center.

UT researchers have successfully developed a system to orally deliver the medicine for hemophilia B patients, human factor IX (hFIX). It’s designed to capitalize on the body’s pH and changes the enzymes inside the gastrointestinal tract for smooth delivery.

(Courtesy/Cockrell School of Engineering)
(Courtesy/Cockrell School of Engineering)

 

“Based on the current capabilities of this system, approximately two capsules would be equivalent to one injection,” said Sarena Horava, the study’s lead author who is a recent PhD graduate from the Cockrell School’s McKetta Department of Chemical Engineering. “However, we anticipate that we will make further improvements to the delivery capacity of the oral delivery system and therefore decrease the capsule amount.”

For families like the Clawsons, it could be a game-changer. “To take a pill and know you can be treated the same way would be a miracle,” said Shelley.

The capsule treatment could also play a big role in delivering better care to those in developing countries. Current treatment is limited by cost and the need for trained medical personnel, as well as possible complications with the needle-based drug administration. In many of these countries, the median life expectancy for hemophilia patients is 11 years because of the lack of access to treatment.

Clawson said throughout this journey, having a support system has been huge. Through the Lone Star Chapter of the National Hemophilia Foundation, she’s found other families in the region dealing with the same worries and emotions.

This September, she held a fundraiser at Board and Brush in Cedar Park, raising money to help find better treatments and cures for bleeding and clotting disorders.

Researchers say it’s too early to know when the capsule will be on the market, but they’re making every effort to accelerate their studies. They must first complete animal and clinical studies, and then get FDA approval.

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