$1.5M grant will fund research into TIPS to aid hemophilia A treatment

NHLBI award given to U at Buffalo researcher Sathy Balu-Iyer

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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A researcher at the University at Buffalo has won a three-year grant worth $1.5 million to develop a way to tame a patient’s immune system and prevent it from rejecting factor VIII as a replacement therapy for hemophilia A.

“My interest in hemophilia started when I read a mother’s story about her son who couldn’t tolerate the infusions to treat his hemophilia,” Sathy Balu-Iyer, PhD, a professor of pharmaceutical sciences, said in a university news release.

Hemophilia occurs when the protein that promotes blood clotting is missing or its levels are low. Replacement therapy is a standard treatment for hemophilia that involves providing the missing clotting factor through regular infusions. The missing factor determines the type of hemophilia a person has. In the case of hemophilia A, it’s factor VIII.

Sometimes the immune system sees the clotting factor as a threat and produces a type of antibody, called an inhibitor, against it. These inhibitors can stop the clotting factor from working properly, compromising the replacement therapy’s effectiveness. This is more common in cases of severe hemophilia A.

Balu-Iyer’s team developed a platform called tolerance-inducing phosphatidylserine (TIPS) that can teach a patient’s immune system to accept factor VIII instead of seeing it as a threat.

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“TIPS essentially teaches the immune system to tolerate something it would normally see as a threat,” Balu-Iyer said.

Earlier work was funded in part by the National Institutes of Health’s National Heart, Lung, and Blood Institute (NHLBI), as is the new grant, to advance the effort and apply it in the context of hemophilia A.

TIPS makes use of tiny particles, called nanoparticles, loaded with phosphatidylserine, a fatty molecule that can induce immune tolerance, when the immune system remains unresponsive toward a threat.

The researchers observed that in mice treated with factor VIII replacement therapy the nanoparticles were able to reduce the production of inhibitors against it by cells of the immune system.

The nonprofit Empire Discovery Institute (EDI) also awarded Balu-Iyer’s team a $1 million grant to support research into TIPS for gene therapy applications. The organization eyes programs focused on developing new therapies, invests in them, and then licenses them to pharma companies.

Autoimmune diseases are another class of disorders that may potentially be treated with this immune tolerance approach. According to Balu-Iyer, the next steps in developing TIPS will include researching its ability to treat type 1 diabetes, which occurs when the immune system attacks and destroys insulin-producing cells in the pancreas.