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Bortezomib can treat chronic GVHD, study shows

Skin biopsy showing GVHD

Credit: PLOS ONE

The proteasome inhibitor bortezomib can treat chronic graft-vs-host disease (GVHD), according to research published in Blood.

The study showed that bortezomib provides better outcomes than existing treatments and does not impair the graft-vs-tumor effect.

“Bortezomib helped a group of patients who desperately needed a treatment, having failed multiple different therapies,” said study author Mehrdad Abedi, MD, of the University of California, Davis.

“The drug fights chronic graft-vs-host disease, and, unlike other GVHD therapies such as steroid, cyclosporine, or mycophenolate, it treats chronic GVHD without dampening the graft-vs-tumor effect, which can be critically important to help patients avoid relapse. In fact, because bortezomib is an anticancer drug, it potentially attacks cancer cells in its own right.”

The researchers first studied bortezomib in mice and found the drug suppresses the donor immune cells that cause GVHD.

“We then tested this concept in patients with chronic GVHD . . . ,” Dr Abedi said. “Almost all the patients who tolerated and remained on the treatment responded. In some cases, individual responses were quite dramatic. We were able to stop their other immunosuppressive medications and keep the patients under control with just weekly injections of bortezomib.”

Dr Abedi added that one patient had severe hemolytic anemia that did not respond to several lines of therapy.

“After receiving bortezomib, the patient’s symptoms improved, and we were able to take her completely off steroid and other immunosuppressive medications,” he said. “Another person had multiple ulcers, which completely healed. These were patients who had been on all different kinds of medications and had no response.”

This research is ongoing. Dr Abedi and his colleagues are now looking at a potential oral version of the drug and a similar agent that would alleviate the need for weekly injections and could have fewer side effects.

This research was funded by the National Cancer Institute, the National Institute of Allergy and Infectious Diseases, and Millennium Pharmaceuticals, makers of bortezomib.

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Skin biopsy showing GVHD

Credit: PLOS ONE

The proteasome inhibitor bortezomib can treat chronic graft-vs-host disease (GVHD), according to research published in Blood.

The study showed that bortezomib provides better outcomes than existing treatments and does not impair the graft-vs-tumor effect.

“Bortezomib helped a group of patients who desperately needed a treatment, having failed multiple different therapies,” said study author Mehrdad Abedi, MD, of the University of California, Davis.

“The drug fights chronic graft-vs-host disease, and, unlike other GVHD therapies such as steroid, cyclosporine, or mycophenolate, it treats chronic GVHD without dampening the graft-vs-tumor effect, which can be critically important to help patients avoid relapse. In fact, because bortezomib is an anticancer drug, it potentially attacks cancer cells in its own right.”

The researchers first studied bortezomib in mice and found the drug suppresses the donor immune cells that cause GVHD.

“We then tested this concept in patients with chronic GVHD . . . ,” Dr Abedi said. “Almost all the patients who tolerated and remained on the treatment responded. In some cases, individual responses were quite dramatic. We were able to stop their other immunosuppressive medications and keep the patients under control with just weekly injections of bortezomib.”

Dr Abedi added that one patient had severe hemolytic anemia that did not respond to several lines of therapy.

“After receiving bortezomib, the patient’s symptoms improved, and we were able to take her completely off steroid and other immunosuppressive medications,” he said. “Another person had multiple ulcers, which completely healed. These were patients who had been on all different kinds of medications and had no response.”

This research is ongoing. Dr Abedi and his colleagues are now looking at a potential oral version of the drug and a similar agent that would alleviate the need for weekly injections and could have fewer side effects.

This research was funded by the National Cancer Institute, the National Institute of Allergy and Infectious Diseases, and Millennium Pharmaceuticals, makers of bortezomib.

Skin biopsy showing GVHD

Credit: PLOS ONE

The proteasome inhibitor bortezomib can treat chronic graft-vs-host disease (GVHD), according to research published in Blood.

The study showed that bortezomib provides better outcomes than existing treatments and does not impair the graft-vs-tumor effect.

“Bortezomib helped a group of patients who desperately needed a treatment, having failed multiple different therapies,” said study author Mehrdad Abedi, MD, of the University of California, Davis.

“The drug fights chronic graft-vs-host disease, and, unlike other GVHD therapies such as steroid, cyclosporine, or mycophenolate, it treats chronic GVHD without dampening the graft-vs-tumor effect, which can be critically important to help patients avoid relapse. In fact, because bortezomib is an anticancer drug, it potentially attacks cancer cells in its own right.”

The researchers first studied bortezomib in mice and found the drug suppresses the donor immune cells that cause GVHD.

“We then tested this concept in patients with chronic GVHD . . . ,” Dr Abedi said. “Almost all the patients who tolerated and remained on the treatment responded. In some cases, individual responses were quite dramatic. We were able to stop their other immunosuppressive medications and keep the patients under control with just weekly injections of bortezomib.”

Dr Abedi added that one patient had severe hemolytic anemia that did not respond to several lines of therapy.

“After receiving bortezomib, the patient’s symptoms improved, and we were able to take her completely off steroid and other immunosuppressive medications,” he said. “Another person had multiple ulcers, which completely healed. These were patients who had been on all different kinds of medications and had no response.”

This research is ongoing. Dr Abedi and his colleagues are now looking at a potential oral version of the drug and a similar agent that would alleviate the need for weekly injections and could have fewer side effects.

This research was funded by the National Cancer Institute, the National Institute of Allergy and Infectious Diseases, and Millennium Pharmaceuticals, makers of bortezomib.

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