Novel Approaches for GVHD on the Horizon Reports Stem Cell Transplant Program Lead

Mar 3, 2020 | Dana-Farber Cancer Institute, Graft-Versus-Host Disease (GvHD), GvHD, Stem Cell Transplantation

Novel Approaches for GVHD on the Horizon Reports Stem Cell Transplant Program Lead

Patients with acute or chronic graft-versus-host disease (GVHD) have several unmet needs, including later-line therapies. Novel agents and other research emerges as well. For the frontline setting, corticosteroids remains the standard of care, firs-line treatment for GVHD. Unfortunately, not all patients respond favorably and some become resistant. Hence, for these patients, physicians may administer JAK1/2 inhibitor ruxolitinib (Jakafi). Upon failure of this treatment, patients begin to run out of options and hence, the world of research becomes imminent. There is no approved “third-line option,” reports the medical director of the Stem Cell Transplant Program with Dana-Farber Cancer Institute.

Graft Versus Host Disease (GVHD)

Characterized by inflammation in various organs, with the specialty of epithelial cell apoptosis and crypt drop out, the GvHD syndrome is commonly associated with stem cell transplants such as those that occur with bone marrow transplants. White blood cells of the donor’s immune system, which remain within the donated tissue (the graft), recognize the recipient (the host) as foreign (non-self). The white blood cells present within the transplanted tissue then attack the recipient’s body’s cells, which leads to GvHD. This is different than a transplant rejection, which occurs when the immune system of the transplant recipient rejects the transplanted tissue—hence, GvHD occurs when the donor’s immune system’s white blood cells reject the recipient. The underlying principle (alloimmunity) is the same, but the details may differ. GvHD can occur after a blood transfusion if the blood products used have not been irradiated or treated with an approved pathogen reduction system.

Corey S. Cutler, MD, MPH, reports that “a number of second-and third-line therapies (for chronic GVHD) that are going to come on over the next few years” with the goal of far more safe and predictable outcomes.

Research Updates

The FDA grated a breakthrough therapy designation to an experimental treatment called KD025 in October 2018 for the treatment of adults with chronic GVHD assuming the failure of lines of systemic therapy, reports OncLive. In the Phase II ROCKstar trial, interim findings reveal that KDO025 generated clinical responses in about two-thirds of patients with chronic GVHD—including three responses. The OncLive presenter, Dr. Cutler, reports that preventative strategies are required along with upfront determination of probable patient response to corticosteroids.

In ROCKstar, a Phase II, randomized, multicenter study, the sponsor and investigators evaluate the efficacy and safety of KD025 in participants with Chronic GVHD after at least two prior lines of systemic therapy. KD025 is being developed by the Kadmon Corporation. Founded in 2009, the biopharmaceutical company, based in New York City, and operations in PA and MA, was launched by Samuel D. Waksal, founder and CEO of ImClone Systems. Eli Lilly acquired the company outright.

The Interview

Follow the link to the OncLive interview to learn more about GVHD research updates from Cutler, medical director of the Stem Cell Transplant Program with Dana-Farber Cancer Institute.

Lead Research/Investigator

Corey S. Cutler, MD, MPH, medical director of the Stem Cell Transplant Program with Dana-Farber Cancer Institute

Source: OncLive

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