Chronic graft versus host disease (cGvHD) is a complication that can occur in about 50% of patients following allogeneic stem cell transplant. Most cases are mild to moderate, but about 10-15 % of patients develop symptoms that are more severe with significant morbidity and mortality.
“In cGvHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are living with cGvHD.”
Patients are typically systemically treated for this debilitating disease with corticosteroids and calcineurin inhibitors (such as cyclosporin and tacrolimus).
The news that the US Food and Drug Administration approved the compound belumosudil (future brand name: Rezurock) on July 16, 2021 and designated the drug as a Breakthrough Therapy, will therefore be welcomed by those patients with chronic GvHD. The drug is approved for “the treatment of adult and pediatric patients 12 years and older with cGvHD after failure of at least two prior lines of systemic therapy.” The compound is an oral drug that is the first in a new class of compounds targeting Rho-associated coiled-coil kinase 2 (ROCK2), a pathway that modulates the inflammatory response.
The drug’s developer, Kadmon, completed 2 Phase II clinical studies of about 65 patients each that were dosed either with 200 mg once a day or 200 mg taken twice a day.
The median time from cGvHD diagnosis was 25.3 months and 48% of patients had four or more organs involved. Patients had cycled through a median of 3 prior lines of systemic therapy and 78% were refractory to their last therapy.
In other words, this patient population was in serious need of a treatment alternative.
about the author
I was diagnosed in 2014 with blood cancer and have been very fortunate to find treatment at the division of Cellular Therapy at the Duke University Cancer Institute. My wife, Vicki, and I have two adult children and a grandson who is the ‘light of my life’. Successful treatment has allowed Vicki and I to do what we love best: traveling the world, albeit it with some extra precautions to keep infections away. My career in the pharmaceutical industry has given me insights that I am currently putting to use as an advocate to lower drug pricing, especially prices for anti-cancer drugs and, very specifically, CAR-T therapies, with recent contributions posted by Health Affairs, the Institute for Clinical and Economic Review and the Centers for Medicare and Medicaid Services.