Kiadis Pharma hosted a May 31 lunch meeting in New York to analyze the risks and benefits of haploidentical hematopoietic stem cell transplants (HSCT) in treating blood cancers.
Two experts, Dr. Stephen Devine of Ohio State University’s Comprehensive Cancer Center and Dr. Denis-Claude Roy, a professor of medicine at the University of Montreal, discussed HSCT’s risks, particularly graft-versus-host disease (GVHD) and cancer relapse, according to a press release. Arthur Lahr, CEO of Kiadis, provided an update on the company’s lead candidate drug, ATIR101 (Allodepleted T-cell ImmunotheRapeutics), currently in a Phase 3 trial for the treatment of acute leukemia.
Physicians generally view HSCT as a potential benefit for patients with blood cancer and inherited blood disorders. However, during HSCT, the patient’s diseased blood and immune system must be destroyed and subsequently replaced by a donor’s healthy system. That makes the treatment very risky, and it may take up to 12 months to recover to a near-normal state.
ATIR101 was developed to provide a single-dose lymphocyte infusion with functional, mature cells from a haploidentical family member – someone who is only a partial match to the recipient, like a family member. That minimizes the risk of causing severe GVHD, which happens when donor immune cells recognize the patient’s healthy cells and tissues as malignant.
Kiadis estimates that 35 percent of patients who are eligible and urgently need an HSCT will not find an adequately matched donor in time. A half-matched parent or child, however, could serve as a donor for nearly all patients, yet would cause severe GVHD due to the infusion of half-matched mature lymphocytes. The ATIR101 therapy being developed by the Dutch company would enable the use of haploidentical transplants without the unacceptable risk of GVHD.
ATIR101 can be given as an adjunctive immunotherapy after a haploidentical HSCT and provides immediate protection against relapse. ATIR101 will also help bridge the time until a patient’s immune system has fully regrown after transplant by fighting infections and remaining tumor cells.
ATIR101 has a pending marketing authorization application to the European Medicines Agency in Europe as an adjunctive treatment in HSCT for malignant disease, as well as an orphan drug designation from both the EMA and the U.S. Food and Drug Administration (FDA).