The US FDA has approved omidubicel, a new cell therapy to reduce the risk of infections in patients with blood cancers undergoing stem cell transplant. Omidubicel is a modified allogeneic (donor) cord blood-based cell therapy, which has been designed to hasten neutrophil recovery. The human allogeneic stem cells from umbilical cord blood are processed and cultured with nicotinamide, a type of vitamin B3.

The approval for omidubicel was based on the results of a randomized multicenter trial of 125 patients, aged 12-65 years, with hematologic malignancies like acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma (NCT02730299). Neutrophil recovery was evident in 87% of omidubicel recipients within a median of12 days after transplant compared to 83% of those assigned to umbilical cord blood transplantation, who attained neutrophil recovery within a median of 22 days post-transplant. After 100 days of the transplant, bacterial or fungal infections were seen in 39% of patients in Omisirge group vs 60% of subjects in umbilical cord blood transplantation group

• Indication:Patients aged ≥12 years with blood cancers planned for umbilical cord blood transplantation following a myeloablative conditioning regimen such as chemo- or radiotherapy

• Dose:Single dose administered intravenously; each dose is patient-specific, containing healthy stem cells from an allogeneic pre-screened donor.

• Adverse effects:Infections, graft versus host disease (GvHD, infusion reactions.

• Warnings and precautions

o   Monitor patients for infusion reactions, GvHD, engraftment syndrome, graft failure.

o   Patients should also be watched for secondary malignancies.

o   There is a potential risk of transmission of serious infections or rare genetic diseases from the donor cells.

o   Omisirge carries a Boxed Warning for infusion reactions, graft versus host disease, engraftment syndrome and graft failure

Sources

1. https://www.fda.gov/news-events/press-announcements/fda-approves-cell-therapy-patients-blood-cancers-reduce-risk-infection-following-stem-cell. April 17, 2023. Accessed on April 18, 2023.
2. https://www.medpagetoday.com/transplantation/transplantation/104063, April 17, 2023. Accessed on April 18, 2023.