The U.S. Food and Drug Administration has recently approved Omisirge (omidubicel-onlv), a substantially modified allogeneic cord blood-based cell therapy. This treatment has been designed to help reduce the risk of infection and quicken the recovery of neutrophils, a type of white blood cell, in adults (aged 12 years and older) and pediatric patients with blood cancers. Blood cancers are a type of cancer caused by the uncontrolled growth of cells in the blood, which can be fatal and have serious and damaging effects on the body.Â
Stem cell transplantation is a standard treatment for blood cancers, and healthy stem cells can be sourced from umbilical cord blood. Prior to receiving a stem cell transplant, the patient may undergo radiation or chemotherapy treatments, which can weaken the immune system and increase the risk of severe infections. Omisirge is administered as a single intravenous dose and contains human allogeneic stem cells from umbilical cord blood that are processed and cultured with nicotinamide, a form of vitamin B3.Â
In a multicenter, randomized study involving 125 subjects with confirmed blood cancers, the transplantation of Omisirge was compared to the transplantation of umbilical cord blood. The study’s efficacy was based on the time needed for neutrophil recovery and the incidence of infections following transplantation.
The results showed that 87% of subjects who received Omisirge achieved neutrophil recovery within a median of 12 days, compared to 83% of subjects who received umbilical cord blood transplantation and who achieved neutrophil recovery within a median of 22 days. Additionally, bacterial or fungal infections by 100 days following transplantation were seen in 39% of subjects receiving Omisirge versus 60% of subjects in the control group who received umbilical cord blood.Â
However, it is essential to note that treatment with Omisirge can potentially cause severe side effects. The product label for Omisirge includes a Boxed Warning indicating potential risks for infusion reactions, graft versus host disease, engraftment syndrome, and graft failure. The most frequently observed adverse effects of Omisirge are infections, infusion reactions, and graft-versus-host disease (GvHD).
Patients who receive this therapy need to be closely monitored for the development of engraftment syndrome, graft failure, the possible transmission of rare genetic diseases or severe infections from donor cells, as well as the long-term risk of developing secondary malignancies.Â
In conclusion, the approval of Omisirge by the FDA is a significant milestone in treating blood cancers. This cell therapy has shown promising results in reducing the risk of infections and quickening neutrophil recovery in patients undergoing stem cell transplantation. However, it is important to note that this treatment is not without risk, and patients should be monitored for potential adverse effects. The development of Omisirge represents a significant step forward in the fight against blood cancers, and further research in this area may lead to more effective and safer treatments for patients.Â
