A New Hope for Patients Lacking Suitable Stem Cell Donors
Introduction
Blood cancers such as leukemia, lymphoma, myelodysplastic syndromes, and multiple myeloma continue to challenge physicians worldwide. In many cases, allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative treatment option. However, for patients who do not have a matched donor, access to life-saving therapy is extremely limited.
In 2025, a new breakthrough therapy named Zemcelpro has emerged as a potential game-changer in this field. Zemcelpro is a cell-based therapy developed from umbilical cord blood stem cells and designed for adult patients with hematological malignancies who lack access to compatible donors.
What is Zemcelpro?
Zemcelpro is an advanced therapy medicinal product (ATMP) developed using umbilical cord blood stem cells. The innovative component, dorocubicel, involves laboratory expansion of these cells to significantly increase their number. This enhancement enables even a small cord blood unit to provide sufficient cells for transplantation in adults.
Indicated Patient Population
Zemcelpro is intended for adult patients:
- Diagnosed with hematological malignancies such as leukemia, lymphoma, or MDS
- Requiring allo-HSCT following myeloablative conditioning
- Who lack access to an HLA-matched related or unrelated donor
Mechanism of Action
Cord blood stem cells are a valuable source for allo-HSCT, but their limited volume and cell count typically restrict their use in adult patients. Zemcelpro overcomes this challenge through dorocubicel – a method to expand a portion of the cord blood cells, thus improving engraftment potential.
Upon infusion, the expanded and unexpanded stem cells home to the bone marrow, reconstituting the patient’s hematopoietic system, and enabling the generation of healthy blood cells.
Clinical Evidence
In pooled data from two open-label, single-arm clinical studies involving 25 patients:
- 84% achieved neutrophil engraftment within a median of 20 days
- 68% achieved platelet engraftment within a median of 40 days
Common side effects among 116 treated patients included cytopenias, infections, hypertension, hypogammaglobulinemia, and engraftment syndrome. Acute graft-versus-host disease (aGvHD) occurred in 60%, and chronic GvHD in 13% of patients.
Regulatory Status in 2025
As of June 2025, Zemcelpro has received a positive recommendation for conditional marketing authorization from the European Medicines Agency (EMA) under its PRIME scheme. This marks a significant step towards making the therapy available across all EU countries, as well as Iceland, Norway, and Liechtenstein.
Currently, Zemcelpro is not yet approved in the USA, UK, Canada, or other regions, but further regulatory submissions are anticipated.
Why Zemcelpro Matters
- Enables transplantation in patients without matched donors
- Faster and more effective engraftment with expanded cell technology
- Addresses critical unmet medical needs in hematologic oncology
- Supported by EMA’s PRIME and orphan drug programs
Conclusion
Zemcelpro represents a promising future for patients battling blood cancers without access to compatible stem cell donors. By harnessing the regenerative power of expanded cord blood stem cells, Zemcelpro has the potential to redefine transplant options and improve survival outcomes for many high-risk individuals.
