BioLineRx’s investigational drug BL-8040 has demonstrated positive interim results from a Phase 2 trial as a monotherapy for the mobilization and collection of donor blood stem cells for bone marrow transplants.
In many blood diseases, bone marrow transplants have become a standard therapeutic procedure. This process requires that a donor’s healthy progenitor, or stem cells, be collected and selected, then infused into the recipient patient.
To achieve a high enough number of stem cells for an effective transplant, and to mobilize stem cells into the blood for easier access, donors usually receive injections of a compound called granulocyte colony-stimulating factor (G-CSF), administered in four to six injections. This is a time-consuming process and is often associated with side effects such as bone pain.
But BioLineRx and Washington University School of Medicine are conducting a Phase 2 trial (NCT02639559) to assess the safety and effectiveness of BL-8040 to replace the standard mobilization protocol of G-CSF.
Clinical and preclinical studies have demonstrated that BL-8040 has the potential to inhibit growth and induce cell death of several types of solid and liquid tumors, including multiple myeloma. Combining BL-8040 with standard of care chemotherapies improved anti-tumor response.
But in addition to its anti-tumor potential, researchers also found that BL-8040 was capable of mobilizing progenitor cells of different subtypes of blood cells, such as colony-forming cells, T-cells, B-cells and NK cells.
Interim results of the Phase 2 trial showed that a single injection of BL-8040 mobilized stem cells similar to levels achieved with four to six injections of G-CSF, the standard protocol. This was performed in 10 donors and was found to be safe, with no significant side effects reported.
“We are very encouraged by these initial results of the Phase 2 clinical trial for assessing BL-8040, our lead oncology and hematology platform, as a single agent for hematopoietic stem cell mobilization for allogeneic transplantation,” Philip Serlin, CEO of BioLineRx, said in a press release.
Patients with advanced hematological cancers who were paired with these donors received the transplanted cells and showed a successful engraftment, meaning the stem cells reached the patients’ bone marrow and started multiplying. They will be followed for one year to assess any adverse side effects or graft failure.
“These results, supporting BL-8040 as a one-day dosing and up-to-two-day collection regimen, for rapid mobilization of substantial amounts of stem cells, represent a significant improvement over the current standard of care, which requires four-to-six daily injections of G-CSF and one-to-four [cell purification] sessions,” Serlin said.
If there are no safety concerns from Part 1 of this trial, Part 2 will get underway, with 24 donor-recipient pairs. The results are expected by the end of 2017.