A regenerative medicine company called Regen BioPharma, Inc., has announced that it received a communication from the U.S. Food and Drug Administration that grants it permission to initiate clinical trials under its Investigational New Drug (IND) #15376.
Granting of the IND gives the green light to Regen BioPharma to begin testing their product HemaXellerate in clinical trials with human patients. HemaXellerate is a personalized stem cell treatment for patients whose bone marrow no longer works (aplastic anemia). It uses fat-based stem cells from a patient’s own belly fat to treat bone marrow that has been damaged. HemaXellerate uses the patient’s own fat-based stem cells as a source of endothelial (blood vessel) cells to heal damaged bone marrow.
Aplastic anemia occurs when the bone marrow stops producing sufficient numbers of blood cells. It is a potentially fatal disease of the bone marrow that leads to bleeding, infection and fever. Patients with severe or even very severe aplastic anemia have a mortality rate of greater than 70%. Current treatments for aplastic anemia include blood transfusions, immunosuppression and stem cell transplantation.
This Phase I clinical trial will treat patients who have been diagnosed with refractory aplastic anemia, which includes those patients with aplastic anemia who were unsuccessfully treated with first-line immunosuppressive therapy. Patients treated with HemaXellerate with be followed for safety parameters and signals of treatment efficacy. Since this will be an unblinded trial, all data will be available as the study progresses.
“Current drug-based approaches for healing bone marrow dysfunction involve flooding the body with growth factors, which is extremely expensive and causes unintended consequences because of lack of selectivity,” said Harry Lander, Ph.D., President and Chief Scientific Officer of Regen Biopharma. “By utilizing a cell-based approach that both modulates the immune system and stimulates production of blood cells, we aim to offer alternatives to the current approaches to treating patients with aplastic anemia. This product will complement our immune-modulatory pipeline that includes a potential novel checkpoint inhibitor.”
If HemaXellerate passes this clinical trial, Regen Biopharma would like to position HemaXellerate as a treatment for bone marrow dysfunction on par with other members of the hematopoietic growth factor market that includes drugs such as Neupogen®, Neulasta®, Leukine® and Revolade®.
“The FDA clearance marks a substantial step for Regen, in that we are now a clinical-stage company. We are grateful to our collaborators and scientific advisory board members who have worked tirelessly in bringing our product to the point where the FDA has permitted treatment of patients,” said David Koos, Ph.D., Chairman and Chief Executive Officer of Regen BioPharma. “We believe the success of today will not only allow for the rapid execution of HemaXellerate’s development plan, but will also allow for more rapid translation of the company’s other immune modulatory products to the clinic.”