Well-established Mouse Model Utilizes T-cell-depleted, Mismatched Donor Bone Marrow Transplants in Conjunction with Veto T-cell Transplants to Correct Sickle Cell Disease with Minimal Risk as Reported in Bone Marrow Transplantation (BMT) Journal
March 12, 2021 – New York, NY – Cell Source, Inc. (OTC: CLCS) (“Cell Source” or the Company”), the world leader in Veto Cell based immunotherapy technologies that can safely facilitate mismatched donor stem cell transplants, e.g. bone marrow transplants (BMT) – in different mouse models for non-malignant hematological disease and organ transplants announced today that its fully mismatched mouse Veto Cell based BMT protocol has overcome significant challenges to durably prevent the onset of the pathological parameters of sickle cell disease (SCD) including complete conversion to normal, donor-derived red blood cells.
“Sickle cell disease diminishes the quality of life and lowers the life expectancy of approximately 100,000 people in the United States alone,” said Dennis Brown, Chairman of Cell Source. “The results of the preclinical study published in the BMT Journal demonstrate that our curative approach to SCD may potentially translate to safe and efficacious treatment in human patients. Our Veto Cell based protocol could overcome the lack of suitable donors, treatment toxicities, and graft-related challenges. This may have the potential to deliver a safe, effective, and durable curative treatment for patients with SCD.”
The study, “Correction of murine sickle cell disease by allogeneic hæmatopoietic cell transplantation with anti-3rd party veto cells,” published online by BMT Journal on March 3, 2021, may be found here: https://www.nature.com/articles/s41409-021-01237-6
As noted in BMT Journal , the Veto Cell protocol was able to safely and efficaciously achieve durable, donor-derived chimerism in mice prone to sickle cell disease (i.e., the donor’s transplanted healthy cells begin to be produced by the recipient mice instead of the host defective cells) and prevent onset of SCD pathology by overcoming several significant challenges, including:
- The need for safer partially or fully mismatched bone marrow transplantation (BMT) protocols
- The challenge of transplant rejection under reduced intensity conditioning (RIC)
- The possibility of graft vs. host disease under RIC
A Phase 1/2 clinical trial is currently in progress testing the safety and efficacy of Cell Source’s Veto Cells in patients with hematological cancers or with non-malignant hematological diseases including SCD.
Information regarding the clinical trial may be located here: https://www.clinicaltrials.gov/ct2/show/NCT03622788
About Cell Source
Cell Source, Inc. (OTC: CLCS) is the worldwide, exclusive innovator of Veto Cell based cellular therapy and immunotherapy platform technologies designed to provide safer and more accessible bone marrow transplantation (BMT) and improve the treatment of malignant blood cancers such as multiple myeloma and leukemia, in addition to correction of nonmalignant genetic blood disorders like sickle cell disease, and to durably treat auto immune diseases such as type 1 diabetes, which are also treatable by the Veto Cell technology. The Company is developing and evaluating its Veto Cell CAR-T platform, which could potentially enable CAR-T vectors to be used in donor-derived (allogeneic) settings with increased safety, efficacy, and persistence. Cell Source’s Veto Cell technologies are also being developed to facilitate safe, mismatched organ transplants (e.g., kidney and liver) without the need for life-long immune system suppression.
The Company is focused on transitioning its globally exclusive allogeneic Veto Cell platform technologies into human studies in order to develop safe and curative BMT from mismatched donors as well as “off-the-shelf,” safe, durable, and efficacious immunotherapy and immuno-oncology products.
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