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Veto T-Cell Bone Marrow Transplant Safely Demonstrates Potential Cure for Sickle Cell Anemia in Preclinical Trials

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Cell Source (OTC: CLCS) has made significant advancements in Veto Cell based immunotherapy, achieving a key breakthrough in treating sickle cell disease (SCD). Their newly developed mouse model protocol demonstrates durable prevention of SCD pathology, with complete conversion to normal, donor-derived red blood cells. Published in the BMT Journal, this study addresses major challenges in mismatched donor stem cell transplants. The ongoing Phase 1/2 clinical trial is testing Veto Cells for safety and efficacy in hematological diseases, offering hope for safe and effective treatments.

Positive
  • Veto Cell protocol showed durable prevention of sickle cell disease pathology.
  • Results indicate complete conversion to normal, donor-derived red blood cells.
  • The study published in the BMT Journal supports potential human application.
  • Current Phase 1/2 clinical trial is underway for Veto Cells in hematological diseases.
Negative
  • None.

NEW YORK, March 12, 2021 /PRNewswire/ -- 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.

Cell Source Investor Resource Center

Cell Source maintains a comprehensive Investor Resource Center including share information, SEC filings, investor relations contacts, and more, located here: https://cell-source.com/investor-center

Cell Source General Information

For general information regarding Cell Source, please visit the Company's website at https://cell-source.com

A PDF presentation introducing Cell Source and its technologies is available here:

https://cell-source.com/wp-content/uploads/2020/12/CLCS-Introduction-Q1-2021.pdf

For details regarding Cell Source's Veto Cell Technology Platform and other immunotherapy innovations, please visit https://cell-source.com/veto-cells

Background information on Professor Yair Reisner, Ph.D, the award-winning Chairman of Cell Source's Scientific Advisory Board, is available here: https://cell-source.com/professor-yair-reisner-ph-d

Dr. Reisner's other published works and additional background information are available via ORCID, here: http://orcid.org/0000-0002-3354-6945

To learn about Cell Source's world-class collaborative partnerships, please visit http://cell-source.com/collaborative-partnerships.

For information regarding Cell Source's Scientific Advisory Board, please visit https://cell-source.com/scientific-advisory-board

Company Contact
Itamar Shimrat, CEO
646.612.7554
ishimrat@cell-source.com

Investor Contact
Michael Briola
Alternative Advisory Group
917.455.0820
mbriola@alternativeadvisory.com

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. With the exception of historical information, the matters discussed in this press release are forward-looking statements that involve a number of risks and uncertainties. The actual future results of Cell Source, Inc. could differ significantly from those statements. Factors that could cause actual results to differ materially include risks and uncertainties such as the inability to finance the company's operations, inability to hire and retain qualified personnel, and changes in the general economic climate, as well as the risk factors disclosed in Cell Source, Inc.'s Form 10-K filed on March 30, 2020. Cell Source, Inc. may, in some cases, use terms such as "anticipates," "continue," "estimates," "predicts," "believes," "potential," "proposed," "expects," "plans," "intends," "may," "could," "should," "might," "will," or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions. Although we believe that the expectations reflected in the forward-looking statements are reasonable, such statements should not be regarded as a representation by Cell Source, Inc. or any other person, that such forward-looking statements will be achieved. Cell Source, Inc. undertakes no duty to update any of the forward-looking statements, whether as a result of new information, future events or otherwise. In light of the foregoing, readers are cautioned not to place undue reliance on such forward-looking statements. This release does not constitute an offer to sell or a solicitation of offers to buy any securities of any entity.

 

Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/veto-t-cell-bone-marrow-transplant-safely-demonstrates-potential-cure-for-sickle-cell-anemia-in-preclinical-trials-301246369.html

SOURCE Cell Source, Inc.

FAQ

What advancements has Cell Source made in sickle cell disease treatment?

Cell Source's Veto Cell based protocol has shown the potential to durably prevent the onset of sickle cell disease pathology in mice.

What are the main features of the Veto Cell protocol?

The protocol allows for safer mismatched bone marrow transplants and tackles challenges like transplant rejection and graft vs. host disease.

When was the study on Veto Cell protocol published?

The study was published on March 3, 2021, in the BMT Journal.

What is the current status of Cell Source's clinical trials?

A Phase 1/2 clinical trial is currently testing Veto Cells in patients with hematological cancers and sickle cell disease.

How might Cell Source's Veto Cell technology impact future treatments?

It could lead to curative treatments for sickle cell disease and other hematological conditions, providing safer transplant options.

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