Sickle Cell Disease (SCD) research project hopes to pave the way for approval of a clinical trial by the US-FDA in the next few years
ALAMEDA, CA and OAKLAND, CA – March 20, 2017 – AllCells, LLC, a leading provider of hematologic tissue, primary cell types & services for life science research, and further manufacturing of cell therapies, has been selected by Children’s Hospital Oakland Research Institute (CHORI) and the Innovative Genomics Institute (IGI) as a partner to process peripheral blood from Sickle Cell Disease (SCD) patients. The research project is funded by the California Institute for Regenerative Medicine (CIRM).
Under the $4.5 million CIRM grant awarded to the investigators at CHORI (part of UCSF Benioff Childrens Hospital) and IGI (a partnership between UC Berkeley and UC San Francisco) researchers are working towards a cure for SCD. Towards the goal of helping researchers advance their work to the clinic, AllCells will be isolating CD34+ hematopoietic stem cells (HSCs) from sickle cell discard blood and providing CD34+ cells from clinically healthy, mobilized donors. AllCells is utilizing its expertise in large-scale HSC isolation and collaborating with Miltenyi Biotec GmbH (Bergisch Gladbach, Germany) to optimize a procedure for SCD CD34+ cell isolation using Miltenyi’s CliniMACS Prodigy® cell separation platform. Purified CD34+ cells from SCD patients will be delivered by AllCells to Mark DeWitt, PhD at the IGI and David Martin, M.D. at CHORI, who will use an ex vivo gene editing (or gene surgery) approach, CRISPR-Cas9, to correct the genetic mutation that causes SCD. If this preclinical research project looks promising, the goal is to eventually move this approach into human clinical trials whereby the genetically corrected stem cells would be returned to the SCD patient as part of an autologous bone marrow transplant cell therapy. This treatment has potential to cure SCD since these corrected stem cells would constitute the patient’s new blood forming system resulting in physiologically normal red blood cells. AllCells previously provided purified SCD CD34+ cells to these researchers, for their gene editing proof-of-concept study published last year in Science Translational Medicine (DeWitt et al, Sci. Transl. Med. 8, 360ra134, 12 Oct 2016).
According to CIRM, SCD affects over 6,000 primarily African-American individuals in California. A survival of <40 years of age was observed in a large cohort of California patients. The estimated lifetime cost of care is $9 million per person. If successful, this cell therapy approach would be a universal life saving and cost-effective therapy.
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Wayne Vaz
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