Cell therapy weekly: first human CAR-T cell therapy patients are cancer-free, 10 years after treatment
This week: first human CAR-T cell therapy patients are cancer-free, 10 years after treatment, researchers engineer human spinal cord implants to treat paralysis and Arcellx raises US$124 million in IPO to advance CAR-T cell therapy.
The news highlights:
First human CAR-T cell therapy patients are cancer-free, 10 years after treatment
CAR-T therapy is a cell-based gene therapy that alters the genes inside T cells to find and destroy cancerous cells. A team of researchers from the University of Pennsylvania (PA, USA) and Novartis Institutes for BioMedical Research (MA, USA) published their pioneering study in Nature, revealing that two of the first human CAR-T therapy patients remain cancer free, 10 years after the initial treatment for chronic lymphocytic leukemia. These findings provide growing support for CAR-T cell therapy in the treatment of certain blood cancers.
“A decade ago, CAR-T cell therapy was a therapeutic approach explored by a very small number of scientists and was considered a fringe approach and unlikely to work,” commented Martin Pule, director of the UCL Cancer Institute CAR-T cell program (London, UK). “This paper shows us that CAR-T cells can give patients with cancers which no longer respond to chemotherapy remissions which last a decade,” he concluded.
Researchers engineer human spinal cord implants to treat paralysis
Researchers at Sagol Center for Regenerative Biotechnology at Tel Aviv University (Tel Aviv, Israel) have engineered and implanted functional human spinal cord tissues, which have been shown to treat paralysis. Results from the study on mice revealed an 80 percent success rate in restoring walking ability in patients with chronic paralysis. These encouraging results provide hope for future clinical trials in humans.
“We hope to reach the stage of clinical trials in humans within the next few years, and ultimately get these patients back on their feet,” commented Tal Dvir, Director of Sagol Center for Regenerative Biotechnology. “The company’s preclinical program has already been discussed with the FDA. Since we are proposing an advanced technology in regenerative medicine, and since at present there is no alternative for paralyzed patients, we have good reason to expect relatively rapid approval of our technology,” he continued.
Arcellx raise US$124 million in IPO to advance CAR-T cell therapy
Arcellx (MD, USA), a clinical-stage biopharmaceutical company developing novel cell therapies for the treatment of cancer and autoimmune diseases, has secured US$124 million in IPO financing to advance its CAR-T therapeutic candidate to clinical trials. The biotech offers deimmunized synthetic binding scaffolds, which are smaller than those currently available in the CAR-T therapy industry. Arcellx’s preclinical research suggested that this smaller size allowed for higher transduction efficiency, higher expression of its binders on the surface of T cells and lower signaling that can lead to off-target activation of T cells.
“Ultimately, we hope to develop multiple therapeutic options incorporating different mechanisms, cell modalities, and targets to treat hematologic cancers, solid tumors, and indications outside of oncology, such as autoimmune diseases,” stated Arcellx in its filing.
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