Cell therapy weekly: Point-of-care CAR-T therapy placed on clinical hold
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This week: Acupuncture inspires method for stem cell differentiation and angina therapy receives RMAT.
The news highlights:
Phase I trial for point-of-care CAR-T therapy placed on clinical hold
Acupuncture-inspired device supports growth of therapeutic stem cells
Refractory angina therapy receives RMAT designation
Phase I trial for point-of-care CAR-T therapy placed on clinical hold
The FDA has placed a clinical hold on a Phase 1 trial evaluating a CD19-specific CAR-T therapy manufactured at the point of care. Pending additional information relating to chemistry, manufacturing and controls in support of the investigational new drug application from Ziopharm Oncology, Inc. (MA, USA), the initiation of the trial may be delayed. Ziopharm is a wholly-owned subsidiary of Intrexon Corporation (MD, USA) and The University of Texas MD Anderson Cancer Center (TX, USA), and a second-generation trial using these cells, based at MD Anderson, continues enrollment and treatment of patients.
“We know what is needed to address the hold issues and are looking forward to responding to the agency in a timely manner,” said Laurence Cooper, Chief Executive Officer of Ziopharm. “We are undertaking cutting-edge science and are on the verge of a paradigm shift based on our approach to very-rapidly manufacture CD19-specific T cells within two days using our non-viral approach to CAR-T therapy based on the Sleeping Beauty platform.”
Acupuncture-inspired device supports growth of therapeutic stem cells
A team of researchers from Hong Kong Baptist University (Hong Kong) have developed a medical device that supports the proliferation and differentiation of neural stem cells (NSCs) without the use of growth factors. The device, which uses ‘physical massage’ to differentiate the NSCs, won the Gold Medal with Congratulations of Jury at the 46th International Exhibition of Inventions of Geneva held in Switzerland in April 2018. An application for a US patent has been filed.
Professor Ken Yung, joint team leader (Hong Kong Baptist University), explained, “The novel matrix enables scientists to cultivate NSCs by adopting the usual method, however with the added advantage of organic compounds (like [sic] polylysine and polyornithine) being excluded from the process, thereby reducing the potential risk of carcinogenesis or inflammation in stem cell therapy. It could provide a safe platform for research into stem cell therapies using the latest, novel nanotechnology, and also help boost the development of regenerative medicine.”
Refractory angina therapy receives RMAT designation
Caladrius Biosciences, Inc. (NJ, USA) has announced the FDA has granted their late-state CD34+ cell therapy program for refractory angina regenerative medicine advanced therapy (RMAT) designation. The license for this program was acquired from Shire plc (Dublin, Ireland) in March 2018.
“We are delighted and encouraged that the FDA has recognized our CD34+ cell therapy program with an RMAT designation. Refractory angina is a serious condition with high morbidity and no known effective treatments. We look forward to working with the FDA to define a path to registration for our therapy with the aim of providing expeditious treatment to patients suffering from this condition,” commented David J. Mazzo, President and Chief Executive Officer of Caladrius.
For more weekly cell therapy news, read previous editions of the cell therapy weekly.