Cell therapy weekly: FDA files for permanent injunctions against two for-profit stem cell clinics

Written by RegMedNet

This week: FDA acts on for-profit stem cell clinics, gene therapy price tags predicted to top US$1 million and an industry-academic partnership seeks to develop an off-the-shelf CAR-T therapy.

The news highlights:

FDA seeks permanent injunctions against two for-profit stem cell clinics
First therapy priced more than US$1 million could be on the horizon, possibly gene therapy for hemophilia
Industry-academia partnership expands to develop off-the-shelf allogeneic CAR-T therapy

FDA seeks permanent injunctions against two for-profit stem cell clinics

The FDA has filed two permanent federal injunctions against clinics marketing and providing unapproved stem cell treatments. US Stem Cell Clinic LLC (FL, USA) and California Stem Cell Treatment Center Inc. (CA, USA) have been served the injunctions to cease marketing their stem cell products until the necessary approvals have been obtained and violations of good manufacturing practice have been corrected. These permanent injunctions follow previous warning letters from the FDA, but US Stem Cell Clinic LLC has already announced its intention to ‘vigorously defend’ this filing.

“[Regenerative medicine] holds significant medical opportunity, but we’ve also seen some bad actors leverage the scientific promise of this field to peddle unapproved treatments that put patients’ health at risk. In some instances, patients have suffered serious and permanent harm after receiving these unapproved products. In the two cases filed today, the clinics and their leadership have continued to disregard the law and more importantly, patient safety….the FDA will continue to take enforcement actions against clinics that abuse the trust of patients and endanger their health with inadequate manufacturing conditions or by purporting to have treatments that are being manufactured and used in ways that make them drugs under the existing law but have not been proven safe or effective for any use,” commented FDA Commissioner Scott Gottlieb.

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First therapy priced more than US$1 million could be on the horizon, possibly gene therapy for hemophilia

In a Leerink (MA, USA) research note obtained by CNBC, biotech analysts are predicting drug prices of over $1 million. In particularly, new therapies for hemophilia, in development by BioMarin (CA, USA), Spark Therapeutics (PA, USA) and UniQure (the Netherlands), could top $1.5 million. Factor replacement therapy for patients with hemophilia is already estimated to cost between $580,000 and $800,000 for adult patients.

“It appears the seemingly impervious million-dollar threshold may be breached with hemophilia gene therapy, which could do so while still creating value for society by reducing the cost of factor replacement therapy,” Leerink analysts Joseph Schwartz and Dae Gon Ha wrote in their research note.

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Industry-academia partnership expands to develop off-the-shelf allogeneic CAR-T therapy

Atara Biotherapeutics, Inc. (CA, USA) has announced expansion of its collaboration with Memorial Sloan Kettering Cancer Center (MSK; NY, USA)  to develop an off-the-shelf CAR-T immunotherapy. Under the agreement, Atara will gain access to a number of MSK’s proprietary technologies and methods for designing these therapies to look at oncology, autoimmune and infectious disease indications.

Michel Sadelain, Director, Center for Cell Engineering, MSK stated, “We are eager to work with Atara to continue advancing promising allogeneic T-cell immunotherapy technologies that originated at MSK. The new CAR T technologies seek to overcome persistent therapeutic challenges, such as safety and tolerability, durability of treatment response, and activity in areas of significant unmet medical need that are underserved by the current generation of CAR T immunotherapies.”

“Our earlier MSK collaboration has been highly productive…[and] the deepening of our collaboration with MSK allows us to rapidly advance novel gene-edited CAR T development programs leveraging our existing off-the-shelf T-cell immunotherapy technology platform, manufacturing expertise and research and development capabilities,” commented Isaac Ciechanover, Chief Executive Officer and President of Atara Biotherapeutics.

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For more weekly cell therapy news, read previous editions of the cell therapy weekly.