Cell therapy weekly: more indications for Kymriah

Written by RegMedNet

This week: more for Kymriah, and treatments for autoimmune disorder, epilepsy and diabetes.

Kymriah receives priority review designation for second indication

Novartis’ application for Priority Review for Kymriah, an autologous CAR-T immunotherapy, has been accepted by the FDA for a second indication. Kymriah is now indicated for patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) or r/r B-cell acute lymphoblastic leukemia (ALL) that cannot be treated, or have been treated unsuccessfully, with an autologous stem cell transplant. In addition, the European Medicine Agency has granted accelerated assessment to the Marketing Authorization Application for these same indications. If successful, Kymriah would become the first CAR-T therapy available for two distinct indications.

“The first approval of a CAR-T therapy truly redefined the future of the cancer treatment landscape, and we are only at the beginning of this new era in cancer care,” commented Samit Hirawat, Head, Novartis Oncology Global Drug Development. “The Priority Review designation and accelerated assessment signal that the FDA and EMA have recognized the potential of Kymriah to provide a much-needed therapeutic option for these patients with relapsed or refractory B-cell ALL and DLBCL. We are now focused on working with these regulatory agencies to bring this potentially transformative therapy to more patients.”

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First serious adverse reaction to iPSC-derived retinal cells reported

In the world’s first clinical trial utilizing allogeneic iPSC-derived retinal cells to treat age-related macular degeneration, the first serious adverse reaction has been reported. The patient, in his 70s, suffered a swollen retina and was operated on to remove preretinal membrane, determined to be the cause. All five procedures planned in this clinical trial were carried out March—November 2017 and iPSCs kept at Kyoto University (Japan) were specially created to avoid immune reactions. It is hoped that this will not affect future clinical studies utilizing this technique.

“We cannot deny the causal correlation with iPS cells,” commented team lead Masayo Takahashi, Project Leader for Laboratory for Retinal Regeneration at the RIKEN Center for Developmental Biology (Kobe, Japan), at a news conference, stressing that although it was a serious case that required hospital admission, it was “neither a matter of great urgency nor life-threatening.”

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ESC-based type 1 diabetes therapy to be trialed in Canada

An implantable medical device containing embryonic stem cell-derived pancreatic cells is to be trialed in Vancouver (BC, Canada). Through vascularization, it is hoped that cells within the implants, developed by ViaCyte (CA, USA), will differentiate into beta cells, sense blood sugar and release insulin as needed. Supported by a CA$500,000 grant from the Stem Cell Network (ON, Canada), the trial could involve 10 or more patients with severe type 1 diabetes who will be followed for 2 years.

“If these replacement cells restore a person’s ability to produce their own insulin when needed, it would prevent dangerous episodes of low blood sugar and lessen the complications resulting from high blood sugar, such as blindness, heart attacks and kidney failure,” commented David Thompson, a principal investigator in the clinical trial and medical director of the Vancouver General Hospital Diabetes Centre.

“Eventually, it might even free people from a lifetime of constantly checking their blood sugar and injecting themselves, transforming treatment of this disease into a more manageable condition.”

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One step closer to a gene therapy for epilepsy

The Cell and Gene Therapy Catapult (London, UK) have signed a collaboration agreement with CombiGene (Sweden) to develop manufacturing processes for CombiGene’s drug candidate CG01. CG01 uses gene therapy vectors to deliver neuropeptide y (NPY) and NPY receptors into brain cells, shown to inhibit epileptic seizures in preclinical studies. Through the agreement, a process will be developed to enable CombiGene to progress to commercial GMP production and, subsequently, clinical trials. 

“We are delighted to be working with CombiGene to accelerate the commercialization of an important gene therapy to treat an unmet medical need. It is testament to our international reputation and the capabilities that we offer that we continue to be the development partner of choice for innovative cell and gene companies,” commented Keith Thompson, CEO, Cell and Gene Therapy Catapult.

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Stem cell transplant could extend life of patients with rare autoimmune disorder

In a new study published in the New England Journal of Medicine, stem cell transplants were found to improve survival of patients with severe scleroderma. Scleroderma is an autoimmune disease that can damage the skin and lungs. The treatment involved chemotherapy and radiation to destroy the patient’s immune system, which is then restored through an autologous blood-derived stem cell transplant. After 72 months, 86% of patients that received the transplant remained alive, compared with 51% of the cohort that had received cyclophosphamide, the most effective existing treatment.

“This is a major advance in the treatment of severe scleroderma,” commented Karen Ballen, co-investigator on the study and director of stem cell transplantation at the University of Virginia Cancer Center (VA, USA).

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