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Cellectis’ CAR T-Cells Based Immunotherapy Faces Clinical Hold

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UPDATE: After the approval of the first gene therapy- “Kymriah” to treat a rare blood cancer in children, a similar CAR-T therapy candidate of CellectisUCART123 faced clinical hold on the study. It is intended to treat two different types of cancer, namely acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).

The hold is placed after the death of a patient having BPDCN who suffered from an adverse event-  cytokine release syndrome (CRS) after treatment with UCART123 and later died due to its complications. CRS is one of the major side effects caused to the patients treated with UCART123. CRS is a life threatening condition that occurs due to fatal inflammatory response of the body against the therapy.


One of the leading CAR T-cells immunotherapy company, Cellectis dosed the first blood cancer patient with the allogeneic, “off-the-shelf” CAR-T therapy, UCART123 in June at Weill Cornell Medicine and NewYork-Presbyterian Hospital.

UCART123: the Universal Cancer Immunotherapy

Gene editing technology has provided the privilege of engineering the genetic makeup of an individual to treat diseases like cancer and to understand the genetic function of humans more precisely.

Cellectis’ UCART123 is an allogeneic genetically modified CAR T cell immunotherapy. It is developed by obtaining T-cells from healthy donors. Later it is prepared as “off-the-shelf” CAR T cells which could be used by a large number of patients without customizing them for a particular patient.

UCART123 is the first U.S. gene-edited, “off-the-shelf” CAR T-cell program. The development of UCART123 is based on the gene-editing technology, TALEN. TALEN are the “molecular scissors” which cut a particular gene specifically in order to reprogramme them so that they can precisely target and kill cancer cells.

UCART123 can be manufactured in large scale from the healthy donor T cells, cutting out the need of patient’s own cells for every case.


Related: Is 2017 the Year of First CAR-T Cells Immunotherapy Approval?


First-in-Human Administration of UCART123

The first patient with acute myeloid leukemia received the UCART123 immunotherapy. UCART123 targets the molecule CD123 that is expressed across AML tumor cells. In the clinical study, the safety and efficacy of UCART123 in patients with AML will be investigated.

AML is an aggressive blood cancer that kills about 10,430 people in the US every year. Despite a high complete response rate of 80 percent in younger patients, most patients die due to relapse of cancer.

 Dr. Loan Hoang-Sayag, Cellectis Chief Medical Officer said, “This first program targeting CD123 will be a paradigm shift for our Company, as it will provide a wealth of valuable additional knowledge and data to drive our gene-edited allogeneic CAR T-cell platform.”

In 2015, another TALEN-based allogeneic immunotherapy, UCART19 showed surprising results in the 11 months old baby girl, Layla who was in the last stage of the incurable, refractory and highly aggressive ALL- an aggressive blood cancer. The girl had not responded to chemotherapy earlier but after her treatment with the UCART19 immunotherapy, her cancer disappeared and she is doing well now.

Another 16 months old baby suffering from ALL became free of cancer after receiving the experimental UCART19 injection.

Editor’s Note: The article was originally published on 30 June 2017 and has been updated for comprehensiveness.

Featured image credit: Bigstockphoto (cart cell human trials)

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