Clinical trials at Hadassah lead to complete remission of common myeloma

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Article in “Doctors Only” website - May 2023. Translation to English.

Impressive results in CAR-T cell therapy for multiple myeloma, with over 90% of patients achieving complete remission. Prof. Polina Stepensky, Director of Bone Marrow Transplantation and Immunotherapy Department at Hadassah, says, "These are dramatic results and provide immense hope for patients".

CAR-T cell therapy for multiple myeloma has achieved impressive outcomes, with over 90% of treated individuals experiencing complete remission of the disease. The advanced treatment, developed by Prof. Polina Stepensky, Director of the Bone Marrow Transplantation and Immunotherapy Department at Hadassah Medical Center in Jerusalem, has led to substantial remission rates among the majority of patients, demonstrating a highly favorable overall response.

The research findings will be presented at the end of this month (May 30th) at the BioMed Research and Industry Conference organized by Hadassah Medical Center. Researchers and physicians from Hadassah will present their studies and developments to industry leaders and the Israeli bio-medicine community.

Multiple myeloma is the second most common hematological malignancy, accounting for one percent of all cancer types and ten percent of blood cancers. The innovative treatment against the disease, which was previously considered incurable, was developed following a series of experiments conducted at the Bone Marrow Transplantation and Immunotherapy Department of Hadassah in recent years, under the leadership of Prof. Stepensky.

"To date, 74 patients have participated in the trial, with a waiting list of over 200 patients from Israel and around the world at any given time," says Prof. Stepensky. "Due to the complexity of the manufacturing process and the treatment itself, only one patient per week undergoes the experimental therapy." In the coming months, the experimental treatment will also be expanded to the United States.

CAR-T cell therapy, based on genetic engineering technology, is developed and produced at Hadassah in collaboration with Prof. Cyril Cohen, Head of the Laboratory of Immunology and Immunotherapy at Bar Ilan University. So far, 90% of the treated patients have shown complete remission without any signs of the disease. "We have highly positive response rates with minimal side effects, and they are mild. These are dramatic results," emphasizes Prof. Stepensky. "It represents immense hope for patients with a disease that previously had no cure."

The concept of using immune system cells to combat cancer cells first emerged several decades ago at the Weizmann Institute of Science in the Immunology Department under the leadership of Prof. Zelig Eshhar. The development and advancement of CAR-T therapies, which involve "programming" the patient's white blood cells to fight tumors by collecting healthy cells from the immune system, has been led by Prof. Stepensky since then.

As part of the treatment, the T cells, which are the active cells in the immune system capable of self-fighting growths, are isolated using an apheresis device that separates red blood cells from leukocytes. Subsequently, the T cells are engineered in the laboratory at Hadassah, which was specially constructed with the strictest international standards in sterile rooms for this purpose.

In the next stage, a process of genetic engineering takes place by adding a virus containing a genetic segment that encodes a receptor against cancer cells. A large number of engineered cells are then injected into the patient. Ultimately, the engineered T cells adhere to the tumors and eliminate them. Until now, this treatment has only been available in China and the United States at a staggering cost of nearly $400,000 per patient, and its availability has been severely limited. Only 20% of those who need it in these countries actually receive it," explains Prof. Stepensky. "With our in-house development, led by researchers in the Leukemia Research Laboratory of Prof. Dani Canfi, we have managed to dramatically reduce the price and make the treatment affordable and accessible. Moreover, Hadassah has developed a sophisticated and advanced treatment that surpasses the current global standards. As the first and only institution in Israel developing, producing, and delivering internal CAR-T therapy, Hadassah is actually leading the field, enabling the development of future CAR-T treatments for patients with other types of cancer." Prof. Stepensky concludes, "The American company Immix Bio has acquired a patent license, and we are on the verge of initiating a clinical trial in the United States. The plan is to reach commercialization and FDA approval as a drug within a year.