Article in “Ha’aretz” – Israeli newspaper - May 2023. Translation to English.
An Israeli experimental treatment for multiple myeloma, conducted at Hadassah Ein Karem Hospital, has yielded highly positive findings, offering significant hope for patients with this incurable disease. The research team leader stated, "These are dramatic results that bring immense hope to patients with a disease that has had no cure thus far."
The treatment, based on Chimeric Antigen Receptor-T cell (CAR-T) technology used for other diseases, was developed by Prof. Polina Stepensky, Director of the Bone Marrow Transplantation and Immunotherapy Department at Hadassah Medical Center, in collaboration with Prof. Cyril Cohen, Head of the Immunology and Immunotherapy Laboratory at Bar-Ilan University. So far, 74 multiple myeloma patients have received the treatment as part of the clinical trial.
"We have witnessed overwhelmingly positive response rates, with minimal side effects that are also mild," said Stepensky. She further emphasized, "These are dramatic results. It is an immense hope for patients with a disease that has never had a cure before." The detailed findings of the trial will be presented at the upcoming Hadassah Research and Biomedical Industry Conference at the end of the month.
Multiple myeloma is a relatively common cancer, accounting for about 1% of all cancer cases and 10% of blood cancer cases. Each year, between 400 and 500 new patients are diagnosed with this disease in Israel, the majority of whom are over 70 years old. The disease originates from abnormal plasma cells, a type of white blood cell, which proliferate and grow uncontrollably. As a result, the disease affects the bone marrow, bones, kidneys, and immune system. Over a decade ago, significant advancements were made in multiple myeloma treatment with the development of new biological therapies, but a curative treatment has not yet been achieved.
The groundbreaking idea of utilizing the immune system's cells to fight cancer cells was first conceived in the 1980s in the Immunology Department at the Weizmann Institute of Science by Prof. Zelig Eshhar. Subsequently, the treatment became known as Chimeric Antigen Receptor-T cell (CAR-T) therapy, which is now considered revolutionary in the field of hematological malignancies. It is also included in the healthcare basket in Israel for the treatment of lymphoma. Cohen and Stepensky sought to develop a similar treatment based on this technology for multiple myeloma patients.
"It's not a disease that can be cured, and that is not our goal either. The development of cancer treatments aims to achieve maximum remission with minimal side effects to maintain the best possible quality of life," said Prof. Stepensky.
"In the context of treatment, a process of isolating T cells is performed, which are the active cells in the immune system capable of fighting tumors. The process is carried out using a device called 'apheresis,' which separates red blood cells from white blood cells. Subsequently, T cells are engineered in a dedicated laboratory at Hadassah, specifically built for this process. In the next stage, a genetic engineering process is performed, during which genetic segments are introduced into the T cells using a virus, rendering them receptive to cancer cells. The receptor on the T cells binds to a protein characteristic of the cancerous cell, validating and destroying it. Ultimately, a large quantity of engineered cells is injected into the patient in a single infusion.
Following a preclinical trial that yielded positive results, in February 2021, the clinical trial at Hadassah Ein Karem began with patients. "To date, 74 patients have entered the trial, with a waiting list of over 200 patients from Israel and around the world at any given time," said Stepensky. "Due to the complexity of production and the treatment itself, only one patient receives treatment per week, which is still conducted as part of the trial." The success data from the trial indicates that a large majority of the patients experienced a positive response and temporary or complete remission from the disease. The shortest remission period observed was approximately three months. The longest-treated patient received the treatment a year and ten months ago and is still in remission. According to interim trial findings, median data show that 50% of the patients are still in complete remission after ten months.
"It is not a curable disease, and that is not the goal," said Stepensky. "The development of cancer treatments aims to achieve maximal remission with minimal side effects, while preserving the quality of life as much as possible. In these parameters, the treatment demonstrates significant success and a dramatic breakthrough for patients." According to Stepensky, "A similar treatment based on the same principle for multiple myeloma patients is currently available only in China and the United States, where it is severely limited in availability, with a cost of approximately $400,000 per patient. Only 20% of those who need it in these countries actually receive it." Stepensky adds that with the development of the treatment, led by researchers at the Danny Kanof Leukemia Research Laboratory, "we managed to dramatically reduce the cost, making the treatment affordable and accessible. Furthermore, the treatment we developed is more sophisticated and advanced than what is currently available worldwide." Following the success of the trial, the American company Immix Bio recently acquired the patent license for the treatment. "We are on the verge of launching a clinical trial in the United States," Stepensky revealed. Now, she and her team are planning to move forward toward the next goal: "to achieve market entry and FDA approval as a drug within a year."
- More about the CAR T program in Hadassah
- About Hadassah Hospital
- About Prof. Polina Stepenksy
- Interview with Prof. Stepenksy
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