A team of Israeli scientists and doctors have developed the country’s first blue-and-white CAR T immunotherapy for the treatment of multiple myeloma, potentially giving thousands of sick patients access to this lifesaving treatment.
Multiple myeloma is a still incurable cancer within the spongy bone marrow.
A Phase I clinical trial kicked off during the COVID-19 pandemic. So far, seven patients have received the treatment, and it is proving safe and effective.
The idea to develop Israel’s own CAR T therapy was spearheaded by Prof. Polina Stepensky, who heads Hadassah-University Medical Center's department of bone marrow transplantation and cancer immunology, along with Prof. Cyrille Cohen, the head of the immunology lab at Bar-Ilan University. Stepensky was inspired by the work of Memorial Sloan Kettering Cancer Center for Cell Engineering Director Michel Sadelain and convinced her team that they could do it.
Over the last few years, Cohen’s lab completed all of the necessary in vitro and in vivo experiments, built laboratories and good manufacturing practice facilities with Hadassah, and received all the necessary Health Ministry approvals to test the treatment on people.
Stepensky and two PhD students, Dr. Shlomit Kfir Erenfeld and Dr. Nathalie Asherie, have been working on the clinical trials.
“I think that it was important to demonstrate the feasibility of the whole process here in Israel, as it was designed from scratch and evaluated preclinically in our laboratory in Bar-Ilan University, with the support of the Adelis Foundation,” Cohen told The Jerusalem Post. “This partnership with Hadassah opens up possibilities for patients who do not have access to big clinical trials or to this kind of therapy that, when approved, can cost several hundreds of dollars.”
CAR T treatment is a type of gene therapy that “marshals the patient’s own T-cells – white blood cells of the immune system. The cells are genetically engineered to include a ‘hunting’ element that seeks out the cancer cells and destroys them. Called chimeric antigen receptors (CARs), these hunters bind to specific proteins and reprogram the T-cells to destroy the cancer,” the Hadassah website described.
The treatment is usually administered alongside chemotherapy.
Until now, few Israelis with multiple myeloma had access to such a treatment, as it was not offered in the country. The only options were to fly to China – if approved – or the United States. But only American citizens could be treated in the US, and the cost was up to $400,000.
Stepensky said the price tag for the Israeli treatment will be much less.
Next steps: the team is planning to test up to 60 patients and then move on to Phase II and Phase III trials, if all goes well.
Moreover, explained Cohen, there are many other applications for CAR T treatment, including for use against the rare disease Amyloidosis, which causes a buildup of an abnormal protein called amyloid in organs and tissues throughout the body. The deposits lead to organ and tissue failure.
“It is not easy to make the jump from bench to bedside, and we did it,” Cohen said. “If it goes the same way it went in mice – or even half as well – we will be able to save many lives.”
Stepensky added: “My aim is that this is only the beginning.”