Hadassah offers the world’s first CAR-T cancer outpatient treatment
The therapy was developed at Hadassah to treat Multiple Myeloma, AL Amyloidosis and other BCMA-positive malignancies.
The treatment involves altering the genes inside specific immune cells so that they can identify and destroy cancer. The therapy is available only in two other countries in the world (the US and China), and, as Prof. Stepensky stressed, it has been yielding amazing results.
An Israeli cancer treatment is en route to becoming the world’s first and only outpatient CAR-T for multiple myeloma, AL amyloidosis and other BCMA-positive malignancies.
Prof. Polina Stepensky at the Jerusalem Post Women Leaders Summit in February 2023. Photo credit: MARC ISRAEL SELLEM.
Prof. Polina Stepensky, Dr. Shlomit Kfir-Erenfeld and Dr. Nathalie Asherie of Hadassah’s Bone Marrow Transplantation Department, in collaboration with Prof. Cyrille J. Cohen of Bar-Ilan University, developed the CAR-T treatment for myeloma. At the end of last year, California-headquartered Immix Biopharma in-licensed the therapy, known as NXC-201, from Hadassah for $20 million in license fees and future milestone payments. It also agreed to sponsor ongoing clinical research and development at Hadassah for more than $12 million over the next four years.
NXC-201 has shown an 85% overall response rate and 71% complete response/stringent complete response at the therapeutic dose from the first 20 patients in an ongoing phase 1b relapsed/refractory multiple myeloma ongoing clinical trial.
The treatment, according to Stepensky, can prolong and significantly improve the life quality of people suffering from myeloma.
“The idea of CAR-T treatment was born in Israel in the 1980s but was implemented mainly in the United States,” Stepensky explained. “In the states, they developed a treatment for lymphoma. Here in Israel, we developed our treatment for myeloma. China is the only other country where this treatment is available at all.”
While Israel typically licenses such treatments from the United States, CAR-T is very expensive and not easily accessible. In the US, it can cost as much as $400,000 for a patient to receive this life-saving treatment. “This is why it was so important for us to develop something in Israel. Not only was it not always available, but it was costly,” she said.
Funded by Hadassah and the Women’s Zionist Organization of America, Stepensky led the charge to change the situation for her patients. As such, all the research, clinical trials and production in a certified Good Manufacturing Practice (GMP) facility are done at Hadassah.
“We treated 65 patients and we saw an overall response rate of more than 90%, and most of them were already after all kinds of therapies and not considered salvageable,” she said. “It’s really exciting to see these patients and to see how the tumor is going away. For our patients, it gives them another chance at life,” Stepensky said, adding that she has a lot of hope for the work that will be done in California.
“Now, they are going to start clinical trials in the US, and we really hope the results will be the same as ours in Israel,” she explained. “Then, we can submit the treatment for final approval by the Food & Drug Administration (FDA). After that, the European Medicines Agency usually follows.”
About Prof. Polina Stepensky
Prof. Polina Stepensky
“I told myself that I could still help patients by becoming a nurse because it’s a fantastic profession,” Stepensky shared, speaking at the Women Leaders Summit in Tel Aviv organized by The Jerusalem Post group on Wednesday.
After a few years working as a nurse, however, Stepensky’s passion for research inspired her to sit the test to enter medical school. She passed. Today, she is the Head of the Bone Marrow Transplant and Immunotherapy Department at Hadassah Hospital, offering CAR (chimeric antigen receptor) T-cell therapy for cancer patients.
Publication Source — JERUSALEM POST.