In an oncological milestone, an Israeli cancer treatment is en-route to becoming the world’s first and only out-patient CAR-T for multiple myeloma, AL amyloidosis and other BCMA-positive malignancies.
The experimental treatment, developed at Hadassah-University Medical Centre, has a 90% success rate at bringing patients with multiple myeloma – the second most common haemotalogical disease and considered incurable – into remission, say the scientists.
It is an “unprecedented achievement” in the treatment of multiple myeloma cancer, which accounts for one-tenth of all blood cancers and 1% of all types of malignancies, reports The Jerusalem Post.
The treatment was developed after a series of experiments carried out in the hospital’s bone-marrow transplant and immunotherapy department in recent years, based on genetic engineering technology, which is an effective solution for patients whose life expectancy was only two years until a few years ago.
They have used a genetic engineering technology called CAR-T, or Chimeric Antigen Receptor T-Cell Therapy, which boosts the patient’s own immune system to destroy the cancer.
More than 90% of the 74 patients treated at Hadassah went into complete remission, the oncologists said.
“We have a waiting list of more than 200 patients from Israel and various parts of the world at any given time,” said Professor Polina Stepensky, head of the department. “Due to the complexity of the production and the complexity of the treatment itself, only one patient a week enters the treatment, which is still being conducted as an experiment.”
According to Professor (emeritus) Yechezkel Barenholz, a world leader in oncology research and head of the membrane and liposome research lab at Hebrew University-Hadassah Medical School, the CAR-T technology is a major achievement that will make the diagnosis much easier and simpler, and treatment possible.
The CAR-T cell treatment was developed and produced by Hadassah in collaboration with Professor Cyrille Cohen, head of the immunology and immunotherapy laboratory at Bar-Ilan University in Ramat Gan.
“We have evidence of a very positive overall response rate with minimal side effects, and they are mild,” Stepensky said. “These are dramatic results. This is a huge hope for patients with a disease that has not yet had a cure.”
The experimental treatment will also be provided throughout the US in the coming months.
What is multiple myeloma?
Multiple myeloma is a type of cancer of the bone marrow, which is the spongy tissue at the centre of some bones that produces the body’s blood cells. The disease was named multiple myeloma because cancer often affects several areas of the body, including the skull, pelvis, ribs and spine. It is frequently suspected or diagnosed after a routine blood or urine test.
At first, it may not produce any symptoms, but as it develops, myeloma causes various problems, including chronic bone pain; weakness, shortness of breath and fatigue resulting from anaemia; high levels of calcium in the blood that can trigger symptoms, including extreme thirst, stomach pain, needing to urinate frequently, confusion and constipation; weight loss, dizziness, blurred vision and headaches; repeated infections, bruising and unusual bleeding; weak bones that fracture easily; and kidney problems.
The disease is more common in people over 60. It is usually diagnosed after 70 and rarely under the age of 40, in men more than women and in people with a family history of multiple myeloma.
“The American company IMMX Bio has acquired a patent licence, and we are about to open a clinical trial in the US,” Stepensky said. “The plan is to reach commercialisation and FDA approval as a drug within a year.”
The groundbreaking idea of using immune-system cells to fight cancer cells was born several decades ago at the Weizmann Institute of Science in Rehovot by Professor Zelig Eshhar’s immunology department.
The development and promotion of CAR-T treatments, whose function is to programme the patient’s white blood cells by collecting healthy cells from the immune system, has since been led by Stepensky.
As part of the treatment, a process is performed to isolate the T cells, which are the active cells in the immune system that can fight tumours by themselves.
This is carried out by apheresis, which takes donated blood components and separates the red and white blood cells. The process takes two to four hours and is similar to a regular blood donation.
The T cells are then engineered in the Hadassah laboratory, which was built especially for this purpose, according to the strictest international standards in clean rooms.
In the next step, a genetic engineering procedure is performed by adding a virus along with a genetic segment that encodes a receptor against the cancer cells. Many engineered cells are then injected into the patient. Ultimately, the engineered T cells target the tumours and destroy the cancer.
Until now, this treatment has been available only in China and the US for nearly $400 000 per patient treatment, and it is very limited in its availability.
“Only 20% of those who need to receive it in these countries actually get it,” Stepensky said. “With the development led by the researchers at our Danny Cunniff Leukaemia Research Laboratory, we were able to reduce the price dramatically and make the treatment affordable and accessible.
“Moreover, Hadassah developed a more sophisticated and advanced treatment than that offered in the world. As the first and only institution in Israel that develops, manufactures and delivers CAR-T treatment, Hadassah is actually leading the field that will enable the development of future treatments with CAR T cells for the benefit of patients with other types of cancer.”
See more from MedicalBrief archives:
Chip-based blood test for multiple myeloma may relegate bone biopsy
Cost of key cancer drug drops after generics victory
CAR-T cell therapy for leukaemia associated with less dangerous side effects
FDA approves CAR-T cell therapy for some large B-cell lymphomas