An experimental treatment developed at Israel’s Hadassah-University Medical Center has a 90% success rate at bringing patients with multiple myeloma into remission.
By JUDY SIEGEL-ITZKOVICH, JPOST)
Hadassah-University Medical Center in Jerusalem’s Ein Kerem has announced an “unprecedented achievement” in the treatment of multiple myeloma cancer – the second-most common hematological disease. It accounts for one-tenth of all blood cancers and 1% of all types of malignancies.
The innovative treatment against the disease, which has long been considered incurable, was developed after a series of experiments carried out in the hospital’s bone-marrow transplant and immunotherapy department in recent years.
“We have a waiting list of over 200 patients from Israel and various parts of the world at any given time.”
Polina Stepensky
“Now, in light of the impressive results of CAR-T treatments, it seems that they have many more years to live – and with an excellent quality of life,” said Prof. Polina Stepensky, head of the department.
The treatment is based on genetic engineering technology, which is an effective and groundbreaking 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,” Stepensky said. “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 Prof. (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 Prof. 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 the blood cancer known as multiple myeloma?
Multiple myeloma is a type of cancer of the bone marrow, which is the spongy tissue at the center 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. Many times, it is suspected or diagnosed after a routine blood or urine test.
At first, it may not produce any symptoms, but as it develops, myeloma causes a wide variety of problems, including chronic bone pain; weakness, shortness of breath and fatigue resulting from anemia; 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 the age of 60. It is usually diagnosed after the age of 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 license, and we are about to open a clinical trial in the US,” Stepensky said. “The plan is to reach commercialization 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 Prof. Zelig Eshhar’s immunology department. The development and promotion of CAR-T treatments, whose function is to program 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 tumors 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 tumors 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 Leukemia 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,” Stepensky said.
@Edgar
Now this is an important qualifier. Unexplained weight loss is often a consequence of cancer, though not exclusively so, of course. Normal cells reproduce in accordance with the body’s need and the energy requirements of the cell are met by breaking down carbohydrates (sugars). Cancer cells, however, reproduce without any regard to the body’s need. The increased cell reproduction causes an increased need of food for the cell. So the cancer cells stimulate new blood vessels to carry more food to it, but it also stimulates the body to break down muscle tissue. The cancer cells undergo a conversion process, the mechanism for which is not fully understood, but the outcome leads to the cancer cells preferring the energy source of protein rather than carbohydrates. This is part of the process called cachexia, and it is responsible for the unexplained weight loss which is often associated with cancer.
I don’t want to unnecessarily alarm you, as unexplained weight loss can be caused by diabetes, gastro-intestinal disorders, kidney issues and a wide host of other conditions besides cancer. The combination of unexplained weight loss and AUE together, however, would lead me to place cancer higher on the possible list of causes to investigate further, which I am sure is already being pursued in the case of your friend.
Regarding the research in the article above, it is currently specifically for multiple myeloma, which is a particularly harsh form of cancer, but it is being considered for research in the treatment of other cancer types. Bear shared an article on the CART-T research about two years ago, and I am glad to see that the research is still being funded and resulted in such successful results.
PELONI-
Thank you for your post. To me it sounds as if he was describing AUE. He has had unexplained weight loss also. He is not old, most likely in his mid 50s; a pediatrician of excellent qualification, and likely with many colleagues at hand. I sent him the announcement in Arutz Sheva which I see reprinted above.
Some great confidence here. I sent it in case it was a blood cancer,
@Edgar
Anemia is a condition in which the red blood cells are lower than normal and its causes are quite varied. Among the anemias, however, exists a subset of cases which defy explanation, ie the cause is not determined even after a thorough diagnostic examination of the blood, the patient, and the patient’s history. These are called anemia of unknown etiology, or AUE. I am not sure if this is what was intended to be relayed to you by your friend, or if he meant that he has yet to have his case fully worked up, in which case he may simply not know what the cause is, yet.
With regards to anemia and cancer, the two are not the same, though cancer can cause anemia, directly and indirectly. Anemia means there exists a deficiency in the red blood cells, while cancer means that there is some tissue, organ or cell type which is growing while defying physiological regulatory mechanisms, ie the cells just keeps reproducing without being prompted to do so. There is nothing in the statement that your friend related to you, or as you have described it here, that would actually suggest to me that he has cancer, just that he has anemia, either AUE or an anemia which is thus far undiagnosed.
AUE are common among the elderly, more so among elderly women, but it can present at any age. Also, the cause may be determined after treatment, on post mortem or not ever. Anemia in general can be caused by a toxin, parasites, organ disease (eg kidney or liver), genetics, malnutrition, malabsorption (eg, reduced absorption of iron or vitamins), vitamin deficiency, immune disorders, chronic disease, and these are just a few of the more obvious ones which come to mind. The diagnosis of AUE is derived after a full work up (extensive lab tests and history and blood cytology) without finding an actual cause for the low blood cell count.
The effects of anemia is that the patient will experience a regularly elevated heart rate, quickly exhausted (often referred to as exercise intollerance), and having a pale complexion.
Unfortunately, anemia is caused by a great many things, and sometimes our best diagnostic tools evade the detection of what the cause might be.
Since the cause of AUE is unknown, by definition, the treatment is usually symptomatic. One of the causes of AUE is reduced iron for an unknown reason. Among the possible reasons is malabsorption as stated above, so iron shots (very painful) can be administered. Dietary iron supplementation can be used if malabsorption is not the cause, but it takes a long time for the dietary supplements to build up in the system. Sometimes, the problem is actually a deficiency in a hormone called erythropoeitan (EPO) which is responsible for triggering the development of new blood cells. Supplementing with a drug which mimic EPO, ie EPO replacement therapy, is another possible treatment employed in treating AUE. A problem arises when a patient is either non-responsive to EPO therapy or when they are decreasingly responsive to the therapy over time. Transfusions can be employed to bump up the red blood cells if the anemia is severe, but the transfused cells are often depleted at a quicker pace than normal – the upside to this is that the depleted red blood cellls allow the patient to have a ready source of iron if iron deficiency is a possible cause of the AUE.
In any event, anemia is a pretty broad topic, and I hope I haven’t confused you too much. Let me know if you have any other questions.
Does this cancer discovery include cancers of the blood, anemia etc. My good friend a doctor working in a foreign country yesterday emailed me that he suddenly found he had an unidentified anemia, I am medically deficient so I assume it is a kind of blood cancer.
Does anyone have an answer for me??