New cancer treatment beats chemotherapy without the toxic side effects


June 28, 2013

Abnormal lymphocyte being attacked by macrophages (upper right), which lead to enhanced growth and progression of many cancers (Photo: National Cancer Institute)

Abnormal lymphocyte being attacked by macrophages (upper right), which lead to enhanced growth and progression of many cancers (Photo: National Cancer Institute)

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If a locked door must be opened, explosives can be used, but normally it is better to use a key. The conventional treatments for cancer, surgery, radiation, and chemotherapy, have a range of terrible side effects that resemble the use of explosives. Now a key has been found to treat various forms of leukemia and lymphoma with only very minor side effects. The drug ibrutinib has proven sufficiently safe and effective in early clinical tests by physicians at Ohio State University that it has been given breakthrough drug status by the FDA.

Both chemotherapy and radiation treatment protocols for cancer have one primary goal – design a treatment that is slightly more lethal to the cancer than to the patient. Chemotherapy began nearly 100 years ago, when mustard gas derivatives were studied following World War I. From this early start, serious research on chemotherapy agents for cancers began around 1950. While chemotherapy is still one of the key weapons to use in fighting cancer, a good deal of pharmaceutical and medical research is presently going toward more targeted agents to minimize the enormous stress of cancer therapy on the patient.

Leukemia and lymphoma are cancers of the blood. Leukemia is the proliferation of immature white cells in the blood, which leads to impairment of the immune system, blood clotting issues, and red cell anemia. Lymphoma is a similar proliferation of lymphocytes, which has similar symptoms, but can also involve solid tumors of the lymph nodes. In both cancers, complete cure is unlikely, but the disease can be managed in most cases by triggering partial or complete periods of remission through chemotherapy and/or radiation treatment. As these treatments are still painful and debilitating, and sometimes ineffective, new forms of treatment are an active area of research.

Ibrutinib is a member of a new class of drugs for use against B-cell blood cancers. B-cells are a type of white blood cell that is active in the body's immune system. They have B-cell receptor (BCR) proteins on the cell surfaces, which binds to specific bodily invaders, thereby allowing the immune system to attack the invaders. B-cells also act as part of the memory function of the immune system, keeping a chemical record of past targets of the immune reaction. Malfunctioning B-cells can cause autoimmune diseases as well as becoming cancerous themselves.

In B-cells, an enzyme called Bruton's tyrosine kinase (BTK) is an important stage in the B-cell maturation and activation of the BCR proteins. Among other functions, this BCR signaling is thought to drive the growth and well-being of many types of B-cell cancers. In short, B-cell cancers have a very difficult time surviving in the absence of BTK.

Ibrutinib is a strong covalent inhibitor of BTK, and in inhibiting BTK triggers B-cells to undergo apoptosis, or cell death, effectively blocking cancer growth and metastasis. Ibrutinib also shows activity in treatment of autoimmune disease, by throttling back the action of the immune system.

A new clinical trial carried out by medical researchers at Ohio State University Comprehensive Cancer Center in cooperation with MD Anderson Cancer Center, investigated the effect of ibrutinib in two groups of patients, one having confirmed recurring or resistant cases of chronic lymphocytic leukemia (CLL) and the other having confirmed recurring or resistant cases of mantle cell lymphoma (MCL). Neither study involved a control group or healthy patients, as the object was to determine the toxicity and efficacy of the ibrutinib treatments.

The CLL trial involved 85 ambulatory patients, 51 of whom received ibrutinib at 420 mg/day, and 34 who received 840 mg/day. Ibrutinib can be absorbed by mouth, so the doses were given orally. The lower dose group included a third group, originally excluded from the study because of high-risk genetic factors, who showed no response to chemoimmunotherapy. The early results of the clinical study proved sufficiently positive that exclusion of the third group was judged to be inappropriate.

Treatment with ibrutinib proved to be very safe, with most participants only encountering annoying side effects that did not require stopping the treatment. Only six patients were forced to halt treatment, primarily due to diarrhea and the associated dehydration, and upper respiratory tract infections.

The treatment was also very effective at stopping progression of CLL, and in most cases (71 percent) causing at least a partial remission. The results were independent of the doses used, which argues toward using smaller doses. The response was somewhat better (85 percent) in patients lacking high-risk genetic mutations. Overall, the 26-month survival rate was 83 percent, with little dependence of age or stage of the cancer at the start of participation in the study.

The MCL trial included 111 ambulatory patients, all of whom received 560 mg/day of ibrutinib. Nearly all of the patients had previously received multiple rounds of chemotherapy, and 86 percent had intermediate or high-risk lymphoma.

The ibrutinib treatment proved quite effective, with 21 percent of the patients experiencing a complete remission of MCL, and another 47 percent having a partial remission. The estimated total rate of survival at 18 months was 58 percent. Again, a remarkable feature of the trial is that ibrutinib helped nearly all patients to one extent or another.

“This is remarkable because the last agent approved by the Food and Drug Administration for MCL had a 30 percent response rate,” says senior author Kristie Blum, MD, associate professor of medicine, and head of the OSUCCC – James lymphoma program. “This trial suggests that ibrutinib could significantly improve the landscape of therapy options for MCL.”

The level of response found to ibrutinib could only be approached through conventional chemotherapy by intensive, multiagent regimes of treatment associated with very high toxicity. Ibrutinib is clearly on the fast track to approval for treating a range of B-cell cancers. Hopefully it becomes a standard therapy option before any reader needs such treatment.

Source: Ohio State University

About the Author
Brian Dodson From an early age Brian wanted to become a scientist. He did, earning a Ph.D. in physics and embarking on an R&D career which has recently broken the 40th anniversary. What he didn't expect was that along the way he would become a patent agent, a rocket scientist, a gourmet cook, a biotech entrepreneur, an opera tenor and a science writer. All articles by Brian Dodson

Dad died of chemo & lung cancer 5-18, I wonder if USC Norris Center knows of this treatment, or had in Use then? Or just research phase. Hope no future victims will undergo Chemo

Stephen Russell

Great article. It is refreshing to see an online writer give detailed information about a topic. There are some of us out there that want to know how the watch works not just what time it is.


I really hope this drug brings relief to all the sufferers of cancer out there, and I can only hope medical breakthroughs will continue for every disease.

Hasham Abbas

This sounds like a great key and a new route to cures. Fantastic.

The only thing about this article, and any other that deals with lethal diseases is this: 1) Quantity: How does the longevity granted with the medication compare with letting the disease run it's course? 2) Quality: How does the quality of life improve when compared with letting the disease run it's course?

I hear a lot about improvements in medical treatments, but with some diseases, is getting treated worth it? It's hard to tell without a statement of the baseline stats. I'm not advocating not being treated... but were I handed a diagnosis, I'd want to know what I'd be facing without treatment.

Ruth Vallejos

Great article. Humans as a collective spend billions on things like testing martian soil for traces of life. It would appear to me we do not have our priorities right. No surprises given human nature.


I would appreciate a point of contact regarding this research. I have nerucosarcoidosis, and I know that the sarcoid lesion that is growing inside of my spinal cord is a result of a faulty immune system. I'm not sure if I'm understanding the article correctly, but if there is even a slight chance that this could help, I would jump on it. Chemo drugs are nasty. Prednisone is almost as bad.


If they can put a patent on it, FDA will approve the treatment. Only expensive treatments are allowed. They prefer treatments with heavy side effects

Stewart Mitchell

What I need to know is which drug company will be distributing this new and wonderful drug. Hopefully it will be a small co. that needs a drug like this to push them over the top. So if you could please let me know who will be handling this drug.

Jerry Hyer

I do not recollect exactly the delivery system used but do remember reading an article in "Readers Digest" more than 30 years ago about a doctor in Germany. He had mastered the method of delivering chemo drugs precisely to the cancerous tumor ONLY with almost zero side effects. At that time the waiting period for getting admitted to his hospital was like more than a year. I wonder whatever happened later.


BTK is required for normal B cells too. Therefore, it is not clear that ibrutinib would do better than anti-B cell therapies. I have worked on B cell receptor and cancer, and now I consider that universal therapies might be better than "personalized medicine" for aggressive cancers.

Daniel Corcos

Stewart Mitchell said "If they can put a patent on it, FDA will approve the treatment. Only expensive treatments are allowed. They prefer treatments with heavy side effects."

This is grammatically poor and foolish. I am not sure who the subject of the conversation is - the "they". They could be patients, whom we can assume do not want treatment with heavy side effects, or it could be doctors - and for what possible reason would doctors want treatments with heavy side-effects? Basically, an insane comment.


Bigolfishy you asked about contact information. This link may help you with contact information. Copy this to your web brouser. My Doctor mentioned that I will be a candidate for ibrutinib in the near future. I go to the University of Penn Perelman Center for Oncology/Hematology. You could also contact the office of Dr. Peter Lebowitz from Janssen the affiliate company of Johnson & Johnson. I'm sure they would direct you to the correct physician. It may be a little work, but it would be worth it for the benefits of this drug.

Jerry - This drug is being handled by Johnson & Johnson Janseen.

pmshah - This drug comes in a pill form.

I'm very excited about this new drug, and I'm looking forward to it's release date. I have stage IV SLL, and this drug will postpone the use of chemotherapy for me. I will be able to avoid some of the extreme side effects of chemo.


Thank you Penni for your reply.

David Griffin

mom started this drug this week, I hope it works like they say it does!!!

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