Why do people participate in clinical trials?

There is exciting and encouraging news coming out of the ASH Conference in Atlanta with regard to new treatments for CLL. Sounds like chemotherapy will soon be a thing of the past for CLL patients because of Kinase Inhibitors (oral agents) like Ibrutinib and GS-1101 (formerly known as CAL-101).

My husband and mother-in-law participated in phase one trials for CAL-101. John began this drug in May 2010 after failing FCR (chemo). Marian began in October 2010 as first line therapy for her CLL. They are both doing extremely well in extended phases of the study. And my mother-in-law, who recently turned 80, was fortunate in having successfully avoided chemotherapy and the side effects. She still goes out dancing on a regular basis. And in a recent phone conversation, she told me how shocked a 72-year-old new friend was when the friend announced her age expecting to be older than Marian. My mother-in-law could pass for a woman in her late sixties. And she is living her life to the fullest, thanks to this drug putting her CLL in remission.

I am so thankful (and, okay, a little bit proud) that I was able to play an important role in her care and treatment decisions. Had she elected to receive local treatment where she lives rather than agreeing to come to Nashville and be cared for by Dr. Flinn, our CLL specialist, she would not have had the opportunity to participate in this trial. When she began treatment, she was developing anemia and her platelets were in a consistently downward trend. She looked fine. Some in our family didn't understand why she was entering treatment at all. But that is one of the challenges of CLL patients. Observers don't understand how sick they are when they don't look sick. But those of us who understand CLL know that treatment decisions often have to be made before the patient shows obvious outward signs of being sick.

I have been reading all morning about the ASH Conference in Atlanta. It's so exciting to see these major developments in CLL. When John began CAL-101, stem cell transplant was the option on the table. And because I was continually reading online about clinical trials, I knew about CAL-101 and aggressively urged John to consider the trial rather than going straight to transplant. Thankfully, he was open to the idea and so was Dr. Flinn. John's immediate response to the smallest dose given thrilled and surprised us all.

When Marian was in the hospital with pneumonia (possibly a side effect of her treatment, but also possibly a result of being misdiagnosed with bronchitis by her Evansville physician), I was asked by a family member why people do clinical trials. I tried to explain what an opportunity it was. And I was frustrated because it was apparent that the family member just didn't get it. Yes, there is risk involved. Not all clinical trials result in the kind of advancement that has come from Ibrutinib and GS-1101/CAL-101. But John didn't have another good option. And chemo could have killed Marian at her age. If her pneumonia was a result of treatment, it's pretty safe to assume that the side effects of chemo could have been life threatening.

When I saw this image on another CLL patient's blog this morning, my mind went back to the question of why people participate in clinical trials. 




http://bkoffman.blogspot.com/2012/12/surprising-results-for-ibrutinib.html


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