However, in perhaps the ultimate case of good news/bad news, the new medications that allow 90% of CML patients to look forward to a normal life span can cost $10,000 to $12,000 per month—every month for life.
Even with insurance covering part of the bill, copays can be $4,000 a month—which is more than most people earn.
To help patients cope with the challenges of living with CML, the Birmingham-based National Chronic Myelogenous Leukemia Society was founded as a grassroots effort to meet patient needs for information, education and support. It serves as an advocate with policymakers and an advisor to patients seeking access to medications they can’t afford, but need to stay alive.
For executive director Greg Stephens, founding the organization was a labor of love built on the experiences his mother Carolyn and their family encountered on their long personal journey with the disease.
“There was very little information we could find when she was diagnosed, and it was almost impossible to connect with other CML patients. There was no way to share information with others who were going through the same thing,” Stephens said. “CML is so rare, many doctors have never seen a case. Few physicians specialize in CML, and there were none in this area, so getting answers took some doing.
“On my mother’s bedside table, there’s still a worn blue spiral notebook full of notes on everything she learned about CML. She always said, ‘It may not help me, but someday it may help someone.’ That information was the starting point for “Carolyn’s Hope,” which grew into the National CML Society.”
Stephens likes to tell the story of how the tenacity of Dr. Brian Drucker led to the release of the first drug to target genetic effects of a particular cancer while leaving healthy cells unharmed.
“Dr. Drucker got tired of only being able to send patients home to get their affairs in order,” Stephens said. “After Dr. Janet Rowley identified the “Philadelphia chromosome,” a translocation of chromosomes 9 and 22, there was a target for attacking CML. Dr. Drucker became involved in research and determined the shape of molecule he thought might work. He persuaded Ciba Geigy to let him go through molecules on their shelf, and from several possibilities he identified STI-571 as the most promising.
“The problem was persuading someone to go forward with a trial. No one he approached seemed to be willing to invest resources on testing a drug for such a rare disease. Finally, he decided to do the trial himself. He received a supply of the inhibitor, and FDA permission for a trial on mostly hospice patients who were very ill.
“The response was so positive and immediate, the FDA rushed approval. Within a week, spleen size was back to normal in some patients. Within 28 days, blood counts were normal. Eventually, the molecular response progressed to the point that CML cells were not detectable even using polymerase chain reaction testing.”
Still, physicians speak of CML in terms of response, not remission. Unlike acute leukemia where remission can be achieved and patients no longer need treatment, CML hides and can return with a vengeance if medication is discontinued.
STI-571 came to the market as Gleevec®, the first of the tyrosine kinase inhibitors. Second and third line drugs have since been introduced for patients who become resistant or need an alternative due to allergies or side effects. As patients survive, more and more are taking these medications, and TKI drugs are also being prescribed for several other types of cancer.
Though the drugs are something of a miracle, they come at a price that may be difficult or impossible for patients to pay.
“Part of the problem is that the Drug Parity Act is buried in committee,” Stephens said. “Oral chemotherapy drugs are not covered by insurance the same way as their IV counterparts. Instead, they come under the prescription drug benefit, which in some cases means the total benefit for a year isn’t enough to cover one month’s prescription.
“Other policies won’t cover a second drug if the patient becomes resistant to the first. We just lost a young father whose policy would only cover a marrow transplant that didn’t go well. We’re also concerned about some states talking about limiting Medicaid coverage to only one brand of drug. They don’t seem to understand that not every drug works for every patient. If the drug that works for you isn’t covered and you can’t pay for it, it could cost you your life.”
The National CML Society puts patients in touch with drug companies that offer assistance to those who can’t afford their medication.
“Approval isn’t automatic. Insurance and other factors are considered. We try to help people understand the information they need to include with their application,” Stephens said.
The foundation’s patient care services are coordinated by Alisa Peinhardt, a nurse who has worked in clinical research at the NIH in Bethesda, Maryland. She spends much of her day helping patients access information and resources.
“Some of the most exciting work I get to do is with our Ask The Experts service,” Peinhardt said. “ Patients can submit any CML question online to our Medical Advisory Board, who are all well-recognized hematologists and CML experts. We can also help patients locate CML specialists in their area, find clinical trials, connect with social and economic resources and in general, navigate the healthcare system, which can sometimes be overwhelming for patients especially when first diagnosed.”
Peinhardt also keeps a close watch on emerging therapies, clinical trials and research that could benefit CML patients.
“Targeted therapies are extremely exciting as research continues,” Peinhardt said. “There are some very promising combination trials, such as those with zileuton at the University of Massachusetts and combining TKIs with interferon at the University of Michigan. Advances in transplant medicine will be very important as patients survive longer and for those who are diagnosed at a younger age. We’ve been in a high speed search for a cure since the first medications came along, and we aren’t slowing down. The science is just amazing.”
Perhaps one of the most comforting services the foundation offers CML patients is connectivity and community. The organization sponsors meet-up style events across the country where patients can talk and exchange information and support.
“It would have meant so much to my mom to be able to talk with others who were walking the same journey,” Stephens said. “She didn’t have an opportunity to meet another CML patient until shortly before her personal journey came to an end.”
Carolyn Stephens was among the small percentage of patients who didn’t get a good response to therapy. She went into an accelerated blast crisis and died in 2007.
“But right up to the end, she kept writing in that blue notebook, jotting down all the information she found and all the answers her doctors gave her,” Stephens said. “She kept saying, ‘It may not help me, but maybe it will help someone someday.’”
