Jim Dean of Bellevue is a prostate cancer patient on a new vaccine called Provenge.
By Sean D. Hamill Pittsburgh Post-Gazette
For four years Jim Dean had been fighting the prostate cancer that had threatened to ravage his body when it was first found in an advanced state, already having spread to bone.
Despite that, he had been winning. Chemotherapy and hormone injections had kept the disease at bay and Mr. Dean, 62, of Bellevue, continued his work as a funeral director.
But this past spring, there were signs that the disease was fighting back.
"My doctor said, 'We're going to have to do something, go back on chemotherapy or something else,' " Mr. Dean recalls. "And right about then I saw on TV that the FDA had approved Provenge."
For nearly a decade, many men with prostate cancer had followed the slow progress of the U.S. Food and Drug Administration's approval process for Provenge. In April it became the first therapeutic cancer vaccine approved by the FDA after clinical trials showed it added, on average, four months to the life expectancy of men who took it.
But because of a host of issues surrounding the drug -- including its high cost, relatively short improvement in life expectancy and a limited supply for the next six months -- on Wednesday the Centers for Medicare and Medicaid Services, or CMS, are going to review the drug.
Exactly what CMS intends to review about the drug and what it intends to do is not clear. Will it try to pay less than the $93,000 Dendreon, its Seattle-based maker, is charging for the three needed treatments? Will it restrict who Medicare will allow to take the drug?
With 217,000 new cases of prostate cancer diagnosed annually, and 32,000 men dying from it every year, patients, their families and doctors alike are waiting to see what CMS decides.
"They will set the standard in terms of how much they're willing to pay and who gets to get the drug," said Ralph Miller, a urologist and director of the Allegheny General Hospital Prostate Center. "They have tremendous power in this."
Because of that power, what CMS decides will likely affect what private insurers decide to pay and whom they approve to take Provenge, too.
Although there was some concern when Provenge was first approved that private insurers would not cover its use because of the high cost, Pittsburgh-area doctors say they know of no cases where that has happened here.
And because the company said it could only produce enough doses for 2,000 patients in the first year, it was also feared some men who were eligible for it would not get it. But that also has not happened, doctors said.
"I'm not aware of any hardships of any patient who has wanted it but not gotten it," said Joel B. Nelson, chairman of the department of urology at UPMC.
Provenge works by boosting the immune system to fight prostate cancer, rather than attacking the cancer itself. The immune boosting approach has long been theorized, and with Provenge's approval it is touted as just the first of a new line of cancer drugs.
For each of three treatments, blood is drawn, the white blood cells are separated out and then incubated with a protein and an immune system booster, and then the treated cells are transfused back into the patient.
But the drug still only has provided a median of four additional months of life -- although a third of men who took it in trials were still alive three years later -- and it isn't the long-dreamed of cure that researchers are working toward.
That's why Dr. Miller said: "I think of it as evolutionary, as opposed to revolutionary.
"But this is unique, and it is a legitimately hopeful thing. But I don't think anyone thinks it's a home run."
Like many drugs, though, it is problematic, said Dr. Nelson.
"There is no way to assess response and no way to know necessarily who will benefit and who won't," he said. "So, from a drug company point of view, it's the perfect drug because you have to give it" to whoever is medically eligible.
John Lech, a urologist with West Penn Allegheny Health System, was part of the clinical trials for Provenge. That fact allowed his practice to be among the first in the country to have access to the drug, although Dendreon has since given UPMC patients access to it, too.
"This is not a cure," he said, "but compared to chemo, it's a heck of a lot better."
In addition to extending life, for most men Provenge has almost no side effects, unlike the typical side effects of chemotherapy -- nausea, hair loss, fatigue.
And all of that has meant that prostate cancer patients have been asking their doctors about the drug nonstop since it was approved.
"I had to tell some guys, 'No, I can't give it to you,' Dr. Lech said. "Some guys with bone pain, which makes them too weak from the cancer, that tends to exclude people.
"And this is not for everyone with prostate cancer, [but] only those with advanced disease. And that's my job: to weed out who is and who isn't eligible. That's the hard part."
He said he has had arguments with some patients, who bring in information or articles they've copied from the Internet about the drug, trying to persuade him to approve them for the drug.
He didn't have to argue with Mr. Dean.
The day he heard about Provenge's approval last April, Mr. Dean said: "I went straight to the Internet to research it," and he came away encouraged.
"You don't want to get too excited about it," Mr. Dean said. "But I brought it up to my doctor, and we agreed it was worth a look."
That resulted in a referral to Dr. Lech, who approved Mr. Dean to take Provenge because his prostate-specific antigen test had shown that Mr. Dean's PSA levels were rising for the first time in a while.
PSA is a protein produced by cells of the prostate gland, and the test measures how much of the protein is present in the blood. A higher result means the cancer may be growing, and Mr. Dean's PSA had risen from 1.7 to 7.
Four months later, Mr. Dean began the three treatments, finishing in October, showing no ill effects "other than having to have your blood drawn," he said.
As for the impact of Provenge: "All I know is it's supposed to work, and I can't tell till I'm further down the road if it worked for me," he said.
Mr. Dean has had much to deal with beyond his own illness in recent years. His wife's multiple sclerosis worsened. In the past two years both of his parents died.
And yet, he said, he couldn't help but laugh in June when friends and family jokingly called him to ask if he died because they heard that Jimmy Dean -- the country singer and sausage-maker -- had died.
"I haven't given up. Who knows how long I'll be around. But it won't be next week that I go," he said. "My philosophy is to live life to the fullest, but keep your bags packed."
While contemplating his own mortality, Mr. Dean, too, wonders about the questions that CMS will deal with Wednesday. Is it too expensive? Who should get it?
He has found himself giving two answers to such questions.
"Well, you get right down to it, I think 90 percent of the prices of medicine are outrageous," he said. "But, whenever you're talking about your own life, nothing is too expensive."