Chemotalk Newsletter, Vol. 22: February 1, 2010
Hello again, and sorry for the earlier mishap. I decided there was no point to repeating the 2 pieces from the earlier iteration, since there is a huge amount of material out there that pertains to chemo; repetition would be a waste, I think... Anyway, the following pieces have NOT appeared here before:
At last, some positive news about one of the most aggressive, difficult to treat cancers:
PANCREATIC CANCER DETECTED BY BLOOD TEST
Study Shows Test for PAM4 Protein Can Reveal Early-Stage Pancreatic Cancer
Researchers say they have developed a blood test that can spot pancreatic cancer earlier, when it is more curable.
The test uses an antibody that works like a heat-seeking missile, homing in and attaching to cells that carry a protein called PAM4 that is present in the vast majority of pancreatic cancers. "This protein appears to be very specific for pancreatic cancer. It's [rarely] found in normal tissue or other cancers," says David V. Gold, PhD, of the Garden State Cancer Center in Belleville, N.J.
Importantly, PAM4 is also seldom detected in pancreatitis, a condition marked by inflammation of the pancreas that is initially often difficult to distinguish from pancreatic cancer, he says.
The antibody also shows promise for treating the disease by acting as a carrier for radiation or drugs that can target and kill pancreatic cancer cells, Gold says. The findings were released in advance of the 2010 Gastrointestinal Cancers Symposium, being held in Orlando, Fla.
Pancreatic cancer is the fourth leading cause of cancer deaths in men and women in the U.S. More than 42,000 new cases and over 35,000 deaths are expected in 2010 in the U.S., according to the American Cancer Society.
"This disease is a killer," Gold says. "Only 2% to 3% of patients will survive for five years." The reason is that most patients with pancreatic cancer are not diagnosed until the disease has spread throughout the body. "The goal of the new test is to provide a tool for the detection of early-stage disease," he says. If cancer is detected early, a patient's chance of surviving five years jumps to 20%.
Currently, only 7% of pancreatic cancer cases are detected at an early stage, before the cancer has spread.
The researchers first tried out the test on blood samples taken from nearly 300 people -- some of whom had pancreatic cancer, some of whom had other cancers, including breast and lung, and some of whom were healthy. "The test was positive in 77% of pancreatic patients, but only 5% of patients with other forms of cancer," Gold says. "Thus we know that if the [test] is positive, there is a large likelihood a patient has pancreatic cancer."
For the new study, the researchers evaluated the PAM4 protein test in 68 people who had pancreatic cancer surgery and 19 healthy people. The test correctly detected 62% of very early-stage pancreatic cancers that were still confined to the pancreas, 86% of cases that had spread only to nearby tissue, and 91% of later-stage cancers that had spread further throughout the body. Overall, the test correctly identified 81% of all pancreatic cancers.
Screening for Pancreatic Cancer
If the findings are validated in larger numbers of people, Gold foresees the test being used to screen people at high risk of pancreatic cancer. This includes people with long-term diabetes, those with chronic pancreatitis, people with a history of tobacco or alcohol use, and those with a family history or genetic factors that place them at increased risk, he says. Also, "if a doctor suspects pancreatic cancer, the test could be used to distinguish between various types of cancer and healthy tissue," he says. The test could also be used to monitor patients who have undergone treatment for signs of recurrence. Gold predicts the test will be available in two to three years.
In a separate study of 21 people with advanced pancreatic cancer, the researchers also tested whether the antibody could be used as a treatment to bring targeted agents to the cancer. The antibody is attached to radioactive isotopes and injected into the body. The idea is that once the antibody homes in on tumor cells, radiation is released, killing the tumor cells while sparing healthy tissue. In the study, tumors shrank in 23% of the patients and stopped growing in an additional 45%, says Gold.
Robert P. Sticca, MD, of North Dakota School of Medicine and Health Sciences, is very enthusiastic about the possibilities. "If we had a blood test that could detect cancer early, we could better manage our patients and we would have few deaths. The added benefit of using it as a treatment option is also very exciting,"
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EXPERIMENTAL DRUG MAY HELP IN BRAIN, PROSTATE CANCERS
An experimental drug called imetelstat shows promise in treating glioblastoma BRAIN CANCER and PROSTATE CANCER, according to the results of preclinical studies in which the drug was tested on human prostate cancer cells and in rodents with glioblastoma.
Researchers from the University of Texas Southwestern Medical Center found that the drug had an effect on most tumor cells, as well as cancer stem cells believed to cause most of a cancer's growth. Tests in mice with glioblastoma also showed that the drug was able to cross from the bloodstream into the brain. Most drugs cannot cross the blood-brain barrier.
The glioblastoma study is published in the January issue of Clinical Cancer Research, and the prostate cancer study was published in the International Journal of Cancer.
Imetelstat (also called GRN163L) is being tested in clinical trials as a treatment for BREAST CANCER, LUNG CANCER, and LYMPHOCYTIC LEUKEMIA. The drug targets a mechanism that allows cancer cells to continue dividing.
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And in another piece about another drug:
EXPERIMENTAL DRUG SHRINKS BREAST TUMORS
An experimental drug can slash the number of chemotherapy-resistant cancer stem cells in women with advanced BREAST CANCER, curbing tumor growth, researchers report.
The findings are in line with the latest theory of what causes cancer, namely that cancer stem cells hiding within tumors fuel their growth. Conventional treatments fail to cure cancer, according to the theory, because they are targeting the wrong cells.
The new compound, called a gamma-secretase inhibitor or GSI, reduced the population of cancer stem cells in 35 women with advanced breast cancer, shrinking their tumors, says Jenny Chang, MD, of Baylor University in Houston. At the San Antonio Breast Cancer Symposium (SABCS), Chang showed a before-and-after photo in which one could see a large breast tumor in a woman who had failed to respond to all other drugs shrinking after treatment with GSI.
All stem cells -- regardless of their source -- share some general properties: They can reproduce and make exact copies of themselves, they live longer than ordinary cells, and they can give rise to other cells in our bodies.
Cancer stem cells are a perversion of other adult stem cells. Chang says that fewer than 10% of breast cancer cells have stem cell properties, but that it is this small number that continually reproduce and fuel tumor growth. "If you can get rid of the 'mother cells' so they can't have offspring, eventually the tumor will go away," said Baylor University's Ken Osborne, MD, who is president of SABCS.
In her first set of experiments, Chang took biopsies of breast tissue from dozens of women, before and after chemotherapy. The number of cancer stem cells shot up after chemo. Then, she injected the post-chemo tissue samples into mice with compromised immune systems. Tumors identical to those in the women rapidly formed.
Then the researchers identified a cellular pathway -- called the Notch pathway -- that regulates self-renewal of cancer stem cells. "These breast cancer stem cells are dependent upon the Notch pathway for survival," Chang says.
Chang theorized that shutting down the Notch pathway would deplete the supplies of cancer stem cells. So she then tested the new compound [GSI] which is known to block the Notch pathway in mice that grew human tumors. It worked. The number of cancer stem cells that would otherwise have remained dropped.
Now, Chang and colleagues are testing the drug in combination with a commonly used chemotherapy drug, in women with advanced breast cancer. So far, 35 women have been treated in the ongoing study. Tissue samples after treatment showed a dramatic drop in the number of stem cells. And after several rounds of treatment, tumors started to shrink. The chemo attacks the ordinary tumor cells, and the experimental compound goes after the breast cancer stem cells, Chang explains.
This study was funded by Merck & Co. which makes the experimental GSI used in the study.
Osborne says that Baylor researchers are also testing a Notch inhibitor in people with leukemia. "In the first patient, we saw a dramatic decrease in cancer stem cells. Then after two to three months, as the mother cells were depleted, the patient began to get better," he says.
William Gradishar, MD, a breast cancer expert at Northwestern University, says that while current therapies shrink advanced tumors, they eventually start growing again. "That's because they are not targeting these cancer stem cells. So this appears to be an effective approach," he says.
The next step is larger studies pitting the combination treatment against standard treatment in women with advanced breast cancer, Chang says.
Gradishar reported that adding the cancer drug Nexavar to standard chemotherapy extends the time until advanced breast cancer progresses. In the study of 220 women, tumors shrank in two-thirds of those treated with the Nexavar plus Taxol compared with about half of those treated with Taxol alone. Also, women given the combination therapy were stable for an average of 8 months vs. 5.6 months for those treated with chemo alone. Nexavar attacks tumors on multiple fronts, starving them of their blood supply, interfering with cell signaling that spurs tumor growth, and preventing cell division. It's already approved to treat liver and kidney cancers. Nexavar "may provide added benefit over what you might expect with chemotherapy alone," says Gradishar.
The study was funded by Bayer and Onyx, which make and distribute Nexavar.
In a third study, researchers reported that a higher dose of the anti-estrogen drug Faslodex works better than the standard dose in postmenopausal patients with advanced breast cancer. Importantly, women given the higher dose did not experience more side effects than those given the standard dose, says Angelo Di Leo, MD, PhD, of the Hospital of Prato, in Italy.
Astra Zeneca, maker of Faslodex, funded the work.
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Who says movement in cancer drug development is slow?! Here's a third story of yet another new drug:
TARGETED BREAST CANCER DRUG SHRINKS TUMORS
Study Shows T-DM1 Helps Patients Who Were Unsuccessfully Treated With Other Drugs
A new targeted cancer drug has been shown to shrink tumors in women with METASTATIC BREAST CANCER after an average of seven other drugs, including Herceptin, failed. The new drug, called T-DM1, combines Herceptin with a potent chemotherapy drug. It's a Trojan horse approach, where Herceptin homes in on cancer cells and delivers the cancer-killing agent directly to its target.
Tumors shrank in one-third of women with metastatic breast cancer given T-DM1, says Ian Krop, MD, of the Dana-Farber Cancer Institute in Boston. In another 12%, tumors stopped growing for at least six months. The women remained cancer-free for an average of seven months -- results unheard of in patients this sick, he says. All the women, who had breast tumors for an average of three years, had cancer that had metastasized, or spread to other parts of the body. They had been treated with an average of seven different therapies, including Herceptin, Tykerb, and Xeloda, and each had failed.
"This is the first study looking at women who have failed so many other treatments," says Krop. "But we think these results are as good as we've ever seen is such a refractory [sick] population".
About 20% of breast cancer patients have HER2-positive cancers -- tumors that have too much of a type of protein called HER2. Herceptin, a man-made antibody, binds to and blocks the HER2 receptor that appears on the surface of some breast cancer cells. But metastatic breast cancer eventually becomes resistant to Herceptin. So researchers have searched for new drugs that target HER2.
T-DM1 is such a drug. The "T" stands for trastuzumab, the scientific name for Herceptin. The "DM1" is derived from an old chemotherapy drug called maytansine that was abandoned several decades ago when it was found to be too toxic for patients, Krop says. Because Herceptin only zeroes in on cancer cells that express HER2, DM1 is delivered only to those cells, he says. "The cytotoxic drug goes right to the cancer cells, so it's not floating around and causing other problems. And Herceptin still does all the things that Herceptin does" to fight cancer, Krop says.
All the women experienced some side effects, typically the fatigue and nausea often seen with chemotherapy, he says. Edith Perez, MD, a breast cancer specialist at the Mayo Clinic in Jacksonville, Fla., says the drug's benefits far outweigh the risks. "The response rate they saw in the study is exceptional in a group of patients this ill," she says.
One patient with pre-existing fatty liver disease and multiple other medical conditions died from liver failure. Perez says that the death due to liver failure is not overly concerning given the women's overall poor health. "These patients are very sick and right now we have no choices to offer them." In other ongoing studies, T-DM1 is being pitted against other cancer drugs in patients with both metastatic and earlier-stage breast cancer, Krop says. Researchers expect T-DM1 will perform even better in women with earlier-stage cancer, he adds.
The study was funded by Genentech and Hoffmann-La Roche, which are developing the new drug.
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ACTEMRA APPROVED FOR RHEUMATOID ARTHRITIS
Actemra (tocilizumab) has been approved by the U.S. Food and Drug Administration to treat RHEUMATOID ARTHRITIS among people who haven't responded to, or who cannot tolerate other approved RA drugs.
Actemra was approved for limited use because of adverse reactions noted in clinical trials, including the possibility of elevated liver enzymes, elevated "bad" cholesterol (LDL), hypertension, and gastrointestinal perforations. Actemra blocks an immune system protein called interleukin-6, which is found in excess in people with RA.
As a condition of the approval, Genentech, the drug's marketer, must conduct additional clinical studies to evaluate the drug's long-term effectiveness and cardiovascular safety, the FDA said. It also is requiring development of a Risk Evaluation and Mitigation Strategy for doctors, to inform physicians on how to monitor patients for possible liver or gastrointestinal side effects.
The most common adverse effects found in clinical testing included upper respiratory infections and other "serious" infections, headache, nasal and sinus inflammation, high blood pressure and increased liver enzymes.
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AMPYRA APPROVED FOR ADULTS WITH MS
Dalfampridine (Ampyra) extended-release tablets have been approved by the U.S. Food and Drug Administration to help adults with multiple sclerosis who have trouble walking.
In clinical testing, people who took Ampyra had faster walking speeds that those who took a placebo, the agency said. Some 400,000 people in the United States and 2.5 million globally have been diagnosed with the disease, the FDA said.
The drug, if given at doses higher than the recommended 10 milligrams twice daily, can cause seizures, the agency warned. The most common reported side effects include urinary tract infection, insomnia, dizziness, headache, nausea, weakness, back pain, nasal or throat swelling, irregularity, indigestion and burning or itchy skin.
People with moderate-to-severe kidney disease shouldn't take Ampyra, the FDA said.
The drug is marketed in the United States by Hawthorne, N.Y.-based Acorda Therapeutics.
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Here's a relevant story that appeared recently in The New York Times:
CANCER CENTER ADS, APPEALING TO EMOTIONS AT A FRAGILE TIME
By Natasha Singer
A print advertisement for prostate cancer surgery at Mount Sinai Medical Center in Manhattan is typical of the way many elite research and teaching hospitals sell hope to the public.
"Our newest prostate specialist, Dr. David Samadi, has pioneered a minimally invasive approach that allows him to retain the highest cancer cure rates with the lowest risk of side effects," says the ad.
Highest cure rates. Lowest risk. What evidence does the medical center have to back up such superlatives?
The Ad's claims are based on the successful results of Dr. Samadi's operations and testimonials from his patients, said Jane Zimmerman, Mount Sinai's chief marketing officer.
In medical science, such anecdotal data would not be considered statistically valid. But ads for nonprofit medical centers are not held to scientific standards of evidence.
"There seems to be a disconnect between the business end of the cancer treatment industrial complex and the physicians on the front lines treating patients," said Dr. John D. Birkmeyer, a cancer outcomes researcher who is a professor of surgery at the University of Michigan Health System.
Some medical centers take a similar approach to marketing their services in specialties like cardiovascular disease and cosmetic surgery. But cancer treatment advertising is particularly fraught with emotion, critics say, because it can play on fears about this disease.
If a drug marker ran an ad for a cancer medicine, Food and Drug Administration regulations would require the company to be able to support any superiority claims with substantial evidence from rigorous clinical studies.
But federal agencies cannot limit the ad claims made by nonprofit medical centers about their ability to cure people of diseases like cancer, according to the government's main ad regulator, the Federal Trade Commission.
Cancer experts interviewed for this article say there are no comprehensive statistics showing that any one elite medical center has better overall cancer success rates than its competitors.
Yet the advertising campaigns of prestigious cancer centers often use superlatives, promote the latest technologies, promise unique care or recount miraculous patient recoveries. Based on such ads, a consumer might reasonably assume that the medical profession has made more progress in the decades-long war on cancer than the more sobering facts would show.
`The problem with many ads is the implication that choosing a particular hospital could be the deciding factor in whether a cancer patient lives or dies, said Dr. H. Gilbert Welch, a medical professor at the Dartmouth institute for Health Policy and Clinical Practice.
People with some more complicated cancers or rarer diseases like leukemia do tend to fare better at comprehensive cancer centers. But Dr. Welch and others worry that such ads could persuade people with localized cases of more common diseases like prostate cancer to travel long distances from their families at great expense to obtain treatment that may be as successful, or unsuccessful, as the treatment available much closer to home.
And Dr. Welch said, the ads may exaggerate the benefits of cancer treatment, implying that a cure is certain.
But marketing executives defend their approach, saying cancer treatment ads tend to play more heavily on emotion than on medical statistics because the ads are not intended to inform people who already have the disease. They are meant to make an impression on future patients, who may decide on treatment years after they have seen an ad, or to sway influential people who might advise a future patient.
"This isn't retail advertising," said Ellis Verdi, president of the DeVito/Verdi Agency in Manhattan.
The agency produced the Mount Sinai ad, which ran in The New York Times, and has created cancer ads for other hospital clients. "This is reputation advertising," Mr. Verdi said. "There is a very big difference."
Ms. Zimmerman of Mount Sinai said the medical center's marketing campaign, including about 30 treatment ads for cancer and other diseases, highlighted the institution's expertise in treating complicated cases. All the information in the ads was accurate, she said.
In the case of the ads with the superlative prostate cancer claims, it was later revised to say that Dr. Samadi's approach gives high rates of success coupled with lowered risks of side effects." Ms. Zimmerman said Dr. Samadi was not available to be interviewed.
"We gave Nick something he couldn’t find anywhere else in the Northeast. Life without cancer."
That was the text of a print ad last year by the Massachusetts General Hospital Cancer Center in Boston, promoting its $50 million center for proton beam therapy, a kind of high-energy radiation to treat brain tumors and other cancers.
The hospital was the only medical center in the region with a proton therapy center, the ad said, enabling doctors there to successfully treat the brain tumor of a young man named Nick.
The ad's concept was that Nick had a greater chance of survival because the precise proton beam could destroy malignant brain tissue while leaving surrounding healthy brain tissue intact, said Jodie Justofin, the marketing director at Mass General's cancer center.
If the marker of a radiology machine had run an ad promoting its technology as superior to other ways of treating brain cancer -- or promising "life without cancer" -- F.D.A. rules would have required the company to be able to support the claim.
But Mass General has no such federal restrictions. And no rigorous studies have shown that proton beam therapy has higher brain-cancer cure rates than other treatment methods, said Dr. Birkmeyer of Michigan. "The ad might be accurate that they are the holy hospital in the northeast with this particular widget," he said. "But it could be misleading that the availability of this particular widget gave this patient better odds of survival."
Dr. Thomas F. DeLaney, the medical director of the Francis H. Burr Proton Therapy Center at Mass General, said he had no involvement in the ad and did not have any information about Nick.
But the advantage of the proton beam, Dr. DeLaney said, is that it delivers about 60% less radiation to normal tissue surrounding a tumor, allowing treatment of certain cancers -- particularly pediatric brain tumors -- that may otherwise be untreatable.
And officials at the hospital say there have not yet been rigorous comparative studies of proton beam therapy because the tumors it treats are extremely rare and, with children, there would be ethical concerns.
It seems like a miracle.
"Cancer. You said I'd never bear children," reads the handwritten letter, held out by a pretty, healthy-looking woman, as a toddler peeks from behind the paper. "My daughter says you're wrong."
That recent print ad from Memorial Sloan-Kettering Cancer Center in Manhattan tells the story of Michelle Rogala, a patient with cervical cancer.
Ms. Rogala's hospital in New Jersey could offer her only a hysterectomy, an operation that would have left her unable to have children. Instead, she went to Memorial Sloan-Kettering, where she entered a clinical trial that was studying less invasive surgery. Ms. Rogala now has a little girl named Maddie.
If a customer had given a testimonial about, say, her amazing weight loss at a for-profit clinic, the F.T.C. would have required the ad to clearly describe the results a more typical consumer might expect. But these federal rules do not apply to patient-testimonial ads for non-profit cancer centers, a spokeswoman for the commission said.
In a recent telephone interview, Ms. Rogala, a 37-year-old meeting planner in Monroe Township, N.J., said that hers had indeed been a special case.
She had early-stage cervical cancer, she said, making her eligible for a novel operation that has now become a standard treatment at the center. After her operation, doctors told her she would need fertility treatments to conceive. But she said she turned out to e one of the few patients in the study who did not need radiation -- which can cause fertility problems. She later became pregnant without medical intervention.
Ms. Rogala said she wanted her ad to inspire women with cervical cancer to investigate fertility-sparing surgery. But if a woman has more advanced cervical cancer, she said, "unfortunately, it may not be an option."
Ellen Miller-Sonet, vice president for marketing at Memorial Sloan-Kettering, said consumers seeing the ads realize that these were individual stories. "They know that no two people are the same," she said.
But Dr. Bruce A. Chabner, clinical director of the cancer center at Mass General, argues that treatment ads should avoid testimonials because any hospital could find a patient who had a positive experience. The nation's major cancer centers, he said, should establish collective advertising guidelines.
"Would I like to see those ads be more technical and less testimonial in their presentation?" Dr. Chabner said, referring to ads by cancer centers, including his own institution. "Sure I would."
Mass General's cancer center is "among the first hospitals in the nation to broadly test tumors for specific genetic mutations, helping us to personalize cancer treatment." So ran an ad, illustrated with a giant fingerprint, this year in magazines like U.S. News & World Report.
The ad promoted Mass General's genotyping research program, which tests lung and colon cancers for genetic mutations that may respond to novel drugs aimed at specific molecular targets.
The intent of the ad, said Ms. Justofin, the cancer's marketing director, "was to set Mass General apart as being on the cutting edge of offering the latest clinical trials."
But the ad did not explain that some patients might be treated with existing drugs while others would have to enroll in a clinical trial to be eligible for the experimental drugs.
The F.D.A. Does regulate clinical trials. And, had the ad been an overt solicitation of volunteers for a trial, F.D.A. Rules would have required a special institutional review board to vet it for balance on behalf of the potential research subjects.
Mass General's review board, for example, has guidelines saying that recruitment ads for research subjects should prominently display the word "research" and should not "'hype' the study."
But medical centers typically do not ask their review boards to vet ads that promote the general idea of access to clinical trials.
Dr. Steven Woloshin, an associate professor of medicine at the Dartmouth Medical School, said that because many clinical trials fail, cancer centers should not promote experimental treatments in their advertising campaigns aimed at consumers.
A few years ago, Dr. Woloshin an colleagues published a study that found that many ads "seemed to place the interests of the medical center before the interests of the patients."
Cancer treatment marketing seems all the more troubling because it could offer false hope to ill patients, said Dr. Lisa M. Schwartz, a co-author and an associate professor of medicine at Dartmouth.
"If you really wanted to give people real information, you would give them statistics," Dr. Schwartz said. "But that wouldn’t be nearly as compelling."
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In another, related story in The New York Times:
FACTORS TO WEIGH IN DECIDING WHERE TO GET CARE
By Natasha Singer
How should cancer patients decide where to receive treatment?
Many experts say that for common forms of cancer without significant complications -- a small prostate tumor that has not spread beyond the gland, for example -- a good community hospital may suffice.
Still, when friends or acquaintances pose the question, Dr. Bruce A. Chabner, the clinical director of the Massachusetts General Hospital Cancer Center in Boston, recommends that they go to a comprehensive cancer center.
Comprehensive cancer centers typically offer patients more treatment options, greater access to experimental drugs and more physicians who specialize in treating one particular kind of cancer than do community hospitals, Dr. Chabner said.
The National Cancer Institute maintains a list of cancer centers it has designated as leaders in integrating research and cancer care.
Moreover, for certain operations on rarer tumors, like esophageal cancer, studies have shown that hospitals that performed more of these operations had significantly better outcomes, said Dr. John D. Birkmeyer, a professor of surgery at the University of Michigan Health System.
But studies on surgical treatments for more common diseases, like a colectomy for colon cancer, have reported only marginal differences in outcomes between higher-volume elite hospitals and regular community hospitals, said Dr. Birkmeyer.
FIND A GOOD FIT. The American Cancer Society recommends choosing a doctor and a hospital with a lot of experience in treating your particular type of cancer.
Dr. H. Gilbert Welch, a professor of medicine at Dartmouth's Institute for Health Policy an Clinical Practice, recommended that patients ask their primary care physicians to find out whether a hospital or specialist they are considering has expertise and experience in treating their particular cancer.
CHECK CREDENTIALS. You may also want to ask about a doctor's credentials, like board certification in surgery, or whether a radiologist has a subspecialty in radiation oncology, according to the National Cancer Institute.
Patients may also ask whether the specialist has published scientific articles in their particular type of cancer, a sign that the physician is a leader in the field, said Dr. Thomas F. DeLaney, director of the Francis H. Burr Proton Therapy Center at Mass General.
AN UP-TO-DATE DOCTOR. Dr. Chabner also recommended that a patient ask whether a doctor has a teaching appointment at a medical school and conducts research, indications that they physician is up to the latest developments in the field.
THE HUMAN TOUCH. Patients, Dr. Chabner said, should choose a doctor who seems comfortable communicating with them, a particularly important skill when discussing cancer treatment options.
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GIVE THESE DONORS A BONE
By John Wagner and Jeff Rowes*
Every year, more than 100,000 Americans discover that they have often life-threatening blood and bone-marrow diseases like leukemia. For many, the only hope is a transplant of blood-producing marrow cells.
Finding someone to donate the marrow is challenging, though, because the cells must be a near-perfect genetic match with the patient's own cells, and those are hard to find. Even siblings have compatible marrow cells only 30% of the time. Most patients must search nationally and internationally for potential donors.
Only 7 in 10 Caucasian patients who need a donor find one. For African-Americans, the odds are longer still; only one in four do. Tens of thousands of Americans have died for lack of a donor.
It would make sense to encourage donation by offering potential donors an incentive -- a gift to a favorite charity, for example, or a scholarship. But federal law forbids doctors, nurses or dying patients to offer any incentives. The intent of the 1984 law, the National Organ Transplant Act, was to prevent the sale of human kidneys for transplant, out of concern that a market in organs could tempt people to risk their health for money by making an irreversible decision to be a donor.
But with marrow donation this is not an issue. Unlike organs, marrow cells -- basically, immature blood cells -- are renewable. The body grows fresh ones quickly enough to replace those extracted for transplant in about a month. And donating marrow cells is now very safe -- in most cases, it's simply a matter of drawing blood from the donor's arm and running it through a machine that skims off the marrow cells. Well under half of donations are conducted the old way, by harvesting marrow cells from the donor's hip.
Interestingly, Congress didn't bar compensation for all human donors. In writing the l984 law, it excluded renewable cells like blood or sperm from the payment prohibition, even as it inexplicably included bone marrow.
We have filed in federal district court a constitutional challenge to the marrow prohibition, because we want to set up a pilot program to ascertain the extent to which certain strategic incentives -- a $3,000 scholarship, a housing allowance, a charitable gift -- could increase marrow-cell donations.
If our suit is successful and incentives are allowed, it would not create a free-wheeling market in bone marrow donation. Marrow donation would, and should, remain anonymous -- and there should be no negotiation with donors. There would be no buyers or sellers, no possibility of market-like transactions.
But people who provide life-giving marrow cells could, in good conscience, get something in return for helping save a life.
* John Wagner is a professor of pediatrics and the director of the blood and marrow transplant program at the University of Minnesota. Jeff Rowes is a senior lawyer with the Institute for Justice, in Arlington, Va.
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Finally, in case we forget how much politics has to do with health care issues, a final piece from a recent (12/15/2009) issue of The New York Times:
PLAN FOR KIDNEY DRUGS SPURS DIVISION
By Kevin Sack
A Congressional proposal to help pay for drugs needed by transplant recipients to prevent rejection of donated kidneys has run into opposition from dialysis providers, drug companies and the National Kidney Foundation.
A groups have emphasized that they support extending Medicare coverage for the drugs. But they oppose the method Congress has identified to pay for expanded coverage for the drugs. But they oppose the method Congress has identified to pay for expanded coverage, saying it would help transplant recipients at the expense of dialysis patients by effectively reducing government reimbursements for dialysis.
The proposal, put forward by leading Democrats, would extend Medicare coverage of immunosuppressant drugs beyond the current limit of 36 months after a kidney transplant. To pay for that extra coverage, a change would be made in the formula used to reimburse dialysis treatment.
The proposal has created a rift between those in the business of providing dialysis and those in the business of performing transplants. The discord is being felt on Capital Hill, and supporters of the measure fear it may make it easy for Congress to kill the provision altogether in the late stages of negotiation.
On Saturday (12/12/2009), a coalition of drug makers, dialysis providers and nephrologists known as Kidney Care Partners informed senators in a letter that "the kidney care community strongly objects" to the payment method.
But the American Society of Transplantation actively supports the provision and fears that opposition from the kidney groups may arrest its momentum.
Although Medicare is primarily an insurance program for older Americans and the disabled, it has since 1973 covered those with end-stage renal disease, regardless of their age or condition.
The federal program now pays for most costs associated with dialysis and transplantation. But for patients younger than 65, coverage of the anti-rejection drugs -- which can run from $1,000 to $3,000 a month -- ends after three years. If patients cannot afford the medications, they may lose their donated kidneys and have to return to dialysis while awaiting another transplant.
The policy is widely regarded as pound-foolish. Medicare spends an average of $17,000 a year on kidney transplant recipients, most of it for the anti-rejection drugs, compared with $71,000 a year on dialysis patients and $106,000 for a transplant.
"It's like buying someone a new car and giving them only enough gas to drive around the block a few times," said Dr. David J. Cohen, medical director of the kidney transplant program at Columbia University Medical Center.
The health care bill that passed the house that passed the House last month includes a provision that would provide Medicare coverage for immunosuppressant drugs for all beneficiaries for life, starting in 2012.
To pay for the expanded coverage, House Democrats embraced a proposal that would set a flat fee for dialysis treatments and related medications that some providers say would not cover costs. The Congressional Budget Office has calculated that the package would save the government $100 million over the next 10 years.
The Senate majority leader, Harry Reid of Nevada, did not address immunosuppressant drugs in the bill now being debated on the Senate floor. But the second-ranking Democrat in the Senate, Richard J. Durbin of Illinois, last week submitted an amendment that replicates the House language, giving the provision a badly needed lift.
Senate democratic aides said there was little chance the amendment itself would receive up or down vote. But they said Mr. Durbin hoped to have it included in a package of loose-end amendments, supported by Mr. Reid, to be presented for a single vote at the end of the debate.
Dialysis providers argue that they bundled-payment mechanism may force some clinics to close. "We believe that the amendment as proposed, while helping one group of patients, would potentially put another very vulnerable group of patients at risk," said the letter from Kidney Care Partners, which was signed by its chairman, Kent J. Thiry, the chief executive of DaVita, a large commercial dialysis provider.
The group instead proposed to pay for expanded drug coverage by delaying when Medicare would start covering kidney patients who also have private insurance.
The National Kidney Foundation, the largest advocacy group for renal patients, signed on to the letter because it shares the group's concerns about bundling of payments. But Ellie Schlam, the foundation's spokeswoman, said its support for extending coverage for anti-rejection drugs would ultimately outweigh ts opposition to bundling.
Shortly after the House legislation passed, 40 Democrats in that chamber wrote to Speaker Nancy Pelosi to express concern about the bundling payment method. Many are members of the black or Hispanic caucus, and the letter cited kidney disease's disproportionate impact on members of minorities.
Three-fourths of the signers had also accepted recent campaign contributions from dialysis providers and drug makers. That included 26 who had received donations in the last three years from Amgen, a California company that makes Sensipar, one of the drugs that would be in the new reimbursement bundle.
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