Targeted Cancer Drugs Keep Myeloma Patients Up And Running


by Richard Knox
February 18, 2013 3:34 AM

http://www.npr.org/blogs/health/2013/02/18/172098789/targeted-cancer-drugs-keep-myeloma-patients-up-and-running

Don Wright got diagnosed with multiple myeloma at what turned out to be the right time. It was 10 years ago, when he was 62.

That was at the beginning of a revolution in treating this once-fearsome blood cell cancer, which strikes around 20,000 Americans every year. The malignancy can literally eat holes in victims’ bones, which can snap from the simple act of bending over to pick up a package.

The first treatment Wright had was thalidomide, the sedative that caused awful birth defects in the 1960s. But it didn’t work for long.

Then he got into a study of a related drug called pomalidomide. Earlier this month the Food and Drug Administration approved the drug, which will be marketed as Pomalyst.

Don Wright was diagnosed with multiple myeloma in 2003 only days after completing his first marathon. Since then he has run 70 marathons in all 50 states.

Not only has the drug kept the Minnesota man alive far beyond the three or four years typical for newly diagnosed myeloma patients not so long ago. But it’s also enabled him to keep running marathons.
In the decade since his diagnosis, Wright has averaged seven marathons a year. He’s training for his 71st, which will take place in March on Cape Cod.

“Yesterday I ran 18 miles inside of a soccer dome,” Wright said recently, “and then came back and shoveled snow for 2 1/2 hours. That was a big day.”

Wright was one of the first patients to get Pomalyst, back in 2006. “For me this has been a miracle drug,” he tells Shots. “It’s kept the myeloma stable. And it’s just a little pill.”

Pomalyst is the latest of a half-dozen myeloma drugs developed in the decade or so since Wright got diagnosed. Another, called Kyprolis, won FDA approval last summer.

Like many so-called “targeted” cancer therapies coming out these days, the new drugs are enormously expensive. Pomalyst will cost about $10,450 a month, or more than $125,000 a year.

Dr. Brian Durie of the International Myeloma Foundation says the availability of these new drugs has been transformative.
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“It really has changed the whole landscape for both the doctor and the patient,” he says. “Ten or 15 years ago, it was very depressing to see a new myeloma patient because you knew that the life was potentially short and it was difficult to be overly optimistic about what might transpire.”

Wright is among the 20 to 30 percent of patients who have a less aggressive type of myeloma. But Durie says virtually all patients are benefiting from the new treatment options.

“We can say that 95 to 98 percent of the time we can pretty much guarantee we have a treatment that works — that the patient will go into a remission,” Durie says.

That’s pretty remarkable. But it’s not a cure. Eventually the first drug regimen — whatever it is — stops working and the cancer comes back.

But researchers think they’re on the verge of blocking myeloma’s return for good.

“We’re close, close, close,” says Dr. Ken Anderson of the Dana-Farber Cancer Institute in Boston. “We’re soon really going to change the natural history [of myeloma] in a major way.”

Don Wright shows the drug pomalidomide, which he has been taking as a participant in a clinical trial. Pomalidomide was recently approved by the FDA approved for treatment of multiple myeloma.

That’s doctor-speak for “cure.” Until specialists achieve that cure, they have an expanding bag of tricks to keep the cancer at bay.

New drug combinations and more novel drugs are coming along. Durie notes that scientists presented more than 700 papers on myeloma at December’s American Society of Hematology meeting.

In addition to the new medicines, many patients get stem-cell transplants in an attempt to obliterate nearly all the myeloma cells. But Anderson is leading an international study to determine whether the new drug regimens are so good that risky transplants aren’t needed any more.

“If somebody comes and sees us in the clinic,” Anderson says, “you can look them in the eye and say, ‘You know, it’s highly likely you’re going to live a decade or more. And frankly, with the maintenance therapy, it could be quite a lot longer.’ ”

Marathoner Don Wright has his own definition of a cure.

“The cure for myeloma,” he says, “is to hang on long enough to die of something else. And that is precisely my hope — and my plan, actually.”

You might say he’s in it for the long run.

* Solace in numbers

http://www.thestarnews.com/chula-vista/solace-in-numbers/

The Star-News

Solace in numbers

Sat, Jan 07 2012 12:00 PM Posted By: Allison K. Sampité

In October 2006, Guadalupe Moreno was taken to the emergency room for severe bone and kidney pain and a fever.  During the second day of her one-week stay at Scripps Hospital in Chula Vista, doctors told Moreno something that would change the rest of her life.

Moreno, now 64, was diagnosed with multiple myeloma, an incurable blood cancer where abnormal cells collect in the bone marrow and form tumors.

It’s the second most common blood cancer after lymphoma, affecting an estimated 750,000 people worldwide.

In July 2007, Moreno went to the University of California, San Diego, where she received a stem cell transplant.

“The transplant worked for 20 months — my cancer was in remission then it began slowly coming back and I started chemo again,” she said.

Moreno, like many people who are diagnosed with multiple myeloma, have never heard of the disease and think of it as melanoma, the leading cause of death from skin disease.

Years ago, multiple myeloma mostly affected men in their 60s and older, but today, diagnosis includes women and those younger than 30.

There are approximately 19,900 new cases of myeloma in the United States each year, according to the International Myeloma Foundation, which works to improve treatment options to improve the quality of life for patients.

Although multiple myeloma is treatable with medication, radiation and chemotherapy, the condition can cause bone pain, frequent infections, dizziness and fatigue.

Moreno takes steroids and two chemotherapy treatments in cycles, which helps minimize her pain, but causes side effects such as nausea.

While symptoms of multiple myeloma can be strong, they are not easy to detect at an early stage.

For some people like Elliot Recht, who was diagnosed during an annual physical 14 years ago, there are no symptoms at all.

Recht is one of two leaders for the San Diego Multiple Myeloma Support Group, but started off as a member when he was first diagnosed, taking over after group founder Fred Gloor passed away.

“I am very fortunate,” Recht said. “I’ve been pretty asymptomatic the whole time.”
Still, patients must manage their symptoms.

Because multiple myeloma affects plasma cells responsible for producing antibodies, it often affects the immune system, making patients more susceptible to infections, colds and viruses. It can also cause build-up of the M protein, which thickens the blood.
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Moreno explained the importance of washing her hands and dishes well and often to rid them of bacteria and how she can’t eat fruit or vegetables that grow underground because of pesticides.

When Recht was diagnosed, the life expectancy of patients was only three years, so he decided to stop working full time as a photographer.

“Why work so hard when you have such a short time to live?” he said.

Dr. Brian Durie, chairman of the foundation’s board, said that in the last decade, six new agents have been created to fight myeloma.

“Novel therapies are much better tolerated and work for a whole lot longer than chemotherapy does,” Durie said. “On average, life expectancy has doubled in the last decade from three to six years … allowing patients to return to a longer, fuller life.”

Chula Vista resident Lanorris Sewell was diagnosed at Scripps Hospital in Chula Vista in April after his back went out. Blood tests, a bone marrow biopsy and MRI confirmed he had multiple myeloma.

Sewell, 49, retired from the Navy in 2003 after 20 years working with chemicals to strip rust off airplanes. Prior to being diagnosed Sewell hadn’t heard of multiple myeloma.

“One thing I have learned about the cancer thing is that you have to stay positive if you want to beat it, otherwise you’re defeated,” he said.

Although the reason why people develop multiple myeloma is unknown, there are man-made risk contributors that include environmental pollution, stress and pesticides.

In Recht’s case, he grew up in Pittsburgh where he said he believes that air pollution, caused by the burning of coal and production of steel and iron, might have contributed to his condition, in addition to medal casting, oil paints, turpentine and clay.

“It’s a fine line between awareness and susceptibility,” Durie said. “Only a small percentage of those exposed have a genetic predisposition to it. People who do get myeloma aren’t able to break down these chemicals because they are slow metabolizers.”

Sewell and Moreno live on disability, unable to work, but attend support group meetings, which offer information on finances, give emotional support, and provide a better understanding of myeloma.

“It helps them realize that they are not alone in this battle and struggle,” Recht said.

“This is a type of cancer where education is the best possible thing.”

Moreno also attends meetings at Sharp Medical Center’s Leukemia and Lymphoma Society in Chula Vista.

“It’s good to learn and talk with other people that are like you and exchange your experiences,” she said. “I think when you have cancer, you have to fight to get well—you have to think positive.”

For information on support groups visit www.multiplemyelomasandiego.org.

* KPBS Radio Clip on Myeloma

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* A Miracle Drug Keeps This 70-Year-Old Cancer Patient Running Marathons

Don Still Going

http://gizmodo.com/5867859/a-miracle-drug-keeps-this-70+year+old-cance

Don Wright was diagnosed with myeloma—cancer in his blood cells and bone marrow—two weeks after running his first marathon. His doctor gave him a five-year survival estimate. Eight years later he has run 60 26.2-mile races in 41 states and takes just one pill per day to keep his cancer at bay.

“I feel wonderful,” Wright says. He and his family run the races together: his wife and daughter run half marathons while he does the whole shebang. His only complaint is a touch of runner’s knee every now and then.

Wright’s miracle drug is called pomalidomide, and it’s still in clinical trials. It’s one of several emerging therapies over the past decade that have doctors stunned and cautiously ecstatic about their effectiveness. Other drugs in this group include thalidomide (infamous for causing birth defects in the 60s) and lenalidomide, which are called immunomodulating agents. While not a cure, they could make treating cancer as relatively manageable as taking insulin for diabetes or a statin for high cholesterol.

Dr. Brian Durie, the co-founder and chairman of the International Myeloma Foundation, is amazed by Wright’s running stamina. “It’s mind boggling, for God’s sake. It’s amazing.”

The key, he says, is to stay on the treatment, just like taking insulin regularly to keep diabetes at bay. It’s a major and welcome shift from the comparatively shorter but heavy-hitting and damaging doses of chemotherapy and radiation that were the baseline treatments for myeloma 10 years ago. Most patients will take a lifetime of taking one pill per day over months of hair loss and severe nausea.

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Nowadays, most newly-diagnosed patients try one of these novel drugs since they work so well for some people. The problem is, the treatments are unpredictable and work differently for everyone. That’s why Dr. Durie and his colleagues are taking samples of patients’ bone marrow and using genetic profiling to determine who will respond best to which drug.

First we sequence the whole genome, then we break it up into smaller sequences about 200 nucleotides long. Then you sequence those, and you end up with literally millions of these little pieces. You line those up and compare the malignant cells with the normal cells.

By comparing these super-detailed sequences of DNA, Durie hopes to one day determine who will respond best to each drug on the list of novel treatments for myeloma.

Meanwhile, Wright hopes to keep taking pomalidomide for as long as it keeps working for him. At 70, he’s beginning to slow down, but only slightly. His goal is to run a marathon in every state—he’ll rack up one more this weekend in Rehoboth Beach, Delaware. He has already won multiple marathons in his age group, his fastest race being 3:36. That’s twice as fast as my fastest marathon, which I ran when I was half Wright’s age. Clearly he loves running more than I ever did:

“I just cruise,” he says. “I coast.”

* Doctors Tame One Of Cancer’s Deadliest Forms

Doctors Tame One Of Cancer’s Deadliest Forms

by Richard Knox

Listen Now   http://www.npr.org/templates/story/story.php?storyId=99970093

or download here http://planetcommunications.us/media/20090129_me_07.mp3

Documentary filmmaker Hardy Jones was diagnosed with multiple myeloma in his late 50s. New drugs have helped him keep the cancer at bay for more than five years.

Morning Edition, January 29, 2009 · The chances of surviving cancer have been increasing in recent years. One of the most dramatic success stories in cancer care involves a little-known cancer called multiple myeloma that had been among the deadliest types of the disease.

Myeloma is a painful bone marrow cancer that affects white blood cells that make antibodies. When these cells become malignant, they crowd out other bone marrow cells. That weakens bones so much that patients can break a bone just stepping off a curb.

Hardy Jones, a documentary filmmaker and recreational surfer, was diagnosed with multiple myeloma when he was almost 60. He knew something was wrong when he lost his usual energy.

“I just couldn’t get that stoked feeling, no matter what I did,” Jones says. “I was just always draggin’.” He could barely trudge up the hill from his favorite surfing beach, carrying his surfboard and wet suit.

His doctor ran some tests and mentioned that Jones might have cancer. While waiting for the results, Jones started reading up on possibilities. Some were scary.

“I vowed that I would go in there and no matter what the diagnosis was, I’d be cool,” Jones says. “And when the doctor said, ‘Well, it’s multiple myeloma,’ I said, ‘Oh, my God!’ I totally cracked. I wasn’t cool. But he said, ‘Stop it! Stop it! This is not a death sentence!’ ”

Not any more.

Fighting Myeloma With Drugs

Now there are easy-to-take drugs that can keep myeloma at bay for years.

“Everybody responds, and the majority have a very significant response,” says Dr. Ken Anderson of the Dana Farber Cancer Institute in Boston. “So it’s clearly a new day in myeloma.”

Anderson says the big breakthrough came a decade ago. Cancer research pioneer Dr. Judah Folkman of Harvard, who died last year, suggested that doctors try treating myeloma with thalidomide. The notorious drug caused an epidemic of birth defects when doctors prescribed it to prevent morning sickness in pregnant women 50 years ago.

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Successes like this touched off an explosion of drugs effective against myeloma, some related to thalidomide and others that work in a different way.

“The excitement here is that we have six new treatment options that we didn’t have only five years ago,” Anderson says. “And we have three additional treatment strategies that are in the last stages, so-called phase three clinical trials, that likely will create additional options.”

Having drugs to mix and match gives myeloma patients hope of remissions even after they relapse — something unusual in cancer treatment.

“It really offers for us the opportunity to treat patients even when their myeloma has come back not once, but perhaps even many times,” Anderson says.

Avoiding Bone Marrow Transplants

Doctors can’t yet cure myeloma, but they’re turning it into a disease that patients can live with for many years. The median survival rate has recently increased from about three years to seven or eight.

U.S. and French researchers are about to launch an international study to see if the new drugs are better than bone marrow transplants, which put patients through a grueling regimen of toxic chemotherapy. Officially, transplants are still the first-line treatment for myeloma, although many patients such as Jones are looking at transplants as a last resort.

“Now the question becomes relevant: Do you actually need a transplant?” Anderson says. “That question couldn’t even have been asked before.”

The picture isn’t all positive. Dr. Brian Durie of the International Myeloma Foundation says more patients are being diagnosed with the disease.

“In the United States, there are approximately 20,000 new patients diagnosed each year,” Durie says. “The incidence used to be 12,000 new cases a year. So it’s a significant upward trend.”

But Dr. Vincent Rajkumar of the Mayo Clinic says the increase in numbers is from aging of the population, not a real increase in the rate of myeloma.

There’s also debate about whether environmental toxins are causing myeloma.

“The commonest chemical that has been linked to myeloma is dioxin,” says Durie. That’s why some Vietnam veterans are thought to have gotten myeloma — from exposure to Agent Orange. The Department of Veterans Affairs has acknowledged the link.

But among the civilian population, the picture isn’t as clear. Some studies are emerging that civilians exposed to herbicides and pesticides do have a higher risk of myeloma. But it’s difficult to pin down people’s exposures to environmental toxins and relate those exposures to the incidence of a rare disease.

The bottom line is that experts say it’s too soon to conclude that environmental toxins are the cause.