Chemotalk Newsletter

Chemotalk Newsletter, Vol. 61: May 1, 2013

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Hello, Spring...Finally!

Yesterday, I did something I always suspected I might, which is erase an entire month's work by pushing the wrong button.  Fortunately, I can re-create most of May's newsletter.  Only one story is gone for good.  In it's place, I'd like to offer a personal experience that might be worthwhile for anyone curious about colonoscopies.

I had a virtual colonoscopy last month, 2 years after a colonoscopy that couldn't be completed.  A colonoscopy under anesthetic is covered by Medicare; a virtual colonoscopy is only covered by Medicare if the procedure, like my first one, can't be completed under anesthetic.

After the "virtual", the doctor asked me if I'd like to see the results.  A ride through my colon...what could be more fun!  To make this personal adventure interesting to readers, I'll just offer my conclusion: the virtual colonoscopy is the more accurate procedure.  It is also by far the cheaper of the two processes, which is why it is vigorously opposed by some doctors who would make a lot less money if virtual colonoscopies became the norm.

Bottom line: if you need a colonoscopy, and if insurance isn't an issue, ask for the virtual version.

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Now, a truly courageous action by a group of concerned medical professionals:


By Andrew Pollack

With the cost of some lifesaving CANCER drugs exceeding $100,000 a year, ore than 100 influential cancer specialists from around the world have taken the unusual step of banding together in hopes of persuading soe leading pharmaceutical companies to bring prices down.

Prices for cancer drugs have been part of the debate over health care costs for several years -- and recently led to a public protest from doctors at a major cancer center in New York.  But the decision by so many specialists, from more than 15 countries on five continents, to join the effort is a sign that doctors, who are on the front lines of caring for patients, are now taking a more active role in resisting high prices.  In this case, some of the specialists even include researchers with close ties to the pharmaceutical industry.

The doctors an researchers who specialize in the potentially deadly bloo cancer known as CHRONIC MYELOID LEUKEMIA, contend in a commentary published by a medical journal that the prices of drugs used to treat that disease are astronomical, unsustainable and perhaps even immoral.

They suggested that charging high prices for a medicine needed to keep someone alive is profiteering, akin to jacking up the prices of essential goods after a natural disaster.

"Advocating for lower drug prices is a necessity to save the lives of patients" who cannot afford the medicines, they wrote in Blood, the journal of the American Society of Hematology.

While noting that the cost of drugs for many other cancers were just as high, the doctors focused on what they know best -- the medicines for chronic myeloid leukemia, like Gleevec, which is enormously profitable for Novartis.  Among the critics is Dr Brian Druker, who was the main academic developer of Gleevec and had to prod Novartis to bring it to market.

Novartis argues that few patients actually pay the full cost of the drug and that prices reflect the high cost of research and the value of a drug to patients.

Gleevec entered the market in 2001 at a price of about $30,000 a year in the United States.  Since then, the price has tripled, it said even as Gleevec has faced competition from five newer drugs.  And those drugs are even more expensive.

The prices have been the subject of intense debate elsewhere as well. The Supreme Court in India ruled recently that the drug could not be patented, clearing the way or use of far less expensive generic alternatives.

Some of the doctors who signed on to the commentary said they were inspired by physicians at the Memorial Sloan-Kettering Cancer Center in New York, who last fall refused to use a new colon cancer drug without being better (See Chemotalk Newsletter, Vol. 55, November 1, 2012).

After those doctors publicized their objections in an Op-Ed article in The New York Times, Sanofi, which markets Zaltrap, effectively cut the price in half.

What impact the new commentary will have remains to be seen.  The authors, however, call merely for a dialogue on pricing to begin.

The leader of the protest is Dr. Hagop M. Kantarjian, chairman of the leukemia department at the prestigious MD Anderson Cancer Center in Houston.

Many of the roughly 120 doctors who were co-authors of the commentary -- about 30 of whom are from the United States -- work closely with pharmaceutical companies on research an clinical trials.  They say they favor a healthy pharmaceutical industry but think prices are much higher than they need to be to ensure that.

"If you are making $3 billion a year on Gleevec, could you get by with $2 billion?" Dr. Druker, who is now director of the Knight Cancer Institute at Oregon Health an Science University, said in an interview.  "When do you cross the line from essential profits to profiteering?"

Gleevec's sales were $4.7 billion in 2012, maing it Novartis's best-selling drug.  A newer Novartis leukemia drug, Tasignam had sales of $1 billion.

Novartis said in a statement, "We recognize that sustainability of health care systems is a complex topic and we welcome the opportunity to be part of the dialogue."

It said that its investment in Gleevec continued after the initial approval, expanding the drug's use to other diseases.  It also said that it provided Gleevec or Tasigna free to 5,000 uninsured or underinsured Americans each year and to date had provided free drugs to more than 50,000 people in low-income countries.

Novartis and the manfacturers of the other drugs for chronic myeloid leukemia say the prices reflect the value of the drug.  While many cancer drugs with equally high prices extend life y only a few months on average, it is widely agreed that Gleevec and rivals are near-miracle medicines that essentially turn a death sentence into a chronic disease like diabetes.

"It is a little surprising that their focus is in a cancer where the small-molecule medicines have had the greatest impact on long-term benefit," said Dr. Harvey J. Berger, chief executive of Ariad Pharmaceuticals which sells the newest and most expensive of the leukemia drugs, Iclsig.

Dr Berger said the price of Iclusig was $115,000 a year, not the $138,000 a year cited in the commentary.  Pfizer said the price of its drug, Bosulif, also was overstated in the piece.  The manufacturers cite the price at which they sell to wholesalers, while the authors of the commentary were referring to a price they say better reflects what is charged by a pharmacy to patients.

The other drugs for chronic myeloid leukemia are Sprycel from Bristol-Myers Squibb and Synribo from Teva.

The commentary noted that despite drug company programs, a minority of the estimated 1.2 million to 1.5 million people in the world with chronic myeloid leukemia were receiving one of the drugs.  In many developing nations, it said, cancer experts were advocating risky bone marrow transplants because that is a one-time procedure that is cheaper than continuous treatment with one of the drugs.

The article also said the survival rate for patients in the United States appeared to be less than it should be, perhaps because costs are forcing patients to not tae their medicine.  Prices for the drugs are twice as high in the United States as in many other countries, which often apply some government pressure or price controls to keep drug costs down.

Even if out-of-pocket costs can be low, health systems in general still must pay for the drugs, the commentary says.  And some patients say assistance programs are not always easy to use.

Raven Riedesel of Winlock, Wash., said she had been turned down by various charities -- though she hadn't yet tried Novartis itself -- because her husband a pipe fitter, makes too much money.  Yet the insurance from his union would require her to pay $1.200 to $1.600 a month as a co-payment for Tasigna.

"It would take everything that we ha left over after buying necessities and paying our bills," said Ms. Reidesel, 28, a mother of two young children.  She is now in a clinical trial allowing her to obtain Tasigna free; the trial will end in November.

Patients in the United States circulated an online petition last year protesting the price of Gleevec, but the effort was dropped after receiving about 400 signatures.

Cheap generic versions could enter the American market as early as 2015 when the main patent on Gleevec expires.  Novartis might try to assert other patents to stave off competition, however.  It is also trying to shift patients to Tasigna, which has a longer patient life.

Dr. John M. Goldman, emeritus professor of hematology at Imperial College in London and a co-author of the commentary, said he knew several researchers who declined to become authors because they feared losing research money from the industry.

Dr. Kantarjian, the lead author, said that was a risk.

"I am sure I am going to be blackballed," he said.  "My research career will be hurt."

But he said it was time to speak out.  "Pharmaceutical companies have lost their moral sense," he said.  Prices, he added, "are getting to the point where it is becoming unsustainable."

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By Gina Kolata

Anyone in the United States who wants to know if she has mutations in two BREAST CANCER genes has little choice of where to be tested.  One company alone has patents on the genes, and that company pretty much controls the market.

The Supreme Court will take up the issue of whether companies can own patents on genes.  But there is another issue, often overlooked, that might make the patent question beside the point  No matter which way the patent decision goes, the company Myriad Genetics, will still own the largest database that tells patients what various mutations mean.

With 17 years of experience, millions of tests looking for thousands of mutations in the genes, and a $500 million investment, the company was able to amass a huge database that tells which DNA changes increase cancer ris and by how much, and which are inconsequential blips in DNA.  And it is keeping that data to itself.

Soe genetics researchers are furious and have now figured out a way to get the data anyway.  Every time Myriad sends out a report on a gene test, it specifies not just the mutations it found but also what they mean.  As a result, Myriad's data on each of the mutations is scattered in millions of reports in the hands of doctors and patients.  If the geneticists could just gather those reports, they say, they can recreate Myriad's database.

So they started a grass-roots project, Sharing Clinical Reports, and are asking cancer clinics and doctors to provide them with all the Myriad data they have from patients who have been tested.

None of the data have names of patients or other identifiers, so confidentially is not an issue, advocates say.  But their task is huge because the amount of data needed is vast.  The project's leader, Dr. Robert L. Nussbaum, chief of the division of genomic medicine at the University of California, San Francisco, estimates that with about 1,000 mutations collected so far, he has only about 1.5 percent of what Myriad has.

"Myriad is probably laughing at me, saying 'Here is this little flea,'" Dr. Nussbaum said.

The story began in 1996, when Myriad got patents on the two isolated DNA molecules known as the BRCA1 and BRCA2 genes and provided a test to determine if the genes carried mutations.  The company realized, though, that it would be crucial to figure out how risky each mutation was.

That sort of analysis requires linking the mutations to people's cancer history.  Obtaining that data is a momentous project, said Mark Capone, president of Myriad Genetic Laboratories, a wholly owned Myriad subsidiary. In 1996, the company classified 40% of mutations as being of uncertain significance because it did not have enough information to know what they meant.  By 2004, the figure was down to 20%.  Now it is just 3%.

Mr. Capone explained how the work is done.  The company finds a mutation and is not certain what it means.  To find out, it needs to see the same mutation in at least 20 other people, asking whether they had cancer and, if so, what type.  So Myriad offers free testing to other family members to get more information.

Until 2004, Myriad posted its data on a site for researchers.  But Mr. Capone said, the company became aware of problems with the way its data were being used.  For example, he said, the person running the database part time updated it only every couple of months.  And the database included risk estimates submitted by laboratories all over the world, not just by Myriad.

"We might classify a mutation one way, and someone else might call it something different," Mr. Capone said.  That is fine if the data were being used as intended -- for research purposes only.  But instead, they were being used to tell patients their cancer risk and to make major medical decisions.

Myriad's database, Mr. Capone said, is highly regulated.  "We can only use our database to provide clinical results for patients who had their genes sequenced in our lab" he said.  So, he said, when the company became aware of how the research database was being used, "we had to act -- we didn't have any choice."

Myriad stopped posting its data.

Dr. Nussbaum does not buy that argument.  "The Myriad approach is a big black box," he said.  "It's a 'trust us, we know best' approach."  And, he said, "it is contrary to the public health."

One thing it does is preclude independent second opinions, said Sherri Bale, managing director of GeneDX, a gene testing company working with Dr. Nussbaum.  Yet the consequences of some mutations are so dire that women may have their breasts and ovaries removed to protect themselves from cancer. "You are going to remove my breasts, you are going to remove my ovaries? Let me ask one other person," Dr. Bale said.

Myriad disagrees, though, saying it has licensed to LabCorp the ability to independently use its technology to search for mutations.  Myriad then uses its ata to say what the mutations mean.

But having one company control the data for genes is contrary to the way medicine is developing, said Heidi Rehm, a Harvard geneticist who also is working with Dr. Nussbaum.

In the not so recent past, she said, when it was difficult and expensive to determine the NA letters that make up a gene, individual laboratories would study one or two genes and become the world's expert on them.  Today, octors and researchers are scanning all of a patient's genes and the old system is crumbling.  No one can know enough to interpret the results without a public database.

"Now it is, 'I have 22,000 genes and have to stay on top of 50 million variants,'" Dr. Rehm said.  "There is just no way a single laboratory can manage that."  And if companies control the data on the interpretation of results, doctors, clinical laboratories and researchers are in a quandary.

Dr. Rehm, Dr. Nussbaum, Dr. Bale and others now are working with the National Institutes of Health to start a public database of variants in all the genes that have been studied.  But first, they are working on the Myriad issue.

The Myriad project began about a year ago when Dr. Nussbaum decided he and other could take matters into their own hands.  Working from home at night and on weekends, with the volunteer help of two genetics counselors, he began contacting geneticist friends and people he knew at large clinics, asking them to send in Myriad reports.

"I would say, 'How about pulling your results?'  They would say, 'It's a big pain,' So I would tell them, 'I will pay you 22 cents for each variant,'" Dr. Nussbaum said, explaining that a clinic woul usually end up getting about $50 if it sent in all of its patients' mutations and their interpretations.  Most then agreed to do it, assigning a student who needed extra money.

Clinics that sent in at least 200 unique genetic variants would paid an iPad mini.

The funds for these inducements were supplied by Peter Kolchinsy, managing director of RA Capital Management in Boston.

Dr. Iolchinsky, a scientist by training, said he would like to see many gene testing labs compete on the basis of cost, speed and customer service. But they would all share data on interpreting alterations in genes rather than creating what he called "gene- or disease-specific trade secret monopolies."

"That works for Coke, not for cancer," Dr. Kolchinsky said.

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Sorry for the shortened effort.  More next month ...

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And if you have any thoughts of how this newsletter could be improved, please email me directly, at

Elaine Jesmer

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