The Cancer Drug Dark Ages Are Coming to an End

Xconomy National — 

Only a couple stories in the past decade have given advocates of personalized cancer medicines much to brag about. There was Genentech’s Herceptin for a form of breast cancer in 1998, and Novartis’ Gleevec for chronic myeloid leukemia in 2001.

But just a few short weeks ago, in August, we saw a flurry of FDA approvals that I believe will go down as a turning point in the history of personalized medicine. Industry has paid lip service to this idea for years, but I’m starting to believe that many in pharma and biotech are getting real about changing their ways, and making drugs that are superbly effective in small niches of patients, rather than settling for mediocre advances for the masses.

Even with an explosion of knowledge in biology over the past 40 years, most of the new cancer drugs produce humbling results. Many drugs shrink tumors for one-fourth or one-third of patients, and nobody knows (or at least not enough have seriously bankrolled efforts to find out) how to pick which patients would fall into the lucky few. These drugs might extend lifespan a couple months, but there’s so much variability it can look like a roll of the dice—sometimes the drug could provide zippo benefit for you, or, it might extend your life a few years.

Two things, though, were certain. Patients would endure some significant side effects, and insurance companies would get some extremely high drug bills.

That’s why this past August was so important, and why people in biotech are still buzzing about what happened. Consider this string of FDA approvals, which the agency granted ahead of its usual legal deadlines, so it could get these life-saving therapies to patients sooner.

On August 17, Roche’s and Daiichi Sankyo/Plexxikon’s vemurafenib (Zelboraf) was cleared for sale as a new treatment for a deadly form of melanoma that has spread through the body. This drug is tailored to treat about half of melanoma patients with a mutated form of a protein called BRAF. Clinical trials showed this treatment reduced the risk of death by 63 percent in this distinct genetic population, when compared to standard chemo. Researchers are still following patients to see how much longer people can expect to live with the new drug. Importantly, the treatment was approved with a companion diagnostic test that will help doctors determine whether patients should get the drug or not. The diagnostic test costs $150, and the drug will go for the premium price of $56,400 for a six-month course of therapy.

On August 19, Seattle Genetics (NASDAQ: SGEN) won FDA approval for brentuximab vedotin (Adcetris) for patients with a couple of rare lymphomas—Hodgkin’s disease and anaplastic large cell lymphoma. This is a targeted drug that acts like a “smart bomb” by delivering a potent dose of chemotherapy directly to cancer cells that carry a signature marker known as CD30, while mostly avoiding healthy cells. The treatment was able to significantly shrink tumors for about 75 percent of patients with Hodgkin’s disease that had relapsed, and it produced an even better 86 percent response rate for very sick patients with anaplastic large cell lymphoma. Researchers are still following patients to see how long they are living, beyond the 2-3 year life expectancies they were given at the outset of the trial. Doctors can easily figure out which patients should get this drug based on their CD30 status, before they prescribe a product that is expected to cost about $108,000 per patient on average.

On August 26, Pfizer showed that even though it made its fortune on mass-marketed blockbuster drugs like Viagra and Lipitor, a significant part of its future will depend on a so-called “nichebuster” in cancer. This one is called crizotinib (Xalkori), which is designed to treat about 3-5 percent of patients with non-small cell lung cancer that overexpresses a protein called ALK. The drug showed it was able to shrink tumors in a majority of patients—50 to 61 percent—and the spread of tumors was halted for close to a year. The responses have been so encouraging for this specific group of lung cancer patients that researchers, again, will have to follow patients for an extended time to get an accurate read on how long it will help patients live. Abbott Laboratories has agreed to sell the companion diagnostic test to determine a patient’s ALK status, so doctors can see which patients should get this $9,600-a-month treatment.

There’s a pattern here. These drugs are going after … Next Page »

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13 responses to “The Cancer Drug Dark Ages Are Coming to an End”

  1. I read with interest your piece today on the potential turning point that the approvals of Zelboraf and Xalkori represent to the future of PM. Certainly the first step that the FDA took in sorting out the means to regulate companion diagnostics by this very elegant turn of a phrase, that the biomarker in question was to be detected using an FDA approved test, is historic. It has made little sense to tell drug manufacturers that the tests were so integral and necessary to the safe and effective use of their targeted therapies that they had to conduct clinical trials to demonstrate their clinical utility and validity – but then allow other companies and labs to make LDTs unregulated and ready for use. This new language in the drug labels for these two drugs perhaps brings an end to this significant disincentive to the drug and test manufacturers.

    However, I write to take issue with a passing comment that you made early in your article because it is one that is made many times over, by many commentators, and in its repetition it does a disservice to the nascent industry. You said that “industry has for paid lip service to this idea for years” in no way acknowledging or alluding to a fact with which you must be intimately familiar – that it takes upwards of 10-15 years to fully research, design and gain approval for a drug. That Xalkori and Zelboraf both followed significantly shorter timelines is simply yet another testament to the power of PM. The human genome was only sequenced in 2000 with this concept of PM coming to light really only in the few years preceding and following – which means that we should see a wave of new targeted therapies seeking approval in the next few years. Certainly our work with these companies indicates that is the future.

    But I must vehemently disagree with you that the industry has given mere lip service to this new development paradigm. In contrast, they have been working diligently to understand how this change in R&D affects their entire process and how to shift a development timeline that has been in place and standardized for the last several decades. They are making great inroads to understand how to integrate this concept across their organizations where the greater majority of people have no experience with or understanding of how this area works. They have even now begun to better understand how the commercial concepts for PM are far broader than for stand alone therapies and how to truly use the test to unlock the value of the drug. I am sure of this because we have worked with, assisted and watched the top 10 major pharma and now many of the smaller pharma working diligently to integrate and bring this new paradigm to life and they are not helped by the repetition of such incorrect phrases as “they are paying lip service” to the also overused phrase that targeted therapies cannot be blockbuster drugs (Herceptin and Gleevec have both put that idea to rest).

    Hopefully the next few years will see a surge in approvals for targeted therapies across a range of therapeutic areas which will put these comments to rest permanently.

  2. Sarah Condit says:

    People always wonder why we can’t cure cancer with all the millions of dollars people are donating. This gives a good explanation on why cancer can’t be cured right now.

  3. Saumitra says:

    Thanks Luke. I believe there would be lot of research in the ‘Pharma graveyard’ looking for molecules which had low favorable response in treating patients who were enrolled in clinical trials. If they can still analyze the genome of patients who survived more than 2 years or so and tag it to the molecule under clinical trial, they still can have a brand new molecule ready for FDA submission. (Not sure if FDA has any regulations on Retrospective studies) I do understand it is much easier said than done, just wanted to share my two cents.

  4. The money is a big issue as well as attending money conferences too. Here’s a site that has some collections with small cap biotech information with interviews with the CEOs. We attend as many of the major biotech conferences in the US and abroad. You might find some information not seen or heard elsewhere.

  5. larry glen says:

    I’m on Medicare-do you think they will approve these outlandish prices? As usual, the treatment and research of cancer is “Clear as mud”!