There have been recent commentaries on the prospects and potential for both what is called "precision oncology" and Vice President Joe Biden's Cancer Moonshot program. Although I have discussed both in the past, the current news, writes Dr. Vinay Prasad in the journal Nature, is quite “sobering.” The news is sobering because the hype and expectations are way beyond the truth. When costs are high—and in the case of precision oncology, they are extremely high—research efforts must focus on areas of likely success, not on high expense with little likelihood of return. Since myeloma is one of many cancers (and not the most common), we are particularly sensitive about the need to fully justify the spending of every research dollar.

Myeloma’s many, many mutations

So what do precision oncology and the Moonshot efforts mean for myeloma research? Precision oncology matches treatments to specific mutations found in the cancer cells. The concept is simple: find the mutation and shut it down. Bingo—problem solved. But not so fast: myeloma cells have many, many mutations and even multiple clones with many different mutations. Shut down one and the others take over. The end result is huge costs to pay for sequencing to find the mutations and develop targeting therapies for what turns out to be very short remissions in 30% of patients—if that.

Slim odds in current data

Dr. Prasad summarizes the most current data. At the MD Anderson Cancer Center in Houston, Texas, he writes, 2,600 patients were studied and “just 6.4% were paired with a targeted drug for identified mutations,” which is called a match. At the National Cancer Institute (NCI), only 2% of 795 patients achieved matches (no myeloma patients). Since only 30% of patients paired with a targeted drug respond to therapy, it is estimated that overall only 1.5% of patients can benefit, with an average remission of 5.7 months (2.3 months in a randomized trial versus standard therapy). Sobering indeed!

Cancer Moonshot’s latest blast sounds familiar

Recently, a blue-ribbon committee of the Cancer Moonshot project announced 10 "transformative approaches poised for acceleration.” Six of the ten areas of research identified have been part of the IMF’s International Myeloma Working Group (IMWG) myeloma research program for more than a decade, if not longer.

  • Number One: Engage patients in the clinical trial process. Check!
  • Number Four: Give top priority to data-sharing to achieve results. Check! Just one of many examples: In 2005, the IMF published the new ISS staging system based upon data from thousands of patients from 26 top centers.
  • Number Six: Prioritize guideline development. Check! So far the IMF/IMWG have published close to 40 international guidelines, with more on the way, including new PET/ CT guidelines.
  • Number Seven: Move to prioritize prevention strategies. Check! Last week, I summarized the new prevention ideas that will evolve from the IMF’s Black Swan Research Initiative-funded Iceland project, iStopMM.
  • Number Ten: Develop new “enabling technologies.” Check! The development of new MRD tests is an excellent example.

One of the Cancer Moonshot initiatives applies only to children, and three include an emphasis on precision oncology (!), which I consider deserving of lesser priority. Lower-priority initiatives include immune-therapy-only trials, which, I think, are not required—better to combine with other therapies—and 3D dynamic maps of mutations.

What is really feasible?

But the more tragic aspect of the Cancer Moonshot is that funding is not as yet secure. Each one of the project’s “transformative” approaches will require hundreds of millions of dollars—and many for each individual type of cancer. In this tense political year, funding is tenuous at best, unfortunately.

Thus, this week I give mostly sobering news. My advice is to beware of the hype. Precision oncology is a rather popular notion. It sounds great. Which patient does not want to have a mutation profile performed and specific therapy to wipe out the myeloma? But what is truly feasible? I believe the best chance we have is to stop myeloma in its tracks BEFORE the dangerous mutations emerge. This is the fundamental idea of our Black Swan Research Initiative: treat early and achieve cure.

The moonshot ideas are great, but they must be tailored to each cancer. Specialists in each cancer, such as myeloma, are already very much focused on achieving the best outcomes or cure for each of their patients. So, reader beware, and stay tuned for further updates!

Image of Dr. Brian G.M. DurieDr. Brian G.M. Durie serves as Chairman of the International Myeloma Foundation and serves on its Scientific Advisory Board. Additionally, he is Chairman of the IMF's International Myeloma Working Group, a consortium of nearly 200 myeloma experts from around the world. Dr. Durie also leads the IMF’s Black Swan Research Initiative®.

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