As the new year begins, important developments that will impact patients have been in the news, ranging from molecular testing to new therapies for myeloma. The depth and scope of these reports reflect what I think are five key trends for 2018 and beyond.

Precision medicine—What does this term really mean and how do different researchers feel about it as we move into 2018? The defining feature of “precision medicine” is DNA sequencing of both people and diseased tissue (such as a tumor or a myeloma bone marrow sample) carried out on a huge scale to gather what is called “big data.” Such a project is being conducted by the Mayo Clinic and myeloma expert Dr. Keith Stewart, who directs Mayo’s Center for Individualized Medicine.

In a segment aired on NPR about the project, Dr. Stewart call it “a very worthwhile investment,” even though the specific value is yet unclear. But a skeptical Mayo Clinic colleague, scientist Dr. Michael Joyner, says he believes that directly studying each disease can be more fruitful than “collecting vast piles of information, hoping to make sense of it later.”

I agree with Dr. Joyner. We need to develop effective treatments, and figure out as we go along why they do or do not work. When treating myeloma, it is important to be aware that each patient has a different pattern of numerous mutations affecting many different genes. As a result, the opposite of the precision-medicine approach is necessary to understand if the same therapy can work well for this broad range of mutations we routinely encounter. I foresee the development of customized mutational analysis panels that will make it possible to screen patients in their doctors’ offices. Thankfully, the new immune therapies, such as daratumumab (Darzalex®), attack antigens expressed on myeloma cells irrespective of these diverse DNA mutations, thus providing a broadly effective treatment approach.

Sequencing is still important—Big-data gathering is one way to collect and use DNA sequence information, with detractors and downsides noted above. But sequence data can be used very effectively in a more focused fashion. A good example in the news this week is the sequence analysis of viruses and bacteria to rapidly identify dangerous new infections. Two companies, Illumina and Oxford Nanopore, have just released new sequencers: the first (Illumina) is a desktop model and the second (Oxford Nanopore) is the size of a USB jump drive!

This new development means that if there is a key mutation, it will most likely be possible to identify important mutations rapidly in the clinic. This is, in fact, a goal of the Iceland iStopMM project, which is funded through the IMF’s Black Swan Research Initiative®: when we identify the driver mutations leading to active myeloma and different patterns of myeloma, rapid clinic screening will guide appropriate treatment selection and possible prevention strategies.

CRISPR: A possible snag?—Gene editing using CRISPR is constantly in the news, and 2018 will undoubtedly see many new advances. For example, gene editing can be used to produce more powerful CAR T cells as a decisive immune therapy. However, a new study cautions that some types of treatment may be ineffective or even dangerous because the body rejects the bacterial Staph aureus Cas9 enzyme, which is the main component of the editing system.

Because of this potential danger, several “workarounds” have already been proposed. First, the gene editing can be done in the lab so that the Cas9 is not given to patients. Second, a Cas9 enzyme from a source other than Staph aureus can be used, thus avoiding what is called “a primed immune reaction.” It is impressive how rapidly (almost immediately) solutions to this potential problem were found and proposed publicly. The momentum to push gene editing into the clinic seems unstoppable at this point.

China is starting to produce cutting-edge treatments—China is jumping into the global drug development scene in a big way. This is something I have touched upon in several recent blogs. Instead of attempting to replicate Western medicines, the Chinese drug industry is now focused on conducting research and developing their own new drugs.  

China’s new drug-development strategy is to:

Commit to a research and development program. For example, the wealthiest resident of Hong Kong, Li Ka-shing, made that commitment and created Hutchison China MediTech. The company started developing cancer drugs in 2005.

Establish a relationship with a global pharma company. China MediTech (now Chi-Med) is linked to global pharmaceuticals giant AstraZeneca. On December 21, 2017, Legend Biotech (based in Nanjing, China) entered into a worldwide collaboration with Janssen to develop CAR T cells to treat myeloma. On January 8, 2018, Jiangsu Hengrui Medicine (based in Shanghai, China) entered into a global licensing agreement with TG Therapeutics for development and commercialization of a tyrosine kinase inhibitor, which works well in lymphoma and CLL.

Obtain Chinese FDA approval first…The first step will be for new drugs/treatments to be approved in China by the Chinese FDA (CFDA). This captures the huge, local Chinese market and creates a revenue stream. In addition, the costs of these drugs will most likely be much lower in China compared to Western equivalents. Already, CAR T-cell therapies are much cheaper in China. How this will affect subsequent US and global pricing remains to be seen.

Second, seek US FDA and other global approvals. This reverses the current model, in which new drugs receive US FDA approval as the first step.

Costs of care—The costs of care will remain a central concern in 2018 and beyond. According to a recent New York Times article,  US health-care spending is $3.3 trillion per year, which is 17.9 percent of the US GDP (gross domestic product) and twice as much per patient as any other industrialized country. The major added cost is at the “point of service”—in the clinic or hospital—and is largely related to drug costs. It seems there is no way around this conclusion, and as I’ve noted in the past, the high cost of drugs must be addressed by bringing all the stakeholders to the table to work out viable solutions.  

The potentially high costs of CAR T-cell therapies may be the ones that “break the bank,” so to speak. The first two approved CAR T-cell therapies (from Novartis and Gilead Sciences, respectively) are priced at $475,000 and $373,000 for a one-time treatment. Bluebird Bio just announced it will seek regulatory approval for an anti-BCMA CAR T-cell therapy by the end of 2019. It will be important to see the competitive impact on pricing from the evolving CAR T-cell therapy development and commercialization in China. As illustrated in a slide presented by Juno Therapeutics at the 2018 J.P. Morgan Healthcare Conference this week and shared on Twitter, the commercial opportunity in myeloma is huge. And for patients, the need is great. I’m hopeful that the enormous energy dedicated to discovering new myeloma treatments this year brings us closer to a cure.


Dr. James Melius, R.I.P.

I was saddened to learn of the January 1st death of extraordinary advocate Dr. James Melius. Dr. Melius played a pivotal role in the establishment of the James Zadroga 9/11 Health and Compensation Act, which includes myeloma among the resulting cancers covered. He was an international expert on workplace medicine and urged Congress to pass legislation that would help those who were affected by exposed to toxins after the terrorist attack of Sept. 11, 2001. His impact was far-reaching: With a small group of other American experts, Dr. Melius travelled to Bhopal, India to investigate and report on the long-term effects of the Union Carbide poison-gas leak. His dedication to individuals are who harmed as the result of their occupations serves as an inspiration to patient advocates everywhere.

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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