Scientific breakthroughs in the news this week promise many practical implications for myeloma patients.

Out with the bad

Dangerous genetic mutations have been edited out (removed) from the genes of human embryos, it was announced on Wednesday, August 2. Dr. Shoukhrat Mitalipov of Oregon Health and Science University, and his colleagues in California, China, and South Korea, repaired dozens of embryos.

If such embryos were allowed to develop into babies (which is currently prohibited by federal law), they would be disease-free and not able to transmit disease to descendants. Pretty amazing!

“It feels a bit like a ‘one small step for (hu)mans, one giant leap for (hu)mankind,’ ” Jennifer Doudna, a biochemist who helped discover the CRISPR-Cas9 gene-editing method used, told The New York Times.  Indeed. The technique could apply to many thousands of mutations.

Mutations that cause MGUS

For myeloma, the implications for the future are huge. In the iStopMM Iceland study, part of the IMF’s Black Swan Research Initiative®, we are looking for the mutations that cause MGUS. More than 4,000 patients with MGUS (monoclonal gammopathy of undetermined significance, myeloma’s precursor) will be studied. It is highly likely that one or two key mutations will be found.

Now there is the possibility that these mutations can be edited out. No more MGUS; no more myeloma linked to these mutations. This would be similar to BRCA mutations for breast and ovarian cancers, which are known and would now be amenable to the CRISPR technique. An incredible future is possible.

But we must be cautious. As the study’s co-author, Dr. Paul Amato, says, “I think it could be widely used, if it’s proven safe.” Much research will be required to evaluate the procedure’s safety and effectiveness.

Problems with DNA sequencing

The need to establish safety and effectiveness through rigorous research is further emphasized in a report from STAT this week: “FDA pushes to bring order to the chaotic world of DNA sequencing.” A “precision FDA” program launched in 2015 has turned up some important problems. In testing Next Generation Sequencing (NGS) methods, only half of the methods were deemed fully reproducible. Apparently, problems arose involving the computer processing speed linked to software programs used to identify specific genetic sequences.

Bringing true precision to genetic testing and manipulation will take time. An article in The Scientist notes that genetic variation between individuals can complicate how CRISPR editing can work. Serious “off target” effects could cause unwanted genetic changes.

There is also a lot to learn about other potential applications. As I reported in a recent blog post, the use of engineered CAR T-cells has been very much in the news. With sophisticated editing, T-cells and other types of immune cells can be produced.  So instead of hitting the myeloma (cancer) and the immune system with a sledge hammer, T-cell populations can be produced to fine-tune these immune therapy approaches: more like repairing the fine mechanisms of a Swiss watch.

A cautionary note: current therapies have been rushing ahead with huge venture capital investment. However, it is important to take a breath and consider more accurate approaches.

Body heat

While on the topic of groundbreaking science, it is worth mentioning two other recently published papers: “Self-Organized Resonance during Search of a Diverse Chemical Space” and “Spontaneous fine-tuning to environment in many-species chemical reaction networks.” These may seem completely unintelligible and uninteresting topics. But they may help us to understand how our bodies work.

Consider: If you stop to get a cup of Starbucks coffee on the way to work, by the time you get to work (especially in LA), the coffee will be cold. However, your body will have maintained its temperature whether you drank the coffee or not.

Most of us take for granted that our heart keeps beating and our lungs keep breathing. But how does the body capture, store, and use energy in an even fashion, almost indefinitely?

Well, researchers at MIT have identified chemical reactions which, instead of running down and stopping, have “energy-seeking” behavior that keeps them going. Technically speaking, these behaviors illustrate “lifelike patterns of collective molecular behavior.” In other words, these researchers have identified a mechanism whereby we can all wake up in the morning ready to enjoy another day, especially if we can finish our coffee before it gets cold.

In a world where we are barraged with disturbing news, I find it reassuring to see such important fundamental research producing important new results. It offers a lesson to us all to create our own personal goals and objectives to move forward each day.  Preferably, with a nice cup of hot coffee.

“What Is CRISPR?” Dr. Durie explains:

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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