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Sixty years ago, President John F. Kennedy delivered a powerful and historic speech about America sending the first man to the moon. 

On Monday, September 12th, President Joe Biden rendered his own speech at the Kennedy Presidential Library in Boston to outline the progress of his “moonshot” vision of bold approaches to end cancer.

According to Pres. Biden, “there are too few ways to prevent [cancer] in the first place” and that there are “stark inequities in cancer diagnosis and treatment based on race, disability, zip code, sexual orientation, and gender identity,” as reported by NPR

"We know too little about why treatments work for some patients, but a different patient with the same disease, it doesn't work for. We still lack strategies in developing treatments for some cancers," Biden further said, adding that not enough is being done “to help patients and families navigate the cancer care system.” 

Pres. Biden emphasized that “one of the reasons why he ran for president” is because of the cancer moonshot program—an initiative that he helped create with the goal of reducing cancer deaths by 50 percent in a span of 25 years. 

In his speech, the president announced that he has appointed Gingko Bioworks Vice President of Business Development Dr. Renee Wegrzyn as director of the new federal agency, Advanced Research Projects Agency for Health (ARPA-H), as reported by the New York Times.

According to NPR, ARPA-H was established in February “to improve the U.S. government’s ability to drive health and biomedical research.” 

“ARPA-H will have the singular purpose to drive breakthroughs to prevent, detect and treat diseases, including cancer, Alzheimer's, diabetes and other diseases and enable us to live healthier lives," said Pres. Biden via NPR.

Additionally, the president announced that he will be signing a new executive order to launch a National Biotechnology and Biomanufacturing Initiative to assure that the technology used to help end cancer will be made in the United States. 

Pres. Biden remarked “that the creation of new technologies for cancer treatments and other things will create jobs and strengthen supply chains,” adding that the U.S. “would not have to rely on anywhere else in the world for that advancement,” NPR further reported.

Impact of Blood Testing

Of major interest for myeloma research is a statement made by Cancer Moonshot Coordinator to the White House Danielle Carnival to the Associated Press: “One of the most promising technologies has been the development of blood tests that offer the promise of detecting multiple cancers in a single blood test and really imagining the impact that could have on our ability to detect cancer early and in a more equitable way. We think the best way to get us to the place where those are realized is to really test out the technologies we have today and see what works and what really has an impact on extending lives.”

As it turns out, this is something that has been emerging in the research done over the past decade by the Black Swan Research Initiative®(BSRI) which is very much focused on blood testing for both early diagnosis and monitoring.  

Within the proposed framework for the new cancer moonshot program, the BSRI’s efforts —what can be considered as the Myeloma Moonshot—are well ahead of schedule.

 

BSRI’s blood testing initiatives

1. Screening blood samples in Iceland

Blood testing is central to the IMF’s Black Swan Research Initiative’s® (BSRI) efforts to achieve both prevention and a cure for myeloma—a true moonshot! 

In 2017, the IMF began collaborating with Prof. Sigurdur Kristinsson, leader of the BSRI-supported iStopMM (Iceland Screens Treats Or Prevents Multiple Myeloma) Project. 

Part of the iStopMM Project is to screen Icelandic residents and subsequently do a randomized trial assessment of myeloma-related monoclonal proteins which might be discovered with screening testing of the serum. Over 85,000 Icelandic residents signed up and participated in this first large-scale screening study in the field of myeloma.

New monoclonal proteins—indicative of the potential for later development of myeloma —were detected in over 4,000 individuals. Follow up on these individuals is revealing how and when progression may occur as well as determining the full impact of easy screening.

Does screening improve survival outcomes and quality of life? Five years of follow up on the randomized effort will provide answers. The identification of more than expected cases of smoldering multiple myeloma or SMM (with a prevalence of 0.5%) indicates that there is an opportunity to introduce early therapies which will improve outcomes overall.

2. Role of Mass Spectrometry testing in blood

In addition to the standard screening —a combination of serum protein electrophoresis (SPEP) test, Immunofixation (IFE), and serum Freelite® testing, the new and highly sensitive Mass Spectrometry (MS) testing was also carried out. 

This MS method is so sensitive that it can detect small monoclonal proteins which occur in response to routine infections such as colds or the flu.

For myeloma patients, this is like looking back in time to the earliest possible moment when an abnormal precancerous clone might emerge. For very low-level monoclonal (spike) proteins, the chance of progression to active myeloma is about 1% per year and for slightly higher-level spikes (in patients with so-called SMM) the chance is higher at ~ 10% per year. It is exciting to have this unique ability to carefully study the first steps of monoclonal gammopathy of undetermined significance (MGUS) and SMM and the potential of progression to active multiple myeloma.

3. Testing DNA mutations in blood

Another area of blood testing is to assess genetic mutations of circulating DNA in blood samples. This technology has been applied as part of the BSRI® project by the team of Prof. Andrew Spencer in Melbourne, Australia.

They have shown that specific mutations (such as RAS mutations) occur at the time of relapsing disease and can potentially become the targets of new therapies to overcome resistance to standard treatments. Initial trials have been promising.

4. Understanding early disease and the potential for prevention

Identifying very early stages of these monoclonal diseases definitely opens up the possibility of interventions to prevent progression to active disease. We already know that simple steps (such as sticking with the traditional Icelandic fish-based diet) can reduce the risk of progression. Much more sophisticated interventions can also be considered. Iceland’s population has had full DNA/gene sequencing performed (germline sequencing)—the only country in the world where this has been achieved.

By comparing individuals with and without monoclonal proteins, a gene profile can be established, linked to the occurrence of monoclonal protein diseases. 

Although the predisposition to active disease may be polygenic (meaning multiple factors are involved), it may be that a few key (or driver) genetic sequences will be detected and will be amenable to a range of interventions— from gene-targeted therapies to Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) type gene editing technology approaches. True prevention can be feasible.

5. Role of blood testing and early intervention

Early detection of progression (or the high likelihood of progression) is essential to direct appropriate early interventions to achieve best outcomes. 

Recent studies have shown that the occurrence of myeloma cells in the bloodstream is a precise quantitative marker for disease progression. The technology involves the use of the minimal residual disease (MRD) test method of next-generation flow (NGF) on blood samples.

An enhancement of this technology by Dr. Bruno Paiva and his team at the University of Pamplona in Spain may lead to sensitivity at the unheard level of 1 in 10-8 (meaning reliable indicate zero or not @ the level 100 million cells screened).

With reliable identification of patients progressing or about to progress to active myeloma even aggressive and potentially curative therapies can be introduced. Two pilot CURE Trials: the CESAR and ASCENT trials have indicated the promising results feasible with screening, assessment, and decisive interventions.

Moonshot Efforts

It would be great if the new Biden Moonshot ARPA-H initiative can enhance our efforts by accelerating approvals and/or refining testing capabilities or access to novel therapeutics for us to move forward at warp speed. In the case of myeloma, the tools are in place to achieve rapid success.

Integration into the healthcare system will be a next important step to ensure equitable access to these innovative approaches. A recent case in California illustrates the cautions required.

CA Attorney General Rob Bonta is investigating healthcare algorithms, “citing a study that found one algorithm referred white patients to get enhanced services at a higher rate than their Black counterparts with similar needs,” as reported by the L.A.Times.

Healthcare algorithms can streamline processes, but unintended inequitable consequences must be avoided to achieve best outcomes for all patients.

Effective and True Innovations 

In California, there is hope that true innovations can be implemented and really make a difference. 

During the recent heat wave, rolling blackouts were avoided. How did they manage to do so? It turns out, California has been able to get key stockholders together to implement a huge battery backup plan for the state’s electric grid system. Thus, despite so many areas of high-level administrative dysfunction, this remarkable project was able to go ahead and protect the state from blackouts.

As pointed out by Mike Perry, research director for UC San Diego Center for Energy Research, this was no trivial feat—the industrial-sized individual battery systems can weigh tens of thousands of pounds and whole systems can be larger than a football field.  This project culminated after 12 years, beginning with the passing of Assembly Bill 2514 in 2010.

This was recently pushed forward strongly by CA Governor Gavin Newsom and the lights stayed on despite the heat wave!


The Bottom Line: Moonshot Lessons

Stay focused and look for every collaboration to implement key ideas. Shoot for the stars with innovations that matter. Success is the constant partner of dedication.
 


Image of Dr. Brian G.M. DurieProfessor of Medicine, Hematologist/Oncologist, and Honoree MD at the University of Brussels, Dr. Brian G.M. Durie is the Chairman of the Board and Chief Scientific Officer of the IMF. Dr. Durie is also the Chairman of the International Myeloma Working Group (IMWG)—a consortium of more than 250 myeloma experts from around the world—and leads the IMF’s Black Swan Research Initiative® (BSRI). 

 

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