The Future of Myeloma Treatment: Insights and Innovations

As we move forward in 2022 and consider Key Myeloma Research Questions for 2022-2026 as we did at the 2022 IMWG Summit, hopes and challenges for the future come into strong focus. Unfortunately, the COVID-19 pandemic is still an ongoing challenge for myeloma patients who are at special risk from infections.



The International Myeloma Foundation (IMF) continues to be resilient in its mission: “improving the quality of life for myeloma patients while working towards prevention and a cure.”



The IMF’s mission has served patients extremely well for over three decades, and it will continue to do so for decades to come. Being flexible and adapting to changing times have always been trademark qualities of the IMF’s programs. I am therefore very optimistic that the IMF will continue to guide the way forward — with deep insight and kindness that are essential in considering every patient’s individual needs.
 
“Knowledge is power” has always been and will continue to be the IMF’s fundamental principle when it comes to patient education and support. Patient feedback has been key in establishing metrics and benchmarks for ongoing improvements; and in achieving best results. 
 
Additionally, the formation of a network of support group leaders across the U.S. and the Annual Support Group Leaders Summit with working groups have been instrumental in providing a venue for feedback and discussions thus, enabling the IMF to head into the right direction when it comes to fulfilling its mission. 



Blueprint for Success 


Our goal of helping patients in every way possible has never wavered. Working as an international organization, it has become apparent to the IMF from the outset that options for diagnostic testing and treatment vary wildly. 
 
In translating information into multiple languages and holding patient and family seminars around the world, it has become clear that everything can be different—and not just the language! Do you tell patients that they have a form of cancer? Should you tell them about options which are unavailable and/or are too costly?

How do you talk to a patient who has used up every option available in their country? Knowing the plight of patients has been transformative for myeloma experts working in these IMF programs—we are keenly aware that we must do more to help. 
 
Sharing vials of medication to help another patient happens in many countries on a day-to-day. Working around import restrictions to have access to lifesaving immunomodulatory drugs (IMiDs) saves many lives. Japan is fortunate to have this happen, thanks to the efforts of the late Aki Horinouchi—an amazing and compassionate patient advocate who founded IMF Japan in 1997. 
 
This led to our decentralized model: one in which guidelines and support structures are adapted to fit local needs. The truth about limited access is very real when treatment options run out, or when they were never there in the first place. 
 
For Asia and Europe, regional guidelines, have been really helpful. Making new therapies available through clinical trials, as being done in the IMF’s Asian Myeloma Network, can provide access where it’s desperately needed. The reality of global inequities is a powerful truth.
 
While speaking of Aki, a fond memory came to mind. On July 17, 2012, I wrote a blog about my visit to the land of cherry blossoms for the IMF Japan Patient and Doctor Meetings. 
 
I remember how the memory of Aki remained strong and vibrant through the course of the 2012 IMF patient and doctor meetings that were held in Kyoto. The meetings were a roaring success—attended by over 500 Japanese myeloma experts. 
 
That year, a new feature was accommodated—eight myeloma experts from Japan made time to give one-on-one consultations with Japanese patients. Because of that, over 300 patients were able to take advantage of a broad range of sessions, including four special “commentary” sessions which I was pleased to present on: bone disease, renal problems, novel therapies, and key questions from 2012. 
 
Additionally, Koyko Joko (who served as chair of the IMF Japan Board at that time) organized many new initiatives—including the involvement of local physical therapy students as volunteers during the patient seminars. Also, fundraisers that year were able to yield one or two research grants (given annually, in honor of Aki) were awarded to deserving top myeloma researchers in Japan.
 
To top it all off, we were also able to spend time with Aki’s courageous wife, Midori who continues to help lead and inspire IMF Japan. It was truly a wonderful experience. 
 

Hopes and Challenges for 2022-2026 and Beyond: The Future is Bright



Immune Therapies


The introduction of so many new immune therapies—such as CAR T cells and bispecific antibodies, in addition to other novel agents—offers great hope. This optimism covers both improving outcomes and potentially achieving a cure for some patients. 
 
The next five years will definitely see a steady increase in lengths of remissions as well as overall survival outcomes. Assessing which options and sequences are best will definitely be a challenge, but these are good challenges to have.



The Black Swan Research Initiative® 


The IMF, through the Black Swan Research Initiative®, is strongly committed to continuing research to explore options for achieving both prevention and cure. 
 
A new understanding of the early evolution of myeloma by the iStopMM (Iceland Screens Treats or Prevents Multiple Myeloma) project in Iceland can lead to prevention strategies, as well as early interventions to optimize outcomes. 

In the search for the cure, the first results of the ASCENT Trial evaluating the use of daratumumab (Dara) plus carfilzomib, lenalidomide, and dexamethasone (KRd) in high-risk smoldering multiple myeloma (HR SMM) will be presented later this year.
 
Results will guide the next steps toward achieving long and sustained minimal residual disease (MRD) negative remissions and potential curve. High technology approaches have been used and further refinements, such as use of artificial intelligence (AI) and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) gene editing plus sophisticated molecular techniques, will enhance what can be accomplished.



Key Next Challenges 



Realistically speaking, these fantastic new therapies will only become available to a privileged few. The top priority is to achieve access to standard of care (SOC) testing and treatments for a majority of patients globally, who do not even have basic options available. 
 
Individual patient stories tell the truth and hopefully, can elicit enough compassion in those who are capable of taking action. It is urgent to explore all possible solutions to help every patient in need.


The cost of drugs is the fundamental underlying problem. Broader introduction of generics may help, but multiple new approaches are required to achieve greater access to standard of care therapies.

COVID-19 Subvariants and the Future



The current spread of the BA.5 omicron subvariant and recent news on the emergence of another new omicron subvariant, BA.2.75 unfortunately, illustrates that the 
COVID-19 pandemic is not over. 

According to Helix (the company conducting viral surveillance of the coronavirus), BA.2.75 has been detected in three U.S. states in the past two weeks, namely California, Washington, and Illinois but additional states have been reported by New York Institute of Technology professor Raj Rajnarayanan who maintains a database tracking COVID-19 variants. Prof. Rajnarayanan reported that the new subvariant has also been detected in New York, North Carolina, Texas, and Wisconsin.

This new mutation of yet another omicron subvariant means that we need to go back to exercising extra precautions to avoid being infected with COVID-19. 
 
Despite vaccination and boosters, the risk of a COVID-19 infection or re-infection is very real. Wearing the best possible mask in all situations of potential exposure, especially indoors, is very important to prevent infection. 
 
In case of possible infection and a positive test result, it is good to be aware of the value of early Paxlovid™ treatment. The U.S. Food and Drug Administration (FDA) has authorized pharmacists to prescribe Paxlovid with certain limitations. Check with your doctor or pharmacy to make sure there are no potential drug interactions, then get a prescription or supply of Paxlovid to have on hand.



As we move into Fall, more variants may still emerge. Fortunately, boosters targeting new variants may become available to ward off new infections. In the meantime, it is very reasonable to ahead and get a third booster (from among currently available ones), 4 months after receiving the second booster. 
 
Additionally, stay aware of the level of COVID-19 in your community and discuss this topic regularly with your doctor during appointments. Avoiding a COVID-19 infection is still an important goal. 

The Bottom Line 

The IMF is here to help with myeloma care decisions. We are committed to our mission and formula for success and will continue to flex and adapt to new challenges, as we continue to guide patients to the best possible outcomes while working hard towards prevention and a cure.