Volume 21 Number 1
Winter 2021
Myeloma Today A publication of the International Myeloma Foundation
FDA Approves New MM Therapies: ABECMA® and PEPAXTO® } Read about PEPAXTO, the first
anticancer peptide-drug conjugate PAGE 4
} Read about Abecma, the first
CAR T-cell immunotherapy in MM PAGE 5
Also in this edition: PAGE 6 } 2021 Brian D. Novis Research Grant
} COVID-19 pandemic updates } New IMWG guidelines highlight role of experts
awarded to Dr. Eugenio Morelli of Dana-Farber Cancer Institute PAGE 11
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} Key Trends in Myeloma Care for 2021
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This edition of Myeloma Today is supported by Amgen • Bristol Myers Squibb • Karyopharm • Oncopeptides • Sanofi Genzyme • Takeda Oncology
International Myeloma Foundation
Founder
President & CEO
Brian D. Novis
Susie Durie
Board of Directors
Chairman Dr. Brian G.M. Durie Christine Battistini Yelak Biru Prof. Dr. Mario Boccadoro Loraine Boyle Susie Durie
Martine Elias George T. Hayum Jason Katz Benson Klein Andrew Kuzneski, III
Dr. Robert A. Kyle Prof. Dr. Heinz Ludwig Dr. Edith Mitchell Charles Newman Dr. S. Vincent Rajkumar
Matthew Robinson E. Michael D. Scott
IMF Executive Team Chief Financial Officer & Chief Operating Officer Jennifer Scarne jscarne@myeloma.org
Senior Vice President, Strategic Planning Diane Moran dmoran@myeloma.org
Senior Vice President, Clinical Education & Research Initiatives Lisa Paik lpaik@myeloma.org
Senior Vice President, Global Affairs Daniel Navid dnavid@myeloma.org
Chief Medical Officer Dr. Joseph Mikhael jmikhael@myeloma.org
Senior Vice President, Philanthropy Lynn K. Green, Ed.D. lgreen@myeloma.org
Senior Vice President, Global Advocacy, Access, Policy & Research Mimi Choon-Quinones, PhD, MBA mchoon-quinones@myeloma.org
Vice President, Marketing Peter Anton panton@myeloma.org
Inventory Control Manager Betty Arevalo marevalo@myeloma.org
Meeting Registration & Guest Relations Abigail Guzman aguzman@myeloma.org
Assistant to Senior Director, Member Events Karla Lemus klemus@myeloma.org
Regional Director, Support Groups Kelley Sidorowicz ksidorowicz@myeloma.org
Director, Regional Community Workshops Brenda Hawkes bhawkes@myeloma.org
Senior Director, Public Policy & Advocacy Robin Levy rlevy@myeloma.org
Donor Relations Sarah Solomon ssolomon@myeloma.org
InfoLine Coordinator Paul Hewitt phewitt@myeloma.org
Senior Research Project Coordinator Amirah Limayo alimayo@myeloma.org
Accounting & Distribution Brando Sordoni bsordoni@myeloma.org
Web Specialist Kevin Huynh khuynh@myeloma.org
Publication Design Jim Needham jneedham@myeloma.org
Assistant to the President Rafi Stephan rstephan@myeloma.org
IMF Staff
Senior Director, Member Events Suzanne Battaglia sbattaglia@myeloma.org Regional Director, Support Groups Nancy Bruno nbruno@myeloma.org Director, Support Groups & Regional Community Workshops Kelly Cox kcox@myeloma.org Director, Public Policy & Advocacy Danielle Doheny ddoheny@myeloma.org Director, Global Myeloma Action Network and European & Middle Eastern Patient Programs Serdar Erdoğan serdogan@myeloma.org Donor Relations Heather Fishman hfishman@myeloma.org Support Group Coordinator Jon Fitzpatrick jfitzpatrick@myeloma.org
Editor-in-Chief, Publications Marya Kazakova mkazakova@myeloma.org
Administrative Assistant, Meetings & Events Meghan O’Connor moconnor@myeloma.org
Assistant Director, Member Events Ilana Kenville ikenville@myeloma.org
Director, Operations Selma Plascencia splascencia@myeloma.org
InfoLine Coordinator Missy Klepetar mklepetar@myeloma.org
Director, Meetings & Events Annabel Reardon areardon@myeloma.org
Manager, Marketing & Communications Sapna Kumar skumar@myeloma.org Accountant Phil Lange plange@myeloma.org
Database Analyst Joy Riznikove jriznikove@myeloma.org
Vice President, Support Groups Robin Tuohy rtuohy@myeloma.org InfoLine Coordinator Judy Webb jwebb@myeloma.org Donor Relations Jonathan Weitz jweitz@myeloma.org
Web Producer Miko Santos msantos@myeloma.org
This free issue of Myeloma Today© (Volume 21, Number 1) is dated March 30, 2021. Myeloma Today© is a quarterly (Spring, Summer, Fall, and Winter) publication of the International Myeloma Foundation, located at 12650 Riverside Drive, Suite 206, North Hollywood, CA 91607 USA
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A Message from the IMF President & CEO Dear Reader, In October 2020, the IMF celebrated our 30th anniversary. It’s hard to believe but the IMF is now in its 31st year, and we have steadfastly remained dedicated to improving the quality of life of myeloma patients while working toward prevention and a cure. Myeloma Today, the journal you are reading is now in its 29th year of publication. This seems like an appropriate time to look back to our very first edition, published in 1992, and to share with you an excerpt of the very first Dear Reader column, written by the IMF’s co-founder, Brian D. Novis. Dear Reader, Welcome to Myeloma Today – your first edition of the new quarterly publication of the International Myeloma Foundation (IMF). For both patients and physicians around the world, this publication will provide a central reference resource that is focused exclusively on the most up-to-date information available on the treatment of myeloma and the latest developments in its research.
in the field of myeloma and how the IMF’s International Myeloma Working Group (IMWG) continues to chart the future of myeloma treatment and care. You will also learn about promising innovative myeloma research fostered by the IMF through the Brian D. Novis Research Grant program. And you will be inspired by the patients, caregivers, advocates, and physicians who are making a difference for the myeloma community, fighting myeloma on all fronts and around the world! The IMF continues to make strides toward a cure, always keeping myeloma patients at the heart of all our work, and I am honored to have dedicated the last three decades of my life to such a worthy cause. Warm regards, Susie Durie, IMF President & CEO
Myeloma Today offers an opportunity for physicians to share the latest in treatment modalities and for patients to find answers to the many questions presented by myeloma management. An international scientific advisory board consisting of preeminent oncologists and hematologists advises the IMF. This group of specialists in myeloma is chaired by Dr. Brian Durie, a professor based currently at the University of London, England, and convenes regularly.
Brian D. Novis, Executive Director
Brian D. Novis died later in 1992. In my mind, he gave his life for the IMF. I have written the Dear Reader column ever since, and I continue to be honored to serve the myeloma community and the IMF, which is now more than half a million members strong! In this edition of Myeloma Today, as in our first edition, you will hear from Dr. Brian G.M. Durie about the latest
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Susie Durie and Brian Novis, 1992
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Scientific & Clinical
PEPAXTO® (melphalan flufenamide) First Anticancer Peptide-Drug Conjugate Approved by the FDA On February 26, the U.S. Food and Drug Administration (FDA) approved Pepaxto® (melphalan flufenamide), an alkylating agent, in combination with the steroid dexamethasone for the treatment of adult patients with relapsed or refractory myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor (e.g., Velcade® [bortezomib], Kyprolis® [carfilzomib], and Ninlaro® [ixazomib]), one immunomodulatory drug (e.g., Revlimid® [lenalidomide], Pomalyst® [pomalidomide], and thalidomide), and one CD38-directed monoclonal antibody (e.g., Darzalex® [daratumumab] and Sarclisa® [isatuximab]). Pepaxto is the first anticancer peptide-drug conjugate approved by the FDA. According to the Journal of Clinical Oncology (March 1, 2021), Pepaxto is a next-generation nitrogen mustard agent, a first-in-class agent evaluated in the phase II HORIZON clinical trial with 157 patients, which resulted in an overall response rate (ORR) of
29% and progression-free survival (PFS) of 8.5 months in responding patients. In the 97 study patients who met the FDA-approved indication of at least 4 prior lines and refractory to at least one proteasome inhibitor, immunomodulatory drug, and monoclonal antibody, the response rate was 24% with a median duration of response of 4.2 months Pepaxto is administered at a dose of 40 mg on Day 1 of each 28-day treatment cycle as a 30-minute infusion via a central venous access device. Most common side effects occurring in more than 20% of patients are fatigue, nausea, diarrhea, fever, and respiratory tract infection. Most common laboratory abnormalities occurring in 50% or more of patients are leukocytes decrease, platelets decrease, lymphocytes decrease, neutrophils decrease, hemoglobin decrease, and creatinine increase. Alopecia (hair loss) and mucositis, which are side effects commonly encountered with conventional chemotherapy, were not seen. MT
What does this mean for patients with myeloma? “Melflufen, which is also known as melphalan flufenamide, is a first-in-class peptide-drug conjugate that targets aminopeptidases and rapidly releases its alkylating moiety directly into myeloma cells. The rationale to evaluate melflufen was to improve upon the specificity and tolerability of melphalan and other alkylators, which are a highly active class of cytotoxics in myeloma. The phase II HORIZON clinical trial evaluated the efficacy of melflufen in combination with dexamethasone in patients with relapsed and refractory myeloma, a population with an important unmet medical need. The combination showed clinically meaningful efficacy and a manageable safety profile in heavily pretreated patients, including those with triple-class-refractory and extramedullary disease, as well as those patients in whom prior alkylator therapy had failed.”
Paul Richardson, MD
Principal Investigator, HORIZON clinical trial “It is wonderful to see myeloma research move strongly forward with the approval by the FDA of melphalan flufenamide. It is important for myeloma patients to have a new treatment option with a unique new mechanism of action. We look forward to further clinical trials with melphalan flufenamide in different combinations of myeloma medications in earlier disease settings.”
Brian G.M. Durie, MD
Chief Scientific Officer, IMF “It is very gratifying to see more options available to patients who have developed triple-class refractory myeloma as this has now become so common. It is also important to note that response to melphalan flufenamide was seen in patients who were previously treated with melphalan in the context of an autologous stem cell transplant or as oral melphalan, validating this mechanism of delivering an alkylating drug to myeloma cells.”
Joseph Mikhael, MD
Chief Medical Officer, IMF
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Scientific & Clinical
ABECMA® (idecabtagene vicleucel) CAR T-Cell Treatment for Myeloma Approved by the FDA On March 29, the U.S. Food and Drug Administration (FDA) approved Abecma® (idecabtagene vicleucel, a.k.a. ide-cel), a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T-cell immunotherapy, for use in adult patients with relapsed and refractory myeloma who have had four or more prior lines of therapy and who have been exposed to all three major drug classes (triple-class exposed), including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody. Abecma is the first-in-class FDA-approved anti-BCMA CAR T-cell therapy for triple-class exposed patients with myeloma. Abecma recognizes and binds to BCMA, a protein that is nearly universally expressed on myeloma cells, leading to the death of BCMA-expressing cells. The FDA approval was based on the results of the pivotal phase II KarMMa clinical trial. Patients in the trial were treated with their own T cells, which were engineered
to target BCMA on the surface of the myeloma cells. Of 140 patients enrolled in the study, 128 received treatment. The majority (72%) of patients achieved rapid, deep, and durable responses. Minimal residual disease (MRD)negative status was achieved in 33 (26%) of treated patients. The safety profile of Abecma is well-established and predictable, including cytokine release syndrome (CRS) and neurologic toxicities that are mostly low-grade with early onset and resolution. Abecma is a personalized immune cell therapy delivered as a one-time infusion, manufactured for each individual patient using the patient’s own T cells. Due to the specialized nature of cell therapy, Abecma will be administered at certified treatment centers through out the country. A Risk Evaluation and Mitigation Strategy (REMS) program will be implemented to support appropriate use of Abecma. MT
What does this mean for patients with myeloma? “In the phase II KarMMa clinical trial, ide-cel elicited rapid responses in the majority of patients, and these deep and durable responses were observed in patients with triple-class exposed and refractory multiple myeloma. As a treating physician, I often work with patients with relapsed or refractory myeloma who are in critical need of new therapies. Now, with the FDA approval of Abecma, we are excited to finally be able to offer patients a new, effective personalized treatment option that is delivered through a single infusion.”
Nikhil C. Munshi, MD Principal Investigator, KarMMa clinical trial “The FDA approval of Abecma (idecabtagene vicleucel) means that myeloma patients have gained yet one more treatment option for this cancer of the blood plasma cells. This is a win for patients! Overall, the results of the clinical trial were extremely promising, with a median response duration of 10.7 months, progression-free survival (PFS) of 8.8 months and overall survival (OS) of 19.4 months. Patients who achieved a complete response (CR) or better had a longer response duration of 19 months. One-third of patients in the clinical trial achieved meaningful responses in the one-to-two-year range or better, resulting in an excellent quality of life period of time off from any type of conventional anti-myeloma therapy.”
Brian G.M. Durie, MD
Chief Scientific Officer, IMF “This is genuinely a wonderful and long-anticipated day for myeloma patients. Although the science behind the Abecma (idecabtagene vicleucel) CAR T-cell therapy is complex, the simple benefit is clear. The response rates are unprecedented in relapsed and refractory myeloma. This is an opportunity for patients with heavily pretreated disease to have truly deep and durable responses. Furthermore, a one-and-done approach allows patients more time outside of the clinic to be with their loved ones and not experience the side effects of ongoing therapy.”
Joseph Mikhael, MD
Chief Medical Officer, IMF
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Scientific & Clinical
COVID-19 Pandemic Updates
Vaccination, mask mandates, and pandemic fatigue By Dr. Brian G.M. Durie IMF Chairman of the Board
Myeloma patients cannot look to the future through rose-colored glasses. The reality is that keeping myeloma in remission – and especially getting there in the first place – requires close attention and expert advice. The same is true for COVID-19.
Implications for myeloma patients Avoiding COVID-19 infection continues to be the top priority, especially for patients with active myeloma requiring new therapy. For patients with MGUS or SMM, avoiding COVID-19 infection is also key but it is really important to emphasize that MGUS and SMM patients do NOT appear to be more likely to develop COVID-19 infection, and have outcomes similar to a matched non-myeloma population based upon age and/or risk factors such as high blood pressure, diabetes, obesity, or underlying lung, heart, or kidney conditions. It is very important for myeloma patients undergoing treatment to maintain the recommended therapy to stay in (or achieve) remission. This is a top priority and arranging safe visits to the hospital or clinic must continue as best feasible to sustain ideal control of the myeloma.
Vaccination Vaccination is strongly encouraged for all myeloma patients. Whichever vaccine is available, please proceed with vaccination as early as possible. All of the vaccines are 100% effective in eliminating the risk of severe disease, which is the main goal. The onedose Johnson & Johnson vaccine is obviously quite convenient. Talk to your doctor if you have concerns about your particular treatment situation. If you are just recovering from an autologous stem-cell transplant (ASCT), then vaccination should be delayed by a month or two. Otherwise, it is safe to proceed. I suggest a commonsense adjustment to your treatment around the timing of vaccination. For example, try to get vaccinated during the week off from maintenance (such as with Revlimid® [lenalidomide]). Do not take more intensive treatment, such as Darzalex® (daratumumab) on the same day as vaccination. However, be very
cautious about any extended delays in treatment, since the top priority is still to keep your myeloma in remission. Adjustments for a few days around the day of vaccination are sufficient.
Masks Even if masks are no longer required in your area, myeloma patients should continue to wear masks. You just do NOT want to take any risk of getting infected. Even as vaccinations are rolling out, the use of masks remains essential for people with myeloma. If you have been vaccinated, chances are any COVID-19 infection will be mild, even if you get infected with one of the new variants. The new guidelines by the U.S. Centers for Disease Control and Prevention (CDC) for those who are vaccinated say it is okay to get together in small groups with grandchildren. This is a really encouraging step forward. However, flying to visit them is still not a great idea, at least until more people are vaccinated and community levels of infection are lower. Italy may have the right idea – recently the country unveiled “COVID-free” trains to tourist destinations.
Pandemic fatigue After a year, everyone is feeling the impact. Our lives are just not the same and the tragic losses in our communities are truly unbearable. The new COVID Relief Bill is definitely a triumph and will definitely help in the day-to-day and ongoing recovery for everyone. “From Vaccine to Variants: The Latest COVID-19 Safety Guidance” is an IMF Patient & Family Webinar available for replay at videos.myeloma.org under the IMF Patient & Family Seminar tab. Panelists include Dr. Paul G. Richardson (Dana-Farber Cancer Institute), Dr. Rafat Abonour (Indiana University), Dr. Warren Brenner (Boca Raton Regional Hospital), and others. This webinar offers myeloma patients and caregivers guidance for staying safe in our COVID-19 world, information on the latest trends on myeloma care and treatment, and tips for reducing stress in these challenging times. Stay safe and be well! MT
Patient and Family Webinar
From Vaccine to Variants:
The Latest COVID-19 Safety Guidance BRIAN G.M. DURIE, MD
PAUL G. RICHARDSON, MD
RAFAT ABONOUR, MD
WARREN BRENNER, MD
DONNA CATAMERO
YELAK BIRU
Live webinar recorded on March 13, 2021
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Scientific & Clinical
International Myeloma Working Group
New relapse guidelines highlight key role of IMWG experts By Dr. Brian G.M. Durie IMF Chairman of the Board
The International Myeloma Working Group (IMWG) has published new guidelines for the treatment of patients with relapsed or refractory (resistant to treatment) myeloma in the March 2021 issue of The Lancet Oncology. Authored by the world’s top myeloma experts, this manuscript analyzes the results of phase II and III clinical trials and creates a rational approach to therapy. As noted by Dr. S. Vincent Rajkumar (Mayo Clinic, Rochester), the co-chair of the IMWG: “Management of relapsed and refractory myeloma is extremely complex, with numerous drugs and regimens to choose from. This guideline provides specific recommendations that will greatly help hematologists and oncologists in managing patients with relapsed myeloma.” A major classification feature is whether or not patients are resistant to prior Revlimid® (lenalidomide) treatment, which is a widely used oral immunomodulatory agent for both primary therapy and maintenance. To fully assess these and other groups of patients, the IMWG team led by Dr. Philippe Moreau (University Hospital Hôtel-Dieu, Nantes, France) examined all clinical trials on relapsed myeloma published in English from 2013 to 2020. The summary and analyses of this large number of manuscripts is a huge resource for practicing hematologists and oncologists, as well as for patients and healthcare professionals overall. Although the major focus is on therapies that have received approval from the U.S. Food and Drug Administration (FDA) in the relapsed setting, newer therapies are also discussed. The recommendations consider both the number of prior therapies, as well as how to deal with both access and costs around the world. If certain combinations are not available or not feasible, then alternative options are provided. The substantial disparities in drug availability around the world represent an enormous ongoing challenge and are increasingly a focus of attention for IMWG members.
Putting the IMWG in perspective There are currently 256 IMWG members who are myeloma experts from around the globe. The ongoing collaborations have resulted in more than 50 publications on myeloma and its related disorders in prestigious journals, including major reference papers such as the following:
•R isk stratification for smoldering myeloma, • Myeloma-related bone disease therapy guidelines. From doctors to patients, the goal of the IMWG publications is to help better understand aspects of myeloma, and to help guide patients and their doctors to treating myeloma in the most effective way for every individual case.
Role of mass spectrometry Another recently published manuscript is the IMWG committee report on mass spectrometry for testing in myeloma and the whole range of plasma cell disorders. This report was sufficient to support appropriate reimbursement for this important test methodology, which will soon replace standard serum protein electrophoresis (SPEP) and immunofixation (IFE) test methodologies for both diagnostic and monitoring purposes. Mass spectrometry can distinguish between very low levels of myeloma protein and blood levels of monoclonal antibodies such as Darzalex® (daratumumab), the anti-CD38 monoclonal antibody used in treatment and potentially confused with residual myeloma protein. Sorting out the distinction allows confirmation of complete remission (the apparent residual “spike” is actually Darzalex, NOT myeloma protein). This confusion can occur in the low range of 0 to 0.4mg/dL of the spike. In addition, a sensitive version of mass spectrometry can also assess myeloma disease at a level similar to MRD testing. This means that a simple blood test can be used for very low-level disease-monitoring, which is much more attractive for patients than repeated bone marrow sampling! MT The mission of the International Myeloma Working Group (IMWG) is to conduct collaborative basic, clinical, and translational research to improve outcomes for myeloma patients while providing scientifically validated, critically appraised consensus guidelines for the myeloma community globally. Creating, supporting, and hosting the important work of the IMWG and its members is a critical element of the IMF’s mission to expand myeloma research and to educate myeloma patients around the world. For more information, please visit imwg.myeloma.org.
• Diagnostic criteria for myeloma, • New criteria for response assessment and the role of minimal residual disease (MRD),
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Scientific & Clinical
ASH 2020: Key Trends in By Dr. Brian G.M. Durie IMF Chairman of the Board
The 62nd Annual Meeting and Exposition of the American Society of Hematology (ASH) was a virtual experience that took place December 5–8, 2020. Nonetheless, the event brought together many clinicians, researchers, patients, advocates, reporters, and business-people from around the globe to hear firsthand the latest advances in the field of blood-related diseases. The total number of abstracts presented at the virtual ASH 2020 was down to 4,800 from 6,000 abstracts presented at the in-person event 2019. Still, many interesting and important abstracts were presented. Virtual ASH 2020 featured 688 abstracts dealing with myeloma, of which 179 were presented orally and the rest presented as posters. By comparison, there were 92 oral presentations in myeloma at ASH 2019. Despite the enormous difficulties of 2020, myeloma patients can take comfort in the fact that the approach to myeloma care continues to evolve rapidly. Important trial data accumulate, and many promising immune and other therapies are emerging. The key trends in myeloma care in 2021 will enhance outcomes, but patients and doctors will face serious challenges in the aftermath of the COVID-19 pandemic that result from the reduction of in-person doctors’ appointments and the slowdown of clinical trials. One bright spot is that it is easier now to get a second opinionconsultation using a virtual platform.
Initial therapy The major lesson from recent clinical trials of newly diagnosed patients is that the doctor’s choice of first therapy has the biggest impact on a patient’s overall response and survival. There is no doubt that “putting your best foot forward” is recommended. Depending on the circumstances, this can take different forms, such as the following: ¡ The preferred option – if it is acceptable to the patient and available, feasible, and manageable – is a three-drug (triplet) or four-drug (quadruplet) combination therapy that can achieve complete response (CR), with or without an autologous stemcell transplant (ASCT), ideally with NO residual disease (MRD negative at the one-per-million level or better, which is associated with best outcomes). ¡ Two-drug (e.g., Revlimid® [lenalidomide] + dexamethasone [Rd]) or three-drug regimens (e.g., Darzalex® [daratumumab] + Rd) are considered for elderly or fragile patients. ¡ Currently, the combination therapy of Velcade® [bortezomib] + Rd (VRd) remains the standard of care. The addition of ASCT produces a higher level of MRD negativity, but the total impact on ultimate longer-term survival is still being clarified. 8
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Main message For a standard-risk patient with myeloma, first remissions of four to five years are routinely expected. This translates into overall survival (OS) beyond seven years, a vast improvement over years past! The introduction of powerful, game-changing immune therapies (e.g., CAR T cells and/or bispecific antibodies) will undoubtedly substantially extend survival.
What about a cure? At a myeloma patient’s initial diagnosis, an optimistic outlook is now part of honest discussions of what to expect. However, a cure remains a work in progress. Through the IMF Black Swan Research Initiative®, two clinical trials have been conducted in patients with very early disease (high-risk smoldering multiple myeloma): 1. The CESAR clinical trial uses Kyprolis® (carfilzomib) + Rd + ASCT. 2. The ASCENT clinical trial uses an even more aggressive approach of Darzalex + Kyprolis + Rd, with or without ASCT. Both of these clinical trials achieve very high levels of MRD negativity in patients, more than 60%. The key is to see if these MRD negative remissions can be sustained beyond the current three to five years of follow-up time frames. It is exciting to await these outcomes! In the meantime, the really good news is that the preferred frontline therapy for patients outside of clinical trials is now very similar to the therapies used in these aggressive clinical trials in patients with early disease. We are very close to having the best strategies overall, with realistic hopes of very long survivals.
High-risk myeloma If high-risk disease features like 17p- or 1q+ are present, there is currently no standard enhanced treatment option. However, clinical trials are now being conducted that incorporate Kyprolis vs. Velcade and immune therapies or alternate novel agents to achieve better outcomes. This is clearly an area of unmet need.
New therapies and strategies Myeloma patients need as many options as possible when their disease relapses. We have been truly fortunate that so many options are available.
Key trends in the relapsed setting
¡ Treating relapse is a decisive effort to achieve an extended next remission. Putting your best foot forward is the way to go. ¡ To achieve the best and deepest response, three-drug regimens are recommended if feasible. info@myeloma.org
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Myeloma Care for 2021 ¡ Personalizing the regimen choice based on prior response is best. • If Revlimid has been used with potential resistance, switching to an alternate immunomodulatory drug (IMiD) like Pomalyst® (pomalidomide) as part of the Darzalex + Pomalyst + dexamethasone regimen, for example, or using an IMiD-free combination like Kyprolis + Darzalex + dexamethasone in the CANDOR clinical trial are clear options. • In another example, the results of the BOSTON clinical trial provide data to support the use of the Xpovio® (selinexor) + Velcade + dexamethasone triplet in the relapsed setting. • Moving forward, cereblon E3 ligase modulator (CELMoD) agents are also alternate choices if resistance to IMiDs has emerged. Full algorithms of choices have been recently presented and published in Blood Cancer Journal. • Early use of a CD38 monoclonal antibody presents a special challenge and is emerging as a major area of unmet need. In this situation, the new powerful immune therapies and other novel agents play a key role.
depth of initial response and enhance longer-term outcomes by producing truly excellent responses as part of the initial treatment strategies. It will clearly take time to develop the best practical regimens that are both highly effective and feasible for the patient in terms of toxicities and costs.
Role of MRD testing Patients who achieve MRD negative status do better, and it is reasonable to set a goal of achieving MRD negativity in ongoing and upcoming clinical trials. If one treatment produces more MRD negativity than another regimen, it can be preferred. MRD negativity typically occurs within 9 to 12 months of starting a particular therapy. This means that it is possible to compare one therapy with another after a year, without waiting to assess ultimate survival many years into the future. MRD testing thus provides a HUGE time advantage in assessing the relative benefit of one treatment versus another.
It has been fantastic to see the promising, very high levels of response with all of these new approaches. Remissions extending beyond one year are being seen, even in patients who have received extensive prior therapies. How best to sequence the various options is a topic for consultation between doctors and patients.
To use MRD as a surrogate in this fashion requires approval by the U.S. Food and Drug Administration (FDA). This approval is the goal of the i2TEAMM, a global consortium of myeloma researchers, through which data from more than 14,000 patients have been gathered and will be submitted to the FDA for review and, hopefully, approval. Feedback from initial submissions will be received soon.
New agents in early disease
Hope for 2021!
As we realize how powerful the new treatments are in achieving very deep responses, we are seeing a trend of their increasing use in early disease. Many clinical trials are planned or already underway to introduce new agents early to improve the
The ultimate key trend for 2021 is HOPE, which not only “springs eternal” but is both realistic and something that can guide us to ongoing improved outcomes for myeloma patients everywhere! MT
Live teleconference recorded on January 14, 2021
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Scientific & Clinical
ASH 2020: Dr. Durie’s Top 10 Abstracts By Dr. Brian G.M. Durie IMF Chairman of the Board
At the 2020 American Society of Hematology (ASH) meeting, the pattern we saw at ASH 2018 and ASH 2019 continued. Many abstracts detailed immune and genetic findings, novel immune therapies, and important updates were presented on key clinical trials whose earlier results have already been reported. This is how you build the future of myeloma care – understand the disease better, introduce or update information on cutting-edge therapies, and consolidate the current standards of care. I have grouped my nominees for 2020 Top 10 ASH abstracts into the most significant categories.
New biology of myeloma 1. Our understanding of myeloma is continuously evolving. Some important new concepts were presented at ASH 2020. The Spanish team brought together the Immunocell Study investigators to sequentially evaluate patients with smoldering multiple myeloma (SMM). They report results for 150 patients, 18 (12%) of whom developed active myeloma. The new observation is that the likelihood of progression correlated with the number of monoclonal plasma cells present in the peripheral blood (PB). This enhanced the International Myeloma Working Group (IMWG) Risk Stratification Model for SMM, which was published in Blood Cancer Journal in October 2020, and this is good news.
cells or greater) were again emphasized, as well as the overall acceptable benefit-risk profile. Other CAR T-cell abstracts included those reporting on the therapies bb21217, CT053, and P-BCMA-101.
Bispecific and related therapies 5. The results of the teclistamab clinical trial were updated. This anti-BCMA (B-cell maturation antigen) x CD3 bispecific antibody again demonstrated encouraging results. Patients have been treated using both intravenous (IV) and subcutaneous (SQ, shot under the skin) routes. A combined 120 patients were evaluable for response, with an ORR of 63.8%. The safety profile was acceptable. 6. The results of a first-in-human study of talquetamab, a GPRC5D / CD3 bispecific antibody, were also presented. This novel bispecific molecule demonstrated encouraging results in 137 treated patients. The ORR was 78% for IV dosing and 67% for SQ dosing, and the safety profile was manageable. Other related abstracts included studies of the therapies MED12228, REGN5458, BFCR4350A, and TNB-383B.
Clinical trial updates
3. The results of the CARTITUDE clinical trial were updated, with 97 patients treated as of May 2020. The overall response rate (ORR) remains high at 94.8%, with 55.7% of patients in stringent complete response (sCR). A key update with regard to adverse events (AEs) was that 10 patients have died during the study: 8 due to AEs and 2 due to disease progression. There is keen interest to better understand the AE profile.
7. The results of the IFM 2009 clinical trial comparing early vs. late ASCT in the setting of Velcade® (bortezomib) + Revlimid® (lenalidomide) + dexamethasone (VRd) induction were presented. With a median follow-up of 93 months (almost 8 years), the initial progression-free survival (PFS) remains improved for those initially transplanted (47.3 months vs. 35 months), but the overall survival is still no different with/ without frontline ASCT. The achievement of MRD-negative status continues to be the major predictor of enhanced outcomes. It is important to note that the ability to perform a delayed transplant is key to allow patients to potentially benefit from that option in the later relapse setting. 8. In the GRIFFIN clinical trial, after a median follow-up of 26.7 months, the addition of Darzalex® (daratumumab) to VRd with/without ASCT continues to contribute to improved outcomes with more and deeper responses. For example, the sCR rate was 42.4% vs. 32%. 9. The FORTE clinical trial of Kyprolis®(carfilzomib) + Revlimid + dexamethasone (KRd), with/without ASCT, which involved 474 previously untreated patients, continues to show added benefit with early ASCT compared to 12 cycles of KRd and Kyprolis + cyclophosphamide + dexamethasone (KCd) + ASCT. Maintenance with KR vs. Revlimid alone also improve PFS. There is much interest in comparing this study to the IFM 2009 study in terms of the role and value of early ASCT, even in the setting of excellent induction therapy with KRd. 10. The TOURMALINE-MM4 phase 3 clinical trial was updated to present the impact of MRD information. This study is important because it illustrates that achieving an MRD-negative status with ongoing therapy improves outcomes vs. patients with persistent MRD-positive disease. There is, thus, value in periodic monitoring during maintenance.
4. The ide-cel study was updated. Abecma® (idecabtagene vicleucel) has since been FDA-approved. The high response rates and the dose dependent benefit (at a dose of 150 x106
Well, there you have it! These are the ASH 2020 abstracts that I believe are important as we build our way steadily to improved outcomes for myeloma patients everywhere. MT
The researchers also noted that serial changes in immune cell subsets in PB occurred in a correlated fashion and will enhance our understanding of regulatory biology as myeloma evolves to an active state. The Spanish team has separately reported on the genetic transcriptional profiling of these circulating plasma cells and identified over-expression of two genes (CENPF and LGALS1) as indicators of more aggressive disease. Additional abstracts at ASH discussed specifically the genomics of plasma cell leukemia, the genetic loss of BCMA expression, and immune heterogeneity.
2. A separate abstract presented an analysis of why some patients are MRD-negative (as measured by NGF in the bone marrow) even though the myeloma protein still persists (as measured by immunofixation in the blood). Several explanations are offered, but further research is required to understand if a small population of cells persists but does not contribute to relapse (not dangerously mutated or actively growing).
CAR T-cell therapies
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info@myeloma.org
myeloma.org
Scientific & Clinical
2021 Brian D. Novis Research Grant IMF supports the innovative research by Dr. Eugenio Morelli of Dana-Farber Cancer Institute
By Susie Durie IMF President & CEO
In 1995, the IMF established the Brian D. Novis Research Grant program in memory of IMF co-founder and my late husband Brian D. Novis. For 25 years, these grants have been awarded annually to further research in all areas of myeloma in an effort to improve patient outcomes. To date, the IMF has funded 143 of the most promising projects in the field of myeloma, which have made contributions that bring us closer to fulfilling the IMF’s mission – a cure for myeloma. In January 2021, the IMF hosted an online event to award a junior grant to Dr. Eugenio Morelli and to thank Sheree and Ron Pask, and Gina Klemm, who organized the Miracles for Myeloma 5K event that is helping sponsor Dr. Morelli’s research. We were honored to be joined by Dr. Morelli and his mentor, Dr. Nikhil Munshi, the Pasks and their family, and Suzanne Battaglia and Ilana Kenville, who run the IMF’s Member Fundraising Program. IMF Chairman Dr. Brian G.M. Durie expressed how important it is for us to be able to continue to fund such important research projects. On behalf of the IMF, Dr. Durie presented the 2021 Brian D. Novis Research Grant to Dr. Morelli, a postdoctoral fellow at Dana-Farber Cancer Institute and Harvard Medical
School. Dr. Morelli earned his medical degree and completed a clinical research fellowship in medical oncology at Magna Graecia University of Catanzaro, Italy. Under the mentorship of Dr. Pierfrancesco Tassone, he gained expertise in molecular biology and experimental therapeutics of myeloma, pioneering the use of primary miRNA therapeutics of human cancer. In 2017, Dr. Morelli joined Dr. Munshi’s lab at Dana-Farber Cancer Institute, where he found the perfect environment to pursue his scientific interests. Please see the sidebar to learn more about Dr. Morelli’s innovative project. Eight years ago, the Miracles for Myeloma 5K was started by organizers who had never fundraised before, and now their event is funding its eighth Brian D. Novis Research Grant, this time supported by proceeds from their first ever Virtual 5K held amid a global pandemic. What a bright light in a dark time! Sheree shared that holding a virtual 5K was no easy task. “But, when I was first diagnosed, I made a commitment to make a difference in the world of myeloma, and with the support of my husband Ron and our children, and our wonderful event organizing committee, we did it! Things might be different right now due to the pandemic, but our commitment to myeloma research and our hopes for the future remain constant.” (continues on next page)
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Images from the virtual award ceremony in January 2021: 1. Dr. Eugenio Morelli. 2. Fundraisers Ron and Sheree Pask. 3. Dr. Brian G.M. Durie and Susie Durie. 4. Dr. Nikhil Munshi of Dana-Farber Cancer Institute.
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Scientific & Clinical BDN RESEARCH GRANT – CONTINUED FROM PREVIOUS PAGE Dr. Munshi added, “I believe that Dr. Morelli will find new ways of treating myeloma and probably lots of other cancers, and his work will set a trend for new directions. And people like Sheree and Ron Pask remind us of the urgency of our work and inspire us to reach for our target of finding a cure. Lastly, on behalf of my colleagues, I would like to say a special heartfelt thanks to Susie and Brian, who for so long have been doing such incredible work for myeloma at every angle.”
As we wrapped up our online grant presentation, we shared a photo of Brian D. Novis, the visionary who co-founded the IMF in 1990 and the man this research program is named after. The aim of the Brian D. Novis Research Grant program has always been to bring us closer to a cure, and we are very grateful to all the people who help support this important work and to all the researchers whose innovative work will get us to the finish line. Brian Novis said, “One person can make a difference, but two can make a miracle.” His words continue to inspire. MT
Characterization of RROL-driven lipogenic signaling in multiple myeloma cells Eugenio Morelli, MD Dana-Farber Cancer Institute, Boston, Massachusetts
Cellular ribonucleic acid (RNA) can serve as precursor for proteins or as effector molecule with active role in malignant transformation. Dr. Morelli and his team have discovered an RNA regulator of lipogenesis (RROL) that promotes the synthesis of lipids in myeloma cells. This unique approach to how myeloma cells grow and develop could result in new targeted ways to 12
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treat myeloma and improve the outcome for patients. The IMF’s Brian D. Novis Junior Research Grant program is proud to support this promising project, enabling Dr. Morelli to decode the molecular mechanism and biologic role of RROL in myeloma, and providing a significant boost to a rapid translation of these findings to the clinical setting. info@myeloma.org
myeloma.org
Education & Awareness
Myeloma Support Group Leaders and Advocates Take Part in ASH 2020 By Robin Tuohy IMF Vice President, Support Groups
community at large the latest information about myeloma research, therapies, and practice strategies.
The IMF has attended the Annual Meeting and Exposition of the American Society of Hematology (ASH) for many years, and we were “there” for the first virtual experience of the 62nd annual event in December 2020. Despite the unprecedented consequences of the COVID-19 pandemic, ASH 2020 brought together thousands of clinicians and researchers, including those who are doing important work in the field of myeloma.
I would like to express my personal thanks to Dr. Brian G.M. Durie and Susie Durie, and to the IMF’s industry partners who sup ported the attendance of myeloma advocates at ASH: Bristol Myers Squibb, Janssen, Karyopharm Therapeutics, and Takeda Oncology.
Myeloma news presented at ASH is important not only to researchers and clinicians, but also to the myeloma patient community. This is why the IMF’s ASH Team always includes patient leaders and advocates who then share with the myeloma
In closing, I invite you to read the articles by Dr. Brian G.M. Durie (see pages 8-10 of this edition of Myeloma Today), in which he writes about key clinical and scientific updates in myeloma. In addition, I would like to share with you the following insights from the support group leaders and advocates who represented the myeloma community and the IMF’s ASH Team:
Jack Aiello
Sheri Baker
Diagnosed with myeloma in 1995, Jack has attended ASH for 15 years. He is a facilitator of the San Francisco Bay Area Myeloma Support Group and a member of the IMF’s Global Myeloma Action Network (GMAN). “ There is no other single conference where so much information is presented about myeloma. I’m grateful to the IMF and their sponsors for registering me to attend… even from my home-office PC. Actually, one of the benefits of attending ASH virtually was to hear presentations that would be scheduled at the same time at an in-person meeting, and you’d need to make a decision which to attend. The virtual platform worked out well, though I did miss connecting face-to-face with others.” 800.452.CURE toll-free in USA and Canada
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Sheri was diagnosed with myeloma in 2011. In 2017, she started the Treasure Valley Multiple Myeloma Support Group in Boise, ID. In 2019, Sheri attended her first ASH meeting with the IMF. “ There are so many options for patients now, and we are lucky to have researchers working hard to find treatments and, ultimately, a cure. The results from clinical trials give myeloma patients hope for the future, hope that we will see our children grow up and get married. Hope that we will become grandparents and that we will live long enough to enjoy retirement. I thank the IMF for allowing me the opportunity to attend ASH and I thank the pharmaceutical companies that support the IMF. I hope we will be able to meet in person next time.” (continues on next page)
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Education & Awareness SG LEADERS & ADVOCATES AT ASH – CONTINUED FROM PREVIOUS PAGE
Yelak Biru
Yelak was diagnosed with stage III myeloma at age 25. Two decades later, he continues to successfully integrate myeloma into his life. Yelak is a member of the IMF’s Global Myeloma Action Network (GMAN) and an advocate for people with myeloma the world over. “ ASH is a premier conference where myeloma clinical trials that can change the approach to treatment are presented. This time, the virtual format allowed us to log in from the comfort of our home, and rewind and replay as needed. The discussions were really outstanding. Recently approved drugs are revolutionizing the treatment outcomes for myeloma patients.”
Cindy Chmielewski
A retired teacher, Cindy now educates a new group of “students” – myeloma patients and caregivers. She is @MyelomaTeacher on Twitter and the patient advocate on the Myeloma Steering Committee of the NCI’s Clinical Trial Network. She serves on the steering committee of the Philadelphia Multiple Myeloma Networking Group. “ I am honored to have attended ASH for nine years with the IMF, thanks to the generosity of our industry sponsors. The IMF understands the importance of education and the value of including the patient perspective at scientific meetings. Much of what I heard at ASH provided me with hope. Many new things are on the horizon!”
John DeFlice, MD
A 10-year myeloma survivor, Dr. DeFlice is a co-leader of the myeloma support group in Santa Fe, NM. He has attended ASH with the IMF for four years. “ The virtual iteration of ASH was a unique experience. One of the silver linings was the option to watch presentations live and then watch them again on replay. Every year we not only anticipate better treatments for myeloma but we also come to find new sources of hope, and this time the exciting immunotherapy developments for myeloma were the highlight of the meeting.”
Linda Huguelet
Diagnosed with myeloma in 2010 at age 46, Linda co-leads the Chattanooga Multiple Myeloma Networking Group alongside her husband. She has attended ASH with the IMF for eight years. “ I’m encouraged by the number of new myeloma therapies and therapy targets. The myeloma treatment landscape continues to evolve and get more complicated. In this case, complicated is a good thing because it means more options for us to tackle our ever-evolving myeloma.”
Teresa Miceli, RN
A 28-year nurse veteran, Teresa is recognized for her extensive experience in autologous and allogeneic transplantation. She is a member of the IMF’s Nurse Leadership Board and is the facilitator of the Multiple Myeloma Sharing Sessions in Rochester, MN. “ Presenters shared specific details of patients who participated in their clinical trials. This personal investment is what drives them to continue the important work towards curing myeloma.” 14
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Jim Omel, MD A retired Family Practice physician who was diagnosed with myeloma in 1997, Dr. Omel’s advocacy has included the NCI Myeloma Steering Committee, peer review and the NCI Director’s Board of Scientific Advisors, FDA Patient Representative, Alliance Cooperative Group Myeloma and Transplant Committees, CIBMTR, ASCO, NCCN Myeloma Guidelines reviewer, and 20 years leading the Central Nebraska Myeloma Support Group. “ One of my main interests is how myeloma experts approach sequencing of treatments for patients. The burgeoning number of choices includes approximately 190 different doublets and 1,140 different triplet combinations, and these numbers will grow in 2021 as new drugs and approaches come to market.”
Valarie Traynham Diagnosed in 2015, Valarie has rallied to educate, inspire, and empower other myeloma patients and caregivers. She is facilitator for the myeloma support group in Aurora, IL. “ The virtual ASH platform enabled me to listen to many of the 500 plus poster abstracts presented on myeloma. This is a lot more than I would have been able to do in the in-person setting. But whether in person or virtually, attending ASH is an invaluable learning experience and I am thankful to be able to have been a part of it.”
Michael Tuohy Michael was diagnosed in 2000 at age 36. In 2001, with the help of the IMF, he and his wife Robin started the Connecticut Multiple Myeloma Fighters Information Group, the first myeloma support in the state. “ I am a 21-year survivor of myeloma, and I am deeply grateful to all the myeloma experts who help us navigate through the complexities of individualized treatment. During one of our ASH support group leader meetings, our friend Jim Omel commented that it’s a blending of art and science. This is why myeloma patients need to see myeloma specialists. After attending the virtual ASH, virtual support group meetings, and virtual Patient & Family Seminars, it will be nice to see everyone in person when this pandemic is over. Until then, I wish you good health!”
Tiffany Williams Diagnosed with myeloma in 2013, Tiffany is the facilitator of the Multiple Myeloma Network for Orangeburg and a co-facilitator of the Charleston Area Multiple Myeloma Networking Group. “ Attending ASH as part of the IMF Social Media Team is a privilege. As in previous years, my mission was to offer hope by sharing my experience. The overall message that resonated for me was, we are inching closer and closer to a cure! Until then, myeloma has more treatment options now than ever before, and it is my hope that all patients benefit from those therapies and advances in care.” MT info@myeloma.org
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Education & Awareness
#W
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M-Power Charlotte is Changing the Face of Myeloma
By Dr. Joseph Mikhael IMF Chief Medical Officer
As discussed in my last column in Myeloma Today on Diversity, Equity, and Inclusion, the IMF is deeply committed to all patients with myeloma. The four pillars of the IMF – Research, Education, Support, and Advocacy – are the framework for all our efforts, including our commitment to reducing disparities in the myeloma community.
myeloma. After the diagnosis is made, myeloma patients can then receive their optimal care from a hematologist/oncologist.
PART 3:
MGUS screening program
The IMF has developed and launched the M-Power program to help empower individuals and communities to change the course of myeloma. This does, of course, imply that the current course of myeloma needs change. And indeed it does. Although we have made tremendous strides in the fight to cure this disease, as highlighted in the great work of the IMF’s Black Swan Research Initiative®, patient outcomes in myeloma remain unacceptable in many communities.
Although we know the incidence of monoclonal gammopathy of undetermined significance (MGUS) and myeloma is more than twice as common in people of African descent as in whites, little is known of the natural history of MGUS in black patients. Led by the myeloma physicians at the Levine Cancer Institute, the large-scale screening study is known as CHAAMP (CHarlotte African-American MGUS Project). We seek to screen 20,000 African-American individuals in the greater Charlotte area, with close follow-up of those identified as having MGUS.
The M-Power program – the “M” stands for myeloma – serves all who have been touched by this disease: communities, patients, caregivers, hematologists/oncologists, primary care doctors, nurses, and other healthcare professionals. M-Power is a longterm strategy for changing the course of myeloma in multiple geographic, racial, and socio-economic communities.
On March 20, the IMF held the first M-Power event in the AfricanAmerican community in the Charlotte, North Carolina region to educate people familiar and unfamiliar with myeloma about the disease, how the disease affects the African-American community, its optimal management and the importance of the patient voice and communication with the healthcare team.
The IMF has joined forces with Atrium Health Levine Cancer Institute’s Disparities & Outreach program to empower people in the Charlotte, North Carolina area to help remove barriers to care and improve both the short- and long-term outcomes of African-American patients with myeloma. In collaboration with Atrium Health and the Levine Cancer Institute, the IMF developed the following three-part program:
Important Facts about Myeloma and African-Americans
PART 1:
Engagement and empowerment Phase one of the M-Power program is an awareness campaign within the African-American community, both the medical and the non-medical. We are educating individuals about myeloma by multiple means to better understand the disease. We are demystifying the disease and encouraging people to seek med ical help if they may have signs or symptoms consistent with myeloma. Furthermore, it will advocate for greater support of myeloma patients and their families as they face this disease by connecting them to the resources of the IMF and Atrium Health.
PART 2:
Primary care education
MGUS and myeloma are TWICE as common in African-Americans. There is a longer time to diagnosis from the onset of symptoms. Survival gains in myeloma over the past decade have not been
seen in African-Americans to the same degree as in Caucasians. Although African-Americans comprise 20% of all MM patients, they represent only 8% of patients on clinical trials. This free, interactive online event featured multiple sessions, from “Myeloma for Patients Who Are Just Getting Started” to the latest in treatments and research. The comprehensive program also included a myeloma patient’s story and an opportunity for the audience to ask questions. We look forward to working with other communities nationwide as we seek to eliminate the disparities that are so prolific in myeloma. Please visit our website to watch a replay of the M-Power workshop at m-powercharlotte.myeloma.org, explore the Myeloma Tool Kit, and then join the M-Power movement and help change the course of myeloma! MT
The majority of myeloma patients are diagnosed by a primary care physician. However, the diagnosis of myeloma is often delayed in African-Americans when compared to Caucasians. A targeted education campaign directed at primary care physicians encourages a more accurate and timely diagnosis of
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Nurse Leadership Board Kevin Brigle, PhD, NP Massey Cancer Center Virginia Commonwealth University Donna D. Catamero, ANP-BC, OCN, CCRC
Myeloma Translational Research Mount Sinai Health System Kathleen Colson, RN, BSN, BS Dana-Farber Cancer Institute Deborah Doss, RN, OCN Dana-Farber Cancer Institute Beth Faiman,
PhD, RN, MSN, APN-BC, AOCN®, FAAN
Cleveland Clinic Taussig Cancer Institute Charise Gleason, MSN, NP-C, AOCNP Winship Cancer Institute of Emory University Michaela Hillengass, RN* Roswell Park Comprehensive Cancer Center *German certified
Patricia A. Mangan, RN, MSN, APRN-BC
Abramson Cancer Center University of Pennsylvania Ann McNeill, RN, MSN, APN John Theurer Cancer Center Hackensack University Medical Center Teresa S. Miceli, RN, BSN, OCN William von Liebig Transplant Center Mayo Clinic – Rochester Kimberly Noonan, DNP, ANP-BC, AOCN
Dana-Farber Cancer Institute Amy E. Pierre, RN, MSN, ANP-BC Memorial Sloan Kettering Cancer Center Tiffany Richards,
PhD, ANP-BC, AOCNP®
MD Anderson Cancer Center Sandra I. Rome, RN, MN, AOCN, CNS Cedars-Sinai Medical Center Mary Steinbach, DNP, APRN Huntsman Cancer Institute University of Utah Joseph D. Tariman,
PhD, RN, ANP-BC, FAAN
College of Science and Health De Paul University Daniel Verina, DNP, RN, ACNP-BC Mount Sinai Medical Center International Affiliates Tracy King, RN, MN Institute of Hematology Royal Prince Alfred Hospital
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The NLB Launches the Myeloma Treatment Discussion Tool and Prepares for the 2021 ONS Symposium By Diane Moran IMF Senior Vice President, Strategic Planning
The IMF founded the Nurse Leadership Board® (NLB) in 2006 as a professional partnership to represent nurse experts caring for myeloma patients. The ongoing mission of the NLB is to improve the nursing care and self-care of patients with myeloma. In 2021, the NLB has been busily working on many projects, including an online video symposium for nurses and a new patient tool to enhance treatment decision-making.
the deciding factor is often the preferences of the myeloma patient and their caregiver(s). This Myeloma Treatment Discussion Tool is designed to support a meaningful conversation to find the right treatment for an individual situation.”
At the upcoming 2021 (virtual) Congress of the Oncology Nursing Society (ONS), NLB members Beth Faiman, Amy Pierre, Donna Catamero, and Charise Gleason will present a symposium, “Case Studies in Multiple Myeloma Care for Challenging Times.” Discussion will include real-life scenarios with rapidly evolving myeloma therapies that use newly approved agents and combinations, symptom management, and infection prevention (including vaccination). The symposium will also broach the topic of care disparities. “The COVID-19 pandemic has revealed healthcare disparities among patients with myeloma, but there are steps that nurses can take to ensure that all their patients receive optimal care,” says Amy Pierre. “As nurses, we know our patients. We can ensure they receive the education and encouragement they need to actively participate in their healthcare. Our patients must have equal opportunities to receive new therapies, stem cell transplantation, and access to clinical trials.” The virtual video symposium is broken into three parts, and nurse participants can claim continuing education credit (up to 1.5 CNE) upon successful completion of the post-test. This engaging NLB symposium will certainly be a highlight at this year’s ONS Congress.
The tool begins with a series of statements to help patients and their caregivers to define their preferences, followed by a series of questions to further ensure that patients and their caregivers have the information needed to be active participants in treatment decisions.
The NLB nurses have also been hard at work on a Myeloma Treatment Discussion Tool that can help align patients and caregivers with their healthcare providers during treatment decisions. This tool, which is now available through the IMF, was created by Beth Faiman, Tracy King, Kim Noonan, Cindy Manchulenko, Charise Gleason, Joseph Tariman, Ann McNeill, and Mary Steinbach.
With the ONS symposium and the Myeloma Treatment Discussion Tool, 2021 is off to a busy start for the NLB! MT
Please visit nlb.myeloma.org to learn more about the work of the IMF’s Nurse Leadership Board
“Unfortunately, many patients, caregivers, and their healthcare providers find it difficult to have a discussion about treatment priorities,” says Beth Faiman. “Myeloma treatment is not one-size-fitsall. With so many excellent treatment options, info@myeloma.org
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Advocacy
IMF Advocacy Priorities for 2021 By Robin Levy IMF Senior Director, Public Policy & Advocacy
I’ve spent much of my career working in Washington, DC. These days, due to the pandemic, I spend most of my time on conference calls with lawmakers. Presidential inaugurations and the excitement that comes along with them, as well as the beginning of a new Congressional term, are some of my favorite times. They embody the rich history of our nation and provide a new opportunity to educate lawmakers about myeloma. I truly enjoy educating them about this disease, as well as the policy changes that would help the myeloma patients I am proud to serve. I am thrilled to share some of the International Myeloma Foundation’s advocacy priorities for 2021. Ensuring that myeloma patients are able to access optimal treatment and care is at the top of our priorities for the new Congress and Administration. First and foremost, the IMF is continuing our work as the leaders of the Coalition to Improve Access to Cancer Care (CIACC). This coalition allows us to advocate for our patients who are taking oral chemotherapy drugs. We are working with Congressional leaders to reintroduce the Cancer Drug Parity Act, a bill which will ensure that patients do not pay more for oral cancer therapies than they do for treatments administered by intravenous infusion. The Cancer Drug Parity Act only has an impact on people with private insurance, but we intend to advocate in other ways for Medicare patients as well. We have joined efforts in advocating for a Medicare out-of-pocket cap, which would place a limit on what patients pay for Medicare Part D drugs over the course of a year. This would give patients certainty and predictability. In the same vein, we have also let lawmakers know that it would be beneficial to spread these costs throughout the year so patients don’t face a large bill at the start of a year. This is known as a smoothing mechanism and would provide patients with additional support. We will also continue to work on anticipated access issues that could have an impact on myeloma patients as new treatments are approved by the U.S. Food and Drug Administration (FDA). The FDA recently approved Abecma® (idecabtagene vicleucel, a.k.a. ide-cel), a BCMA-directed CAR T-cell immunotherapy, for use in adult patients with relapsed and refractory myeloma who have had four or more prior lines of therapy and who have been “triple-class exposed.” (Read more about Abecma on page 5 of this Myeloma Today edition.) We have learned a lot from colleagues who work with other forms of cancer, for which CAR T-cell therapies have already been approved. In the past, we have advocated for this issue to the Centers for Medicare and Medicaid Services and have also educated Members of Congress. We will continue to work to ensure that all new therapies are accessible to patients when they are approved, and we look forward to educating new lawmakers about this important issue. Another crucial part of our work is to lobby for myeloma research. Each year, we lobby for increased cancer research funding through several avenues. In addition to the National Institutes of Health (NIH), there are other sources of cancer 800.452.CURE toll-free in USA and Canada
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research funding. For example, the Department of Defense (DoD) conducts research on diseases that are service-connected through their Congressionally Directed Medical Research Program (CDMRP). Congress specifies which diseases are eligible for research funds and we work to ensure blood cancers are included. I am looking forward to talking to new Members of Congress about myeloma and why it is an important disease to research. While most of our agenda is proactive, there are many instances where we must react quickly to proposed changes. In 2020, much of our advocacy agenda pivoted to ensuring myeloma patients’ perspectives were heard during the discussions surrounding the COVID-19 pandemic. We talked to lawmakers and built coalitions of cancer advocates to educate Washington about access to vaccinations, the triaging of care, and other issues. We anticipate this to continue and we pledge to make sure that the voice of the myeloma community is heard. We will educate lawmakers about myeloma and provide them with real-world examples of how patients and caregivers could be impacted. These are just some of our plans for 2021. We will bring you new opportunities to engage your legislators and invite you to sign up for our newsletter at myeloma.org/myeloma-action-center. We are looking forward to working with you this year! MT
SUPPORT FOR VETERANS Get Your VAM Guide to VA Benefits
YOU ARE NOT ALONE. THE IMF IS HERE TO HELP. VETERANS.MYELOMA.ORG
The Veterans Against Myeloma (VAM) Guide to VA Benefits assembles information from various resources into one cohesive narrative specific to myeloma. This VAM Guide is intended to help Veterans with myeloma, their caregivers, and family members to navigate the VA claims process, myeloma service connections, understanding authoritative bodies, and VA claim appeals. If you have trouble working with the VA or using this guide, please contact the IMF InfoLine at 800-452-CURE, Monday through Friday 0900–1600 Pacific Standard Time.
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Shop for the Cause! Whether you shop on Amazon for special occasions or for your daily needs, please designate the International Myeloma Foundation as your preferred charity. At no added cost to you, Amazon will donate to the IMF a percentage of your purchases if you access your account through smile.amazon.com – IT’S THAT SIMPLE! Same products. Same prices. Same service.
smile.amazon.com
PARTNERS IN PROGRESS Supporting Myeloma Research Toward a Cure The IMF is improving the outcomes of myeloma patients and moving towards a cure. With clinicians and researchers from top institutions around the world working together, the IMF is taking the first steps to identifying curative treatments in subsets of patients. With philanthropic support, you can help the IMF change the trajectory of myeloma. If you would like to learn how to partner with us, please contact:
Lynn K. Green, Ed.D.
Senior Vice President, Philanthropy
334.332.0888 – lgreen@myeloma.org TOGETHER WE CAN MAKE “MYELOMA HISTORY!”
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Please visit myeloma.org for the latest information from the IMF. For news from our international partners, please visit these websites:
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