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Myeloma Voices

"Keep Smiling" my friend!


Pat Killingsworth was a fighter, an advocate, someone who always spoke his mind, and was always there to help others.   I'll mostly remember Pat as a friend. 

Gerry Landy, co-leader of the Stillwater, MN Myeloma Support group shared with me the following:  

"As you know, Pat was a very active member of the Stillwater, MN support group before he moved to Florida.  In fact he would pepper my wife, Annie, with questions about blog writing, website editing and self publishing.  We knew he was up to something but I don't think even he had any inkling as to how popular (and influential) he would become in the Myeloma community.  It was an honor to know him and a pleasure to see his success.   As was the case with the passing of Mike Katz, it's up to the rest of us to honer him by carrying on with his work."

The IMF helped Pat and his co-founders to start the Nature Coast, Florida myeloma support group in 2010.   Pat was a quick learner and even back then wanted to help as many people as possible.  We invited Pat to come to the American Society of Hematology meetings in San Diego in 2011, and although his back hurt every day, he soldiered on and attended as many oral and poster sessions as possible so he could bring the information back to other patients and caregivers.  Pat came to ASH with the IMF in 2011 (San Diego), 2012 (Atlanta), and 2013 (New Orleans).  Each ASH program was chock full of hope that Pat reported back by blogging and sharing his observations and opinion.

In 2012 Pat came with the IMF to the annual Support Group Leader Summit.  He was a 1st timer there, and looking back at a blog he wrote, I'd like to share a quote from our dear friend:

"Sure, the primary goal of the Support Group Leader Summit was to bring back lots of ideas and information to our respective support groups.  But in one short weekend, I was able to turn a dozen former acquaintances--along with more than twice that many survivors, caregivers and IMF team members that I just met--into at I hope will be life-long friends."

Here's Pat's full blog from the Summit: 


Pat was tireless, always asking questions and sharing his knowledge.  His good work helped so many myeloma patients and caregivers.  Rest in Peace my friend, knowing that your life mattered.

"Keep Smiling" my friend!

Robin Tuohy

I met Mike Katz in 1993, when he came to the first ever Patient & Family Seminar. He walked over and introduced himself to me and told me that he and my late husband Brian Novis had spoken on the phone. I was so surprised and touched that this man took the time to fly all the way to Los Angeles to tell me about that conversation. I didn't realize that he, too, had myeloma because he was young, handsome and looked just great. We became great friends.

Mike Katz was many things: hero, friend, IMF board member, opera enthusiast, family man, lover of roller coasters, possessor of a great sense of humor, intolerant of BS, and champion extraordinaire for myeloma patients. Mike was laid to rest on Monday after waging a 25-year battle against myeloma.

SusieMike.pngFrom the moment I met Mike, my life changed--as did that of patients all over the world. Mike was a force to be reckoned with, and it was always best to be on the side he fought for. He quickly realized the value of having patients communicate with one another, share experiences, share information, and share their fears, so they could overcome them. Then they could share hope, too. Mike was also a computer geek who built the IMF website, developed our database and provided a key portal for patients and caregivers to access information and support. He did everything and more for the IMF and the entire myeloma community, patients first and foremost, but he also worked closely with doctors and nurses. In other words there was no area that was off limits to Mike, and we all benefited from his passion and his tenacity.

A survivor of both multiple myeloma and rectal cancer, Mike showed us all how to live with life-threatening illness. "I've always chosen to live my life as if I didn't have cancer," he once said. "I just face forward and try to do everything I want to do, working around symptoms and treatment side effects." For 25 years, Mike worked as a patient advocate across a broad spectrum of cancers, serving as Chair of the Patient Representatives Committee at the Eastern Cooperative Oncology Group (ECOG), Chair of the NCI's Director's Consumer Liaison Group, a Patient Consultant for the U.S. Food and Drug Administration (FDA), and on the Association of Cancer Online Resources (ACOR).

He very passionately advocated to lower the standard dose of dexamethasone, which at that time was 40 mg for four consecutive days--a high dose of dexamethasone that today, thankfully, no patient has to endure. Mike worked with the chair of the ECOG Myeloma Committee and proposed the idea of a head-to-head trial of standard dose and low-dose dexamethasone. To no one's surprise, the patients on low-dose dexamethasone did much better and didn't have to endure the horrible side effects. And many a marriage was saved!

Mike was also the principal driver behind design and implementation of the comprehensive Myeloma Manager Personal Care Assistant application in 2008. This application is designed to help myeloma patients and caregivers manage their care, and can be downloaded free of charge on the IMF website.

At major cancer conferences around the world, Mike recorded video interviews with leading physicians, which were posted on the IMF website and other social media outlets for patients to view directly.

Mike also helped establish a link between bisphosphonates and osteonecrosis of the jaw (ONJ), and he helped change National Cancer Institute (NCI) policies regarding institutional review boards that removed serious delays in initiating clinical trials.

In 2014, the American Society of Clinical Oncology (ASCO) recognized Mike with the 2014 Partners in Progress Award for his exceptional achievements, outstanding work, and dedication as a myeloma advocate. "Working with the IMF and others to help patients and caregivers and to advance myeloma research is an important and an incredibly rewarding part of my life. I am grateful to the community and to ASCO for this very special honor," Mike said at the time.

It is we who are grateful to have known Mike and to have been the beneficiaries of his wisdom, support, keen mind, problem-solving skills, honesty, and sharp wit. Mike loved to laugh and we had many laughs over the years as we traveled the world together. He was my dear, dear friend and I just can't imagine life without him.

Our hearts go out to his wonderful wife, Susie, their three sons Jason, Jeffrey and Jonathan, and their families, which now include many grandchildren.

Mike, we miss you, we love you and will remember you always.

Susie Novis
International Myeloma Foundation


Beautiful tribute to Mike, Susie. He was one of a kind. He did so much for so many people. Ken and I are sending our sympathy and prayers to Mike's family and the IMF family. Thank you Mike.

What a wonderful inspiration to MM patients, I only wish I could have met him!

Susie, thank you for your moving and love-filled tribute to a man for all seasons. Mike Katz's energy and appearances at the
seminars are memorable. He gave us hope and laughter in the midst of our confusion. His presence made us more eager to learn about multiple myeloma, to live with this cancer and to enjoy each day. Diagnosed with MGUS in 1994 and MM in December,2001,
I have celebrated life every day. Thanks, Mike for the inspiration.

Mike was such an inspiration and I learned from his presentations at several IMF Seminars, not to live life as a cancer patient but to look to the future with confidence. He inspired me and reinspired me every time I was fortunate to hear him speak!
He will be sorely missed by all of MM patients.

I am so sad to learn of Mike's passing. I attend a few support group meetings at St. Vincents when I was newly diagnosed and was always amazed at the amount of knowledge Mike possessed. He was so kind and gave me hope. I have spoken about him many times over the years. My heart goes out to his family and friends.

Mike was an inspiration to ALL. I always read everything he posted. His battle was faught with dignity and his many contributions to the lives of cancer survivors are outstanding. My deepest sympathies to his family. He will be misses by ALL! Joan Beauchamp, MM, 9 years

Thank you, Mike. Thank you for returning my call that late evening, in January 2006. You generously provided me with your connections at Saint Vincent's Hospital. This lovely gift was the hope we so desperately needed.

At the time my mother was given 3-6 months to live, we instead were blessed with 4 years.

I am ETERNALLY thankful.

When I discovered I had Mgus I went to st Vincent pne15th street in N Y I met mike there . He headed our group and gave us all hope. He was a wonderful person and will be very missed. Shirley Lustig

When I was diagnosed in 1999, the term multiple myeloma was foreign to me, having never heard it before When exploring the Internet, I encountered Mike who was a life saver to me. He was patient explaining terms and accomplishments at that time, he will always have a special place in my heart. He was a rare, thoughtful, very intelligent, kind, hard-working person.

We missed Mike at the Patient Seminar in SF this spring--sad to hear of his passing. He was a great advocate for just living your life, in spite of MM and all the treatments we go through...

Great tribute, Susie. I will add that the portal Mike developed for patient and caregiver support included the Listserv which he closely monitored for years and more recently smartpatient.com which he also closely monitored and offered great advice. These chat sites provide(d) an invaluable way for patients and caregivers to exchange critical information such as good myeloma specialists in specific geographic areas, how patients are being cared for with various providers around the country and sometimes the world. They are lifelines for the myeloma community. He gave a lot of information on something I posted on smartpatient as recently as March 12 so he was still helping the myeloma community to the end.

There are a lot of expressions of gratitude, tributes and condolences on the smartpatient.com website.

I've been to more than a handful of Patient Conferences and witnessed Mike's presentations and cheerleading in quite a few of them. So I was one of the many whom Mike inspired and it grieves me to hear of this loss. I'm going on my tenth year (in August) of my myeloma diagnosis and I'm in awe of how much longer Mike endured the disease and the horrible suffering the chemo meds cause. I often get to the point of contemplating giving up. I wonder if he had those moments too, but -- like me -- decided to persevere. Whether or not he despaired, he definitely persevered. And used his special skills and talents and wisdom to keep the light shining for many, many others. And I hope there's a way Mike can have known how much I appreciated his unique drive to live and thrive and how much I appreciated his graciously sharing his determination to live fully and well with us all. There's no replacing him. Thank you as well, Susie, for the beautiful and touching tribute, and for letting us know of his death. While I'm waxing appreciative, let me say that I appreciate your similar spirit, as well as your wonderful husband's, in keeping hope alive for me and others, and for how matter-of-fact your generosity -- and Brian's -- feels to me. As did Mike's.

What a great guy he was. The Myeloma Manager is a Godsend!!

I am so very sad at hearing this news, I wasn't aware he was not doing well, trying to read every correspondence he had, he was so knowledgeable and being so new to this disease, 4 years, he was a great inspiration, and an even greater hope. I pray for the family to find peace in the knowledge that he was admired and loved by many who never had the honor to meet him. He has done so much for this community, a great man and surely will be missed.

Along with everything listed above (I had no idea), I will always be grateful to him for demonstrating that a diagnosis of myeloma need not be the end of my retirement travel dreams.


I echo your sentiments about Mike. He really was a hero to all Myeloma patients everywhere. I knew him for many years and also appreciated his outlook on dealing with his disease. He will truly be missed by all who knew him. The Myeloma community will be at a loss without him.

My condolences go out to his entire family.

Marilyn Alexander
Philadelphia Multiple Myeloma Networking Group

He was truly an inspiration. His polite and respectful mannerism was adorned with an amazing inner strength. I feel honored to have met him and gotten to know him.

What a beautiful tribute, Susie. Mike will be missed terribly in our tight-knit MM community.

Mike was an awesome individual that had a huge effect on patients and caregivers alike at the many Myeloma functions that he participated in. Jerry and I had a great respect for him and his incredible knowledge of Myeloma and ways to help everyone with handling this disease. Thanks, Mike for being you and being willing to reach out to everyone else and being an great example of how to share the hope of beating Multiple Myeloma. Marcia Sawyer

Thank you for sharing your personal story of Mike, Susie. You both were pioneers in the field of myeloma. He will be remembered fondly by many.

I was fortunate to attend an IMF patient and family seminar in Chicago in 2001 where Mike was a presenter. The other patient speaker that day was Judy Goldman. I had been officially diagnosed in 1998 and this was my second seminar. For me that weekend was a critical turning point. Mike and Judy gave me hope that just maybe I could live long enough to experience the births of many grandchildren and that I could establish goals and make plans. Because of them I knew that I truly did have a future. Up to that point I had not yet sought out the advice of an MM specialist but Mike and Judy clarified for me that this should be my first priority. I had been researching the list of those who specialized in Myeloma and their medical facilities, but I lived half way between Indianapolis and Cincinnati and at that time there were no MM docs in either city. I was stalling trying to figure out if I should head south or north or maybe way out west. That weekend took me from floundering around to a well defined and life-saving path. Drs. Seema Singhal and Jayesh Mehta were presenters and were in the process of establishing a Myeloma department at Northwestern Medical Foundation in Chicago, my hometown. Over the years I have been fortunate to hear Mike give his
inspiring presentations and to watch him interact with patients. He was an angel among us, a very caring man and also attentive to those who needed his advice or help. I was in awe of the technology and ideas that he brought to the IMF and we patients to help us manage our condition. Because of Mike our medical partners began rethinking how to better manage dosing of our meds to prevent side effects that so robbed us of quality of life. And then I was amazed again when he developed the device that would allow we patients to provide feedback at IMF seminars that, in turn, MM researchers used to our advantage. Thank you, Mike. I don't think that we ever really talked, just exchanged pleasantries, but whenever plans were made to attend a seminar I would look for you. Always had to see if you were moving around as quickly as ever and as determined as ever and always with a smile....well except for those times when there was a techno glitch. But you always got it fixed, just like you helped "fix" so many of us. Blessings to your family and my hope is that the memories created by you and your family will sustain them in the years ahead. Such a legacy you have left for them and for us. Thank you.

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A Myeloma Patient Seminar, Italian Style

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More than 100 people gathered in the town of Udine, Italy last week for a lively and informative myeloma patient event hosted by the Italian blood cancer patient group AIL Pazienti (Associazione Italiana contro le Leucemie, Linfomi e Mieloma).

The Udine group is an extremely active chapter of the AIL, which has more than 400 volunteers working throughout the year organizing events, fundraising and raising awareness of multiple myeloma.

The April 18th event was led by Maria Grazia Santuz, President of AIL Udine, and Giuseppe Gioffre, the organization's chief administrator, and focused on educating myeloma patients and their loved ones. An impressive lineup of speakers was followed by lively Q&A sessions.

Included in the day's proceedings were presentations from Dr. Renato Zambello of  Padova, who spoke on myeloma therapies for the young; Dr. Filippo Gherlinzoni of Treviso, who addressed therapy options for elderly patients; Dr. Norbert Pescota of Bolzano, who spoke about treatment options; Dr. Fabio Barban of Udine, who spoke about putting patients at the center of the cure process; Vittorio Meneghini of Verona, who spoke on treatment options; Margherita Venturini of Udine, who covered the management of treatment side effects and supportive therapy; and Francesca Patriarca of Udine, who concluded the meeting by explaining new clinical trials, drug treatments and cellular therapy for multiple myeloma.

A poetry reading by actress Sonia Cossettini served as a lovely antidote to the day's strong focus on myeloma treatment, education and awareness. As with any successful program of this kind, the AIL event was a highly collaborative, drawing speakers from across Italy to share their expertise and insights for the benefit of the multiple myeloma patients. Special thanks to Paola Angaroni and Melania Quattrociocchi of AIL Rome for their support.

Nadia Elkebir
IMF Director of Europe and the Middle East

Dr. Renato Zambello

Dr. Filippo Gherlinzoni

Dr. Norbert Pescosta

Dr. Vittorio Meneghini

Dr. Francesca Patriarca

Maria Grazia Santuz

Fabio Barban

Renato Fanin

Nadia Elkebir, IMF

AIL Volunteers

Guiseppe & Nadia

Paola & Melania

Photos courtesy : Dario Paoli 


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Donkey in Albany.jpg
Myeloma can be a stressful diagnosis - not only for patients but also their loved ones. Graphic novelist Sarita Johnson learned this first-hand in 2010 when her partner Susan was diagnosed with the disease.

Sarita and Susan had just spent a month on vacation in New Mexico, exploring what life would be like in the small town where they hoped to retire. Susan was experiencing mysterious pain in her back and numbness in her legs. It was so bad, they cut the trip short. Shortly thereafter she was diagnosed with cancer. The news was "devastating," Sarita said. While Susan went in for tests and treatments, Sarita turned to the one thing she knew best: art. 

"While hanging out in the hospital with her and racked with anxiety, I decided that documenting the experience would be good therapy for me," Sarita said.

Sarita began sketching her experiences, chronicling the hospital visits, the tests, the sleepless nights, the worry, and the love - all in a new and unique graphic novel she titled A Donkey in Albany: A Year with Multiple Myeloma. The title comes from an image Sarita said calmed her in the earlier days of diagnosis, when the future seemed bleak. The story spans the first year after Susan's diagnosis, from diagnosis to steam cell transplant.

"I remember saying to myself, 'Gosh, I wish I could just ride a donkey along an empty desert highway, with the sun painting everything warm,'" Sarita said.

"I have a chapter called Myeloma 101 in which I explain in the simplest and clearest of terms what the disease is. It's one of my favorite chapters because not only is it visually enjoyable, but also it helped clarify for me what exactly was going on with Susan. The real focus of the book is actually my experience as a caregiver and how I found strength such a harrowing year," Sarita said.

Sarita has published her comic, page by page, on her blog (http://www.donkeyinalbany.com) with the hope that it will help other myeloma patients, whether they are looking for an escape or want to learn more about the disease by way of one family's myeloma experience. Sarita welcomes comments and feedback from other myeloma patients and their family members. She also hopes a publisher will soon pick up the title so it can touch and inspire even more lives.

"I want to make sure that this particular story gets out and read as soon as possible," Sarita said. "There is still no cure for myeloma, which means that families are still suffering because of it. So, I think it could be helpful and validating to others who are also going through this disease."


We are going through a similar journey. It is 'comforting' to know we are not alone. A comic strip is a 'refreshing' way of documenting a long, stressful and uncertain journey. Where can we get the complete work?

Good luck on your journey. You can find Sarita Johnson's wonderful story-in-progress here: http://www.donkeyinalbany.com/

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The answer is a definite maybe! As myeloma evolves, it does so within the complex immune microenvironment of the bone marrow. The most well-known impact is the reduction in normal plasma cells and reductions in normal immunoglobulin production, causing reduced normal gamma globulins as part of the SPEP (serum protein electrophoresis) pattern. But there are many other effects of the disease which result in reduced or abnormal immune responses. The most well-documented feature of myeloma is the reduced ability to fight infections. Fortunately, this is often not too severe, despite reduced immunity and immunoglobulin levels, while some patients with recurrent infections can benefit from intravenous gamma-globulin infusions, others may benefit from ongoing antibiotic use.

So how can normal immune function be recovered in myeloma patients? This has been extensively investigated for several decades. For example, when it was first demonstrated that interferons (α and β) were helpful in myeloma treatment, chemicals (such as poly ICLC) were developed to trigger the body to produce more helpful interferons. This proved to be of limited value because the immune system became exhausted: only so much interferon could be produced. Likewise, many vaccine approaches attempting to trigger the immune system to reject the myeloma failed because complex inhibitory factors could not be overcome.

Now we have many new immune approaches emerging. One that is the farthest along is elotuzumab (anti-SLAM F7 monoclonal antibody), which triggers natural killer (NK) cells in the bone marrow to attack myeloma cells. This antibody has not worked by itself, but is working well in combination with Revlimid® plus dexamethasone, as well as with Velcade® and dexamethasone (in smaller studies). It seems that increased destruction of the myeloma, which occurs with the combination approaches, is key. This is also what we are seeing with the anti-CD38 monoclonal antibodies (daratumumab, SAR 650948, and MOR 202). With these anti-CD38 antibodies, there is direct killing of the myeloma cell plus recruitment of surrounding NK cells and macrophages to help mop up damaged myeloma cells.

So what we are seeing are new immune methods to reduce the myeloma rather than just modulate the immune response. Reducing the myeloma is actually the key to success. Reducing the myeloma decreases production of all the complex immune inhibitor factors and allows the immune system to bounce back. In patients who have excellent response, after ASCT, we see bounce-back of the normal plasma cells with production of mini monoclonal spikes (called oligoclonal spikes), which is a good sign - actually a very good sign - excellent response and potential long remission. Another feature of the recovering immune system is an increase in T-lymphocytes, which produce interleukin-17 (IL-17): these are called Th-IL-17. This is not something that is normal. This is a reaction to myeloma and successful treatment. Whether this reaction can be usefully enhanced has not been investigated. Whether additional immune enhancement can convert excellent response to cure is a tantalizing but unanswered question.

So is it possible to boost the immune system? Yes, by treating the myeloma and removing inhibitory factors! Whether tweaking, triggering or enhancing the immune system as a sole form of treatment can work remains to be seen. But it is very exciting that immune methodologies are providing a new way forward and allowing us to understand how immune manipulation can help. I am sure there will be many updates to come on this topic! 

Dr. Durie sincerely appreciates and reads all comments left here. However, he cannot answer specific medical questions and encourages readers to contact the trained IMF InfoLine staff instead. Specific medical questions posted here will be forwarded to the IMF InfoLine. Questions sent to the InfoLine are answered with input from Dr. Durie and/or other scientific advisors and IMWG members as appropriate, but will not be posted here. To contact the IMF InfoLine, call 800-452-CURE, toll-free in the US and Canada, or send an email to infoline@myeloma.org. InfoLine hours are 9 am to 4 pm PT. Thank you.

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In the current issue of the Mayo Clinic Proceedings, Hagop Kantarjian, MD and S. Vincent Rajkumar, MD discuss the high cost of cancer drugs and propose some solutions. The high cost of cancer drugs is widely known. Unfortunately, this new article does not provide solutions that have an immediate chance of implementation and/or success.

For example, the authors propose government action to allow Medicare to negotiate on drug prices, which is unlikely to occur in the current political environment.

What is needed instead, I believe, is a discussion involving all the stakeholders--pharmaceutical companies and insurance carriers, but also health economists and policy advisors to mediate and guide the discussions. In this setting, realistic solutions can hopefully emerge which transcend finger-pointing. We need specific action items and, since each cancer is different, we need recommendations tailored to the myeloma community. This requires input from both myeloma doctors and patients.

Focus on best outcomes

What constructive suggestions can be made by the myeloma community? First, I would argue, there must be a focus on the best outcomes. It is helpful that we now have a focus on earlier diagnosis, which leads to more decisive treatment choices to achieve the maximum response. For example, triple therapy to achieve VGPR or ≥ CR or even MRD negative status is highly feasible. An optimal treatment package, a pathway or guideline, can be developed incorporating induction, ASCT if appropriate and consolidation and/or maintenance.  We  can lower the cost of myeloma treatment by creating a competitive environment in which physicians see best outcomes, patients can opt for a tolerable and effective approach, and regulators can see a manageable cost structure with clear value.

A drug holiday?

An increasing subset of patients can achieve CR or better and avoid indefinite treatment. We already know that good risk patients achieving excellent responses can be safely followed off therapy. The result?  A treatment-free interval - a drug holiday - a sought-after goal for many patients, which is also attractive to regulators who manage total costs. For now, many patients need and benefit from ongoing therapy. For those patients, a price structure is needed that does not confer a huge burden on the individual or the healthcare system. This is where creative solutions are required.

Another question that must be raised in any discussion of cancer drug costs is: What do we do about drugs that offer only limited increases in survival at significant costs and toxicities? There must be a way to prioritize the search for new drugs that really make a difference - those that achieve improvement in survival of years, not months. It is to everyone's advantage to improve both outcomes and value. Such drugs should receive a competitive edge.

Clearly, costs cannot continue to skyrocket. Good health is not a commodity like a widescreen TV. A widescreen TV can sometimes fit in the family budget. But, healthcare costs must fit within both the family and total healthcare budgets. Yes, profits to recoup drug discovery and development are fair. But there has to be a limit that stops short of bankrupting the whole healthcare system.

How to achieve this in an equitable fashion is the challenge - but a challenge which we can no longer ignore. All parties must work together to come up with solutions which work and can be implemented.

If you have ideas about what can work, let us know. At the IMWG Summit in June, a working group will debate the topic. But there must also be many great ideas out there which can help chart a way forward. So let the dialogue begin!

Dr. Durie sincerely appreciates and reads all comments left here. However, he cannot answer specific medical questions and encourages readers to contact the trained IMF InfoLine staff instead. Specific medical questions posted here will be forwarded to the IMF InfoLine. Questions sent to the InfoLine are answered with input from Dr. Durie and/or other scientific advisors and IMWG members as appropriate, but will not be posted here. To contact the IMF InfoLine, call 800-452-CURE, toll-free in the US and Canada, or send an email to infoline@myeloma.org. InfoLine hours are 9 am to 4 pm PT. Thank you.

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On Saturday and Sunday, the IMF was honored to chair a workshop in Tokyo, Japan, which introduced Next Generation Flow Cytometry to more than 160 researchers and technicians eager to learn about this super-sensitive automated new myeloma measuring method.

Next Generation Flow (NGF), as I've written about here, here and here, was developed with the support of the IMF's signature Black Swan Research Initiative® and is a crucial step in our ability to chart a pathway to a cure. (Watch IMF's video about the groundbreaking new technology here.)

Susie Novis, IMF president, welcomed workshop attendees on behalf of the organization, and was followed by Hirokazu Murakami (current President of the Japanese Society of Myeloma) and Kenshi Suzuki (Deputy Director of the Red Cross Medical Center), who greeted the audience and thanked the IMF for organizing the workshop. The opening session was co-chaired by myself and Dr. Kazuyuki Shimizu. Dr. Hiroyuki Takamatsu of Kanazawa University set the stage for the presentations to come by describing the perspective of minimal residual disease (MRD) testing in Japan.

Up to now, the key effort in Japan has been to assess NGS (Next Generation Sequencing), which measures MRD using molecular data. As such, Dr. Takamatsu presented a recap of his NGS presentation at the ASH 2014 meeting in December in San Francisco. However, Japanese investigators at the workshop were very keen to hear about Next Generation Flow testing, which can be equally sensitive and much more practical for broad use in Japan.

Throughout the day, Dr. Alberto Orfao of the University of Salamanca in Spain and Dr. Jacques van Dongen of Erasmus Medical Center in The Netherlands examined the current, conventional flow methods of detecting MRD in myeloma as a backdrop to their presentations on the Next Generation Flow technology details, including panel design, data analysis and interpretation, and cross-platform applicability. The day's closing remarks (by myself, Dr. Orfao and Dr. van Dongen) concentrated on the vast improvement in disease detection NGF represents.

It was really heartwarming--and very much appreciated--to hear Dr. Takamatsu, who started the day talking about NGS, tell Dr. Orfao: "I am convinced about the sensitivity and practicality of the NGF method, and look forward to implementing NGF at my center as soon as possible!"

In addition, Dr. Suzuki indicated similar enthusiasm and a desire to adopt the NGF broadly for JMS investigators.

The Japanese were actually the first to hear that based upon the most recent testing, the new NGF method is even more sensitive than originally anticipated: able to detect one myeloma cell in a million or even one in 10 million! In addition, as Dr. Orfao explained, the computer software program for automatic data analysis has been improved since our last workshop was presented in New York at Memorial Sloan Kettering Cancer Center, with a data analysis time of now only 12 minutes--reduced from several hours! This means that samples can easily be analyzed with same-day results, which is a huge advantage.

On the second day of the workshop, participants attended "hands-on" training, courtesy of Juan Flores-Montero of the University of Salamanca. Dr. Suzuki had done a fantastic job in facilitating the "hands-on" session, which went very smoothly and generated tremendous interest from the participants. Especially helpful was a practical, step-by-step video, subtitled in Japanese, which had been prepared in advance by the Salamanca team to demonstrate sample preparation.

Members of IMF's Asian Myeloma Network (AMN) from Japan, China, Korea, Singapore, Taiwan, and Thailand engaged in a spirited discussion of the role NGF might play in their respective myeloma treatment practice and research.

Speaking on behalf of the AMN team, Dr. Wee Joo Chng said that the new NGF method represents a major step forward for the myeloma community and provides a reliable, accessible method for routine MRD testing throughout Asia. According to Dr. Chng, who chairs the AMN Clinical Trials Group, the plan is to integrate NGF into upcoming trials.

The IMF's Tokyo Next Gen Flow Workshop ended on a very positive note, with plans to move forward in the near future. The concrete plans at the end of the workshop were many, ranging from multiple individual contacts with the visiting NGF team as well as broad commitments for collaborations with the JMS. Dr. Kazuyuki Shimizu, past President of the Japanese Society of Myeloma and Chairman for the IMW Congress held in Kyoto in 2013 noted that: "Now I believe most Japanese doctors can understand the importance of NGF... I believe that the landscape of the [use of] flow testing in Japan will be changed." So stay tuned for further developments!


I continue to receive thoughtful comments on my recent blogabout crowdfunding. This new approach to fundraising deserves careful consideration with an equally thoughtful response to those who have taken the time to write.

Finding a cure for myeloma requires a coordinated effort. It is like solving a complicated puzzle. Although each individual effort helps and every dollar counts, I believe it is better to work together to reduce the possibility of wasted time and effort. What has been an issue up to now is that many investigators have focused on the same part of the puzzle leaving many other parts incomplete. Looking at the whole picture provides the perspective to assign efforts appropriately and utilize the best expertise of individual investigators.

Collaboration makes a huge difference in finding the answer faster. The IMF, the International Myeloma Working Group (IMWG) and the Black Swan Research Initiative® provide a framework within which all interested parties can contribute, suggest new parts of the puzzle to solve or to contribute to ongoing active areas of research.

It has been amazing to learn from open discussions that many parts of the puzzle are completed and to avoid duplication of effort it is important to focus efforts elsewhere.

Collaboration, communication and a team effort bring knowledge and energy to this effort to achieve a cure, which will undoubtedly succeed.

Dr. Durie sincerely appreciates and reads all comments left here. However, he cannot answer specific medical questions and encourages readers to contact the trained IMF InfoLine staff instead. Specific medical questions posted here will be forwarded to the IMF InfoLine. Questions sent to the InfoLine are answered with input from Dr. Durie and/or other scientific advisors and IMWG members as appropriate, but will not be posted here. To contact the IMF InfoLine, call 800-452-CURE, toll-free in the US and Canada, or send an email to infoline@myeloma.org. InfoLine hours are 9 am to 4 pm PT. Thank you.

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This week's news of interim results from the ENDEAVOR trial, a head-to-head comparison of "real world" schedules of Kyprolis versus Velcade, reveals an impressive benefit of carfilzomib over bortezomib in relapsed myeloma. Indeed, the 929-patient phase III trial showed a progression-free survival (PFS) of 18.7 months for Kyprolis versus 9.4 months for Velcade--basically a doubling of PFS or remission duration.

In the ENDEAVOR trial, Kyprolis was given in the usual schedule of two IV infusions per week for three out of four weeks, with dose escalation to 56 mg/m2. Velcade was also given according to the standard schedule, with subcutaneous (versus IV) administration allowed at the discretion of the investigator. (Sub-Q administration occurred more than 75 percent of the time.)

The results in this large trial follow on the heels of the impressive results in the 792-patient ASPIRE trial, which showed the superiority of Kyprolis plus Revlimid/Dexamethasone (Rd) over Rd alone. A head-to-head comparison of Kyprolis versus Velcade in the frontline setting is ongoing in a trial by the Eastern Cooperative Oncology Group (ECOG). Impressive results with the Kyprolis/Rd combination in the high-risk smoldering myeloma (HR - SMM) setting were presented at the 2014 Annual Meeting of the American Society of Hematology (ASH).

A strong case is emerging to place Kyprolis within the treatment paradigm for myeloma. One must applaud Onyx/Amgen for the direct comparison trials, which allow assessment of both efficacy and toxicity (concerns over which hampered panobinostat's initial approval in 2014). In the case of Kyprolis, although there are some increased toxicities versus Velcade--including cardiac and renal issues as well as dyspnea and hypertension--these certainly do not preclude use of the drug with normal cautions. Although oral formulations of both Kyprolis and Velcade are in development, it seems their primary role will be in the ongoing therapy or maintenance settings.

Kyprolis is already under review by the FDA in the one-to-three relapse setting based upon the ASPIRE trial results. Future reviews will likely consider the other settings in which Kyprolis can add value--from HR-SMM, frontline, and early relapse to the already approved relapsed/refractory situation. The increases in both depth and length of remissions are definitely important. The twice-weekly IV schedule is a challenge for patients and there is much interest in the ongoing once-weekly schedule study results.

Full data from the ENDEAVOR trial will be submitted for presentation at the 2015 American Society of Clinical Oncology (ASCO) Annual Meeting. In the meantime, this important emerging option is great news for patients. As always, it takes time to determine the best new treatment paradigm--but head-to-head comparison data help considerably. Stay tuned for what will be many updates to come.

Dr. Durie sincerely appreciates and reads all comments left here. However, he cannot answer specific medical questions and encourages readers to contact the trained IMF InfoLine staff instead. Specific medical questions posted here will be forwarded to the IMF InfoLine. Questions sent to the InfoLine are answered with input from Dr. Durie and/or other scientific advisors and IMWG members as appropriate, but will not be posted here. To contact the IMF InfoLine, call 800-452-CURE, toll-free in the US and Canada, or send an email to infoline@myeloma.org. InfoLine hours are 9 am to 4 pm PT. Thank you.

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Please define "relapse" in the context of this trial. It seems to be a difficult to define concept for patients to understand. Is there a number at which the M-spike is a critical mass? Or a ratio of kappa/lambda light chains that must be reached? I am seven years along, on Velcade/dex, stable for the most part, but the numbers keep creeping up, slowly and surely. My only distinct symptom is anemia. Do I wait for bone involvement or kidney issues? What is a relapse?

Thank you,



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In a surprise move this week, the US Food and Drug Administration (FDA) approved Farydak®, despite the 5-2 vote by the FDA's Oncologic Drugs Advisory Committee (ODAC) recommending against approval in November 2014. The approval is obviously good news for the myeloma community. There is an unmet need for new active agents.

It appears that the FDA circumvented the prior controversy regarding less than impressive efficacy and some serious toxicities by focusing on a pre-specified subset analysis of patients treated previously with bortezomib and an iMiD (thalidomide and/or Revlimid ®). For these 193 patients, the median progression-free survival (PFS) was 10.6 months for the panobinostat combination versus 5.8 months with placebo: a 4.8 month advantage. This trumped the prior numbers which ranged from 1.7 to 3.9 months of PFS benefit. The overall response rate was also higher: 59% versus 41%.

The FDA handled the serious toxicity aspect using a "Boxed Warning" approach with a specified Risk Evaluation and Mitigation (REMS) plan for severe diarrhea and potential severe and fatal cardiac events, arrhythmias and ECG changes. The side effect profile is thus acknowledged with warnings about how to best prevent or alleviate these problems.

The willingness of the FDA to consider Farydak® in this fashion is very positive, I think, as it makes agents available that confer some benefit in the randomized trial setting as long as use is accompanied by careful risk assessment. This is preferable to either disapproval or a much looser "compassionate use" protocol prior to adequate efficacy/risk evaluation, such as what is proposed in the so-called "Right to Try" legislation that has passed in some states. So, kudos to the FDA for choosing this middle ground and trusting the treating physician to be aware of the efficacy profile and the "Boxed Warnings." It is certainly hoped that judicious dose reductions of panobinostat as needed will be accompanied by retained efficacy and a reduced side effect profile.

For relapsed patients, each new response is a blessing; each new response is a bridge to further new options. Let's be optimistic that several new agents in the pipeline will likewise receive approval and be available in the most appropriate relapse niche. 

Dr. Durie sincerely appreciates and reads all comments left here. However, he cannot answer specific medical questions and encourages readers to contact the trained IMF InfoLine staff instead. Specific medical questions posted here will be forwarded to the IMF InfoLine. Questions sent to the InfoLine are answered with input from Dr. Durie and/or other scientific advisors and IMWG members as appropriate, but will not be posted here. To contact the IMF InfoLine, call 800-452-CURE, toll-free in the US and Canada, or send an email to infoline@myeloma.org. InfoLine hours are 9 am to 4 pm PT. Thank you.

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In a highly anticipated decision announced this week, the US Food and Drug Administration (FDA) approved the use of Revlimid® in the frontline setting based upon the results of the FIRST trial (which I have discussed in the IMF's presentation on news from the 2014 American Society of Hematology meeting here). Ongoing Revlimid/dexamethasone was clearly superior to a previous standard of care, Melphalan/Prednisone/Thalidomide (MPT), as well as shorter-duration use of Revlimid/Dexamethasone (Rd).

This approval consolidates what has been widespread off-label use of Revlimid in the frontline setting in the US for several years. Practitioners and patients in the US are very fortunate that the off-label mechanism has allowed flexible use of Revlimid, including its use in the popular Velcade®/Revlimid/dexamethasone combination for induction therapy.

The FDA has also added much more detailed guidance than in the past, recommending stem-cell harvesting after the first four cycles of therapy, if possible, and much closer blood-count monitoring. There is also a particular caution about the potential risk of second primary malignancies (SPMs) in the post-transplant Revlimid maintenance setting. With these caveats it is important to note that ongoing use of Revlimid (so-called continuous therapy) is supported by this decision.

The likely approval of frontline Revlimid in Europe* in the coming weeks is much more significant for European patients and physicians. Right now, frontline use is not possible. The pending approval will allow use specifically of Revlimid plus low-dose (weekly) dexamethasone exactly as used in the FIRST trial. This is a key point--the triple combination of VRd will not be immediately approved. Use is restricted to the FIRST trial protocol schedule only. This restriction is becoming an increasingly troubling problem because of the difficulty of completing multiple trials to encompass all the potential treatment combinations. Regulators use the lack of data to restrict the expense of broader use. This is an artificial constraint in that the efficacy and safety of many other combinations is widely known and well documented in the medical literature.

Implications of drug approvals in 2015 and beyond

What is frequently not so clear is the exact comparative efficacy and safety of different combination regimens. For example, how does VRD (Velcade/Rev/DEX ) compare to VCD (the CyBorD regimen) as a primary induction treatment?  Without a direct head-to-head comparison, one can say that the percentage and depth of responses and overall outcomes are rather similar. Both three-drug regimens are also safe, well tolerated, and more active than the two-drug combinations of Rev/dex (just approved) or Velcade/dex, also previously approved. Regulators, however, can point to the substantial cost savings of using VCD versus VRD, as well as the benefit of saving Revlimid for use in an approved relapse setting after VCD.  How best to combine and sequence drugs is a legitimate question without an immediate, clear answer, for sure!

In the US,  selecting among available therapy options falls into the realm of the treating physician working within NCCN guidelines and currently allowed off-label use, neither of which requires validation based on clinical trials of direct comparators. In Europe and elsewhere around the globe, the American scenario  cannot occur. Regulators require trial comparisons.

Thinking about the number (and potential cost) of trials needed to resolve all these questions is mind-numbing! In the interim, cost and regulatory control become the final arbiters limiting new drug use to the exact trial schedule(s) - IF the cost can be justified using trial endpoints and quality-of-life indicators.

Layered on top of comparative drug efficacy are safety concerns in particular circumstances. In the FIRST trial, ongoing Revlimid use as a frontline approach is both safe and very efficacious. But does this mean that similar ongoing use of Revlimid as post-transplant maintenance can be viewed equally? Lingering concerns remain about how best to use Revlimid as post-transplant maintenance. Is Revlimid maintenance recommended for many patients, but not all? Should maintenance be indefinite or for a defined period to maximize benefit? Revlimid used along with oral melphalan or as consolidation and maintenance immediately after high-dose intravenous melphalan, as in the IFM trial, may carry an extra risk of SPMs, including otherwise unexpected lymphoid malignancies such as ALL. It is thus difficult to give blanket approval for the use of any drug in all circumstances--no matter how efficacious and safe overall. 

Should treatment decisions fall within the realm of the primary physician, as they do here in the US? Or should options be left to the discretion of regulators, as is the case elsewhere in the world?

There is much to celebrate as Revlimid is fully available in the frontline setting, but many issues that will limit ideal global access and use remain to be resolved.

*February 20, 2015 UPDATE:

The anticipated frontline approval of Revlimid by the European Commission has occurred today (press release here), just two days after the FDA ruling in the US. This rare rapid approval by both agencies reflects the strength of the submitted packages indicating the value and safety of ongoing Revlimid/low-dose dexamethasone in the non-transplant population. The ability to achieve long-term disease control is an important step forward, especially for unfit or frail patients for whom transplant is never an option. It is hoped that broad access to Revlimid throughout Europe will follow. -- Brian G.M. Durie, MD

Dr. Durie sincerely appreciates and reads all comments left here. However, he cannot answer specific medical questions and encourages readers to contact the trained IMF InfoLine staff instead. Specific medical questions posted here will be forwarded to the IMF InfoLine. Questions sent to the InfoLine are answered with input from Dr. Durie and/or other scientific advisors and IMWG members as appropriate, but will not be posted here. To contact the IMF InfoLine, call 800-452-CURE, toll-free in the US and Canada, or send an email to infoline@myeloma.org. InfoLine hours are 9 am to 4 pm PT. Thank you.

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What are the after effects of Revlimid? Would the kidneys or liver be affected?

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