Dr. Raphael Pollock welcomes the next generation of sarcoma specialists

Dr. Raphael Pollock

Sarcoma Alliance volunteer Kalyse Engebretson interviewed Raphael Pollock, MD, PhD, a sarcoma surgeon and director of the Ohio State University Comprehensive Cancer Center in Columbus. 

What does a typical day look like for you? 

Many mornings I have a 6 a.m. or a 6:30 a.m. administrative meeting, followed by scheduled meetings, conference calls, one-on-one appointments and finishing typically with a work-related dinner probably three out of five nights because my role right now is I’m the director of the Ohio State University Comprehensive Cancer Center. I have a budget of $120 million, more than 300 faculty and 4,000 employees, and approximately 2 million square feet of research and clinical space that I am responsible to administer. I do it with a very large group of other people. They are extremely competent in what they do as an administrative team. But the bulk of my energies now are focusing on that responsibility. However, I still have time for a very small clinical practice and laboratory research practice -- both of which focus on sarcoma as they have for the past three decades.

How many patients do you have in general?

I have a very large practice but I no longer do this myself because of my other demands. We have asked two other surgeons to become my partners, Dr. Valerie Grignol and Dr. Joal Beane, both surgical oncology fellowship-trained practitioners, both of whom have research interests in the disease as well. So we have an integrated practice. Overall the practice is probably caring for upwards of several thousand sarcoma patients. ... The last time we looked about a year ago, we’re seeing about 400 sarcoma patients a year between the Wexner patient center and the Nationwide Children’s center.

Do your patients come from all over the U.S. then?

All over the U.S. and worldwide. It’s very gratifying to be able to reach so many people from so many places.

What do you find most interesting and/or enjoyable about your job? 

Probably the most enjoyable aspect, given that I’m going to be 69 years old next week, is being able to pass the baton onto the next generation and really help them get firmly grounded in sarcoma medicine, broadly defined, helping to build multidisciplinary teams, clinics and research platforms. Being able to see my personal ambitions for sarcoma programs going forward with the active recruitment and involvement of younger individuals. So the sense that the baton is being passed and feeling very positive about that happening and trying to facilitate it. That’s probably the very best part.

Earlier, when I was involved directly and with a much higher commitment to patient care, it was an opportunity to help people improve their lives through what we could deliver in our understanding and also it’s a very pleasurable part of what we did and still do. Our research programs are based on patient tissue acquisition. Describing what we’re trying to accomplish, seeing patients come to a quick understanding of that, and being so eager to participate and help us reach those goals, is also just extremely gratifying. One of the things that we routinely do, if the patient is interested, is to bring them to our research lab so they can meet the team that’s going to be working with the tissues that they’re donating. [This gives them a] better understanding of the questions that we’re trying to answer and is reinforcing for the people in the lab as well, as they get a chance to meet the people who are donating the tissue they’re going to be working with.

I remember a patient from Mexico who donated tissue and we were able to ultimately establish a sarcoma cell line from his tissues. He came and visited us in the lab after he had recovered from surgery, after the cells had started to propagate, and we were able to sit him down in front of a microscope and he could see his own cells moving around in culture. ... To him it was just fascinating, and to us it was the height of gratifying to see someone be able to connect those dots in such a dramatic way. Very moving.

One other thing that I would mention that I find really gratifying, that people may not know about me, I have chronic lymphocytic leukemia. [I have] gone through a clinical trial and will be on a targeted therapeutic for the rest of my life. My situation is such as I will probably never go into remission but I’m living in balance with the disease. So I get a lot of personal gratification from being able to talk from a personal perspective with my patients about issues such as clinical trials, what it feels like to have cancer fatigue -- which I experience on a daily basis -- confronting your own mortality, those are also very gratifying in their own ways.

And another very gratifying part of what I do is, I have 5 children, one of whom is a second-year medical student here at Ohio State and one is an undergraduate at Ohio State and is applying for medical school here as well. Two daughters, they both are very, very close with each other. It’s a wonderful experience, just as it’s wonderful being able to talk to patients directly about cancer fatigue in clinical trials, it’s also wonderful to have children who are interested in careers in academic medicine and talking with them about how that can best be accomplished. Helping them write research reports and things of that sort also has tremendous validation and meaning to me. ... Again, it’s the next generation coming up. I think we are all going to be very well-served by such talented youngsters as they mature into academic physicians.

What might most people be surprised to learn about your job? 

Probably the amount of time that is spent in necessary, but not particularly useful, meetings. There’s a lot of it. These are not things that you can avoid but they are not particularly interesting or gratifying compared to some of the other possibilities we just discussed. ...

This morning I got up at 4:45 to go to an early morning meeting. It was 12 degrees outside when I left my home.

Wow! What time do you go to bed? 

Usually I’m something of an insomniac, maybe that’s something that a lot of people might not be aware of. But I’ve been living on about 4 hours of sleep ever since the middle of high school.

How do you survive?!

That’s a good question.

What’s the most challenging part of your job?

I would say that there are several things: Helping patients who are beyond the possibility of cure, but providing help and insight in a very compassionate and humane manner is an awesome responsibility and it’s implicitly challenging to be able to be sensitive to the needs of other people in that particular life-defining context. It’s not something that can, or should, ever be done in a casual manner. It’s implicitly challenging.

A different type of challenge in an administrative context is, frequently, because of my leadership position, I’m put in the awkward position of two people wanting the same thing and only one can have it -- sort of a zero-sum proposition [in which I am] trying to see how we can negotiate between these two people to come up with a solution that is workable for them.

There are things that people feel very passionate about -- the extent of their surgical privileges vis-à-vis another practitioner, for example, access to operating rooms, resources that are finite to be used to expand a research program where several people are competing for the same dollars. Those can be very vexing, particularly when you have a situation where the two people are people you have worked with for a long time, you consider them friends, and you know for example that they are actually friends with each other. And yet, here they are, coming before you in a somewhat adversarial, zero-sum context and how do you adjudicate that? How do you make this whole and have everyone come forth satisfied is a real challenge, and one that we confront on a constant basis.

What keeps you inspired/motivated?

Seeing this next generation of younger people, developing, rising up, eager to try to move the edge of the envelope forward is one thing that is very inspiring. That, coupled with the tremendous bravery that our patients demonstrate, quietly, on a day-to-day basis. The things they have to cope with, the difficulties, and yet the strong will to not only survive but to surmount these challenges is truly inspirational and keeps my engines fully revved to try to be of help in that.

Is there anything else that you want to add or that you want people to know?

None of this will be possible without the strong connection with the community at large and their philanthropic generosity. Grants do not cover many areas that are critical in career development and research opportunities. The work of your group and the people in the community who are supporting the efforts of the Alliance is really critical to achieving our overall success. We’re just very, very grateful not only to your group, but also to the people supporting your group.

At the end of the day, it’s people helping people and you don’t get any better than that.

His sarcoma patients inspire Dr. William Tseng

Dr. William Tseng

Sarcoma Alliance volunteer Kalyse Engebretson interviewed William Tseng, MD, a surgical oncologist at Keck Medicine at the University of Southern California in Los Angeles. 

What do you find most interesting and/or enjoyable about your job?

The most interesting aspect of being a sarcoma surgeon is the fact that each case is different and requires a lot of thinking and planning to do what is best for the individual patient. The treatment usually involves surgery (that’s what I do!) but no two operations are the same and sometimes it may even be best to do other therapies first. There is nothing routine or straightforward about my job!

What might most people be surprised to learn about your job?

Sarcoma is all that I do. Although it is very rare, to me, it isn’t just a unique case I had a few months ago or even just a part of my practice along with one or two other diseases – it is the entirety of my job. I think that it is an important obligation to the sarcoma patient to be a true specialist and not just someone who “dabbles.”

What’s the most challenging part of your job?

Telling a patient that I can’t remove the sarcoma, that it is “unresectable.” I still think it is important to be honest and direct and I make every effort to help them understand why.

What keeps you inspired/motivated?

My patients and their strength and courage. I can’t take away their sarcoma diagnosis, but I strive to educate and inform them, relieve their anxiety, and give them hope -- along with treating their disease. What motivates me is being able to take sarcoma patients from new diagnosis to successful treatment, being able to say several months later, based on a follow up CT or MRI: “There is no evidence of disease.”

For more information on Dr. Tseng: 
https://www.keckmedicine.org/meet-william-tseng-md-clinical-surgery/

Help us make Sarcoma Awareness Month official!

By Suzie Siegel

The campaign to ask Congress to name July as Sarcoma Awareness Month has started early this year, and we already have bipartisan support. Rep. Sean Duffy, R-WI-7, introduced House Resolution 188 on March 7 with Rep. Sheila Jackson Lee, D-TX-18, as cosponsor.

Please ask your House representative to join as a cosponsor. We have a real chance of passing this resolution if we all work together! You can find out who your representative is at this website. You may have to email, write a letter AND call. Remember, it's OK to nag someone for a good cause!

Here's what the resolution says:

Expressing support for designation of July as National Sarcoma Awareness Month.

Whereas sarcoma is a cancer of connective tissues, such as nerves, muscles, joints, fat, bones, and blood vessels, and it can arise anywhere in the body;

Whereas 15,000 people in the United States were diagnosed with sarcoma last year, more than 6,000 died, and 50,000 are struggling with the disease at any one time;

Whereas 1 percent of cancers diagnosed in adults and 20 percent of cancers diagnosed in children each year are sarcoma;

Whereas more than 50 subtypes of sarcoma have been identified;

Whereas the National Cancer Institute recognizes that sarcoma is often misdiagnosed and underreported; and

Whereas July would be an appropriate month to designate as National Sarcoma Awareness Month to raise awareness of the disease and encourage more individuals to get properly diagnosed and treated:

Now, therefore, be it Resolved,

That the House of Representatives supports the designation of National Sarcoma Awareness Month.

The failure of olaratumab

By Suzie Siegel

Sarcoma specialists and patients are struggling to make sense of the failure of olaratumab (Lartruvo) in a phase 3 clinical trial.

Dr. Breelyn Wilky

“Like all sarcoma trials, there are very likely patients who individually had benefit with olaratumab, that we can't account for or identify in a large trial where everyone is averaged together,” said Dr. Breelyn Wilky of the University of Colorado in Aurora. “The problem is that we don't understand enough about olaratumab, how it works, or what the targets are, to try to pick out those patients.

“What we learned is that at least for now, the collective benefit across all sarcoma patients is not enough to recommend … adding it to doxorubicin. If a patient is currently on olaratumab with or without another drug and is having benefit, at least the early statements from Lilly are that they will continue to allow that patient to continue with olaratumab. But new patients will not and should not be started on olaratumab.”

Doxorubicin, an old drug also known as adriamycin, is often the first chemotherapy offered to patients whose sarcomas have spread. It’s considered a “first-line” or "frontline" drug. A phase 2 trial compared doxorubicin by itself to dox combined with olaratumab. It found the combination helped sarcoma patients live longer.

The FDA gave “accelerated approval” to olaratumab, allowing it to be prescribed even to patients who were not enrolled in the phase 3 clinical trial. This was the first time in decades that the FDA had approved a new first-line drug for a wide range of sarcoma subtypes.

I think most sarcoma doctors expected the phase 3 clinical trial to confirm the results of the phase 2 trial. But Lilly, the maker of the drug, announced Jan. 18 that the phase 3 trial could not confirm that olaratumab helped patients live longer. Sarcoma specialists on Twitter expressed shock and dismay.

“Sarcoma docs all depressed today,” tweeted Dr. Melissa Burgess of the University of Pittsburgh.

Dr. Brian Van Tine

“I am a sarcoma doctor,” tweeted Dr. Brian Van Tine of Washington University in St. Louis. “I was one yesterday and will be one tomorrow. I will not let the setbacks of today prevent me from trying to change the outcomes for my patients. Clinical trials are the only way things will ever change.”

Dr. Seth Pollack of the University of Washington in Seattle asked: “Do you think this could scare off companies thinking of developing their drugs [in] sarcoma?”

Dr. Sam Blackman, a pediatric oncologist involved in drug development, replied: “Not any company I work for.”

Critics said the FDA shouldn’t have given accelerated approval to olaratumab. But Dr. George Demetri of Dana-Farber Cancer Institute in Boston praised the FDA for taking a gamble on a drug that seemed to have so much promise.

“In brief, the classic adage ‘hindsight is 20/20’ applies, and we are all sad for the patients and families who depend on us all to make things better,” he tweeted.

Dr. Winette van der Graaf

This isn’t the first sarcoma drug to fail in a phase 3 trial after looking good in an earlier one, noted Dr. Winette van der Graaf of the Royal Marsden Hospital in London and the Netherlands Cancer Institute in Amsterdam. This also happened to palifosfamide and evofosfamide.

“The biology behind the benefit of [olaratumab] in the randomised phase 2 [trial] was difficult to understand and we had already concerns,” she tweeted. The FDA can approve drugs that work even if no one knows exactly why. An example is trabectedin (Yondelis).

Discussing olaratumab, Dr. Demetri tweeted: “There was always a red flag about the phase 2 data. Why no big PFS benefit with putative huge OS benefit?”

Progression-free survival (PFS) refers to how long patients can live without their cancer growing. Overall survival (OS) is how long they live, whether or not they’re still dealing with disease.

The FDA has approved drugs, such as trabectedin, on the basis of PFS. The goal (“endpoint”) of the olaratumab trial was OS. Overall survival sounds like the best goal — we all want cancer drugs that will help us live longer! But the problem of using that as the goal in clinical trials is that there may be other reasons someone lived longer than another person. Some of the most obvious reasons include being insured and in good health otherwise.

Dr. Cesar Serrano Garcia

“OS is a challenging endpoint,” tweeted Dr. Cesar Serrano Garcia of Vall d’Hebron Barcelona Hospital. The impact of the micro-environment of the soft-tissue sarcoma and immunology “are not yet completely understood and might be fooled by the intrinsic complexity of STS and its management.”

Dr. Patrick Schoffski of University Hospitals Leuven in Belgium noted that overall survival for people treated with doxorubicin continues to climb as patients get better care from multidisciplinary sarcoma doctors. This makes it harder for new drugs to compete against it.

The olaratumab trial had two “arms”: Patients in one arm got doxorubicin + olaratumab. Patients in the other arm got doxorubicin by itself. Doctors didn’t decide which patients got which. Patients were assigned randomly. This randomization is supposed to make both arms equal, instead of one arm full of people who have a better chance of survival. But sarcoma clinical trials can’t enroll as many patients as the more common cancers, and thus, we have fewer unique individuals to randomize.

Because sarcoma is rare, clinical trials often lump together people with different subtypes, instead of splitting them up into separate trials.

“We need more splitting rather than lumping in clinical trials. [It’s] too difficult to predict and statistically account for behavior of 50+ sarcoma subtypes,” said Dr. Victor Villalobos of the University of Colorado Cancer Center.

Dr. Anthony Conley of MD Anderson Cancer Center in Houston tweeted: “A review of all prior frontline trials in metastatic soft tissue sarcoma should occur to determine causes of these problems. I agree that splitting, rather than lumping, may be necessary moving forward and we should critically appraise endpoints and response tools.”

Dr. Tom Wei-Wu Chen

The olaratumab trial did this, but it also kept track of data on leiomyosarcoma, which was expected to respond better than other subtypes. But Dr. Tom Wei-Wu Chen of the National Taiwan University Hospital and Cancer Center noted that even leiomyosarcoma can be broken into subtypes.

Dr. Demetri suggested we just need better drugs. “After all, olaratumab did not fail simply because of sarcoma disease heterogeneity — it failed even in the single subtype of leiomyosarcoma. Complete fail.”

A challenge will be asking drug companies and the National Cancer Institute to fund clinical trials with smaller subsets of sarcoma patients, Dr. Wilky tweeted. She said we need more biomarkers and more evidence of how a drug works before a clinical trial begins.

Dr. Bill Tap of Memorial Sloan-Kettering in New York was the principal investigator of the olaratumab trial. He and Lilly representatives are expected to discuss the data at the annual meeting of the American Society of Clinical Oncology in June.

Ironically, this will probably be the most important sarcoma data presented at the meeting, Dr. Wilky tweeted.

“Do you think it will amount to a ‘shoulder shrug’ or will we … really learn something that moves the field forward, maybe even helps future studies?” asked Dr. Pollack.

“I hope [there] was enough data captured during the course of this trial for a proper ‘post-mortem’,” tweeted Dr. Herbert Loong of the Chinese University in Hong Kong. “We owe this to all [patients] who have been on trial and countless more who are on it commercially. It's important to establish whether or not it's a failure in trial design vs. drug itself.”

Despite this setback, a number of experimental drugs remain in the pipeline. Doctors are also using drugs developed for other cancers to target sarcomas with similar gene mutations and fusions.

Physics and math bring more precision to treatment

By Suzie Siegel

Dr. Jeremy Mason

My mentor at a cancer conference has taken only one biology class in his life, and that was when he was a freshman in high school. Nevertheless, his knowledge can change the way you think of cancer — and the way doctors treat it.

“Math, physics and computer science have a profound impact on the field of cancer research today,” said Jeremy Mason, who has his doctorate in engineering. “Other scholars with little to no experience in cancer are applying their own knowledge and skills to build detailed and relevant models of the event prediction, cellular birth/death processes, therapeutic responses and much more.

“One day, these models will be used for the common goal of improving patient care and extending lives.”

Dr. Mason, assistant professor of oncology at the University of Southern California in Los Angeles, came from the heart of Cajun country and went to college in Georgia.

“I grew up in a region where diabetes, stroke, and heart attack were discussed more than cancer, so I started down this path with virtually no prior knowledge on the subject. Instead of having to correct any misconceptions I had, I was forced to learn everything from the ground up. Fortunately, since I was classically trained as an engineer, applying math and physics to cancer research allowed me to view the challenges that were arising within the field and helped to quantify the complexity in a manner that made sense to me.

“Cancer research has interested me in a number of ways. It presented a unique challenge that does not have a definitive solution, but has profound impact on numerous people. Both patients and their loved ones. Also, every day I get to work with some truly amazing people.”

Dr. Caligiuri with me

Dr. Mason works on the Convergent Science Initiative in Cancer with USC Professor Peter Kuhn, who has his doctorate in physics.  This is the second year that I won a scholarship to attend the American Association for Cancer Research’s annual meeting as part of its Scientist↔Survivor Program. Drs. Mason and Kuhn were the scientific mentors for the team I was on. We had to do a short presentation on math, physics and evolution in cancer research.

The meeting in April drew 22,500 participants to Chicago. The survivor program gives participants a chance to meet VIPs, such as Michael Caligiuri, MD, then president of AACR, in social settings. Dr. Caligiuri is president of the City of Hope National Medical Center in Duarte, Calif.

Dr. Shihong Zhang

Most doctors and scientists work on carcinoma. I always ask about their research: “Is this the same or different for sarcoma? How could this apply to sarcoma?”

Because sarcoma is rare, I could read the summaries of all the sarcoma research presented. For example, I met Shihong Zhang, PhD, who works on immunology at Fred Hutchinson Cancer Research Center in Seattle. She presented a poster on the use of interferon gamma radiation to heat up the cold microenvironment of synovial sarcoma and myxoid-round cell liposarcoma, both of which express the protein NY-ESO-1. The goal is to make them more susceptible to immunotherapy.

Dr. Jianguo Huang 

Jianguo Huang, PhD, of Duke University Medical Center in Durham, N.C., presented a poster on how long, noncoding RNA NEAT1 promotes lung metastasis in soft-tissue sarcoma.

“Metastasis is the major cause of death from cancers, including sarcomas,” said radiation oncologist David Kirsch, MD, PhD, principal investigator on the NEAT1 study. “We seek to understand how sarcomas spread from their primary site to other parts of the body, such as the lungs. By uncovering the way that sarcomas spread, we hope to develop new approaches for preventing or treating sarcoma metastasis.”

Dr. Kirsch, a Duke professor, thinks it would be a stretch to include this presentation in my topic. Dr. Kuhn did not.

Dr. Peter Kuhn

“Physicists are often trained as high-complexity problem solvers, a k a fancy plumbers with duct tape,” Dr. Kuhn said. “It is not necessarily just about light and energy but really just as much about the body as a complex system (space) that experiences many factors of evolution and lifestyle (time). Understanding this space-time complexity and starting to determine what we need to measure to predict it, is exactly what the physics and math guys are going after.”

“The same mechanisms that allow organisms to adapt and survive are the same mechanisms that cause cancer,” said pathologist Carolyn Compton, MD, PhD, a professor at Arizona State University in Scottsdale and chief medical officer of its Complex Adaptive Systems Institute.

Dr. Carolyn Compton makes a point

Cell mutations drive evolution, she said. They allow some animals to adapt and survive in their environment. But mutations also help cancer cells grow out of control in different environments in our bodies. Thus, humanity takes the bad (cancer) with the good (evolution).

Many people have cancer cells in their bodies, but they don’t know it because the cells haven’t found a hospitable environment in which to grow. It takes about a billion cancer cells to make a tumor that is 1 centimeter cubed, Dr. Compton said. The cells in a cancerous tumor may not be all the same, she said, and some may survive treatment.

“Cancer is the ultimate complex, adaptive system. It does new things that you did not expect. If we think of weather, there’s all this math modeling to make predictions, but we’re not there yet” in cancer research.

Sometimes treatment isn’t strong enough to kill cancer. Sometimes doses are stronger than they need to be, causing unnecessary damage to healthy cells, she said. Sometimes cancer treatment can cause so much damage that people develop new cancers.

Dr. Kuhn is working to develop liquid biopsies — blood samples that could make treatment more precise and predict a patient’s future. These samples would be more accurate in screening for cancers. They could predict which treatment would work for a patient, monitor the cancer for resistance to treatment, and predict what treatment might work if the cancer returns.

For many sarcoma patients, treatment can feel like a gamble. I'm glad that math and physics can give us better odds.

We loved the Sarcoma Exchange!

By Suzie Siegel

The Sarcoma Exchange proved so successful that even before it ended, survivors were asking about the next one. 

“We were so encouraged by the outpouring of participation by the sarcoma patient, caregiver and survivor community. We had 147 attendees, but many more expressed that they wanted to attend,” said Alyssa O’Driscoll, executive director of the Sarcoma Alliance. “We’re planning to make this a regular part of our programming should funding continue.” 

The Alliance, founded in 1999, is an international nonprofit dedicated to the education, guidance and support of people affected by sarcoma. Although survivors and families meet each other at Alliance events, this the first time it has held a patient-education conference. 

The conference was July 20-22 in Atlanta. Patients and caregivers came from 29 states and represented 21 subtypes. The patient who had survived the longest was Susie Wright of Cairo, GA, who was diagnosed with synovial sarcoma 48 years ago. The subtype with the most survivors in attendance was leiomyosarcoma (LMS).  

LMS survivor Sharee Whitmer (left) and Lisa Kessler, whose husband survived LMS, but their son did not.    

"The information gleaned from the sarcoma oncologists and researchers empowered me in a way that really let me feel I have some options that I have not exhausted," said Sharee Whitmer of Madisonville, VA, an LMS survivor. She came with her husband and their therapy dog, Stella. "It reassured us that the sarcoma oncologist and team that I am with now are staying well-informed and that I am indeed in good hands. And when and if the time comes to move on and find another team, I have many other choices.

"For us to have some idea of how up-do-date our care and our options, including trials, really are, it is imperative to attend conferences aimed at educating and empowering patients. Not everyone is seen at a large sarcoma center.

"The BIG take away from the conference for us was to really be able to connect in person with other sarcoma patients and their caregivers that we have met through online support groups. And during the conference to be able to meet so many more that we would not have met if it were not for attending the conference. Knowledge is incredibly valuable for patients like us, and having the opportunity to exchange our experiences while enduring this diagnosis and the treatments that follow are invaluable."  

Mary Prince, Lea Custer and me

Mary Prince of Portland, OR, came from the farthest away. Her sister, another LMS survivor, lives in Murrayville, GA. 

“Attending this wonderful event with my longterm-thriver/ BFF / sister, Lea Custer, was the highlight of the year for me. Meeting other LMS survivors and meeting all the wonderful medical professionals and hearing what's coming up on the horizon made it an incredible conference. Love was everywhere; you could feel it!" Mary said. "A special shout out to Dr. Gina D'Amato, Lea's primary sarcoma physician, who made it all happen.”  

Dr. D’Amato, a Sarcoma Alliance board member, practices at Northside Hospital’s Cancer Institute in Atlanta. She’s grateful that sarcoma specialists from across the South volunteered to speak, along with her colleagues from Northside.

The medical oncologists who spoke were Dr. Robert Benjamin and Dr. Shreyaskumar Patel from MD Anderson Cancer Center in Houston, Dr. Jonathan Trent from the Sylvester Cancer Center in Miami, and Dr. Richard Riedel from Duke Cancer Institute in Durham, N.C. Dr. Scott Davidson, a surgical oncologist, and Dr. Hamilton Williams, a radiation oncologist, attended from Northside.

Breakout sessions covered patients in treatment, survivors and caregivers.

Amanda Smith with the late Roger Henderson

"Never quit the fight. That was my mindset," said Amanda Smith of Havelock, NC, who facilitated the caregiver section. She lost her boyfriend, Roger Henderson, to chondrosarcoma. An LPN, she's in school to become an RN. 

Sarcoma Alliance board member Marites Tullius, a nurse practitioner, spoke on palliative care. From Northside, registered dietitian Rebecca Perez spoke on nutrition; social worker Myra Bazell, stress management; RN Debbie Bickes, patient navigation; and occupational therapist Melora Rennie, exercise and fatigue.

Osteosarcoma survivor Woody Roseland, whose "S#!%Cancer Patients Say” brought knowing laughs, entertained us one afternoon. That night, hypnotist Ricky Kalmon evoked a great deal of silliness from his targets.  

Sponsors were drug companies Lilly, Eisai, Ignyta, Novartis, Immune Design and Epizyme as well as  Northside Hospital’s Sarcoma Program. Thanks to them, we had free rooms at the Marriott Perimeter Center Hotel and delicious meals. 

  

Presentations from the conference are posted here: https://sarcomaalliance.org/event/sarcoma-exchange-2018/ Here are a few tidbits:

Dr. D'Amato with Dr. Benjamin

"Stage 4 has a bad connotation. I don't even use it in clinic,” Dr. D’Amato said. The problem with "stage 4" or "terminal" is that these labels include people whose sarcoma has spread far and wide as well as those with oligometastatic disease, which means they have only a few small tumors in one or two distant locations. Some people think "stage 4" or "terminal" means they are going to die soon. They may decline treatment that could help them live longer.

Dr. D’Amato noted that "remission" applies to people who no longer have any evidence of sarcoma after their initial treatment. The term for people who have no evidence of disease after they have been treated for metastatic sarcoma is NED. For example, my original leiomyosarcoma was 2C. Soon after, one small lung metastasis was found; 18 months later another appeared. Thanks to radiation, chemo and surgery, I’m NED from oligometastatic disease.

Dr. Riedel

“When you’re educated, you’re empowered,” said Dr. Riedel, who spoke on immunotherapy. “The future is bright.” 

Don’t let community doctors take you off of a drug just because your tumors aren’t shrinking, he said. His mantra is: “Stable disease is a good thing.”

“Patients want their sarcomas to shrink and disappear, but doctors are happy with stability,” Dr. D’Amato stressed. Sarcoma specialists may try new treatments only if tumors start to grow. 

Some times the tumor stays the same, or even looks larger, but is actually dead after treatment. Some tumors shrink, but not enough to meet the definition of shrinkage in clinical trials. 

Researchers refer to “progression-free survival” (PFS) when tumors stay stable. Some people don’t think the FDA should approve drugs based on stability. They want clinical trials to prove that the people who got the new drug lived longer, which is called “overall survival” (OS). 

Dr. Benjamin thinks PFS can be a good sign that a drug is working. The problem with OS is that it measures  things that weren’t part of the clinical trial, he said. For example, the clinical trial on doxorubicin (adriamycin) + olaratumab (Lartruvo) compared it with dox by itself. But people who got olara may have gone on to get better treatment, improving their survival.  That’s why MD Anderson is doing a clinical trial to compare dox + olara to the older combination of dox + ifosfamide.

Dr. Patel

Dox + ifos is a common treatment for sarcomas, and some community doctors may try to treat all sarcomas with it, Dr. Patel said. They shouldn’t, he added. They need to look at each subtype individually because some, such as alveolar soft part sarcoma (ASPS), don’t respond to this chemo combo. 

        

Because immunotherapy is in the news, he spends a lot of time educating patients on why chemo may be better than immunotherapy for their particular sarcomas. He also discusses the advantages of a clinical trial vs. standard treatment.

“Patients must have information to make a decision,” Dr. Patel said.

A variation on doxorubicin, called aldoxorubicin, “clearly has less cardiac toxicity. It’s a very good drug,” Dr. Benjamin said. We await the results of clinical trials and a decision by the FDA. 

Dr. Trent

Dr. Trent joked about the crazy names that pathologists give to sarcoma subtypes, such as dermatofibrosarcoma protuberans (DFSP). Nevertheless, medical oncologists like Dr. Trent greatly respect sarcoma pathologists. You can’t get the best treatment without an accurate diagnosis. 

For example, most sarcoma cells look like spindles under the microscope, Dr. Benjamin said. If pathologists cannot identify the tumor’s subtype, they may use the catch-all term of “spindle-cell sarcoma.” 

Instead of taking a tissue sample, some doctors are looking at tumors’ DNA circulating in the bloodstream (called ctDNA). They want to see which treatments are working and which might work for the patient in the future. Although these “liquid biopsies” are still experimental, Dr. Trent said: 

“This is what the future is.”

Let's make Sarcoma Awareness Month official!

Sarcoma nonprofits are seeking U.S. senators to introduce a resolution naming July as Sarcoma Awareness Month. We have been working on this since 2007 and have been successful in getting recognition from the Sarcoma Alliance for Research through Collaboration, National Cancer Institute, American Association for Cancer Research, the American Society for Clinical Oncology and the American Cancer Society.

U.S. Rep. Kathy Castor, D-FL, has introduced the resolution in the House in past years, to no avail. We’ve also tried White House petitions, but that didn’t work either. That’s why we’re focusing on the U.S. Senate this year.

We would be grateful if you would contact your U.S. senator! Here’s the suggested wording for the resolution:

A resolution declaring July to be Sarcoma Awareness Month

WHEREAS, Sarcoma is a cancer of connective tissues, such as nerves, muscles, joints, fat, bones and blood vessels, and it can arise anywhere in the body; and 

WHEREAS, the American Cancer Society estimates 13,040 cases of soft-tissue sarcoma will be diagnosed this year, with 5,150 Americans expected to die from it. 

WHEREAS, 3,450 new cases of bone sarcomas are estimated for this year, with 1,590 deaths.

     

WHEREAS, 1 percent of the cancers diagnosed in adults and 20 percent of cancers diagnosed in children each year are sarcoma; and  

     WHEREAS, More than 50 subtypes of sarcomas have been identified; and

WHEREAS, Nonprofit sarcoma organizations want to raise awareness in hopes that more patients will get earlier diagnosis and treatment, and these organizations have agreed upon July as Sarcoma Awareness Month; and

     WHEREAS, the National Cancer Institute, the American Society of Clinical Oncology, the American Association for Cancer Research, the American Cancer Society, the Sarcoma Alliance for Research through Collaboration, the National Comprehensive Cancer Network and many others recognize July as Sarcoma Awareness Month;

NOW, THEREFORE,

Be It Resolved by the U.S Senate: 

That the Senate recognizes July as Sarcoma Awareness Month. 

Big Data research includes sarcoma

By Suzie Siegel

Dr. Bill Dalton is betting on Big Data.

In 2006, when he was CEO of the Moffitt Cancer Center in Tampa, he led the development of the Total Cancer Care Protocol and M2Gen, a for-profit subsidiary.

As part of Total Cancer Care, more than 140,000 patients — including 3,392 with sarcoma — have given permission to have their blood and tissue samples stored and analyzed at Moffitt and to be followed throughout their lives. This produces huge amounts of data that researchers and M2Gen can analyze further.

“We have one of the oldest and largest databases of its kind, especially in molecular genomics,” said Dr. Dalton, MD, PhD, now executive chair of M2Gen, a health-informatics company. “We are continually updating patients’ data to try and learn from each patient’s experience.”

Researchers group patients' de-identified data by their similarities, and then break out the groups with more and more similarities. For example, they might start with patients whose cancers had NTRK gene mutations, and then create a subset that have Ras mutations, too. Because some patients may have the same genetic mutations, patients with different kinds of cancer might be able to take the same drug.

Sarcoma is rare, and doctors often struggle to enroll enough patients in the clinical trials needed to gain FDA approval of a new drug. We may benefit from trials that enroll patients with different cancers who have the same mutations. Targeting these mutations in a person’s cancer is called precision or personalized medicine.

In sarcoma, the first targeted treatment was imatinib (Gleevec) for gastrointestinal stromal tumor (GIST), which often shares the same mutation as chronic myeloid leukemia (CML). The FDA approved imatinib for CML in 2001 and GIST patients followed soon after.

In the past, GIST had often been classified as gastrointestinal leiomyosarcoma (GI LMS). Although my LMS was vaginal, I was checked for the mutation when I was diagnosed in 2002, just in case. My first sarcoma oncologist, Dr. Andy Burgess, now retired from MD Anderson Cancer Center in Houston, told me that imatinib for GIST was the best thing that had ever happened to him in his life.

One of Dr. Dalton’s sons was diagnosed with GIST last year and is doing well on imatinib. “I’m becoming a student again,” Dr. Dalton said, as he learns more about sarcoma. As a physician, he treated patients with multiple myeloma.

My tour to learn more about Total Cancer Care began at Moffitt, where I peered into the pathology lab near the operating rooms. If possible, a diagnosis is made while the patient is in surgery to help the surgeon know how to proceed. With sarcoma, for example, surgeons try to take some normal tissue around the tumor — just in case some microscopic cancer cells have spread that far. In ovarian cancer, surgeons often take much more.

Tissue donated for research is snap-frozen, usually with liquid nitrogen. When tissue is needed for patient care, hospitals store tissue in wax, technically called formalin-fixed paraffin-embedded (FFPE) tissue blocks, said Michelle Fournier, manager of Moffitt’s central biorepository (also known as a tissue bank).

“But that isn’t the best for genomic sequencing,” she said. Ten years ago, Moffitt couldn’t extract good quality genetic information from paraffin blocks, but it now has the technology, Dr. Dalton noted. This matters because Moffitt often receives FFPE samples from other hospitals when patients seek second opinions.

If a patient may benefit from genomic testing, most Moffitt doctors use FoundationOne, Fournier said, which is not always covered by insurance. She said Moffitt wants to create its own genomic-testing lab.

Samples of blood and other bodily fluids are also important parts of Total Cancer Care, she said. Like their colleagues elsewhere, Moffitt researchers hope to develop technology to allow the use of liquid biopsies. Drawing blood for a biopsy would be easier on patients than minor surgery to remove tissue.

Genesis Blanco, left, and Michelle Fournier, right

“I have a lot of patients who say they came to Moffitt because of the research,” said Genesis Blanco, a Total Cancer Care research coordinator.

Total Cancer Care draws from electronic medical records (EMR) created by clinic staff, as well as results of surveys from patients. This two-part system helps ensure accuracy, Dr. Dalton said. “I’d sometimes trust patient-reported outcomes for such things as patient’s pain over EMR.”

M2Gen, built on 30 acres near the main Moffitt campus, promotes research using the Total Cancer Care data. The data are de-identified so that researchers don’t know who the patients are. M2Gen partners with five pharmaceutical and biotech companies, which provide funding for further analysis of the data.

M2Gen also looks into the future. Researchers use statistical algorithms to predict how someone’s cancer might change if it spreads and what treatment options might be available.

In 2014, Moffitt and the Ohio State Comprehensive Cancer Center in Columbus, cofounded the nonprofit ORIEN (Oncology Research Information Exchange Network). M2Gen manages the network.  Seventeen cancer centers participate.

"We’ll be up in Canada next month, and then in Europe,” seeking more partners, Dr. Dalton said.

M2Gen is leading the Avatar Research project within ORIEN that does whole exome sequencing, which looks at the part of DNA that encodes protein, as well as RNA sequencing on tissues collected as part of TCC. Dr. Dalton would like to create a portal so that patients could learn how their donations of tissue and data are advancing research.

ORIEN is using resources on a focused group of Total Cancer Care tissues. “For Avatar we’re looking at a subset of high-risk patients who may be in need of clinical trials,” explained Erin Siegel, MPH, PhD, Scientific Director of TCC.

In this Avatar Research project, researchers in and outside of the ORIEN network can ask to use the data for specific projects. For example, Dr. Damon Reed is looking at teenagers and young adults up to age 40. Dr. Reed, MD, is director of the Adolescent and Young Adult Program at Moffitt and medical director of its Sarcoma Department. Working with Dr. Reed, Moffitt will include over 250 sarcoma tumors in the Avatar project.

"We need champions in this research, like Damon,” Dr. Dalton said.

Sarcoma Awareness Month needs your help

By Suzie Siegel

Once again, we need your help to get Congress or the White House to recognize July as Sarcoma Awareness Month.

Many of us had never heard of sarcoma before our diagnosis. Our first doctor may have seen few, if any, cases. We may have felt alone, with no idea about the resources available to patients and their families.

Increased awareness could help patients get a correct diagnosis and better treatment and support earlier.

July may seem far away, especially for those still digging out from winter, but we need to start contacting Congress and the White House now.

Here's how to contact the White House: https://www.whitehouse.gov/get-involved/write-or-call/. Here's how to find your Senator: https://www.senate.gov/general/contact_information/senators_cfm.cfm. If you don't know who your U.S. House Representative is, put your zip code into the box on the top righthand corner of this page: https://www.house.gov/representatives.

Please keep all communication positive. Remember that we need bipartisan support. If you can't talk directly with your Representative or Senator, you may want to call their offices to see if they have a staff member who handles health issues. After talking to someone, follow up with an email or letter. Check back with them periodically to see if a decision has been made. Visit them in person if possible.

Get your friends in on the fun. Put this on Facebook, Instagram, etc. Talk to your bridge club or your religious institution. Be creative.

Here are facts to mention:

• Sarcoma nonprofits decided at a meeting in 2007 to support July as Sarcoma Awareness Month. We don't know anyone opposed to this. 
• The National Cancer Institute, the American Cancer Society, the American Association for Cancer Research, the American Society of Clinical Oncology, the Sarcoma Alliance for Research through Collaboration and the Connective Tissue Oncology Society recognize July as Sarcoma Awareness Month. But no one in Congress or the White House has chosen to make this official ... yet!
• Sarcoma is one of the major types of cancer. Although it represents only 1% of adult cancer cases, it is 20% of childhood cancers. 
• Sarcomas occur in connective and supportive tissues, including bone, muscle, fat, nerves, etc., anywhere in the body. There may be more than 200 subtypes. People of all ages can get sarcoma, including babies in the womb. Lifestyle choices, such as smoking and drinking, have not been linked to sarcoma.
• The American Cancer Society estimates 13,040 cases of soft-tissue sarcoma will be diagnosed this year, with 5,150 Americans expected to die from it. For bone sarcomas, the figures are 3,450 new cases and 1,590 deaths.

The movement to designate a special time for sarcoma awareness started in 2001 with the late Suzanne Leider, founder of the Sarcoma Alliance. She proposed a Sarcoma Awareness Week in June. Its sister organization, the Sarcoma Foundation of America, adopted that, as did Sarcoma-UK and other nonprofits and sarcoma centers. Then Bruce Shriver, founder of the Liddy Shriver Sarcoma Initiative, decided to have an International Sarcoma Awareness Week in July.

In 2006, I wondered why we couldn't all agree on the same time period. Bruce didn't want to change his week in July. So, the Sarcoma Alliance and others switched to July for the sake of unity. Sarcoma nonprofits agreed on July at the Connective Tissue Oncology Society (CTOS) meeting in Seattle in 2007.

I created a Care2 petition on the topic. A legislative liaison at Moffitt Cancer Center in Tampa helped me draft the wording for a House resolution, and U.S. Rep. Kathy Castor, D-FL, sponsored it 2011.

The former Republican leadership of the House wanted no more resolutions recognizing days, weeks, months, etc. I talked with the leadership’s staff to no avail. The result was we couldn’t get Rep. Castor’s resolution out of committee.

I was a journalist before being diagnosed with leiomyosarcoma. In 2012, I wrote an editorial for my former newspaper, the Tampa Tribune. The next year, I wrote this. In 2015, I wrote another editorial and created a White House petition. The Sarcoma Foundation of America took over the petition in 2016 and 2017.

At the CTOS meeting this November, sarcoma advocates decided to consider collaborating on the effort to gain federal recognition of Sarcoma Awareness Month. The National Leiomyosarcoma Foundation has partnered with Rare Disease Legislative Advocates.

Of course, the Sarcoma Alliance and Sarcoma Foundation of America will work hard on this issue once again -- with your help! Please tell us who you've contacted in comments on this blog post or on the Sarcoma Alliance Facebook page: https://www.facebook.com/groups/sarcomaalliance/