What We've Learned
We formed the Liddy Shriver Sarcoma Initiative in November 2003, two months before our daughter Liddy passed away. In the eleven years since then, we have travelled with many hundreds of sarcoma families down the uncertain path leading from diagnosis through treatments and beyond. We have shared the joy of those who now experience no evidence of disease and the grief and sorrow of those who have lost a loved one to sarcoma.
We have learned much in our personal journey with sarcoma, in the sharing of the journeys of others, through the publishing of ESUN, and in the funding of sarcoma research. As this is the last issue of ESUN which we will publish (see "Preparing for a Transition"), we want to share with you some of what we've learned as well as some of our conjectures and suggestions.
• Being a physician or nurse specializing in oncology, particularly in rare cancers, is an incredibly demanding physical and emotional career. Many sarcomas can be very aggressive. Many are advanced or metastasized when they are diagnosed, making them very difficult to treat. We are constantly amazed, given the scores of patients sarcoma specialists see each month, that there is not more physician and nurse "burn out" under such stressful conditions. Just as each of us has our good days and bad days, so they too, being human, have good days and bad days. This is not said to justify ongoing "bad bedside manner" and insensitivity." It is an observation of human behavior that each of us has experienced.
• Some people can sense the emotions and feelings of others better than the average person can. They can see, hear, or feel anxiety, despair, or fear just as easily as they sense pride, admiration and joy. Those who have this gift have varying levels of ability to use it to help others and to help themselves. Sometimes just listening to what someone has to say without rendering judgment or advice can be the very best help and support you can give them.
• Facing the diagnosis of a rare cancer is often a daunting proposition. The lack of nearby oncologists who have experience with the disease, the financial burden that the treatments place on the family, the myriad of insurance issues that arise, navigating through the hospital and treatment bureaucracy, and the lack of support groups for the particular type of rare cancer the person has been diagnosed with are among the problems which immediately appear. Single parent families and families in which both parents work have additional difficult circumstances to deal with. A diagnosis of sarcoma affects the entire family. We have seen it bring families together. We have also seen it tear them apart.
• Many sarcoma patients never meet or talk with another sarcoma patient during the course of their treatment and experience an extreme sense of loneliness or isolation. No one can be strong all of the time or all by themselves. We urge sarcoma centers to provide a mechanism whereby sarcoma patients can meet one another.
• It takes many years for a basic scientific discovery to go from the bench to animal studies to human clinical trials to ultimately become part of an approved treatment protocol. We know only too well that progress in research is frustratingly slow, and that many who are dealing with sarcoma today have limited treatment options. We know that sarcomas are often diagnosed in young people who face potentially dangerous, long-term complications. We know that federal funding to support research in sarcoma is woefully inadequate. Sarcoma advocacy organizations must support sarcoma research until this changes and they need your help to do this.
• There are over sixty different subtypes of bone and soft-tissue sarcoma. To the best of our knowledge, there does not exist a non-invasive method to definitively diagnose any of them. That is, there are no salvia, urine, stool or blood tests that can be used to diagnose a sarcoma. Tissue samples, obtained from either a biopsy or an excised tumor, must be analyzed by a skilled and experienced pathologist in order to render a diagnosis. While the symptoms a patient presents with, the associated medical history, and the results from a physical examination and various imaging tests (x-rays, CT scans, MRI scans, PET scans and ultrasound images) may suggest to an oncological team the diagnosis of a sarcoma, it can only be confirmed by a pathologist's analysis of tissue samples. Because sarcomas are rare cancers and many physicians may only encounter one or two sarcomas in their lifetimes, a diagnosis of a possible sarcoma isn't the first thing which "springs to mind" when examining a patient, ordering tests and developing a treatment plan. It is not surprising then that sarcomas are often misdiagnosed or diagnosed too late. This leads, in some cases, to a surgery to remove a mass thought to be benign only to find out that a cancerous tumor, sometimes a sarcoma, has been discovered. There is an urgent need not only to develop non-invasive diagnostic tests for sarcoma, but tests that will identify specific bone and soft tissue subtypes. Such tests will reduce the number of misdiagnosed sarcomas and "oops" surgeries and aid the oncological team in developing an appropriate treatment plan for the patient.
• "Personalized medicine" and "precision medicine" will hopefully lead to targeted therapies which will, for the specific patient, be more effective, have fewer side effects, control the tumor growth and possibly cure the patient's cancer. However, tests often have limitations on their usefulness. There is currently a disconnection between the mutations identified by molecular profiling and the activity of drugs supposedly targeting these mutations. In vitro chemosensitivity and chemoresistance tests often lack the full range of agents that could be capable of inhibiting the identified targets. A number of sarcoma oncologists we have talked with believe the use of these tests is premature and the results cannot be used in the clinical setting. They state that in vitro chemosensitivity testing has been studied for more than two decades and has not been shown to reliably predict response to treatment.
• The incredible amount of information being revealed by next generation sequencing does not appear to be leading directly to answers about treatment, but rather is revealing new degrees of complexity in sarcomas — within a patient's own tumor and between tumors of the same type from different patients. This phenomena, which is referred to as tumor heterogeneity, poses important problems not only in the research setting, but in the clinical setting as well.
• Since tissue banks are central to cancer research, one might question if there is a national or an international bank of sarcoma tissues. Given there are no national tissue banks for any disease, you probably already know the answer to this question. It is our belief that access by sarcoma researchers to tissue banks is increasingly important in the study of sarcoma and may lead to significant insights into the diagnosis and treatment of this family of diseases. Some institutions are known to have created "large" sarcoma tissue banks over a period of time. However, since sarcomas are rare and there are so many different types of them it is probably impossible for individual institutions to collect sufficient numbers of comparable samples in any reasonable amount of time to be of use to the sarcoma community at large. We believe there is a significant need for either a national sarcoma tissue bank or a well-defined process for institutions to share tissues for research purposes. One can easily conjecture the need for an international sarcoma tissue bank or process for accessing specimens for research purposes internationally.
• We have believed, from the very beginning of our organization, that progress in developing newer, more effective treatments for sarcoma can be accelerated by: (i) funding quality basic and translational research projects internationally; (ii) encouraging collaborative research; (iii) embracing Open Science principles; and, (iv) establishing and maintaining a quality scientific peer-review process. These and other details of our research grants program can be found in the article, "A Model Research Grants Program for Advocacy Organizations Funding Research in Rare Cancers."
As we end the publishing of ESUN, we owe a significant debt of gratitude to all of those who have given so freely of their time, expertise, and experience to help support our efforts. Many of the members of our Medical Advisory and Editorial Board have, over the years, been of significant help. Scores of authors have submitted articles to ESUN for consideration. There have been over 175 sarcoma oncologists and investigators who have been involved in the peer-review process of these articles. Scores of investigators have submitted grant applications seeking funding to support sarcoma related research. Over 275 sarcoma oncologists and investigators have been involved in the peer-review process of these applications. Taken together, both of these processes have involved an extraordinary gift of time from those whose schedules are already overflowing with commitments to their patients, their families and their profession. The results of their work are evident in the quality of the articles published in ESUN and the quality of research that we have funded. We have also been the beneficiaries of the incredible generosity of thousands of people who have made donations to help fund sarcoma research. We are proud of our record of transparency and accountability to these donors and the sarcoma community. We are indebted to the small group of volunteers who have made all this possible. The accomplishments of the Liddy Shriver Sarcoma Initiative are based on the generosity of others.
Deciding to end the publication of ESUN and phase out our Research Grants Program has been a difficult and bittersweet decision for us, but we felt the time had come to make it. We still have quite a bit of work to do for the Liddy Shriver Sarcoma Initiative, including processing over a dozen grant applications, processing articles that have been submitted to ESUN which are still in the peer-review process, publishing the experimental plans and research reports of research grants that have already been awarded or will be awarded in the next few months. We plan to wind down by next May or June and then move on to the next chapter in our lives.