August 19, 2009
Over the last week, we have received many inquiries on the recent study “Multiple Myeloma in World Trade Center Responders: A Case Series” that indicates 9/11 emergency responders have an increased risk of developing multiple myeloma, a rare and fatal blood cancer. What this study shows is that out of more than 28,000 emergency workers who have been followed as part of a health monitoring program “9/11 WTC Health Registry“, eight cases of multiple myeloma were diagnosed. In the general population, six or seven cases would have been expected. Multiple myeloma in the general population is typically diagnosed in patiens who are at least in their 60s, but in this population, four of the cases were in workers who were less than 45 years old - in the general population, only one case in such young patients would be expected.
All of this said, it is important to understand that follow-up of these workers is limited, and more data are needed to better understand whether there may be a causal link. However, because multiple myeloma has been linked to exposure to very high levels of extremely toxic chemicals like Agent Orange or high levels of radiation exposure from the atomic bomb explosions in Japan, it is unfortunately not surprising given that these workers were exposed to what has been called a “toxic chemical soup” “9/11 Responders May Be At Raised Myeloma Risk“, which included known carcinogens, for up to several months.
The good news is that more progress than ever is being made in the disease - in recent years, survival from diagnosis has doubled - and so these and other patients who are diagnosed today will have many more treatment options in the future than those who sadly lost their battle with this as yet fatal disease.
To learn more about multiple myeloma, click here: http://www.themmrf.org/living-with-multiple-myeloma/newly-diagnosed-patients/what-is-multiple-myeloma/
June 29, 2009
This year was a historic meeting for the Drug Information Association (DIA) – thanks to the visionary perspective of chairperson Nancy Smith, PhD, former Director, Office of Training and Communications, FDA, for the first time, the patient perspective was presented with representation from the MMRF and from the International Alliance of Patients’ Organizations (IAPO).
Last year, Kathy Giusti delivered the keynote address, which led to my participating in two panels to represent the patient perspective on drug development at this year’s meeting. My remarks focused on how the MMRF educates the myeloma community to optimize current therapies as well as ensures that the community has safe and effective therapies in the future. With me on both panels was Ken Kaitin, PhD, Director of the Tufts Center for the Study of Drug Development, who described the MMRF as “changing the landscape in drug development and patient care”. Kathy had the incredible foresight and insight to recognize that the MMRF’s goal mirrored that of industry – to bring new (and of course effective) treatments to patients as quickly as possible – but that was more than 11 years ago! In today’s world, collaboration and transparent communications among all key “pillars” (ie, patients/patient groups, academia, industry, and regulatory authorities) are critical to ensure safe and effective treatments are developed and made available to patients as quickly as possible, and we are more than happy to continue to play the role of congregator and broker to maximize these partnerships.
June 3, 2009
The data presented at the Annual ASCO meeting on new investigational drugs are of interest to all oncology researchers, and the opportunity to gather with large groups of oncology researchers to review and discuss these results is a critical part of the information exchange process in this field. There were approximately 175 abstracts presented from studies in multiple myeloma, including results from clinical studies ongoing within the MMRC clinical trials network. In addition, there were very informative presentations and discussions on early clinical trials of new drugs against pathways and targets that are relevant to myeloma, even if the study under discussion did not include patients with multiple myeloma. It is these early data that help the researchers in myeloma decide whether to investigate some of these emerging drugs in trials specific for multiple myeloma.
The theme of this year’s congress was “Personalized Medicine “. The entire field of oncology clinical research is at an important crossroads. We now understand enough of the biology of cancers, and have new drugs emerging that allow us to begin to tailor specific drug therapy against the molecular pathways to which a particular tumor, or indeed, a particular patient’s tumor, is especially “addicted” for growth. This will not be a short term effort. The tools for genomic and proteomic analysis need to be applied in a more widespread manner, in carefully constructed clinical trials, with the goal of matching patients with particular drugs or drug combinations. The field of myeloma research is well equipped to pursue this goal of personalized medicine. The scientific and clinical researchers who are our partners in the quest to develop new drugs to improve patient outcomes, and ultimately attain cures, in multiple myeloma, have already been collecting bone marrow samples for important analyses. The next step is to organize a large effort in partnership with our research partners and stakeholders, to design these clinical studies where patient treatment is assigned according to biologic markers and risk factors for a given patient. We cannot stress enough how important it will be for patients to participate in clinical trials as we attempt to move this field forward.
In addition to the efforts around personalized medicine, there were clinical trial results and discussions about important therapeutic issues in myeloma presented at ASCO. For example, with the improved outcomes reported as a result of treatment with the newer drugs that have become available for myeloma in the past 4 years, there are now questions raised about the optimal timing of autologous stem cell transplant—which patients, when and after how much initial therapy? All patients should continue to engage in these important discussions with their physicians.
June 1, 2009
On Friday, the MMRF hosted its fifth CME symposium for physicians at the Annual ASCO Meeting. This year, the esteemed faculty included Chair of the Program, Ken Anderson, Dana-Farber Cancer Institute; Sergio Giralt, MD Anderson Cancer Center; Vincent Rajkumar, Mayo Clinic – Rochester; and Paul Richardson, Dana-Farber Cancer Institute. The program was entitled “Treatment of Multiple Myeloma: Cure vs. Control”, based on a thought-provoking paper from Dr. Rajkumar published last year in the Mayo Clinic Proceedings (http://www.mayoclinicproceedings.com/content/83/10/1142.long).
The presentations each addressed the question of whether, at each point in the disease course, the goal of therapy should focus on cure or control. There is consensus on the long-term goal being cure – that much is clear – but at each point, particularly as the disease progresses, there is far from unanimity on how aggressively clinicians should go for the cure – or at least a complete response – at the risk of increased toxicities and impaired quality of life. There does seem to be greater consensus with respect to high-risk patients, as they are more likely to relapse and data indicates that inducing a sustained complete response is critical in these patients. Similarly, there are emerging data to indicate that patients who do not necessarily achieve a CR can benefit from a sustained partial or even minimal response, as shown from a small number of patients who remained stable for a number of years on Revlimid as a single agent – without dex - despite never achieving a CR on therapy. Somewhat counter intuitively, it appears to be more beneficial to achieve a lower but durable response than a transient CR that is associated with rapid relapse (http://www.nature.com/nature/journal/v459/n7246/full/459508a.html).
The question of cure vs. control again emerged subtly in the Sunday morning oral presentations, and it is clear that there is not yet substantial data to help us understand which regimens can maximally delay disease progression and increase survival. It is clear that newer combinations – particularly three drug combinations such as RVd (Revlimid-Velcade-dex) and VMP (Velcade-melphalan-prednisone) are associated with 80-100% response rates, and up to 40% CR rates when used front-line, which is an exponential increase over older combinations such as MP and VAD, but these trials are so young that for many, median survival and time to disease progression, never mind overall survival, have not yet been reached.
Until we have these answers it is important that patients, particularly those who are newly diagnosed and untreated, consider one of the many clinical trials available (www.myelomatrials.org) so that we can answer these critical questions.In the meantime, it is also important to understand that three and four drug combinations are likely to confer the greatest benefit to patients, the trials have very short follow-up, and many have limited patients analyzed to date, so it is premature to make any conclusions about what the optimal combination, at any point, may be.