Archive for the ‘cancer’ Category

Turning the Tide Against Cancer: Patient-Centered Care and Value

September 24, 2014

On October 9, the Personalized Medicine Coalition, the American Association for Cancer Research, and Feinstein Kean Healthcare will be convening the second Turning the Tide Against Cancer Through Sustained Medical Innovation national conference in Washington, D.C., to continue the ongoing dialogue which began at the initiative’s inaugural conference in June 2012.

This year’s conference will bring together leaders from across the cancer community to help identify specific policy solutions to the challenges of supporting the shift to patient-centered research and care and addressing the value and cost of cancer care — two key themes that have emerged through the initiative’s ongoing work.

The Age of Personalized Medicine editorial team sat down with each of the initiative co-conveners to talk about the current cancer research and care landscape,  the upcoming conference, and what progress has been made since the start of the Turning the Tide Against Cancer initiative in 2011.

Our conversation with Edward Abrahams Ph.D., president of the Personalized Medicine Coalition can be viewed below. Stay tuned for additional video interviews with Margaret Foti, Ph.D., M.D. (h.c.), chief executive officer of the American Association for Cancer Research and Marcia A. Kean, M.B.A., chairman of Feinstein Kean Healthcare.

Visit the Turning the Tide Against Cancer website to register for the conference and learn more about ways you or your organization can support the ongoing initiative. The Age of Personalized Medicine will also be tweeting live from the conference on October 9.

Required Reading: August 2014

August 28, 2014

Great stories are published daily about the impact personalized medicine is having on individual patients, and the medical community as a whole, but it can be a challenge to stay on top of the news. With that in mind, we bring to you a monthly roundup of the three to five most thought-provoking articles we are reading, sharing and discussing with our colleagues.

This is the August 2014 installment of Required Reading.


The Price of Personalization by Timothy Gower, Proto Magazine

This article explores the growing debate over the cost and value of personalized medicines and identifies ways that the healthcare system may need to adapt to accommodate the development and use of increasingly more targeted therapies that work for smaller patient populations.

FDA to Regulate Thousands Of Cancer, Genetic, and Other Diagnostics by Matthew Herper, Forbes

Earlier this month, the U.S. Food and Drug Administration (FDA) announced plans to regulate laboratory developed tests, many of which are diagnostics developed as result of the exploding field of genetics. The new regulatory framework proposes that any test used to diagnose a disease or to decide on a course of treatment will need to be cleared by FDA before it can be utilized.

It’s Time for Us to Think About Cancer Differently by Paul Mejia, Newsweek

A recent genomic study published in the journal Cell suggests that 1 in 10 cancer patients could be more accurately diagnosed if cancer were defined by molecular and genetic characteristics, rather than by where it is located. Researchers believe that reclassifying cancer by identifying the type of cell that caused the disease, instead of the tissue type, could ultimately lead to better treatment in the future.

RNA Combination Therapy for Lung Cancer Offers Promise for Personalized Medicine by Kevin Leonardi, MIT News

Early research at the Koch Institute for Integrative Cancer Research at MIT offers promise for personalized cancer treatments using RNA combination therapies to improve therapeutic response. The development of an efficient delivery system of individual or combined small RNAs to solid tumors could help regulate genetic mutations underlying a given patient’s cancer.

Required Reading: June 2014

June 24, 2014

Great stories are published daily about the impact personalized medicine is having on individual patients, and the medical community as a whole, but it can be a challenge to stay on top of the news. With that in mind, we bring to you a monthly roundup of the three to five most thought-provoking articles we are reading, sharing and discussing with our colleagues.

This is the first installment of Required Reading.


Drugmakers Find Breakthroughs in Medicine Tailored to Individuals’ Genetic Makeups by Brady Dennis, The Washington Post

One-size-fits-all blockbuster drugs are giving way to treatments tailored to individuals’ genetic makeups. This article highlights the advantages of these targeted treatments, posing important questions that regulators, drug makers, insurers and patients alike need to consider as more specialized drugs come to market, including who will benefit and how to ensure patients have access to life-saving treatments.

In a First, Test of DNA Finds Root of Illness by Carl Zimmer, The New York Times

Diagnosis is a crucial step in medicine, but it can also be the most difficult. A new DNA sequencing technology, called unbiased next-generation sequencing, can identify a pathogen by testing DNA samples from a patient and then comparing fragments with genetic sequences stored in online databases. This technology has the potential to not only provide speedy diagnoses to critically ill patients, but could lead to more effective treatments for diseases that can be hard to identify.

The Perfect Drug by Erin Biba, Newsweek

This article highlights how continued innovation in our understanding of cancer and the emergence of companies providing personalized treatments helped change one patient’s life by turning his cancer diagnosis from a death sentence into a managed chronic illness.

Keeping the Patient at the Center of Cancer Innovation by Amy Abernethy, M.D., Ph.D. and Arif Kamal, M.D., The Hill’s Congress Blog

Hot on the heels of the 2014 American Society of Clinical Oncology annual meeting and Institute of Medicine’s National Cancer Policy Forum earlier this month, this op-ed focuses on the role of medical innovation and patient-centered approaches in the growing discussion on the cost and value of cancer research and care.

Prescribe by Price, But at What Cost to Patients?

June 16, 2014

In recent months, there has been increased discussion about the cost and value of cancer care. These conversations are important, but it’s crucial to make sure the patient’s unique perspective on value isn’t lost in the mix. We must also be sure we are considering each patient’s unique diagnosis, as well as the heterogeneity of their disease.

It is clear that targeted therapies play an important role in delivering personalized care to patients, and in particular, those with many forms of cancer. A recent article in Newsweek profiled a patient who, through a new tumor identification method, was able to receive a highly-targeted treatment for his sarcoma. As noted in the article, “the slow and steady march of innovation in our understanding of cancer and the emergence of companies providing personalized treatments have not only saved Rose’s life but allowed him to turn his cancer from a death sentence into a chronic illness that simply needs regular managing.”

These stories are becoming more common, yet as new targeted treatments work through the regulatory pathways, we are faced with ongoing questions: How do we ensure patients have access to these life-saving drugs and the diagnostics used to select them? And how do we ensure the system incentivizes research and development broadly, so more people benefit from personalized medicine?

Recently, the American Society of Clinical Oncology (ASCO) announced that it will be developing a scorecard, slated to come out this fall, to evaluate cancer drugs based on their cost and value, as well as their efficacy and side effects. This signifies a potential shift in the way that physicians make decisions about their patients’ health, encouraging them to consider the financial implications of interventions alongside patients’ needs. As this framework is developed, it will be important to ensure that the full value of innovative interventions is recognized.

Scientific advances have the potential to provide earlier and better diagnoses, more effective treatments and even possible cures for patients. Personalized approaches to care could provide a patient with more or better quality time to spend with their family or friends, and overall greater long-term value.

As Edward Abrahams, president of the Personalized Medicine Coalition told The Washington Post, these targeted treatments also bring a greater long-term value to the healthcare system as a whole by cutting down the frequency of unnecessary treatments and procedures, and avoiding unnecessary side effects.

Still our continued focus must be on providing high-value care and creating a system that improves patient outcomes and does not place the focus on prescribing drugs based on cost or financial incentives. The Personalized Medicine Coalition, along with the American Association of Cancer Research and Feinstein Kean Healthcare, continues to engage the cancer community to address these questions of cost and value through the Turning the Tide Against Cancer initiative.

Through ongoing dialogue and discussion as part of this initiative, two key themes continually resurface as components that should be considered when defining value: advancing approaches that are patient-centered, and developing new approaches to value assessment that align with and incentivize scientific progress and innovation.

On October 9, 2014, the Personalized Medicine Coalition, the American Association of Cancer Research and Feinstein Kean Healthcare will re-convene the cancer community in Washington, D.C., for a second Turning the Tide Against Cancer national conference to advance the conversation on the value and cost, while supporting a shift to patient-centered cancer research and care.

From The ASCO Meeting In Chicago: A Focus On Cost, Value, And Financial Toxicity Of Cancer Care

June 9, 2014

In the following post, Dr. Leonard Lichtenfeld, Deputy Chief Medical Officer for the American Cancer Society, shares his thoughts on the discussion at the 2014 American Society of Clinical Oncology (ASCO) annual meeting regarding the cost and value of cancer care and treatment and mentions an article recently published in Clinical Cancer Research that he co-authored with other members of the Turning the Tide Against Cancer initiative.


At the annual meeting of the American Society of Clinical Oncology (ASCO) here in Chicago, something vitally important is happening: there is an increasing recognition of something no one really wanted to talk about in polite company until now. It is the fact that the costs of many of the new treatments being developed are extraordinary.

The headlines about cost and value of cancer care greeted me when I walked into the McCormick Center in Chicago for the opening sessions of the meeting. This is the leading cancer meeting in the world, and what happens here makes news worldwide, significantly impacting the lives of patients with cancer wherever they may be.

Now there is an increasing recognition of the elephant in the room: the costs of these new treatments are extraordinary. No matter how one chooses to slice and dice the arguments, these drugs are expensive with costs per month of $8000 and upwards getting a lot of attention and increasing concerns, especially at this meeting.

For years, it was whispered in hallways, muttered about with reporters, but now it’s out in the open. The headlines say it, the lecturers say it, the attendees are talking about it. There is an increasing awareness surrounding the value, cost and toxicities–both medical and financial-of cancer treatment. Even as I write this I am sitting in a session devoted to the topic of how we make the choices so necessary for the welfare of our patients and how we should guide those choices.

Some say the costs of this brave new world of cancer care are justified by the advances they bring to the clinic and the lives of patients. The cost of the drug should reflect the value they provide to society says one company. Others say the costs are justified to allow continued research to advance new drugs. But increasingly, another voice is being heard: one that says the costs are unconscionable and not sustainable. Patients are going bankrupt and some of the drugs are quite toxic or don’t bring sufficient value to the patient (such as meaningfully extending life, or reducing side effects).

This argument and the facts behind it are not going to go away. And they shouldn’t. There are too many forces at work here to suggest that this discussion be tabled. Right now, camps are developing on either end of the spectrum, with many if not most cancer professionals are in a large undecided middle. This war of words includes pharmaceutical company press releases promoting drug development, lectures describing how organizations are trying to develop ways to measure the true value of particular treatments-not just the cost, but what the drug really brings to the table, how many side effects does it have, and what is its “financial toxicity” (the lecturer’s words, not mine) of the medication.

And as this debate rages, I can assure you it’s only the beginning. Listen to the experts and you hear that we are moving forward rapidly to develop new equipment to more rapidly analyze the cancer genome. Another expert exhorts us that we only have drugs to address 5% of the genetic abnormalities we have identified that make a cancer cell a cancer cell. We are looking at a future where literally thousands of drugs may potentially be available to treat the various genetic abnormalities we find in any one person’s cancer (each cancer has a number of these changes, and then with treatment the cell is smart enough to develop a “workaround” which would require a different drug).

One expert made the very appropriate comment that this level of individualization means we are moving toward a day when “every cancer is a rare cancer.” Imagine cancer cells being like fingerprints: each one an individual, except that unlike fingerprints, cancer cells can change their stripes. And we need to meet each of those changes wherever those cells reside in the body.

A couple of months ago I was privileged to be a coauthor on an article published in Clinical Cancer Research talking about these issues. The article was written by members of the Turning The Tide Against Cancer initiative, and described what we have to do to change the system. Refocusing on the entire ecosystem of research, translation of drugs from lab to bedside, clinical trials, clinical practice and how we care for patients was the topic, and all of those have to be addressed. It will be an immense effort that will require intense focus and commitment from many participants to reform what we do and how we do it and then make it work for our patients, wherever they may be. Ultimately, patients must be our focus and always be the center of our attention. Whatever we do must be through the lens of the patient and their families.

What bothers me about all of this? While it’s great this issue is finally getting some attention, I don’t yet see the passion I would expect to see given the serious barriers standing between us and our promised future. We have spent decades getting to this point. We need to get the new effective treatments to patients as quickly as possible, wherever that patient may be. We need new trial systems to evaluate drugs, and perhaps rethink how we study drugs to learn as much as we can from each patient.

And then we need to figure out how to pay for all of this, supporting our research enterprise, pay for the care, and afford the new drugs. Many people, organizations, and corporations have to rethink how they do business. Society must make this process work.

Here we sit, but is it on the edge of a precipice or the edge of an opportunity? Who will take the lead? Who will truly define value? Who will afford the costs?

So many questions, and hopefully some answers are coming. We need a new paradigm, we need a better future. Business as usual is not going to get us where we need to be. If we want to make this cancer’s last century we had best take hold of the opportunity and make it happen. We may not get another chance if we don’t act now, and make our future and our vision our reality.


A version of this blog originally appeared in Dr. Len’s Cancer Blog at the American Cancer Society.

Finding My Future, or, How to Coexist with Cancer

March 24, 2014

In October 2007, my identity was stolen.

In September 2007, I was a 39 year-old wife and mother of two young girls. Then, suddenly, I was a 39 year-old wife, mother of two young girls, and Stage IV lung cancer patient.

At the time of my diagnosis, I learned a majority of late-stage lung cancer patients die within one year. Just one year. One birthday. One summer. Would I make it to another Christmas? I didn’t know. Already a “glass half empty” kind of person, I wondered if this was my death sentence as the sense of a future ahead of me drifted away.

For the first six months, I tried traditional treatments, and I felt terrible. I was finding out what it felt like to experience the decline of death.

But here I am. More than six years later, alive and kicking. And personalized medicine is the reason.

My experience with molecular testing and targeted therapies
My first line of treatment was the classic carboplatin-taxol combo, combined with bevacizumab—the first of the newer, targeted therapies. Even though I was epidermal growth factor receptor (EGFR) negative and might not respond, I fit the common demographic for success closely enough that my doctor and I decided to try erlotinib with bevacizumab. That run lasted over two and a half years—precious time. I spent that time with versions of the most common side effects but otherwise felt pretty normal and lived life actively, something I never expected to do again.

During that time, I learned about the clinical trial for crizotinib on the news, and after three different people contacted me to pass along the story, I considered it an omen of sorts and asked to have my tumor specimen tested to see if I was a fit for this new personalized treatment.

When I found out I was ALK positive—and therefore likely to respond to the treatment—I felt relief. I knew how promising crizotinib already was and now I had my next plan in place. I have been on this targeted therapy for more than two years and I have been living a virtually symptom-free, normal existence with my daughters, with the hope for more.

Targeted therapies have been invaluable to my treatment. I know that I am very fortunate and remain in the minority to have my disease controlled so well. Frankly, that fuels my feeling of responsibility to do something productive to change things. So many—too many—die so quickly.

From my perspective, there are two important considerations for targeted therapies.

  1. We should use molecular testing as a way to inform treatment—not exclude access to drugs: I am enthusiastic about the value of molecular testing, and I also believe that its value lies not in its exclusionary potential, but in informing the priorities of a treatment plan.

    Having benefited over a period of almost three years from erlotinib when I tested negative for the genetic marker, I’d hate to see that same possibility be denied outright to others. I know that for some cancers and other illnesses, there is a concern about “overtreatment,” but in the lung cancer world, we’ll take whatever we get because our odds are so poor.

  2. We should better keep healthcare providers educated and up-to-date on the latest in molecular testing: I have heard horror stories about oncologists who don’t know much about the testing, never mention it to their patients, and trudge down the traditional paths without pause.

    We need better approaches to ensure doctors have access to the latest research and tools for diagnosis and treatment. I worry about the patient that doesn’t have the tools to advocate for their own healthcare.

Millions of people are depending on scientists, business people, and policymakers to keep working hard and collaborating to bring access to promising therapies to those in need and accelerate medical breakthroughs.

Now, I live with Stage IV lung cancer. It is a part of my body and part of my identity. My outlook on life has changed dramatically since my diagnosis, but my ability to take care of my daughters and live my life hasn’t at all. And that is incredible.

Molecular testing and personalized medicine gave me my life back, and my sense of a future back. While I’m realistic enough to know that my daughters are fairly certain to lose their mother before they are grown, I also know I have tools to fight with, and a responsibility to share my story.

We need earlier detection.
We need the tools to cure.
We need to increase survival for those, like me, who live with cancer.

New Approaches for Practical Challenges in Personalized Medicine

January 27, 2014

When I received the invitation to speak at the Personalized Medicine World Conference (PWMC), I was excited. When I saw the program and faculty, I was honored. When I received my “assignment,” I was intimidated. To share the podium with many of the luminaries in the field of genomics — even to hear them speak — is tremendously exciting. In contrast, how can they really be interested in hearing me speak on the real-world challenges providers and payers face in bringing genomics advances from bench to bedside more quickly?  I cannot answer this question. Although based solely on the number of conferences to which I am invited on this topic, someone must care. They should. Making the science “work” is only the first step in moving from bench to bedside.

As I was preparing my talk, I reflected on the story Sid Mukherjee, M.D., Ph.D., tells in his fabulous book, The Emperor of All Maladies: A Biography of Cancer. I’m not usually a big non-fiction fan, but if you are involved in cancer care in any way, this is a must read. Perhaps the book resonated because my professional career spanned many of the seminal events described in the book. That, and I share Dr. Mukherjee’s general perspective on the challenges of making meaningful advances in cancer treatment. The bottom line: cancer is tough. Just when we think we’ve made some great progress, it teaches us humility. Still, it is impossible not to be excited and enthusiastic about how far we have come in our understanding of the genetics of cancer. I really think we are experiencing a sea change. We must begin to think about navigating these changing currents in the world of health care reform in order to take best advantage of that progress.

So what are the practical challenges? There are many. And many share the underlying theme that the old paradigms do not work so well. Traditional methods of quality control in test performance, i.e. analytical validity, are a big problem. Proficiency testing and standards do not currently exist. To be honest, most doctors do not think about them when ordering a test. The billing and coding of molecular tests have been undergoing dramatic evolution but is still not suited to multi-analyte assays. Perhaps the biggest challenges lie in the area of clinical utility, which impacts providers, payers and regulatory agencies. Patients are impacted in a huge way. Most people have an idea of where we need to go, but we have a shortage of ideas about how to get there. Finally, all of this occurs in the setting of unsustainable growth in health care spending, and the near uniform agreement that we need to spend our money in a more intelligent, impactful way.

We have a lot of work to do together. It is important to remember we are all on the same side. We have a chance to make a huge difference, but we need to do it right.

What do you think our greatest challenges and opportunities are today? I look forward to the discussion in California.

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Read additional posts from speakers and participants of the 6th Annual Personalized Medicine World Conference on January 27-28, 2014. For more information and the full agenda, visit: 2014sv.pmwcintl.com.

Ten Years into Personalized Medicine: What We’ve Learned and What’s Next

January 15, 2014

Ten years ago, the sequencing of the entire human genome, along with the development of aggregate “omics” technologies began giving rise to a fundamentally new capability for the practice of medicine – the ability to predict and track disease risks on a personalized basis, to understand diseases mechanistically, and to target therapy to treat an individual’s specific disease.

Based on the potential impact of these technologies, I predicted in my 2002 Chairman’s address to the Association of American Medical Colleges, that medicine would be transformed from being disease-focused and reactive to being proactive and personalized. I believed that the “one-size-fits-all” approach to disease care was outmoded and would soon be replaced by one that would prevent and treat disease on a personalized basis.

Since its inception ten years ago, many of those predictions have been realized; personalized medicine has begun to have major impacts on components of medical practice and has engendered health care industries estimated to grow to $450 billion by 2015 with $42 billion related to drugs, devices, and diagnostics. Molecular diagnostics have gained traction in cardiology, rheumatology, transplantation, endocrinology, and, in particular, oncology. Targeted therapies have revolutionized cancer therapy and whole genome sequencing is providing insights into baseline health risks and understanding of some diseases. However, while the use of personalized medicine tools to treat disease is gaining traction, the transformation of medical practice to being proactive, strategic, and personalized; i.e., personalized health care, has been slow to develop.

There is, however, evidence that this movement is gaining momentum and with increases in health care consumerism, a more predictable regulatory environment, and changes in medical reimbursement to reward better outcomes, the adoption of personalized health care is inevitable. Personalized medicine will go beyond the use of technologies to individualize disease care to finally transforming the approach to care itself by enabling individuals and care providers to foster proactive, personalized care. In turn, the clinical adoption of proactive, personalized care will broaden the need for personalized medicine technologies thus spurring the growth of this industry.

Appreciating the value of a ten-year review of personalized medicine, the Personalized Medicine World Conference will host, and I will moderate, a panel discussion titled, “Ten Years into Personalized Medicine: What We’ve Learned & What’s Next” on January 27, 2014 in Mountain View, Calif., with luminaries including Kim Popovits of Genomic Health, Randy Scott from InVitae, Brook Byers of KPCB, and Jay Flatley from Illumina, opining on what they initially anticipated, what they’ve learned, and what’s coming next.

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 Look for additional posts from speakers and participants prior to the 6th Annual Personalized Medicine World Conference on January 27-28, 2014. For more information and the full agenda, visit: 2014sv.pmwcintl.com.

2013: A Year of Innovation, Inspiration

December 20, 2013

As we look forward to 2014, we engage in the time-honored tradition of reflecting on the year of activities and progress as we bid 2013 farewell.

For personalized medicine, it was another year with big advances, and big questions. The following captures highlights from The Age of Personalized Medicine Blog for 2013.

We started the year with a call to action from Amy Miller, PMC vice president, public policy, about the power of personalized medicine, the need for “accurate and actionable” data, and for a shift in traditional healthcare models. As Amy noted:

…The power of personalized medicine lies not only in treatment, but prevention. The best scenario for personalized medicine in action will come when patients are able to see not only the implications of their genetic dispositions, but also are motivated and empowered to use that information in preventative care.

In May, personalized medicine took the international spotlight as Angelina Jolie shared with the world her genetic prognosis and personal decision to pursue a course of preventative care. The high-profile case inspired much discussion of the value of personalized medicine, as well as the need for access to diagnostic testing for all:

Ms. Jolie’s contribution demonstrates that without patients who are educated, empowered to get tested and to act, the progress and innovation could be for naught. We need to make sure that patients are knowledgeable enough to pursue personalized options for their own health but also so that they are motivated to support policies that foster continued progress in this area.

Kristin Ciriello Pothier, Diagnostics and Life Science Practice lead at Health Advances, shared key take-aways from the PMC/BIO Solutions Summit panel she moderated on “Evidentiary Standards and Data Requirements for Payer Coverage.”

Ms. Ciriello Pothier examined the challenges facing the personalized medicine diagnostics industry and summarized her panel’s conclusions:

First, panelists agreed that there must be more education for all stakeholders so that each stakeholder can actually evaluate novel products appropriately, a key finding echoed throughout the day. Second, the emphasis on outcomes must shift from only clinical outcomes to clinical outcomes and quality of life for patients. Finally, all panelists agreed the ideal situation is open, trusting lines of communication and split of the responsibility according to expertise.

William Chin, M.D., identified research, collaboration, and innovation as critical components to advancing personalized medicine, while Stephen Eck, M.D., Ph.D., cited the need for funding for basic research, diagnostics testing research, increased healthcare provider education, and reimbursement for diagnostic testing as key.

At the mid-point of 2013, pressures to reduce federal spending elevated the need for policy that will support biomedical research and innovation. PMC responded to these concerns through comments submitted to the Centers for Medicare and Medicaid Services (CMS) regarding the charge on regional Medicare Administrative Contractors (MACs) to set new prices for molecular diagnostic test reimbursement through a process called “gapfilling”:

The Personalized Medicine Coalition (PMC) submitted comments to CMS on the Gapfill Payment Amounts and CLFS, iterating the concerns of PMC members that insufficient payment amounts threaten the sustainability of the laboratory industry and continued investment in the developing field of personalized medicine. As a consequence, this policy has the potential to stifle innovation and progress in healthcare and possibly eliminate the potential for lowering overall costs through the elimination of unnecessary and or ineffective treatments.

In 2013, PMC also released a set of policy principles key to advancing personalized medicine in a deficit-reduction environment. Debuted at a Capitol Hill Briefing, these principles lay the groundwork for consensus-building around key issues to ensure that efforts to contain rising healthcare costs do not undermine continued progress in personalized medicine and protect innovation, the physician-patient relationship, patient values, and choice.

The reality of personalized continues to be realized with patient triumph stories bringing new advances in science, technology, and research to life:

The promise of personalized medicine is very real. Personalized medicine is not an abstract concept for the future of medicine. It is here, it is now, and the true promise has been realized in the lives of Kellie and Stephanie, and the precious days, weeks, months, and years they have taken back from their disease.

I encourage you to read more about Kellie and Stephanie, and to draw inspiration from their strength.

At the 9th Annual Personalized Medicine Conference in November, it was an honor to present Kathy Giusti, founder and CEO of the Multiple Myeloma Research Foundation (MMRF) with the 2013 Leadership Award. Ms. Giusti’s story of ingenuity in the face of adversity has led to a redefinition of the role of patient advocacy organizations in research:

Clinically, MMRF has funded research and paved the way for FDA approval for six multiple myeloma treatments in 10 years, and doubled the lifespan for many patients. Today in her remarks, Kathy shared that her daughter, who was just one year old when Kathy received her myeloma diagnosis, is now a 19-year-old college student with a younger brother. Incredible results.

2013 was a year of progress and inspiration. We look forward to the new year ahead and personalized medicine advances on the horizon, bringing new treatments and improved outcomes to patients.

Personalized Medicine Community Gathers in Boston for the Ninth Year to Share Insights and Innovation from the Field

October 29, 2013

Personalized Medicine was an emerging field of medicine in 2005 when we first held what was to become an annual event, our personalized medicine conference. The importance of personalized medicine was given a boost by the then U.S. Secretary of Health and Human Services, Michael Leavitt, who said in January 2005, “I believe we are moving into a remarkable and powerful new era in medicine and particularly in prescription drugs.  I’d refer to it as an era of personalized medicine.” Since then many different names have been given to this approach to medicine. They include “Precision Medicine”, “Genomic Medicine”, “Genetic Medicine”, “Individualized Medicine”, and “P4 Medicine” to name a few.

This year, we gear up for our 9th Annual Personalized Medicine Conference on November 6th and 7th, in Boston, arguments can be made about what name adequately describes the enterprise in which genetic and genomic information informs us about a person’s risk for human disease; about a clear diagnosis on which treatments depend; about prognosis for a disease such as cancer; about genetic variants in an individual patient that may determine that person’s ability to metabolize a particular chemical entity; about genetic and genomic changes that might inform physicians about the appropriate therapeutic approach; and about many other aspects of health and medicine where genetics may play a role. Some people in the scientific community contend that the use of such a diversity of names for the same enterprise is natural and reflects the evolution of the field. What we cannot ignore is the direction that the science is leading us – toward precision diagnosis and treatment of disease at the molecular level.

As I reflect on the past nine years, some aspects of personalized medicine have changed rapidly and others are relatively unchanged. One of the biggest and most exciting changes involves the growing commitment of drug developers to the development of targeted therapies. At the beginning of the 21st century, there were only a few drugs that could be considered “targeted” therapies. Now, in cancer, for example, the development of such targeted therapies is becoming the norm because many of these drugs have few adverse effects and the response rates of patients, whose tumors have the molecular target for the drugs, are very high compared to other non-targeted therapies. We can find similar “personalized” approaches to treatment in the areas of infectious disease, cardiovascular disease and other areas.

Today, much of personalized medicine is fueled by new technologies related to DNA and RNA sequencing. It has been estimated that the cost of sequencing has dropped by a factor of 100 from the beginning of this century. The amount of DNA or RNA required to sequence whole genomes has also been reduced by orders of magnitude and there are now technologies in development that promise to sequence single molecules of DNA in a matter of few hours. As the technologies improve and associated costs decline, the benefits of genome sequencing become more apparent. It is easy to imagine a not-too-distant future when people around the world have their genome sequenced as part of the standard of care. Despite present concerns about the cost to analyze all of these genomic data, I am certain that newer algorithms will enable us to automate much of the analytical and interpretive processes to propel us toward a new level of understanding regarding the prevention and treatment of disease.

As the science evolves and entrepreneurs continue to innovate, we are faced with new challenges.

Open and informed discussions about issues related to personalized medicine are critical for better understanding of the successes, failures and promises of this relatively young medical enterprise. Our Conference in Boston provides one such forum and we hope that you will be able to join us.

The future of medicine is before us.

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Harvard Medical School, Harvard Business School and the Partners HealthCare System in Boston will convene the 9th Annual Personalized Medicine Conference on the campus of Harvard Medical School this November 6 and 7. As has been the practice since the Conference’s inception, this year’s meeting is in association with the Personalized Medicine Coalition (PMC). For the first time, we also welcome the participation of the American Association for Cancer Research (AACR), a member driven organization that has been dedicated for more than a hundred years to promoting cancer research and cancer care, as a co-convener.

For more information and to register for the 9th Annual Personalized Medicine Conference, please visit www.personalizedmedicineconference.org.


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