Archive for the ‘Personalized Medicine Conference’ Category

Harvard Personalized Medicine Conference Showcases Progress in the Field

December 3, 2012

Great themes emerged at the Harvard Personalized Medicine Conference last week. This is the third year that I’ve attended and there’s a palpable sense of progress. Examples abound on how precise molecular profiling tools are now being used in clinical practice and the potential going forward is huge.

But this transformation is causing a number of new considerations to come into focus. Working in industry, we have to think about what do these opportunities mean for business? Where can we best add value; what parts of medicine are currently underserved? How can we bring industrial and operational strengths to the emerging new paradigm? And how is the regulatory environment likely to change…for therapies and for diagnostics?

The opportunities and the challenges are breathtaking. Contributors spoke of the opportunity for sequencing data integration into the Electronic Medical Record (EMR) and the subsequent effect on medical decision making. And, the emerging Personalized Medicine paradigm should provide opportunity for the sector to make the case of the value of innovation, both clinical and economic. But what will people make of a new generation of EMRs containing contextual individual information across a continuum of patient care…from predisposition, through screening, diagnosis to therapy selection and monitoring.

The audience-sourced data demonstrated how peoples’ acceptance of precision medicine has increased over the years in which the conference has been held. And although oncology still dominates, pharma representatives described case studies in cystic fibrosis and cardiology. Those same pharma contributors acknowledged the absolute necessity for powerful diagnostics to complement the delivery of personalized medicine.

On the first day of the conference, I moderated a session entitled: Business Models for Genetic Information.” Thought leaders from Siemens, PerkinElmer and Oracle described their companies’ visions for the molecular and digital age.

We had interesting audience feedback. Over 50% of the respondents felt that DNA sequence will become a routine part of an individual’s medical record within the next 10 years. Also interesting was the fact that only about 10% felt that availability of science or technology was the biggest obstacle to the adoption of personalized medicine in the clinic.

We discussed how personalized medicine can become mainstream. This isn’t about enabling personalized medicine for the few, but using technology and scale to deliver to the many. Doing this right not only will benefit patients, but input at the conference from regulators, drug manufacturers, and payors, told us there are a lot of parties interested in the success of this new paradigm. 

Making sense of all this data is a real challenge – how to format; how to integrate? Some contributors spoke of a data-driven industry transformation as has happened in other industries – financial services, retail, and communications. The result has been efficiency and transparency and the appearance of new interested parties – such as those capable of integrating workflow and data. 

The need for workflow integration will continue to be important as well. There are many players out there making individual contributions – from platforms, to molecular markers, to infrastructure, ordering, fulfillment, billing, and reimbursement. But who should take on the task of integration – which is surely needed if the benefits and efficiencies of personalized medicine are to be realized?

Personalized Medicine Is Waiting on the Shelf

November 16, 2012

While there’s a tendency to paint the future of personalized medicine as sunny, there are a number of issues persistently clouding the horizon. Bob Langreth and I examine one of these concerns in a story you can read at Bloomberg entitled, “Life-Saving DNA Test Overlooked in Rise of Colon Cancer.” Provider education has long stood in the way of patient access to genetic tests for Lynch syndrome, an inherited cause of high cancer risk. While the testing has been available for more than 10 years, enjoys recommendations from clinical and public health groups, and has been shown to save lives in studies, many providers remain unaware of its availability and power. The test costs as little as $300 in families where the mutation has already been identified. People who test positive can take preventive action with frequent monitoring, perhaps having vulnerable tissue removed.  Yet in too many cases it remains on the shelf.

How are we going to get the benefit of full genomes when simple, straightforward tests like this go unused?

John Lauerman will be participating in the panel discussion, “Impact of Genome Sequencing and Health,” at Harvard’s 8th Annual Personalized Medicine Conference, November 28-29, 2012, in Boston, Massachusetts.

The 8th Annual Personalized Medicine Conference

November 14, 2012

On November 28th and 29th, Partners HealthCare, Harvard Medical School, and Harvard Business School will host the 8th Annual Personalized Medicine Conference in Boston. As has been customary since the conference began, the meeting is also the venue at which the Personalized Medicine Coalition presents its leadership award. This year, it will go to Randy Scott, Ph.D., a founder and former CEO of Genomic Health, Inc.

During the past seven years, we have seen many changes in personalized medicine. Much of the excitement about personalized medicine continues to be driven by the ever decreasing cost of sequencing DNA and RNA. Whole genome sequencing, estimated to cost a billion dollars or more at the beginning of the century has gone down to a few thousand dollars, and the so called $1,000 genome is not too far behind. All in all, the 20,000 or so genes of the human genome only add up to less than two percent of the genome. If the sequencing cost is directly proportional to the size of DNA that is being sequenced, the cost of sequencing all genes should one day be no more $100. We are not quite there yet, but the rapid decline in costs is fueling the evolution of personalized medicine.

There is also a rapid evolution in our understanding of the genes and genomes of many organisms, including humans. Understanding how a normal gene functions and how changes in it (mutations) alter the cellular function is increasing at a rapid pace. As a result, sequencing whole genomes, whole exomes (all of the coding portions of genes), and subsets of genes for medical purposes is becoming ever more common. The fruits of all of these exciting developments are evident in many specialties of medicine but especially in prenatal testing, diagnosis of newborn and pediatric disorders, and cancer.

This year’s conference will highlight the genomic advances in science and medicine, and how they are being applied to diagnose and treat many disorders. The rapid development of technology also raises many questions about the clinical utility of these advances; about the policy and regulatory issues around what, when, who, and how to regulate; and about how the costs are reimbursed.

Personalized medicine is not just an American issue. Many nations of the world are developing strategies to implement personalized medicine in their own countries, and a number of those efforts will be featured at the conference. Professor Richard Hamermesh of the Harvard Business School and Norman Selby of Perseus LLC developed a business case on companion diagnostics that promises to be thought provoking and, based on previous years’ experience, a highlight of the conference.

Please join us at the end of November and participate in the dialog about how personalized medicine is evolving and what its implications are for your health and that of all peoples of the world.

Register online at www.personalizedmedicineconference.orgFollow the conversation online at @HarvardPMConf and #PMConf.

Less May Be More

November 12, 2012

Personalized Medicine has come to be strongly associated with drug-diagnostic combinations (companion diagnostics).  While this is a very important aspect of personalized medicine, it is not the goal.  The goal of personalized medicine is to find the best possible care for each individual patient so as to maximize the likelihood of the individual achieving his/her personal life goals.  While this may seem obvious, this principle has become increasingly harder to identify in every day medical care.

A variety of different motivations has encouraged the healthcare enterprise to view illnesses, or even everyday complaints as the opportunity – if not obligation – to do something for the patient.  The better approach is to ask: What is the best thing one can do for a patient, including the possibility of doing nothing?

As we develop better drug-diagnostic combinations, this will become increasingly apparent.  Soon we will have the ability (through use of precise molecular diagnostics) to know with reasonable certainty whether there is little or no benefit for a particular individual to pursue a therapy which had historically been the standard of care.

But, are we truly ready to forgo a potential therapy even if such a decision is based on strong scientific data?  Or, will we continue to use a therapy on the off chance it might work?  Our desire to do something has to include the possibility that the best treatment might be no therapy, based on the risks to the patient, the likely benefits, and the life goals of the individual.  In the end, patients want their healthcare providers to help them make the best choice and personalized medicine can be a strong tool to do that.  This will provide the largest benefit for patients as they avoid the risks that come with all therapies when no benefit is realistically to be had.

Dr. Stephen Eck will be moderating a panel discussion on Wednesday, November 28, 2012 at the 8th Annual Personalized Medicine Conference in Boston, Massachusetts.  Join the discussion at #PMConf. 

Still Skeptical about Personalized Medicine?

October 29, 2012

Over a century ago Sir William Osler, M.D., stated: “Variability is the law of life and as no two faces are the same, so no two bodies are alike and no two individuals react alike and behave alike under the abnormal conditions which we know as disease.” Despite our deep and long-standing understanding of the heterogeneity of disease and the variations in response to treatment, we are slow to adopt the notion that despite its complexity, the heterogeneity of human illness is decipherable. Skepticisms that we can actually deliver on the promise of personalized medicine is understandable, since converting such variables as severity of illness, uncertain vulnerability to side effects, co-morbid conditions and our cultural environment, to name a few, to precise algorithms for care seem daunting.

So, why has it taken so long for medical science to unravel this heterogeneity and why should we be optimistic that personalized medicine will happen? In part, our current views stems from where we have previously focused our attention. “Bergkrankheit “(mountain sickness) was known in the 14th century as an affliction of metal ore miners in Europe. By the 20th century we knew this as lung cancer and attributed it to a variety of environmental exposures. In this century, we have further refined our description of this disease to specific aberrations in molecular pathways, which if not entirely causative, account for much of the disease biology.

By contrast, medicinal chemistry as a science started much later than clinical medicine and is now closing the gap between knowing precisely what causes an illness to precisely what to do about it to improve the outcome for individual patients. No doubt we have a long way to go, but the current pace of personalized medicines suggests that it is becoming everyday reality for many lung cancer patients.

Join Dr. Stephen Eck on November 28, 2012, when he moderates a panel discussion at the 8th Annual Personalized Medicine Conference hosted by Partners HealthCare Center for Personalized Genetic Medicine, Harvard Medical School, and Harvard Business School. 

Adriana Jenkins and the Case for Personalized Medicine

December 12, 2011

Adriana Jenkins passed away on February 9, 2011 after battling breast cancer. Known for her tenacious advocacy on behalf of targeted therapies, Kelly Lindenboom, honored her passionate work within the personalized medicine community at the Boston Reception hosted by the Personalized Medicine Coalition on November 8, 2011.

Adriana Jenkins had an intoxicating personality and lived every day to the fullest — until, earlier this year, when she died from a rare form of breast cancer at age 41. Adriana was in the prime of her life — she had a thriving career in biotech public relations, was an incredible artist and had an extensive network of friends, that to Adriana, were her family. I’m part of her family.

At age 32, Adriana was engaged to be married, getting ready to start a new job with an up-and-coming biotechnology company and excited for what was to be the best time of her life. And then came the phone call from her doctor that changed everything. With a diagnosis of stage 3B inflammatory breast cancer — a rare, aggressive form of the disease — she had limited options to consider.

With less than a 50 percent chance of survival beyond five years, Adriana was desperate to explore any and all options that might give her better odds. Unlike the majority of people who are diagnosed with cancer every year, Adriana possessed a unique and intimate understanding of the pharmaceutical industry and was able to be her own best advocate after her diagnosis. When a colleague suggested she look into an investigational therapy — Herceptin — being tested locally at the Dana Farber Cancer Institute (DFCI) in Boston, she jumped at the chance. And as it turned out, she was a perfect fit for the trial and was enrolled in the study.

While the clinical trial was not always easy, Adriana responded wonderfully to the Herceptin, which was eventually approved by the Food and Drug Administration and is used today to treat many women with the same type of genetic marker, HER2, that was present in Adriana’s cancer.

Because of her success with Herceptin, one of the first so-called personalized medicines to be approved for use, Adriana was able to beat the odds. Really beat the odds. She credited personalized medicine treatment for giving her the nearly 10 years she was told she wouldn’t have. And she was thankful for every day that she had.

Unfortunately, despite encouraging results, personalized medicine is still a rarity in most cancer treatments. In her article “A Dying Wish,” published in Forbes magazine, written shortly before her death, Adriana made an eloquent appeal for the broad adoption of personalized medicines for cancer and other diseases based on her own incredible, nearly decade-long fight with breast cancer. In the article, she posed a question related to personalized medicine:

How do we convince drugmakers to focus their shrinking R&D budgets on this area of scientific discovery?

And then offered this potential challenge to our nation’s pharmaceutical companies and lawmakers:

“One idea is to create an incentive for drugmakers comparable to that in the Orphan Drug Act. Passed in 1983, it encourages companies to develop drugs for diseases that have a small market (fewer than 200,000 patients in the U.S.). Under the law, companies that develop such a drug may sell it without competition for seven years, in addition to often receiving quicker “fast track” regulatory review… A comparable law could push drugmakers to develop PM drugs for cancer and other deadly ailments.”

At the end of the article she offered this plea:

I urge patients, physicians and insurers to create a similar group to support the commercialization of personalized cancer drugs.

After her diagnosis with cancer, Adriana had the word “hope” tattooed onto the inside of her wrist as a constant reminder to herself for how she wanted to embrace life.

In the years that followed her remission, Adriana remained a strong advocate for the potential of personalized medicine, putting her public relations know-how to work by partnering with Herceptin’s developers to share her experience with the media and bring awareness to other women receiving a new cancer diagnosis.

The week that Adriana passed away, her article about the power and potential of personalized medicine was published in Forbes. And today, her friends are supporting her vision — keeping her “hope” alive — through the Adriana Jenkins Foundation for Personalized Medicine and a fundraising team with Stand Up To Cancer. Formed in Adriana’s name, the goal of the group is to raise awareness and be a proponent for development of personalized medicines, like the one that gave Adriana the nearly 10 years she never expected she would have.

Cancer is an extraordinarily complicated problem, and will only be solved through new approaches and ideas. Personalized medicine is one of them.

These remarks were first made by Ms. Lindenboom at the Personalized Medicine Coalition Boston Reception on November 8, 2011. They were also posted on HuffingtonPost.com.

Personalized Medicine: Celebrating Progress and Looking Ahead

September 26, 2011

The release of the American Association for Cancer Research (AACR) Cancer Progress Report last week, reminded me of the progress that has been made in cancer care since the passage of the National Cancer Act in 1971.  When President Nixon signed the law committing significant U.S. funds to cancer research, little was known about the disease and it was thought that a one-size-fits-all approach might eradicate cancer–what we know today is more than 200 different diseases; and that number will likely grow.  The personalized approach necessary to combat cancer was then not yet imagined, let alone understood.

But the progress in personalized cancer care, and in personalized medicine more broadly, has been recent.

For my September column in Personalized Medicine, I interviewed Harvard Medical School Professor of Genetics and Medicine Raju Kucherlapati, Ph.D., to discuss how personalized medicine has evolved since he launched Harvard’s annual personalized medicine meeting in 2005.  He explained that at first, business leaders and policymakers were skeptical that personalized medicine would have any impact in the near term, but that the many examples of personalized medicine products and new tools to speed innovation have changed minds and forever improved medicine.

No one better exemplifies progress in this field than President and Co-founder of the Institute for Systems Biology, Leroy Hood, M.D., Ph.D., who will be presented with PMC’s Award for Leadership in Personalized Medicine at this year’s Harvard conference. Dr. Hood envisioned the integration of scientific understanding into clinical practice by developing the first DNA and protein sequencer and synthesizer–laying the foundation for the development of personalized medicine.

I hope you will join me at this year’s Harvard conference, November 9-10, 2011, in Boston and at PMC’s cocktail reception to kick off the conference on November 8, 2011.  We will both celebrate the progress personalized medicine has made, thanks to the leadership of visionaries like Raju Kucherlapati and Leroy Hood; and discuss how we continue to move the paradigm of personalized medicine forward through scientific innovation, collaborative business partnerships, and supportive public policies.

For BioPharma Enterprise It’s Full Speed Ahead into the Age of Personalized Medicine

November 23, 2010

As more is understood about diseases and the why and how of their effects on people through advances in biomarkers and genomics, personalized medicine becomes a natural result for biomedical science and a natural trajectory for the innovation-based biopharma industry.   While the majority of those in attendance at last week’s Personalized Medicine: Impacting Healthcare  Conference are deeply immersed in driving this transformation, I was pleased to present the results of  a new Tufts CSDD Impact Report that provides the first look at a year-long effort by the Tufts Medical Schools Center for the Study of Drug Development (Tufts CSDD) to measure progress and prospects in the field by means of interviews and a survey of some 20 companies on the front lines of the movement towards personalized medicine. The company sample consisted of 9 top biotech companies with an average market value of $22B and 12 Big Pharma firms with an average market value of $93B.

While many recognized the challenges ahead, considering the current level of scientific knowledge, all believe that eventually a personalized medicine approach will help to streamline the R&D process, tip the benefit/risk ratio in favor of product approval, and provide options that help reduce overall costs and side effects. Confirming firms’ commitment to push ahead, companies increased their investment in personalized medicine by a median of 30% from 2006 to 2010 (even in a “down” economy), and plan to do so again from 2011 to 2015. Structural change follows the funding flow and all companies have experienced some level of “internal paradigm shifts” in order to integrate the concepts of personalized medicine into their development process, but not without disruptive change in the way decision-making occurs and at what levels.

Regulatory and reimbursement hurdles loom large among the most immediate challenges affecting the advancement of personalized medicines.  Thus, 100% of companies say their discovery strategy involves biomarkers and/or targeted therapies, but having a biomarker was not a requirement to move into clinical development. In fact, the percentage of the pipeline relying on biomarker data decreased as products move downstream, from 60% in preclinical to 50% in early clinical development, and finally 30% in late development. Oncology is much further along with genomic targeting of medicines, but  other therapeutic areas ahead of the curve include cardiovascular, central nervous system and immunologic therapies, while in metabolic, virology, and respiratory research,  personalized medicine is growing but incipient.

The dichotomy of where the industry is at present from where it needs to be is evident from the fact that while 90% of companies say they are investing in personalized medicine, only 10% of compounds in late clinical development actually have companion diagnostics. Nonetheless, the industry commitment in terms of funding, research collaborations, technology uptake, organizational change and unanimous resolve to push forward could signal that rapid advances are on the horizon.

 To read more or purchase the full Tufts Impact Report visit:  http://csdd.tufts.edu/reports/description/ir_summaries

Using Cancer(s) as a Model for Advancing Personalized Medicine

November 17, 2010

I am pleased to be attending this year’s Personalized Medicine Conference taking place this week and appreciate the opportunity to discuss why I, and many others, believe that cancer, or to be more accurate “cancers”, are an ideal model for the discovery, translation and delivery of personalized medicine.  Sheer numbers alone are reason enough to highlight the need for developing a personalized approach to cancer care: 1 out of 2 men and 1 out of 3 women will develop cancer in their lifetime in the United States.

The sequencing of the human genome has unraveled many mysteries as to how a normal cell can go awry and become cancerous. Further understanding of not only the genetics of cancer, but also the biology and metabolism of cancer, have increased our knowledge of biologic systems that support cancer progression, and this new knowledge has been translated into novel strategies for early detection, prevention and treatment.  And yet, these new discoveries that have heightened expectations of success have, in large part, fallen short in delivering cures anticipated by society.  The reality is that we have learned that cancer is actually an array of many diseases masquerading under the single name of “cancer.”   We need to learn to embrace the complexity of this disease we call cancer and stop the attitude of tunnel vision to cure cancer, and instead focus more on caring for the patient, the individual.  National policy must promote the search for solutions, not just cures.  Ultimately by providing solutions, we will reduce, and in many cases, eliminate death and suffering due to cancer.

In order to accelerate what is a continuum of discovery, translation and delivery of personalized medicine, or in this case personalized cancer care, multiple stakeholders must come together to pursue and deliver this common goal.  These stakeholders include researchers, clinicians, administrators (including policymakers and regulators), and of course, patients themselves.  This week’s gathering of the personalized medicine community in Boston is an example of the value of physically bringing together these stakeholders, and is an important forum for the discussion, exchange of ideas, and aligning of objectives that are crucial to the advancement of personalized medicine.

Total Cancer Care: An Approach to Creating Solutions for the Advancement of Cancer Research and Improved Care

Building on the themes of this year’s event – Personalized Medicine: Impacting Healthcare – I would like to describe to you one approach to personalized cancer care that is having a significant impact on the lives of patients. Nearly eight years ago, the Moffitt Cancer Center in Tampa, Fla., launched the Total Cancer Care™ initiative with the goal of identifying all the needs of a patient and developing a means to meet those needs.  By focusing on solutions to meet individual needs, we believed we could reduce death and suffering due to cancer, and that in order to do so we needed to develop strategic partnerships to perform the five following aims:

  1. Create a system to identify the needs of individual patients
  2. Identify markers that would predict needs and risks so that interventions could become preemptive
  3. Identify molecular signatures for patients who are not likely to respond to standard of care
  4. Utilize clinical characteristics and molecular profiling techniques to match the right patient to the right treatment at the right time and the right place
  5. Raise the standard of care for all patients by integrating new technologies in an evidenced based approach to maximize benefits and reduce costs

Critical to the pursuit of these solutions was the development of a large regional cancer biorepository in parallel with the development of a relational data warehouse and an information system containing patient’s clinical data and molecular data. 

We soon recognized that although Moffitt had a large patient population to study and engage in this endeavor, to accomplish our goal, we ultimately needed hundreds of thousands (if not millions) of patients to study, and we sought the advice and support of our Florida network of hospitals and physicians.  To our pleasure, there was universal enthusiasm from our statewide colleagues to participate in what became the Total Cancer Care™ Protocol.  Dr. Tim Yeatman at Moffitt was the original Principal Investigator of this IRB- approved protocol which enrolled its first patient in 2006 and basically asked for patients’ consent to do three things:

  1. Can we follow you throughout your lifetime by collecting and storing your clinical data and information?
  2. May we study any excess tumor or normal tissue using molecular profiling techniques?
  3. May we re-contact you?

What began at Moffitt within a year had been extended to eight different communities in Florida.  Within two years, this effort expanded to nine more communities in 10 different states, for a total of 18 participating sites.  Together, the 18 different sites form the Total Cancer Care™ Consortium with the aim of informing and consenting patients to the Total Cancer Care™ Protocol.  As of November 1, 63,754 patients have consented to the Total Cancer Care™ Protocol; 21,331 tumors have been collected and stored in a high technology biorepository located at Moffitt; and 15,093 tumors have been profiled using gene expression profiling technology.  To my knowledge, this effort makes Total Cancer Care™ one of the largest, if not the largest, prospective observational studies with tumor collection in the world. 

Ultimately, our community colleagues are not only contributors to establishing the foundation of personalized cancer care, but also the beneficiaries by being able to use the information system as a clinical decision tool, and as a means of quality performance and comparative effectiveness research.

In coming weeks, I am looking forward to further discussing the role of comparative effectiveness research in Total Cancer Care ™, and how the patient is not only a participant, but also the ultimate beneficiary of everything we do.

Personalized Medicine Coalition Award for Leadership Awarded Moffitt Cancer Center CEO

November 17, 2010

Today at the Personalized Medicine: Impacting Healthcare conference, the premier gathering of leaders in the personalized medicine field, the Personalized Medicine Coalition presented its annual award for Leadership in Personalized Medicine to Moffitt Cancer Center President, CEO and Center Director William Dalton, Ph.D., MD.

The award, which recognizes an individual whose contributions in science, business, and/or policy have helped advance the frontiers of personalized medicine, is presented annually at the personalized medicine conference sponsored by Partners HealthCare Center for Personalized Genetic Medicine, Harvard Medical School and Harvard Business School.

Giving this award to Dr. Dalton sends the message that personalized medicine is no longer a promise of the future. It currently has the capacity, especially in cancer, to ensure that the right patients receive the right treatments at the right time.  Under Dr. Dalton’s leadership, Moffitt is paving the way towards a new era of customizing treatments based upon individual characteristics.

Dr. Dalton is also receiving the award in recognition of his leadership in advancing public policies designed to support personalized medicine innovation and clinical application and for Moffitt’s pioneering work to implement personalized medicine concepts both to improve patient outcomes and lower costs.     

He is a leading advocate for health policies that support personalized medicine for the benefit of patients and currently serves as the President-Elect of the Association of American Cancer Institutes (AACI) and is Chair of the Science Policy & Legislative Affairs Committee of the American Association for Cancer Research. 

Dr. Dalton’s work is on the leading edge of personalized cancer care and patient-centered outcomes research. Moffitt’s Total Cancer CareTM is an approach to enhancing access to evidence-based, personalized cancer treatments and information/decision tools for patients and clinicians. Total Cancer CareTM is one of the largest cancer tumor bio-repositories and data warehouses in the United States dedicated for use in development of personalized medicine. 

As the recipient of the PMC award, Dr. Dalton joins a group of innovators well recognized for their leadership in personalized medicine. Previous recipients of the award include Dr. Janet Woodcock, Director of the Food and Drug Administration’s Center for Drug Evaluation and Research, Dr. Elizabeth G. Nabel, former Director of the National Heart, Lung and Blood Institute at the National Institutes of Health, Michael Leavitt, former Health and Human Services Secretary, Dr. Ralph Snyderman, Chancellor Emeritus of Duke University and founder of Proventys, Inc., and, most recently, Brook Byers of Kleiner Perkins Caufield & Byers.


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