Archive for the ‘targeted therapies’ Category

Angelina Jolie Lends Her Voice and Experience to Personalized Medicine

May 16, 2013

It was great to see Angelina Jolie’s eloquent op-ed in the May 14 edition of The New York Times. She carefully wove her personal experience with the importance of genetic testing and using the resulting information to make an informed health decision. I applaud her decision to go public with her story as a great example of patient empowerment but also as a powerful example of personalized medicine in action.

Despite all the advances we have made in recent years, we all know that the concept of personalized medicine is still foreign to most Americans. PMC recently conducted focus groups which strongly reminded us of the uphill battle that remains to gain public understanding of and support for personalized medicine. In our groups only one or two out of 52 participants were able to correctly describe the concept.

We found that people are very enthusiastic about personalized medicine once they understand it but it is a complex concept which is often misunderstood. It turns out that giving clear, specific examples of personalized diagnostics and treatments is the best way to explain what personalized medicine is and the benefits it can bring.

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.

Angelina Jolie’s op-ed is an important and high-profile contribution to the discourse about patient experience and personalized medicine.  Her voice echoes the words of Adriana Jenkins, who courageously shared her battle against breast cancer and joins other advocates like Donna Cryer, who works tirelessly to remind all stakeholders about how personalize medicine is a patient-centric approach to healthcare. Courageous individuals, like Ms. Jolie, who publicly share their personal stories have the power to bolster both public and media discourse around patient empowerment, for example with CNN “Early Start” co-anchor Zoraida Sambolin’s disclosure of her own breast cancer diagnosis and treatment decision live on air.

I hope that Ms. Jolie’s op-ed raises awareness of personalized medicine and the options patients have to take control of their healthcare.  No single organization alone can raise public awareness on the scale that we need but through a steady stream of individual op-eds, blog posts, conversations, etc., we in the personalized medicine community can spotlight more stories like this and begin to educate the public.  I look forward to hearing more voices from the community and hope that their contributions will support personalized medicine research, patient and provider engagement, and thoughtful policy.

Shaping the Future of Personalized Medicine with TEDMED

March 22, 2013

I had the opportunity recently to participate in a TEDMED Great Challenges panel discussion entitled, “Shaping the Future of Personalized Medicine.” My fellow panelists and Challenge Teammates included representatives from 23andMe, Foundation Medicine, Illumina, and InformedDNA.

As we discussed the challenges, opportunities and benefits of personalized medicine, three themes emerged.

First, personalized medicine, at its root, is about empowering patients to participate in their own healthcare. Trends in popular culture are meshing with advances in technology to allow consumers access to their health information and the ability to make informed decisions, and our culture is changing in that many now demand to be an involved party in their healthcare.

Amber Trivedi of InformedDNA noted that the power of personalized medicine lies not only in treatment, but prevention. As a genetic counselor, the most common questions asked by her patients are:  “What does my genetic information mean to me, and what will it mean to my children?” 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.

Second, as Michael Pellini, the CEO of Foundation Medicine said, data has to be “accurate and actionable” to have value. However, we cannot discount the potential future benefits of the data discoveries that are underway today. We must find a balance between supporting ongoing data discovery and analysis, while also pushing for data that are actionable now.

Third, traditional healthcare models need to continue to shift to aid in the advancement of personalized medicine. Research cannot continue to solely focus on large population studies; payers need to develop innovative approaches to improve reimbursement policies; and healthcare professionals need training and resources to enable the adoption of personalized medicine in the clinical world.

Finally, when asked what we found to be the most invigorating about personalized medicine, my fellow panelists were quick to share the advances they see on the horizon, including an explosion in targeted therapies aided by new technologies; the shift of cancer to a chronic disease; revolutionizing the treatment of infectious disease through the application of lessons learned from personalized medicine advances in cancer; and deeper data mining enabled by technology currently used in other fields.

Regardless of our individual areas of focus within the world of personalized medicine, this Challenge Team is energized and optimistic about the future of medicine.

Follow the Great Challenges conversation, and submit your questions or comments on Twitter using #GreatChallenges, or at TEDMED.

Progress in Cancer Highlighted by NEJM Retrospective; Turning the Tide Conference to Catalyze Comprehensive Dialogue on How to Sustain Cancer Innovation

June 8, 2012

In honor of the New England Journal of Medicine’s (NEJM’s) 200th Anniversary, the journal examined how medicine has evolved over the last two centuries, looking in particular at oncology diagnosis, prevention, and treatment. But while there has been tremendous progress in cancer, questions remain:  Where do we go from here?  And how do we get there in an era of immense fiscal discipline?  These are questions that we plan to address on Tuesday at our conference, Turning the Tide Against Cancer Through Sustained Medical Innovation.

In a similar vein to what Siddhartha Mukherjee, M.D., a special guest speaker at the conference, lays out in his book The Emperor of All Maladies: A Biography of Cancer, the authors of the NEJM article “Two Hundred Years of Cancer Research” provide a timeline of major discoveries and advances in cancer research and care.

They show how each milestone is built on the ones that came before it and trace the evolution of cancer progress from the early efforts to control the disease through surgery, advances in radiation, chemotherapy, and the targeted therapies that are redefining cancer treatment today.

The underlying science that made these treatment advances possible takes years to translate into clinical benefits for patients, but the original investments pay off. Our understanding of the genetic basis of cancer became possible only after decades of work on the basic biology of DNA beginning in the 1940s and 50s, but it was not until after the sequencing of the human genome that researchers were able to begin to translate genetics knowledge into new medicines.

Genetic understandings of cancer have led to breakthrough new medicines such as Xalkori® (for non-small cell lung cancer) and Zelboraf® (for melanoma) and more targeted therapies are on the way.  A new report issued by the Pharmaceutical Research and Manufacturers of America (PhRMA) found that there are 981 new medicines and vaccines for cancer in development today, many of which are likely to be personalized medicines.

Tomorrow’s progress in cancer therapies and treatment approaches depend on today’s policy makers recognizing the need for policies that holistically support cancer research and innovation.  In advance of next week’s conference, a Discussion Paper “Sustaining Progress Against Cancer in an Era of Cost Containment” coalesces the views of the conference advisory committee and other leading cancer experts about new models for cancer innovation, how to define value in cancer care, and how policy can support continued progress against cancer.

Levin, O’Kelly Provide Perspective on Decades of Personalized Medicine Progress, Urge Action and Support

May 22, 2012

On Tuesday, May 8, 2012, the Personalized Medicine Coalition (PMC) welcomed our chairman, Stafford O’Kelly, President of Abbott Molecular and keynote speaker, Mark Levin, Partner and Co-Founder of Third Rock Ventures to its Eighth Annual State of Personalized Medicine Luncheon. The event brought together PMC’s members, partners and other stakeholders involved in realizing the future of personalized medicine for researchers, industry leaders, patients, caregivers, advocates and policymakers.

Stafford O’Kelly reminded us, in his introductory remarks, of the progress and success we’ve made toward achieving personalized medicine in practice within the past year alone. Just last week we saw that Xalkori is showing progress in fighting certain childhood cancers. O’Kelly pointed out the assurances by the FDA that co-approval of drugs and diagnostics will occur more frequently. He also challenged attendees to think about what they can do “to accelerate the shift toward personalized medicine in a way that will improve treatment outcomes for the patient while, at the same time, lower overall costs for our health care system.”

Mr. Levin sought to use his significant experience and background in venture capital, product development and marketing to answer O’Kelly’s provoking question during his keynote address. He urged attendees to support personalized medicine. He explained that “personalized medicine is one of the most important things in medicine today” and lauded its potential to reduce safety challenges, increase efficacy, and improve productivity in the pharmaceutical industry.

I agree that we are at a point where the scientific and clinical progress made in personalized medicine is undeniable. But to echo Stafford O’Kelly’s call to action:  “We need to get the right stakeholders involved to help develop pathways for accelerating growth in the field….We need commitment from payers, providers and patients.”  In order to continue to make progress against disease and improve outcomes for patients, it is necessary as O’Kelly and Levin said, for the entire ecosystem of players to focus efforts in the direction that scientific discovery points us – toward targeted, patient-centric approaches to research and care.

We hope to continue the discussion around these themes and the future of innovation, specifically in cancer research and care at “Turning the Tide Against Cancer Through Medical Innovation,” a national conference that the Personalized Medicine Coalition, American Association for Cancer Research and Feinstein Kean Healthcare are co-hosting on June 12, 2012.  I encourage you to join us in Washington, D.C. as we look to identify and build support for an environment that sustains innovation and drives our evolution toward personalized cancer care.

Understanding the Complexities of Cancer and Progress in Personalized Medicine

March 14, 2012

Personalized cancer care holds much promise; it gives us confidence that targeted treatments will eliminate cancers and spare us the one-size-fits-all cancer treatments of our past. For the sake of simplicity, we want to think of personalized cancer care as a lock and key; each tumor has a specific puzzle interface, and when the puzzle piece is identified then the cancer melts. This simple message of “lock and key” is comforting because it suggests that we can achieve a new reality where we assess a tumor and choose a treatment in a very simple way, based on a tumor’s genetic makeup.

We have many exciting new targeted cancer treatments in our toolbox – consider, crizontinib (Xalkori®) for non-small cell lung cancer and vemurafenib (Zelboraf®) for BRAF mutation-positive metastatic melanoma. These therapies have been effective in patients with specific gene mutations. At the same time, as the science advances, research continues to reveal the underlying complexities of cancer and the diversity of tumors.

In the March 8 issue of The New England Journal of Medicine, the authors of “Intratumor Heterogeneity and Branched Evolution Revealed by Multiregion Sequencing” provide an exquisite roadmap of how heterogeneous and complex one individual’s cancer can be with many mutations lurking within each tumor mass, and the mutations themselves evolving over time. The picture presented by Gerlinger and colleagues, suggests the need for many keys to a myriad of locks, each tailored for a particular tumor at a particular point in time. The simple message of lock-and-key personalized medicine, where simple one-to-one decisions can be achieved, would not hold true.

This may create concern that a personalized medicine approach has created a false sense of hope. I don’t think that it is false hope, but rather a false sense of the simplicity.

There is hope. While a single needle biopsy may not create a complete picture of a cancer, biopsies are still a crucial part of characterizing tumors, using growing scientific knowledge to understand which treatments might work best. Biopsies are a starting point. A number of institutions are conducting genetic and genomic analyses on tumors. This is probably not the Holy Grail, but rather an iterative step in a long trail of scientific advances that will support personalization of cancer medicine.

In addition, new technologies are needed to improve on the information gathered, including methods to understand whole tumor heterogeneity. If we believe a personalized approach is simple, we are at risk of not building the systems needed to deal with the deluge of complex information that will need to be sorted, understood, and applied clinically in real time.

We need both the science and the information technology solutions to keep improving. New understandings highlight new opportunities to target more central cancer mutations, and to create more thoughtful treatment cocktails.

The underlying message here is that although we have made great progress, we are still early in our journey. In reality, this is a very comforting message. It would be disheartening if we had already exhausted the targeted treatment toolbox and that the best we could achieve is delayed cancer progression but not cures. By embracing the complexity of more individualized information for any particular patient and tumor, we really do have the hope of achieving personalized cancer care.

As we uncover new and unimagined pathways during the scientific discovery process, we should not be deterred or disheartened. We just aren’t there yet. We need to ensure that public policies support the continued investment of energy, attention to detail and resources required to achieve a vision for personalized cancer care.

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.

Our Health Policies Can’t Ignore Where Science Is Leading Us

September 27, 2011

Do we want continued progress in personalized medicine, or don’t we? Based on events last week, the answer might be ‘both.’ I attended a press conference here in Washington that celebrated the achievements made by the health sector in our efforts to turn cancer from a sure death sentence into a chronic disease. Simultaneous to this, the Medicare Payment Advisory Commission (MedPAC) proposed policies to offset the cost of a physician payment fix under Medicare. The disconnect between the two was striking. Unfortunately, the MedPAC proposals have the unintended effect of heavily impacting those on the leading edge of personalized medicine. Of the $233 billion in 10-year savings, $114 billion would come out of the biopharmaceutical and clinical laboratory sectors, thus possibly robbing seniors of access to the latest targeted therapies and the diagnostics used to guide them. The proposal includes savings from giving the Centers for Medicare and Medicaid Services (CMS) new authority to impose “least costly alternative” payment, which would result in locking us into our current one-size-fits-all medical paradigm and weakening physicians’ power to tailor care using diagnostics and targeted therapies.  Another troubling aspect of these suggestions is that they propose cuts by sector, ignoring the reality that personalized medicine brings efficiencies to the system of health care.

The proposal appears to reflect a lack of awareness of the impact that personalized medicine is making in health care and underscores the importance of the Personalized Medicine Coalition’s legislative work.  One of our policy suggestions, released over the summer, calls for inclusion of a personalized medicine representative on MedPAC and the creation of a new personalized medicine advisory committee to foster alignment of the policies across the entire Department of Health and Human Services with the science of personalized medicine. Personalized medicine challenges the status quo in medicine – the blockbuster business model, diagnostic coding and payment, or health care delivery systems. Maybe it’s time it challenged MedPAC, too.

For Cancer Therapies, It’s Getting Increasingly Personal

June 6, 2011

Last November, the Tufts Center for the Study of Drug Development reported that more than 90% of biopharmaceutical companies are investing in personalized medicine, with 12-50% of compounds in the drug development pipeline qualifying as personalized medicines. According to the report, personalized medicine is having an impact across disease areas, but is most evident in new therapeutic approaches to oncology.

The latest research reported at this year’s annual meeting of the American Society of Clinical Oncologists taking place over the weekend in Chicago reflects the same momentum and enthusiasm for the potential of targeted therapies in advancing the treatment of cancer. A front-page article by Ron Winslow in today’s Wall Street Journal, “Major Shift in War on Cancer,” explores some of the exciting personalized medicine therapies under development for cancer, highlighting new data presented at ASCO.

Winslow notes, “New research is signaling a major shift in how cancer drugs are developed and patients are treated—offering the promise of personalized therapies that reach patients faster and are more effective than other medicines.” The article nicely illustrates the promise of the science as well as some of the challenges facing researchers, companies and regulators.

This Thursday, PhRMA President and CEO John Castellani will continue the discussion of promising advances on the horizon for personalized medicine at the Personalized Medicine Coalition’s Seventh Annual State of Personalized Medicine Luncheon at the National Press Club. We hope to see some of you there. To register for the luncheon, visit: http://www.personalizedmedicinecoalition.org/events/State-of-PM.

Personalized Medicine Opens New Pathway to Get Needed Treatments to Patients

April 26, 2011

Should the Food and Drug Administration (FDA) provide post-Phase 1 study accelerated review of biomarker-linked cancer medicines? In a recent, provocative article in the New England Journal of Medicine, Bruce Chabner, M.D., argues that they should. Dr. Chabner, former director of the National Cancer Institute’s Division of Cancer Treatment argues that our growing ability to target therapies to patient subgroups based on molecular or genetic diagnostics necessitates a shift to earlier approval.

In his editorial, Early Accelerated Approval for Highly Targeted Cancer Drugs, Dr. Chabner, now at Massachusetts General Hospital Cancer Center, highlights research on two targeted therapies to illustrate how biomarkers can be used to match therapies to patients most likely to benefit and/or least likely to suffer harm from an experimental treatment:  PLX4032 (for patients with BRAF mutated melanoma) and crizotinib (for patients with EML4-ALK fusion in non-small cell lung cancer).

Both of these treatments were able to progress directly from Phase 1 to Phase 3 studies through use of validated biomarkers, Dr. Chabner says. But with the typical cancer drug taking seven years to complete clinical trials and gain FDA approval, the process is still “expensive and time-consuming, usually taking two to three years to reach survival end points.”  In addition, phase 3 trials still require a control group to receive the standard of care, depriving some patients of the experimental treatment with potentially better response rates and less toxicity.

While other accelerated paths provide patients access to promising therapies (i.e., compassionate-use protocols and accelerated review), if we are able to define populations of patients with high response rates in phase 1 trials, prolonging drug testing and approval raises important issues. A patient with an incurable disease and the right biomarker will want access to a new therapy. Dr. Chabner argues that the new age of molecularly-guided medicine is cause for an even more rapid path to approval: “Given trialists’ ability to define patient subgroups with responsive tumors in phase 1 trials, I propose that for diseases lacking therapies that meaningfully extend survival, the FDA should set flexible standards permitting accelerated approval of new drugs after phase 1.”

Dr. Chabner’s proposal illustrates the growing and likely dramatic impact that personalized medicine will have on research and development as well as regulatory and payment standards. It also offers a potential new pathway for addressing the serious challenge of how to get potentially life-saving medications to patients as expeditiously as possible while maintaining FDA’s gold standard for safety and efficacy. His suggestion reinforces the critical need for science and policy to work hand in hand in the advancement of personalized medicine. As the science leads the way in increasing our understanding of what therapies work best for which patients, regulatory policy will need to evolve as well.

The Age of Personalized Medicine Celebrates the 10th Anniversary of the Human Genome Project

June 9, 2010

On June 26, 2000, Francis Collins and J. Craig Venter announced the completion of the draft sequence of the human genome. Although it would be another three years until the project was complete, the event was nothing short of history-making, and has been foundational in the pursuit of personalized medicine.  This year marks the 10th anniversary of that landmark achievement, and indeed there is much to celebrate. And while remarkable achievements have ensued, the promised revolution in our understanding and approach to treating disease is still just beginning.

At the White House event where Dr. Collins and Dr. Venter made their announcement, then President Bill Clinton made bold predictions of its import. “Genome science will have a real impact on all our lives—and even more, on the lives of our children. It will revolutionize the diagnosis, prevention, and treatment of most, if not all, human diseases.”

Today, the list of targeted therapies and treatments is growing, and over 200 product labels recommend genetic testing or point to the influence of genetic variation on drug response or safety. All of these discoveries have been guided by an ever-increasing understanding of the molecular underpinnings of disease and the genetic predisposition of the patients they affect.

The Human Genome Project required an investment of over $3 billion and 13 years to complete. Today, enabled by innovation in high-speed sequencing technology, the $1000 genome appears to be within reach. With these advances, genomic sequencing has made its way out of the lab and into the hands of clinicians and consumers. Genetic tests are available to guide treatment decisions in the clinic for drugs like Selzentry® (maraviroc) for the treatment of HIV and Tarceva® (erlotinib) for the treatment of lung cancer, and direct-to-consumer genetic tests that can provide information about an individual’s predisposition for some health conditions are also on the rise. As these tests become increasingly available, we must work to ensure that they are scientifically grounded, and that they are supported by the appropriate policy and regulatory framework to govern them and protect patient interests.

These examples are the tip of the iceberg when it comes to the accomplishments, challenges, and opportunities that have succeeded the Human Genome Project.  In the coming weeks, leaders and visionaries from across the personalized medicine landscape will share their perspectives on the impact of the Human Genome Project, and how it continues to reverberate in our efforts to align policy, science, and clinical care to enable personalized medicine.

I invite you to join in the discussion. As the conversation unfolds this month, please share your thoughts about where the Human Genome Project has brought us, and the path that lies ahead.


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