by BioQuick Editor & Publisher Michael D. O'Neill (http://www.bioquicknews.com/node/34)
Shortly after 11 am EST on Friday, January 30, 2015, United States President Barack Obama announced his $215 million proposed “precision medicine initiative.” This is a massive personalized medicine effort centered on the use of next-generation DNA sequencing that Obama said represents the “greatest opportunity for medical breakthroughs that we have ever seen." It will enable physicians to administer the “right treatment at the right time, every time, to the right person." "The time is right to unleash new wave of medicine," Obama said. A jointly authored commentary on the proposed initiative, from NIH Director Dr. Francis Collins (M.D., Ph,D.) and National Cancer Institute (NCI) Director Dr. Harold Varmus (Ph.D), is posted on the New England Journal of Medicine web site (http://www.nejm.org/doi/full/10.1056/NEJMp1500523). Please also see links to related popular press articles provided below. Dr. Collins is a former Director of the National Human Genome Research Institute, leader of the Human Genome Project, and longtime physician-scientist-researcher, whose numerous seminal discoveries include identification of the gene for cystic fibrosis. Dr. Varmus is co-winner of the 1989 Nobel Prize for Physiology or Medicine for discovery of the cellular origin of retroviral oncogenes. Dr. Varmus is also a former President of Memorial Sloan-Kettering Caner Center (MSKCC) and former Director the NIH.
As an immensely moving example of the power of precision medicine, President Obama was introduced by 19-year-old Harvard student and recently published scientiest Elana Simon (Elana's story: http://newswire.rockefeller.edu/after-rare-cancer-diagnosis-teen-works-t...)(More of Elana's story: http://www.gastrojournal.org/pb/assets/raw/Health%20Advance/journals/yga...)
At the tender age of 12, Elan had received the devastating diagnosis of a rare and often deadly type of genetic liver cancer called fibrolamellar hepatocellular carcinoma.
Elana, the daughter of a biophysicist at The Rockefeller University, underwent surgery to remove much of her liver and this surgery left her cancer-free. Incredibly, however, the teenager then went on, with colleagues including her father, to conduct research using next-generation DNA sequencing techniques to identify the precise mutation that caused her own cancer, as well as those of others with her same rare genetic liver cancer.
Elana's research achievement of identifying the specific DNA mutation causing fibrolamellar hepatocellular carcinom was published on February 28, 2014 in the journal Science(httpp://www.sciencemag.org/content/343/6174/1010) when she was just 18.
Elana is currently pursuing further research on this rare genetic cancer gene at Harvard.
OBAMA THANKS ELANA & STARTS TO DESCRIBE HIS PROPOSED $215 MILLION "PRECISION MEDICINE INITIATIVE"
No longer, do we want “accidents of birth determining our fate,” President Obama said. Such accidents, as genetic mutations “are not our destiny; we can remake it,” he said. “That is the power of scientific discovery. We now have the chance to remake our world continuously, offering great promise for future generations.”
The president will include this proposed $215 million initiative in the budget he will be submitting to Congress on Monday, February 2, 2015, and he believes it will garner strong bipartisan support.
In his Friday address, President Obama cited four key features of the initiative: to work closely with the NCI to develop new and effective treatments for cancer; to have the FDA evaluate next-generation DNA sequencing tests in different ways than older technologies have been evaluated; to have the NIH assemble a large group of volunteers who can provide a significant amount of informative data on the DNA bases of health and of disease; and finally, to work to protect patient privacy from day 1.
NIH director Francis Collins was in the audience at the White House and he was among many science and medicine luminaries introduced by the President.
President Obama reminded everyone that Dr. Collins had previously been the director of the Human Genome Project (HGP), and Obama further noted that, so far, $140 has been yielded back to the U.S. economy for each $1 dollar invested in the HGP.
Also in the distinguished audience at the White House announcement were Steven M. Altschuler, M.D., CEO of the Children's Hospital of Philadelphia (CHOP), and 9-year-old patient Emily Whitehead, who is now cancer-free thanks to a personalized treatment called T cell therapy, developed at CHOP
“I am thrilled that President Obama recognizes the promise of our nation’s research efforts by making this investment. Today’s announcement signifies his commitment to bringing this issue to the forefront of our national agenda,” said Dr. Altschuler.
He continued, “As a world leader in precision medicine, The Children’s Hospital of Philadelphia has made significant investment in its Center for Applied Genomics with the goal of diagnosing and curing pediatric disorders. We have already made strides harnessing this technology to better understand and treat cancer, autism, asthma, diabetes, and many other childhood illnesses. Emily Whitehead’s personal story is a testament to the real impact this groundbreaking research can make in saving lives.”
President Obama emphasized that the rapid advance of next-generation technologies and the ever-declining costs of these technologies are strong drivers behind the advance of "precision" or "personalized" medicine. He noted that when the human genome was first sequenced, the overall cost of the effor to completely sequence just one human genome was approximately $3 billion. Today, a single human genome can be sequenced for $1,000 to $2,000.
BIL ELDER, A 27-YEAR-OLD CYSTIC FIBROSIS VICTIM, NOW BELIEVES, FOR THE FIRST TIME IN HIS LIFE, THAT HE WILL LIVE TO SEE HIS GRAND-CHILDREN
President Obama closed his address by introducing 27-year-old Bill Elder to the audience. Mr. Elder was first seen by many when he attended the 2015 State of the Union address as the special guest of Michelle Obama.
Mr. Elder had been diagnosed with cystic fibrosis some 20 years ago. He was determined to be in the 4% of cystic fibrosis patients whose disease is caused by a particular specific mutation in the cystic fibrosis gene. The FDA fast-tracked a drug targeted at this specific mutation and, in 2012, Mr. Elder took that drug for the first time.
When he woke up the next morning, he reported an entirely novel experience. He was able, for the first time in his life, to breathe out of his nose. Now 27 (at one time the median age of survival for cystic fibrosis patients), Mr. Elder is in his third year of medical school.
And now, for the very first time in his life, Mr. Elder believes he may live long enough to be a grandfather.
That, President Obama suggested, is the power of precision medicine.
SELECTED EXCERPTS & QUOTES FROM WASHINGON POST & WHITE HOUSE RELEASES & THE WHITE HOUSE'S DR. JO HANDELSMAN
In reporting on this major development, the Washington Post quoted Dr. Collins, as saying, “This research will dramatically advance our knowledge of diseases, how they originated, and how we may prevent or treat them.”
The Washington Post also noted the following commentary from Jo Handelsman, Ph.D., Associate Director for Science at the White House Office of Science and Technology Policy. First, let us provide some background on Dr. Handelsman.
Before coming to the White House in June 2014, Dr. Handelsman was the Howard Hughes Medical Institute Professor and Frederick Phineas Rose Professor in the Department of Molecular, Cellular, and Developmental Biology at Yale University. She had previously served on the University of Wisconsin-Madison faculty as a Professor in Plant Pathology from 1985 to 2009, and as Professor and Chair of the UW-Madison Department of Bacteriology from 2007 to 2009.
In 2013, Dr. Handelsman served as President of the American Society for Microbiology. From 2002 to 2010, she was the co-founder and co-director of the Wisconsin Program for Scientific Teaching, the Yale Center for Scientific Teaching, and the National Academies Summer Institute on Undergraduate Education in Biology, programs focused on teaching principles and practices of evidence-based education to current and future faculty at colleges and universities nationwide.
In 2011, Dr. Handelsman received the Presidential Award for Excellence in Science, Mathematics, and Engineering Mentoring from President Obama. (http://news.yale.edu/2011/01/21/yale-s-jo-handelsman-receives-presidenti...)
Dr. Handelsman received a B.S. from Cornell University and a Ph.D. in Molecular Biology from the University of Wisconsin-Madison.
“It [the Precision Medicine Initiative] holds the potential to revolutionize the way we approach health in this country and, ultimately, around the world. Precision medicine is about moving beyond [the] one-size-fits-all approach to medicine and, instead, taking into account people’s genes, their microbiomes, their environments, and their lifestyles.”
President Obama noted the precision medicine initiative in his 2015 State of the Union address to Congress on January 20, 2015. A release from the White House at that time and prepared by Dr. Handelsman, read, in part:
”Last night, at his 2015 State of the Union Address, President Obama announced that he is launching a new precision medicine initiative that will help deliver the right treatment to the right patient at the right time. Many of you may be wondering: What exactly is 'precision medicine,' and how can it transform medicine as it is practiced today?"
"Today, most medical treatments have been designed for the “average patient.” In too many cases, this “one-size-fits-all” approach isn’t effective, as treatments can be very successful for some patients, but not for others. Precision medicine is an emerging approach to promoting health and treating disease that takes into account individual differences in people’s genes, environments, and lifestyles, making it possible to design highly effective, targeted treatments for cancer and other diseases. In short, precision medicine gives clinicians new tools, knowledge, and therapies to select which treatments will work best for which patients."
"Cancer treatment is also entering a new era as a result of precision medicine. Patients with breast, lung, and colorectal cancers, as well as melanoma, are now routinely undergoing DNA testing as part of their care, enabling their physicians to select treatments based on this information that improve their chances of survival and reduce their exposure to adverse effects."
"Precision medicine is not just about genomics. Health and disease are influenced by many factors. Precision medicine aims to also leverage advances in medical imaging, such as MRI and 3D X-ray technologies, and utilize advances in health information technology, as well as other fields, to better understand each of these factors and to apply this knowledge in the development of new treatments. The potential for precision medicine to improve care and produce new treatments has only begun to be tapped. Translating initial successes to a larger scale will require a coordinated and sustained national effort."
"The time to begin that effort is now. We look forward to announcing more details soon."
COMMENTS FROM LEADING SCIENTISTS & PHYSICIAN-SCIENTISTS
DR. SHINYA YAMANAKA, 2012 NOBEL PRIZE WINNER
Shinya Yamanaka, M.D., Ph.D., shared, with the UK's Sir John B. Gurdon, the 2012 Nobel Prize for Physiology or Medicine for demonstrating that mature cells can be reprogrammed to become pluripotent.
Born in Osaka, Japan, Shinya Yamanaka received his M.D. degree from Kobe University and his Ph.D., in Pharmacology, from Osaka City University Graduate School of Medicine, under the direct mentorship of Katsuyuki Miura in the laboratory of Kenjiro Yamamoto. Dr. Yamanaka's post-doctoral work was done at the Gladstone institutes of Cardiovascular Diseases in San Francisco, California, under the tutelage of Thomas Innerarity, Ph.D., a senior investigator focused on cardiovascular disease.
Dr. Yamanaka has descrebed his decision to work at Gladstone "as one of the best decisions I ever made in my life. Gladstone provided an almost perfect environment for an ambitious new researcher like me, thanks to its skillful technicians and the provocative discussions I had about science with colleagues."
Later, he returned to Japan and, in 1999, became an Assistant Professor with his own laboratory at the Nara Institute of Science and Technology (NAIST) in Nara Prefecture. in 2004, he moved to the Institute of Frontier Medical Sciences at Kyoto University as a Professor.
In 2006, he and colleagues published in Cell, an article describing the successful generation of iPS cells in mice using four factors. In November 2007, Dr. Yamanaka's group reported the generation of human iPS cells from human fibroblasts by introducing the same four genes used in 2006, via viral vectors. This work was recognized in 2012 with the awarding of the Nobel Prize.
Currently, Dr. Yamanaka holds dual appointments as Senior Investigator at the Gladstone Institutes in San Francisco and Director and Professor at the Center for iPS Cell Research and Application (CiRA), at Kyoto University in Japan.
Dr. Yamanaka was kind enough to provide his comments on President Obama's $215 million proposed "precision medicine initiative to BioQuick Online News. The Gladstone Institutes' Communications and Media Relations Specialist Dana Smith was instrumental in obtaining Dr. Yamanaka's comments and having them transmitted in a timely fashion to BioQuick Online News.
“Most treatments to combat diseases are based on studies using a small, but statistically relevant, number of patients. For this reason, while treatments will work for many patients, there is always a select group that will not respond. Because they come from the patient, induced pluripotent stem cells (iPS cells) are an excellent model to study drug and other disease treatments for that individual," Dr. Yamanaka said.
"As such, I am very happy to see that in a time of budget constraints around the world, the U.S. government is committed to this Precision Medicine Initiative and hope other countries will follow. Combining the research findings gained in the initiative with iPS cell technology will help researchers better understand mechanisms of a variety of diseases and accelerate drug discovery to cure many intractable diseases.”
DR. MARY-CLAIRE KING
Legendary human geneticist Mary-Claire King, Ph.D., was kind enough to provide BioQuick News with some of her thoughts on President Obama's proposed initiative. Dr. King has carried out seminal research on the identification of the BRCA1 and BRCA2 genes for breast cancer and ovarian cancer. She has also studied the genetics of hearing loss and deafness. Dr. King is currently Professor of Genome Sciences and Medical Genetics at the University of Washington in Seattle.
Dr. King is an acknowledged giant in human genetics. In 2014, her body of work was recognized by the awarding of the Albert Lasker Award (Special Achievements), often a prelude to the Nobel Prize, and also the HudsonAlpha Life Sciences Prize. In 2013, she received the Paul Ehrlich and Ludwig-Darmstaedt Prize. She is a past President of the American Society of Human Genetics (ASHG).
In addition to her work on cancer genetics and the genetics of deafness and hearing loss, Dr. King is also recognized for demonstrating that humans and chimpanzees are 99% genetically identical and for applying genomic sequencing to the identification of victims of human rights abuses. In Argentina, for example, in 1984, Dr. King began working with Abuelas de Plaza de Mayo (Grandmothers of Plaza de Mayo) in identifying children who had been stolen from their families and adopted illegally under the military dictatorship during the so-called “Dirty War” (1976-1983).
In communicating with BioQuick News regarding Obama's proposed $215 million initiative, Dr. King said the following.
“President Obama's precision medicine initiative offers us the opportunity to put the results of the human genome project to work for patients. For example, it is now possible, by applying an intelligent combination of knowledge and technology, to identify women at very high genetic risk of breast and ovarian cancer. With this information, women can take action that decreases those risks. The limiting factor to preventing these inherited cancers and saving women's lives is resources to put into practice what we know. The President's precision medicine initiative is a positive step in providing those resources.”
DR. GEORGE CHURCH
World-renowned DNA technology wizard George Church, Ph.D., also had comments for BioQuick News regarding President Obama’s proposed “precision medicine initiative.” But first, let’s provide some background indicating the expertise that Dr. Church brings to his comments.
Currently, Dr. Church is Professor of Genetics at Harvard Medical School, and Professor of Health Sciences and Technology at Harvard and MIT. He is also Director of the Personal Genome Project at Harvard (http://www.personalgenomics.org), which provides the world's only open-access information on human genomic, environmental, and trait data (GET)(http://www.getconference.org).
The Personal Genome Project (PGP) was launched in 2005 and is dedicated to creating public genome, health, and trait data. Participants in the PGP believe that sharing data is critical to scientific progress, but has been hampered by traditional research practices—the approach of the PGP is to invite willing participants to publicly share their personal data for the greater good.
In addition his work for the PGP, Dr. Church is also Director of the NIH Center for Excellence in Genomic Science (http://ccv.med.harvard.edu/).
Over the years, Dr. Church has achieved numerous spectacular innovations that have contributed to nearly all "next-generation" genome sequencing methods and companies (CGI, Life Technologies, Illumina, Nanopore).
These efforts, plus chip-based DNA synthesis and stem cell engineering have resulted in the founding of additional application-based companies spanning the fields of medical diagnostics (Knome, Alacris, AbVitro, Pathogenica) and synthetic biology/therapeutics (Joule, Gen9, Editas, Egenesis, enVolv, WarpDrive).
Dr. Church’s 1984 Harvard Ph.D. work included his development of the first methods for direct genome sequencing, for molecular multiplexing, and for DNA barcoding. These methods led to the achievement of the first genome sequence (that of the pathogen Helicobacter pylori) in 1994.
Clearly, Dr. Church’s comments on Obama’s proposed initiative carry significant weight. These comments, made to BioQuick News, are provided below.
“It's great to see, in the presidential briefing, the words ‘new way of doing research through engaged participants and open, responsible data sharing,’" Dr. Church noted.
“At the PGP, we have one of the few proven consenting methods compatible with such open access to large, individual, precision medicine datasets. The PGP achieves this via focused learning and testing on open sharing (rather than by merely signing ten pages of legalese that protects mainly the institution, not the patient, and disingenuously implies lack of data escape and re-identification).”
“After a wide search, the NIST-FDA, for its Genomeinabottle (GIAB) reference material, came to the conclusion that, of the various genome and cell resources in the world, only the PGP participants were adequately consented for long-term use as reference materials. The first six Coriell cell lines (two trios) are listed here (http://www.sites.stanford.edu/abms/content/want-help-characterize-first-...).
Dr. Church did note that not everyone is gung-ho on the Obama initiative.
He remarked that an anonymous colleague had stated the following: "It's clear that the cancer-allocated money is a bit of a lost cause, at least with respect to engaged participation (continuation of The Cancer Genome Atlas [TCGA] without many changes)."
DR. JAN LOTVALL
Expressing some reservations about the Obama initiative was clinical allergist Jan Lotvall (http://www.gu.se/english/about_the_university/staff/?languageId=100001&u...), M.D., Ph.D., Professor at the University of Gothenberg in Sweden and President of the International Society for Extracellular Vesicles (ISEV) (http://www.isev.org/). Dr. Lotvall is also the Director of the Krefting Research Centre (http://krefting.gu.se/KRC) at the University of Gothenburg and does much work on translational research, chiefly in the field of asthma.
One OF Dr. Lotvall’s most recently published articles is “MicroRNA in Exosomes Isolated Directly from the Liver Circulation in Patients with Metastatic Uveal Melanoma”(http://www.biomedcentral.com/1471-2407/14/962).
Another is “Aspirin Intolerant Asthma in the Population: Prevalence and Important Determinants,” (“http://www.ncbi.nlm.nih.gov/pubmed/24961377).
Dr Lotvall is a world-renowned expert on “exosomes,” small sub-cellular information-carrying vesicles that are produced and secreted by cells throughout the body and are believed to offer enormous clinical potential in a wide variety of areas, particularly as tools for enabling non-invasive “liquid biopsies.” Dr. Lotvall’s comments on President Obama's proposed precision medicine initiative, as provided to BioQuick News, are presented below.
“I have mixed feelings about the 'precision medicine initiative' that was proposed by President Barack Obama two weeks ago. Specifically, I am skeptical that sequencing a million genomes will help the development of precision medication.
"Understanding the diversity in the genome may be an interesting question in its own right, but the last twenty years of genome research have shown that very few diseases are directly connected to specific sequences or “single-nucleotide polymorphisms” in our DNA."
"To develop novel precision medicines, we rather need to understand the diversity of specific diseases, including asthma, cancer, diabetes, cardiovascular diseases, neurological diseases, and inflammatory diseases, which scientists today call 'disease phenotypes' or 'endotypes.'
"By investing in understanding the molecular mechanisms involved in subgroups of disease, more personalized and efficient precision medications can be developed for future generations. Genetics will not be the answer, but more comprehensive and translational approaches are necessary, first describing molecular mechanisms of subgroups of disease, thereafter developing treatments targeting those specific mechanisms. That would be a true precision medicine investment.”
It should be noted that this year's annual meeting of the International Society for Extracellular Vesiccles will take place in the Washington, D.C. area (Bethesda, Maryland) from April 23-26, 2015 (http://www.isev.org/).
DR. MUIN KHOURY
Many of the above comments, excluding Dr. Lotvall's, focus on the envisioned salutary effects of precision medicine and the great health and economic benefits it promises.
But perhaps a more realistic and sobering assessment of the current and near-future state of precision medicine is provided by Muin Khoury, M.D., Ph.D., Director, Office of Public Health Genomics, Centers for Disease Control and Prevention (CDC) in Atlanta, Georgia, in his January 29, 2015 Genomics and Health blog, entitled “The Success of Precision Medicine Requires a Public Health Prespective” (http://blogs.cdc.gov/genomics/2015/01/29/precision-medicine/.
That entire blog is quoted below, with Dr. Khoury’s permission, because BioQuick News believes that it conveys very important thoughts on the present state and the future of precision medicine, thoughts that are particularly relevant to anyone contemplating President Obama’s recently proposed $215 million “precision medicine initiative.”
Before quoting the blog, however, let us give some background on Dr. Khoury.
Dr. Khoury received his B.S. degree in Biology/Chemistry from the American University of Beirut, Lebanon, and his medical degree and Pediatrics training from the same institution. He received a Ph.D. in Human Genetics/Genetic Epidemiology and training in Medical Genetics from The Johns Hopkins University. Dr. Khoury is board-certified in Medical Genetics. In 2005, Dr. Khoury received the National Cancer Institute (NCI) visiting scholar award for leadership and vision in genetic epidemiology and public health.
Dr. Khoury has published extensively in the fields of genetic epidemiology and public health genetics. He has over 500 scientific publications including articles, books, and book chapters.
In 1993, he published a textbook entitled: "Fundamentals of Genetic Epidemiology." In 2000, he was the lead editor for the book entitled: "Genetics and Public Health in the 21st Century: Using Genetic Information to Improve Health and Prevent Disease." His 2004 book entitled "Human Genome Epidemiology" illustrates the applications of epidemiologic methods and approaches to the continuum of genomic information from research to practice. In 2010, he published a completely updated second edition of "Human Genome Epidemiology"
Dr. Khoury is also an Adjunct Professor in the Departments of Epidemiology and Environmental and Occupational Health at Emory University Rollins School of Public Health and an Associate in the Department of Epidemiology at Johns Hopkins University Bloomberg School of Public Health.
Clearly, Dr. Khoury is eminently qualified to comment on the present and the future of precision medicine, and, when contacted by BioQuick News, Dr. Khoury referred us to his aforementioned Genomics and Health blog, entitled “The Success of Precision Medicine Requires a Public Health Prespective” (http://blogs.cdc.gov/genomics/2015/01/29/precision-medicine/) .
Dr. Khoury’s blog post is presented in its entirety below.
“The announcement of a new major US Precision Medicine initiativeExternal Web Site Icon (http://www.whitehouse.gov/blog/2015/01/21/precision-medicine-improving-h...) comes more than a decade after the completion of the Human Genome Project (http://www.nature.com/news/specials/humangenome/index.html), the ambitious project that culminated in sequencing all 3 billion base pairs of our genome. Continuous improvement in the quality of sequencing, dramatic reduction in price, and ongoing advances in multiple sectors of biotechnology all promise a new era of medicine known variably as personalized medicine, genomic medicine, and, more recently, precision medicine (http://www.plengegen.com/blog/personalized-medicine-precision-medicine/)."
“With conventional medicine, patients are treated individually, but typically with the same treatment that everyone else with that condition receives. Thus an opportunity may be missed: certain medical interventions can be more effective or cause fewer side effects for some patients than for others, making it important to identify in advance which patients are more or less likely to benefit from the intervention. This is where precision medicine comes in.”
“Precision medicine takes into account individual differences in the genes, environments, and lifestyles of people allowing the design of targeted disease interventions from the start. While genomics is often suggested as the leading driver of personalization, other factors may be equally as important. For example, health information technology can be used to integrate medical history into patient-centered approaches to improving health and treating disease.”
“As paradoxical as it may seem, while precision medicine focuses on individualized care for each patient, its success truly requires a population-based perspective.“
“First, it is important to learn what works and what does not for one person, but it is impossible to infer causality by working with one person at a time. To be informative, data on an individual need to be compared with data from large numbers of people to recognize important individual characteristics and to identify relevant population subgroups that are likely to respond differently to drugs and other interventions.”
“Second, collecting information from large numbers of people is far more informative when these people are representative of the underlying population from which individuals are drawn. Using data from convenience samples—i.e., collected without regard to important factors such as race/ethnicity, age, and sex–can lead to substantial selection bias and unreliable disease prediction models. A strong epidemiologic foundation is needed to interpret genomics and other “big data” for applications to healthcare.”
“Third, while precision medicine is currently focused on treatment, a compelling case can be made for giving even more attention to early detection and disease prevention. Although personalized treatments can help save the lives of people who are already sick, disease prevention applies to all of us. 'Precision prevention' (http://www.ncbi.nlm.nih.gov/pubmed/?term=25362191)
then may be useful in using both science and limited resources for targeting prevention strategies to subsets of the population. “
“For example, recent data suggest that knowing the speed with which some people metabolize nicotine, based on genetic and other factors, could lead to personalized smoking cessation interventions (http://www.cdc.gov/genomics/public/features/smoking.htm) to complement the highly successful public health efforts that have resulted in reduction in smoking over the past few decades. Another approach to precision prevention is increased screening of people at greater risk of cancer. Family health history collection is an inexpensive tool for identifying individuals and families that require earlier and more intensive screening for breast and ovarian cancer (http://www.cdc.gov/genomics/resources/diseases/breast_ovarian_cancer.htm) or colorectal cancer (http://www.cdc.gov/genomics/resources/diseases/colorectal.htm).”
“Finally, implementation of precision medicine requires the full participation and education of patients (all of us), communities, physicians, payers, and the healthcare community. This should be guided by strong “translational” implementation sciences which go beyond the traditional bench to bedside model [see recent paper on this topic (http://www.sciencedirect.com/science/article/pii/S2212066114000313].”
“Society has a stake in assuring that the national investment in precision medicine research leads to tangible health benefits for all and does not worsen existing health disparities. This is where strong public health-healthcare partnerships (http://blogs.cdc.gov/genomics/2014/08/21/is-genomics-widening/) are key in assessing the needs of individuals and communities, developing appropriate policies and guidelines, ensuring that all people have access to the intended benefits of technology, and tracking effectiveness and cost effectiveness outcomes in the real world.”
“An often used example of early success in precision medicine is targeted therapy for a small subset of patients affected by cystic fibrosis, (http://www.cdc.gov/genomics/public/features/cystic_fibrosis.htm), a common genetic disorder that leads to premature disease and disability. However, the price tag of the drug (Kalydeco) can be around $300,000 per year per patient( http://www.medpagetoday.com/Pulmonology/CysticFibrosis/42018). Economic considerations can have major implications for differential access to such treatment for families, communities, and society at large.”
“These are the early days of precision medicine. The road ahead is long (http://www.plengegen.com/blog/letter-president-obama-precision-medicine/).”
“Let us make sure that a public health perspective is included at the outset to ensure the success of research and ultimately the effective and responsible implementation of new scientific discoveries for the benefit of all.”
INDUSTRY--PATRICE MILOS (CLARITAS), JAY FLATLEY (ILLUMINA), JAMES JOYCE (AETHLON)
Comments from the biotech industry included the following: “We are thrilled to see President Obama and NIH Director Collins further advancing our country’s healthcare system with their bold new Precision Medicine Initiative. It is fully aligned with our shared goals of bringing genomic information to patients and families,” said Patrice Milos, President and CEO of Claritas Genomics (http://www.ClaritasGenomics.com) in Cambridge, Massachusetts.
Jay Flatley, CEO of Illumina, Inc., a leading manufacturer of next-gen DNA sequencing technology, was one of the invited guests at the Obama announcement, and Flatley said this:
“Illumina is encouraged by the Obama Administration’s proposed precision medicine initiative that will use personalized genetic information to help treat diseases like cancer and diabetes. With the cost of sequencing an entire human genome recently lowered to $1,000, advances in genomic medicine are now more accessible. We applaud an endeavor of this magnitude and recognize this is a significant step toward realizing the full potential of unlocking the power of the genome to improve public health and wellness.”
James A. Joyce is Founder, Chairman, and CEO, of Aethlon Medical (http://www.aethlonmedical.com/). Under Mr. Joyce’s leadership, Aethlon has transformed the concept of a selective therapeutic filtration device (The Aethlon Hemopurifier®) into the reality of treating both HIV and hepatitis C patients in a clinical setting. Follow-on research has further validated the ability of the Hemopurifier® to capture a broad-spectrum of bioterror and pandemic threats, as well as immunosuppressive cancer exosomes.
Mr. Joyce has testified before Congress on issues related to Project BioShield legislation and the deployment of the Aethlon Hemopurifier® as a countermeasure against biological weapons. His efforts on Capital Hill were instrumental in expanding the definition of treatment countermeasure in Project BioShield legislation to include medical devices.
In May 2011, the Company introduced the Aethlon ADAPT™ system to advance Mr. Joyce’s vision of an expansive device platform that converges affinity drug agents with plasma membrane technology to create new candidate therapies against life-threatening disease conditions.
In addition to his impressive biotech career, Mr. Joyce played foot1ba1ll for the Denver Broncos professional football team in the NFL. He graduated from the University of Maryland.
Below are Mr. Joyce’s brief comments on the Obama proposed $215 million precision medicine, as provided to BioQuick Online News.
"The premise underlying the President's proposed precision initiative is right on target. However, it seems rather naive to believe that $215 million would fuel meaningful results. As an example, consider that our government has allocated almost $50 billion on traditional biodefense countermeasures since 2001, yet we remain naked against a vast majority of the bioterror and pandemic viruses that pose the greatest threat to mankind."
GENETIC ALLIANCE--SHARON TERRY
Comments from genetic disease organizations were similarly enthusiastic about the Obama proposal. Genetic Alliance President & CEO Sharon Terry was present for the announcement and noted afterwards that “This is what the Genetic Alliance has been working towards for years. I am delighted by the President’s recognition of the power of participants in this initiative. Together with other advocates, citizen scientists, and research participants, we are thrilled to roll up our sleeves and get started!”
The Genetic Alliance (http://www.geneticalliance.org/) is one of the world’s leading non-profit health advocacy organizations. Its network includes more than 1,200 disease-specific advocacy organizations, as well as thousands of universities, private companies, government agencies, and public policy organizations. The network is a dynamic and growing open space for shared resources, creative tools, and innovative programs.
Since 1986, the Genetic Alliance has defined the intersection of health and genetics. Originally founded as an alliance for support groups, the Genetic Alliance's work has evolved along with the growing health advocacy movement and the rapid advancement of genetic technology. While the organization's roots are in genetics, it recognizes that meaningful solutions that improve health must be multidisciplinary. To that end, the Genetic Alliance applies solutions in health and disease, practice and prevention, and on the local and global level. Through its broad and diverse partnerships, the Genetic Alliance has created a dynamic network that serves as an open space for sharing valuable resources, creative tools, and innovative programs.
Michael Goldman (firstname.lastname@example.org), Ph.D., commented on the precision medicine initiative from the academic / research perspective. Dr. Goldman is Professor & Chair, Biology Department (http://biology.sfsu.edu/), San Francisco State University. Not only that, but since 2008, Dr. Goldman hss organized and run a highly successful annual conference on personalized medicine (http://personalizedmedicine.sfsu.edu/pastConf.html).This year’s conference (Personalized Medicine 8.0) has the provisional title, “Genetic Screening & Predictive Medicine,” and is scheduled for Thursday, May 28, 2015, (8 am to 6 pm) at the South San Francisco Conference Center.
“One of the most intriguing aspects of personalized or precision medicine is the ability to predict disease before symptoms even begin. At its most rudimentary, that means assigning individuals a risk for future disease based on genetics (DNA sequence) or epigenetics (DNA sequence with modifications such as methylation), then targeting preventive measures at the people who need them most. We might also target the specific interventions that will be most effective in the individual—broccoli, red wine, or Lipitor."
"The 'right treatment at the right time, every time, to the right person…' will certainly revolutionize medicine and reduce expenditures on medicines that don’t work as well as the cost and loss of life due to adverse drug reactions. But preventive medicine can eliminate the need for those treatments entirely. We cannot afford NOT to have personalized medicine.”
Dr. Goldman teaches general genetics for Biology majors, developmental biology for Cell & Molecular Biology majors, and an interdisciplinary course on the ethical issues in science and technology. His research is focused on chromatin structure and the regulation of gene expression during mammalian development. Of special interest is the relationship between chromosome structural elements called nuclear matrix attachment regions (MARs) as boundaries of functional chromatin domains, studied through molecular methods and bioinformatics.
Dr. Goldman sees the public understanding of science as a key need if science and society are to thrive. He speaks to professional and general groups whenever possible, and he loves to talk about various issues, like stem cell biology, for TV and radio call-in audiences.
He has also written Op-Ed pieces or letters for the Los Angeles Times, the Wall Street Journal, the Sacramento Bee, the San Francisco Chronicle, and the New York Times, as well as a variety of technical articles, including those appearing in Science and Nature Genetics.
Dr Goldman is an Associate Editor for Chromosome Research and a contributing editor to Bio-IT World. He believes that the public learns much about science and bioethics from fiction, and, therefore, he also reviews novels addressing various aspects of genetic science and its implications, in venues like Nature, Science, Nature Genetics and the San Francisco Chronicle.
Clearly, Dr. Goldman is eminently qualified to provide very meaningful comments in the area of precision or personalized medicine.
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[Forbes article on GOP response to Obama proposal] [Politico article] [White House fact sheet on Precision Medicine Initiative/a>] [NIH web site on Precision Medicine Initiative /a>] [White House blog #1] [White House blog #2] [Illumina press release] [Commentary from The Genetic Alliance] [Wall Street Journal article] [NY Times article] [Washington Post article] [U.S. News & World Report article] [USA Today article] [Forbes article]