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Posts Tagged ‘precision medicine

Science Policy Around the Web – June 23, 2017

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By: Saurav Seshadri, PhD

Drug Policy

Trump’s New Policy to Tackle Sky-High Drug Prices Makes Sense — Sort Of

Tackling high prescription drug prices was a repeated promise of the Trump campaign. The Trump administration has now taken its first step towards fulfilling this pledge, outlined in a blog post by Food and Drug Administration (FDA) commissioner Scott Gottlieb. The agency will pursue a Drug Competition Action Plan, whose goal will be to eliminate obstacles to the development of cheap generic drugs – particularly those caused by loopholes in existing FDA policies, which are exploited by pharmaceutical companies to extend their patent exclusivity period and maximize profits. An example of such ‘gaming’ the system, cited in the post, is the practice of limiting access to branded products for comparative testing by generic developers. Ultimately, the FDA will work closely with the Federal Trade Commission (FTC) to address such issues, since directly regulating business practices is outside its mandate.

On its face, the FDA’s effort is a step in the right direction. Availability of generics reduces the cost of medications by over half within the first year, and according to a recent Congressional report, manufacturers state that ‘competition…is the primary driver of generic drug prices’. However, it ignores evidence that the real driver of increased drug spending is new, branded medicines, not overpriced generics. In fact, early indications are that Trump’s policies will favor the pharmaceutical companies that produce such medicines, by reducing regulations and apparently abandoning his promise to enable the government to negotiate drug pricing through Medicare. Overall, these actions signal a commitment to promoting free market mechanisms in the pharmaceutical industry; time will tell whether this approach will actually lead to more affordable drugs. (Julia Belluz, Vox)

Cancer

In a Major Shift, Cancer Drugs go ‘Tissue-Agnostic’

With the landmark approval of Keytruda in May, the Food and Drug Administration (FDA) appears to have ushered in a new era of cancer drug development.  So far, cancer treatment and drug evaluation have largely used the tumor’s tissue of origin as a starting point. Keytruda (an immune system enabling drug developed by Merck and approved for melanoma in 2014) marked the first departure from this approach, receiving priority approval to treat any solid tumor containing a mutation in the mismatch repair pathway, regardless of context. Recently released data suggests that another tissue-agnostic cancer therapy is on the way: larotrectinib (a cell growth inhibitor developed by Loxo Oncology) showed high efficacy for any tumor with a certain biomarker (TRK fusion). Several other such drugs, whose indications will be based on tumor genetics rather than location, are in the clinical pipeline.

Although these advances have generated significant excitement in the cancer community, some caveats exist. First, identifying the patients that could benefit from tissue-agnostic treatments will require individual initiative and depend on the cost of screening, particularly when considering markers that are rare for a certain tumor type. A potential solution is suggested by the NCI-MATCH trial, part of the NIH’s Precision Medicine Initiative (PMI) – in it, patients can enroll in one of several parallel clinical trials if a corresponding drug-targeted mutation is found in their tumor’s genome. If these trials prove effective, patients could eventually be regularly matched with a personalized, tissue-agnostic, biologically valid treatment, based on a standardized screen.  Second, researchers caution that tissue-agnostic studies should have a strong scientific rationale and/or breakthrough-level efficacy. Otherwise, such efforts ‘could actually slow drug development if there are differential effects across tumor types by diverting resources from enrolling patients in a predominant population or in the tumor type most likely to respond’.

Despite these concerns, the tissue-agnostic paradigm offers great promise for cancer patients. NIH-funded resources such as The Cancer Genome Atlas could be invaluable to this field moving forward. (Ken Garber, Science)

Scientific Publishing

US Court Grants Elsevier Millions in Damages from Sci-Hub

A New York district court has awarded academic publishing giant Elsevier $15 million in damages from Alexandra Elbakyan, founder of the website Sci-Hub, for copyright infringement. Elbakyan, a 27-year-old neuroscientist turned programmer, started Sci-Hub in 2011 with the goal of ‘remov[ing] all barriers in the way of science’. The site allows users to download research papers that would normally be blocked by a paywall, by obtaining credentials from subscribing institutions and using them to access publisher-run databases like ScienceDirect. Over 60 million papers are posted on Sci-Hub, and users downloaded 28 million articles in 2016.

Elbakyan’s case is reminiscent of Aaron Swartz, another high-profile champion of open access to scientific research. Faced with federal charges related to his hacking of journal archive JSTOR, Swartz tragically committed suicide in 2013. Both Elbakyan and Swartz found publishers’ ability to profit from restricting access to scientific literature, effectively withholding knowledge from anyone outside of a privileged inner circle, as well as the legal protection provided to this system, to be deeply unethical. Their willingness to act upon these convictions has earned each a sizable following in the scientific community.

For their part, publishers claim that fees go towards overhead, and point to significant efforts to expand free and open access programs. While judges have so far been sympathetic, Elsevier’s legal battle has been largely one-sided. Elbakyan has been ignoring rulings requiring her to shut down Sci-Hub since 2015, opting to simply change domains instead, and since she is currently based in Russia and has no American assets, she is unlikely to pay any damages. (Quirin Schiermeier, Nature News)

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June 23, 2017 at 11:00 am

Science Policy Around the Web – June 16, 2017

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By: Emily Petrus, PhD

Source: pixabay

Science and Politics

Politics in Science – It’s Not Just the U.S.!

Romania is a country in eastern Europe that joined the European Union (EU) in 2007. Scientists there are few and far between; research spending only accounts for 0.49% of GDP, the lowest in Europe (the US spent 2.7% in 2016). After joining the EU, Romanian researchers were encouraged to apply for European merit-based grants and sit on international review boards such as the National Research Council and the National Council of Ethics. It seemed that research was making slow but steady progress, but the new administration elected this year has shaken things up in all facets of government, including scientific research.

The new research minister, Serban Valeca, removed the international members appointed to government councils that oversee research funding, ethics, innovation and science policy, and replaced them with city council members, government-loyal union members, researchers from second tier Romanian institutes and even a surgeon being investigated for embezzlement. Grant review panels have been shuffled to remove international scientists and replace them with domestic researchers, but only if they have a certificate saying their university approves of their participation. These changes mark a departure from welcoming international input into Romanian proceedings and a movement towards scientific isolation.

To combat these changes, Romanian scientists have formed an organization, Ad Astra, which calls on researchers to boycott grant evaluations. Combined with the shuffling, the councils have been suspended for 3 months, which delays funding and puts already under-funded researchers in peril. The European University Association calls the policies deeply concerning, and although the current president may disagree with the research minister’s handling of the situation, his political ties ensure he won’t hold much sway over how this plays out. A computer scientist at the University Politehnica in Bucharest, Costin Raiciu, is concerned that the policies will affect talented researchers who have returned to Romania and says, “Without [merit-based] funding, people would either give up research altogether or move out of the country”. This is an all too familiar scenario in which it is apparent that policy and science must cooperate to produce ideal outcomes. (Alexandra Nistoroiu, ScienceInsider)

Mental Health

Clinical Trials Down, Basic Research Up at NIMH

Mental health is a notoriously tricky field. The development of the Diagnostic and Statistical Manual of Mental Disorders (DSM) in the 1950s has historically been a way to diagnose patients with mental health issues, and then give appropriate treatment. This has proved to be an imprecise treatment strategy, because within a category of diagnosis there is a broad spectrum of behaviors, and underlying this behavior there may be multiple causes. The NIH’s Precision Medicine Initiative (PMI) seeks to characterize 1,000,000 people by behavior, genetics, environment, and physiology. Researchers from the NIMH will send questionnaires evaluating behavior to detect mood and reward responses for this group of people. When this mental health evaluation is combined with information about their genetics, lifestyle and environment, scientists can characterize mental health disorders more specifically.

Many clinician researchers are upset by the steep decline in clinical trial research funded by NIMH, which has become higher profile with director Joshua Gordon’s arrival in 2016. NIMH seeks to route funding to study mental disorders using a basic research approach before spending time and money on costly clinical trials which too often lead to inconclusive or disappointing results. In 2011 NIMH launched the Research Domain Criteria (RDoC), which encourages research proposals to include a hunt for the mechanism underlying mental health studies. Since the initial call to include a RDoC perspective in grant applications, the incidence of RDoC appearing in funded applications has increased while mention of the DSM has decreased. Other buzzwords that are present in funded grants include biomarker, circuit, target and mechanism.

These data represent a shift in how funding decisions will proceed in mental health but may have broader reaching implications for other areas of research. In a blog post Dr. Gordon writes, “the idea that RDoC will facilitate rapid, robust and reproducible neurobiological explanations for psychopathology (as observed within and across DSM disorders) represents a hypothesis”. It remains to be seen if RDoC is an effective metric to evaluate successful grants. (Sara Reardon, Nature News)

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Science Policy Around the Web – August 19, 2016

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By: Ian McWilliams, PhD

Photo source: pixabay

Climate Change

Melting ice sheet may expose cold war base, hazardous waste

During the Cold War, the US Army Corps began a top-secret mission to determine the capability of launching nuclear missiles at Russia from a base in Greenland. The military base constructed for this mission, named Camp Century, lies approximately 125 miles inland from the Greenland coast and was later abandoned in 1964 after the Joint Chiefs of Staff rejected the plans to create a nuclear base. When soldiers abandoned the base, it was thought that leftover fuel and waste material would be safely interred, buried under ice for thousands of years.

However, climate change has now threatened those plans. The increased ice melt could reveal the base as early as 2090 and it is estimated that tens of thousands of gallons of diesel fuel, wastewater, sewage, and other chemicals could be exposed. Adding to concerns is the nuclear generator housed in the frozen base. Although the base never became a site for nuclear weapons, the low-level radioactive coolant from the nuclear generator is still stored in the base. If ice melt continues to occur at an accelerated rate, some have expressed concern that these chemicals could be released into the environment by seeping into waterways causing a potential environmental catastrophe. (Stephen Feller, UPI)

Microbiome

Mouse microbe may make scientific studies harder to replicate

Reproducibility is an issue that has been the subject of much debate in the scientific community recently. Now, scientists are concerned that the microbiome may further complicate the issue. The collection of commensal microorganisms that reside on or within the body is referred to as microbiota, and it is now well known to affect the health of the host. Although researchers have taken meticulous steps to ensure that experimental animals are housed in identical conditions, including sterile bedding, strict temperature control, and standard light cycles, determining experimental variability due to differences in their microbiome have remained elusive. As researchers explore the issue further they have found that mice from different vendors have very different compositions of bacteria in their gut that could explain some inconsistencies in researchers’ experiments.

Although it is not mandated, taking steps to control for microbiome may aid in the reproducibility crisis. Segmented filamentous bacteria (SFB) have been identified as a notable concern, and some vendors are providing SFB positive or SFB negative animals separately. Although it is unlikely that SFB is the only culprit for differences in studies, researchers continue to explore new variables in rodent husbandry in an effort to improve reproducibility of scientific results. To add to the dilemma, because the species that constitute the microbiome are constantly changing, it is difficult to characterize, and impossible to standardize. Since mice share their microbes through eating each other’s feces, cage-mates can have similar microbiomes that provide natural microbiota normalization for littermates. (Kelly Servick, Science)

Precision Medicine

Spiking genomic databases with misinformation could protect patient privacy

New initiatives, like the Precision Medicine Initiative (PMI), are helping to cultivate the human genome into usable sets of data for research purposes. This pursuit is founded upon the willingness of participants to allow their genetic information to be pooled for analyses, but many have expressed concerns over the privacy of this genetic information. It has previously been shown that individuals can be identified from their anonymized genomic data and this has prompted researchers to look for additional security measures. Computer scientists Bonnier Berger and Sean Simmons have developed a new tool to help achieve this goal by using an approach called differential privacy. To increase privacy, a small amount of noise, or random variation, is added to the results of a user’s database query. Although the information returned would provide useful results, it would make it more difficult to conclusively connect this data to a patient’s identity. A similar method has been used by the US Census Bureau and the US Department of Labor for many years.

However, some scientists, including Yaniv Erlich, have concerns that adding noise to the dataset will reduce users ability to generate useful results. Erlich stated that “It’s nice on paper. But from a practical perspective I’m not sure that it can be used”. In the search for privacy, free form access to the data is limited. This “privacy budget” limits the number of questions that can be asked and excludes hundreds or thousands of locations in a genome. Additionally, because noise naturally increases error, it weakens the overall conclusion that can be drawn from the query. Simmons expects that answers will be close enough to be useful for a few targeted questions. The tradeoff for increased security is that databases protected this way could be instantly accessible and searchable, which cuts down on getting access to databases such as those managed by the National Institutes of Health. Simmons added that this method is “meant to get access to data sets that you might not have access to otherwise”. The group plans to continue to refine this method to balance the needs of researchers for access to these data sets while maintaining patient privacy. (Anna Nowogrodzki, Nature)

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August 19, 2016 at 11:08 am

Science Policy Around the Web – June 21, 2016

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By: Fabrício Kury, MD

Photo source: pixabay.com

Personalized Medicine Costs

The Paradox of Precision Medicine

Precision medicine has been hailed by President Obama as a multi-hundred-million “moonshot” meant to revolutionize medicine in a way never seen before. Its rationale derives from the recent field of research called Genome-Wide Association Studies (GWAS), which seeks to discover, in large and accelerated scale, the genetic basis of disease, novel targets for drugs, and what treatments work for which patients and at what moments and doses. This very rationale, however, can be self-limiting in a capitalist market where economics of scale is required to provide patients with access to otherwise prohibitively expensive treatments. In this lucid review, Janeen Interlandi from Scientific American demonstrates that old-fashioned, non-personalized treatments have recently been demonstrated not only be tremendously cheaper than “bespoke” drugs, but also just as clinically effective. (Janeen Interlandi, Scientific American)

Research Ethics

Scientists Are Just as Confused About the Ethics of Big-Data Research as You

Dubbed “the fourth paradigm” of science (book available for free download here), big data research poses novel ethical questions that might not be appropriately addressable by the current paradigm of ethics centered on the Common Rule and oversight by Institutional Review Boards (IRBs). A study can be ruled exempt from IRB approval if it only utilizes publicly available data – but what is it “publicly available,” exactly? In this article, Sarah Zhang from Wired magazine reviews recent cases of controversy in utilization of large datasets for studies, such as the Facebook Emotion Experiment, and suggests that IRBs might need new sets of skills to safeguard human subjects in the evolving landscape of research. (Sarah Zhang, Wired)

Data Science

The Doctor Who Wants You to Be a Research Parasite

After the editor-in-chief of the New England Journal of Medicine published in January, 2016 a stingy editorial affirming that some clinical researchers regard data scientists as “research parasites,” a wave of controversy exploded and culminated with personalities such as U.S. Chief Data Scientist DJ Patil and National Academy of Sciences President Marcia McNutt publicly using the hashtag #IAmAResearchParasite in defiance. In this article, Taylor Mayol from Ozy introduces Dr. Atul Butte, recently-appointed head of Clinical Informatics at the University of California, who sustains a bold call for more “research parasites” in health care, while additionally characterizing lack of entrepreneurship among academics as “a tragedy” because it is “the right way to truly change the world, by going beyond writing papers.” (Taylor Mayol, Ozy)

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June 21, 2016 at 9:00 am

Science Policy Around the Web – May 27, 2016

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By: Sophia Jeon, Ph.D.

Photo source: pixabay.com

Drug regulation and rare diseases

FDA delays decision on whether to approve Sarepta drug for Duchenne

The Food and Drug Administration (FDA) is wedged between a rock and a hard place to decide whether or not the agency should approve the controversial drug, eteplirsen, for Duchenne muscular dystrophy (DMD). DMD mainly affects boys and is considered a rare disease as it affects fewer than 200,000 US cases per year. As the name implies, it is a neuromuscular condition in which symptoms include frequent falling, trouble getting up or running, and learning disabilities. Average life expectancy for those afflicted with DMD is about 25, and there is no treatment. Considering these devastating factors, it is easy to understand why DMD patients, and their parents would want to hasten the approval of a drug that could potentially save their lives.

On the other hand, it is also easy to understand why the FDA is hesitant to approve this drug. The FDA is a regulatory agency and one of their missions is to evaluate drugs to determine whether or not they are safe and effective to be in the market. The issue is with a Phase III study that Sarepta Therapeutics did to test the efficacy of eteplirsen – that the trial was not well-designed making it difficult to come to a definitive conclusion that this drug works. The trial only involved 12 patients, without a placebo control group. If the FDA makes a decision to approve this drug under political pressure from various stakeholders, a drug that potentially could be ineffective for many kids with DMD only gives them and their family false hope and decreases the motivation for pharmaceutical companies to develop more effective DMD drugs.

Understanding patients’ needs, FDA has an expanded access program that, with the FDA’s approval, allows patients to try experimental drugs. In addition, the Orphan Drug Act gives pharmaceutical companies more incentive to develop drugs for rare diseases. However, it is clear that patients whose lives are on the line do not think the drug development is happening fast enough and are willing to try any option they have available. In 2015, there was even a bill introduced, called Right to Try Act that allows patients to have access to an experimental drug without the FDA’s approval. How much should public input or influence be taken into account in a drug approval process? Should FDA have better strategies in effectively communicating and engaging with patient groups? These are some good questions without definitive answers. (Ed Silverman, STATnews)

Research evaluation and bibliometrics

The pressure to publish pushes down quality

Let’s look at our current research culture. Whether or not you get an academic position, a grant, or a renewal of a grant all depends on how much you publish. “Publish or perish” is a phrase that is frequently used in academic science and it definitely rings true for many researchers in the US. People evaluate your research and productivity based on the number of your publications and the impact factor of the journals you publish in. Daniel Sarewitz recently wrote in Nature about the negative consequences of promoting this “publish or perish” culture.

The first problem is that increasingly everyone in research, whether conscious of it or not, seems to be contributing to this culture, and we need a cultural shift, which does not happen overnight. However, there are efforts to change the way we evaluate science. For instance, Declaration on Research Assessment (DORA) was initiated by the American Society for Cell Biology with a group of editors and journal publishers to try to make that cultural shift and start the movement for everyone in science to realize that merely an impact factor or how many times your paper has been cited should not and cannot accurately reflect your productivity, assess your work’s value or even define your career.

The second problem that Daniel talks more about in this article is the problem of rigor. Some researchers are so pressured to publish that they end up do things that are unethical or produce hard-to-replicate findings by experiments that are not rigorously designed. Researchers also sometimes exaggerate the importance of their findings in order to publish or hand-wave at any inconsistencies in their discussion sections. The real harm is done not only when other researchers waste time trying to chase a false lead but also when these not-rigorously-tested studies accumulate to adversely affect public health. A popular example is the study done by Andrew Wakefield who published his (false) claim linking the MMR vaccine to autism. These problems should not just stop at being “concerns,” instead it is time to re-think the strategies of evaluating science and doing science. Scientists could do better quality science by spending more time thinking and rigorously testing hypotheses than strategizing how to write an attractive story to publish more in a “high impact” journal. (Daniel Sarewitz, Nature Comments)

Clinical trial design and personalized medicine

Personalized medicine: Time for one-person trials

Biomedical research is in a very unique position right now. Recent technological advances have allowed scientists to easily and economically perform activities such as whole genome sequencing (WGS), big data analysis, mobile health data tracking and tissue and cell engineering. These technologies, especially when used in combination, can be a powerful tool that not only offers scientific insight into human biology but also brings up a number of exciting opportunities for prevention and treatment of diseases. These are a few of many reasons the President’s Precision Medicine Initiative (PMI) is gaining much attention.

With these advanced technologies, scientists are beginning to realize that personalized medicine, not just genetic counseling but one that includes a number of other measures, such as your metabolic profile, lifestyle factors, environmental exposures, etc., is the future of biomedical science. One-person trials hope to address a number of issues that the current clinical research or trial designs cannot address sufficiently such as the fact that people have different responses to drugs, and that there has been a lack of inclusion of minority or health disparities populations in many clinical trials taking place in the US. N-of-1 trials would not only address that issue but also could reduce any ethical concerns for placing patients in a randomized placebo control group especially when there is no standard-of-care, because everyone in the trial would be getting the experimental drug for a certain period of time, wait for the effects to wear off, and then a placebo for another to examine their responses to the drug. (Nicholas J. Schork, Nature Comment)

 

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May 27, 2016 at 1:30 pm

Science Policy Around the Web – November 10, 2015

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By: Daniël P. Melters, PhD.

Infectious disease

Cattle trial cuts human sleeping sickness

In addition to HIV and malaria, sleeping sickness is another serious infectious disease causing major health problems in sub-Saharan Africa, resulting in many thousands of infections each year. In total over 65 million people are at risk of infection. The disease is caused by the protozoan parasite from the genus Trypanosoma, where Trypanosoma brucei gambiense accounts for more than 98% of all reported cases. The parasite is transmitted via tsetse flies. The people most affected by this parasite live in rural areas, where they are in close contact with life-stock. These life-stock hold an important step in the life-cycle of Trypanosoma. To make matters worse, diagnosis and treatment require specifically skilled staff, resulting in only about 30% of all infected individuals receiving treatment following a diagnosis.

A collaboration between the University of Edinburgh (UK), Makerere University (Uganda), and the Ugandan government has tried to tackle the problem by injecting 500,000 cows with a parasite killing agent in addition to regular fumigation with insecticide to qualm the number of tsetse flies. The number of people diagnosed with sleeping sickness went down by 90%. Following this successful trial the program will be expanded to cover the whole of Uganda, including the treatment of 2.7 million cattle. (SciDev.Net)

Precision Medicine Initiative

Privacy Risks from Genomic Data-Sharing Beacons

One of the corner stones of President Obama’s Precision Medicine Initiative is the broad sharing of medical data between many scientists, albeit in a responsible manner. In their recent report, the NIH Precision Medicine Initiative Cohort Program (PMI-CP) workgroup advised the creation of a “hub-and-spoke” model that has a Coordinating Center to provide safeguards to facilitate data access, data normalization, and participant engagement. Part of this dataset is genomic data from patients. One major concern about genomic and genetic data is that this can be used to identify the donor, even when the genomic data is made anonymous early on. A recent article by Shringarpure and Bustamante in the American Journal of Human Genetics provides evidence that it is not only possible to re-identify to whom an anonymous genetic ‘beacon’ belongs to, but also identifies their relatives with just 1000 single-nucleotide polymorphisms (SNP)s. A beacon is a web server that answer allele-presence queries in a binary manner. This might pose a serious privacy-concern for potential participants in the PMI-CP. This concern is not limited to the PMI-CP either. Recently the American Association for Cancer Research (AACR) rolled out their Project GENIE where US and European research institutes will share their cancer genomes to catalyze the development of more precise cancer treatments. Nevertheless, Shringarpure and Bustamante do make several suggestions to continue to safeguard patient privacy. (American Journal of Human Genetics)

Direct-to-Consumer Genetics

Another Genetic Testing Company in Hot Water with the FDA

In November 2013, the US Food and Drug Administration (FDA) warned the direct-to-consumers health testing company 23andMe that they needed to comply with federal regulations with respect to approval for medical devices (section 201(h) of the Federal Food Drug and Cosmetics Act). 23andMe offered a saliva-based genetics test that provided participants with an ancestry-based analysis of some of genetic markers, in addition to various health-related genetic variations (SNPs). The FDA is of the opinion that the latter one required approval by them as a medical device. Seven months after their warning, the FDA received an application from 23andMe. Recently, they obtained the federal seal of approval for a few of their health-related genetic tests.

23andMe is maybe the most well known of these direct-to-consumers genetic testing companies, but they are certainly not the only ones. On November 2nd, the Louisiana-based DNA4Life Company received a similar notification from the FDA. Just like 23andMe, DNA4Life has held the position that they do not need FDA approval to sell their genetic test kit. However, the FDA maintains that the genetic test, which predicts how patients will respond to 120 of the most common medication, meets the definition of a “medical device” and requires that the company either provide evidence of FDA approval or present why they do not need approval. DNA4Life has not yet publicly responded to the FDA notification.

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November 10, 2015 at 12:00 pm

Science Policy Around the Web – June 9, 2015

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By: Cheryl Jacobs Smith, Ph.D.

Drug Policy

FDA Backs Experimental ‘Female Viagra’

Amidst previous accusations that the Food and Drug Administration (FDA) maintains a gender bias when it comes to treating sexual dysfunction, there may finally be a drug on the market intended to treat female sexual dysfunction. Flibanserin (proposed trade name: Addyi), produced by Sprout Pharmaceuticals, has been recommended for approval by the FDA. The recommendation is lauded as a victory by advocates for a female version of the blockbuster male sexual dysfunction drug, Viagra. Finally, there is a product on the market tailored to specific biological sexual dysfunction in women—but the FDA warns that it should be considered cautiously.

For years the FDA has struggled to approve female sexual dysfunction drugs in part due to lackluster effectiveness and safety issues. In a report released by the FDA from the Bone, Reproductive and Urologic Drugs Advisory Committee (BRUDAC) and the Drug Safety and Risk Management (DSaRM) Advisory Committee, individuals on the committee show interest in getting flibanserin approved; however, they are hesitant due to the interactions flibanserin has with alcohol consumption. The agencies assert that although the observed treatment benefits are significantly beneficial when compared to those individuals who took a placebo, the safety concerns outweigh the risks. Those risks include that co-consumption of flibanserin and alcohol can lead to heavy sedation, and that there are cardiovascular issues, such as syncope and hypotension, that can lead to low blood pressure and fainting. “We really know almost nothing about the actual clinical effects of using this product together with alcohol,” said Dr. Tobias Gerhard of Rutgers University. “We have some indication that there is clearly a concern from very small studies.”

Despite FDA’s caution concerning flibanserin’s drug interactions, there is still a desire to take flibanserin even to achieve the drug’s expected modest results. “I want to want my husband, it is that simple,” said Amanda Parrish, a mother of four from Nashville Tenn. “For us, flibanserin is a relationship-saving and life-changing drug.” (Matthew Perrone, Huffington Post)

Precision Medicine

NIH researchers sequence healthy volunteers’ DNA and find they aren’t so healthy after all

The National Institutes of Health (NIH) conducted a study a few years ago where they sequenced the genome of 1,000 healthy volunteers. The volunteers appeared to be free of disease as assessed by blood tests, echocardiograms, and self-reported systems. The sequencing result of these 1,000 healthy volunteers shocked the NIH team.

Many of the presumably healthy volunteers’ DNA contained mutations that made them more susceptible to certain conditions such as cancer and kidney disease. Interestingly, these some of individuals, or their family members with the same genetic background, were living with the disease mutation but did not have any clinical indication or outward sign of the disease. All of the participants in the study were adults, ranging from 45 to 65 years of age. The researchers who designed the study assert that this age range is prime to observe many signs of genetic disease, so it was interesting to observe volunteers whose diseases had not manifested despite their genetic susceptibility.

NIH researchers did contact participants about their genetic findings concerning disease and one volunteer shared the information with his sister. The sister sequenced herself and found that she shared the disease mutation that predisposed her to breast and ovarian cancer. She decided to undergo preventative surgery for ovarian cancer and during the procedure the doctors found a tumor.

Such findings are important to understand in light of DNA testing and in the wake of the 21st Century Cures Initiative that aims to put personalized medicine in the forefront of patient care. (Ariana Eunjung Char, Washington Post).

Global Science and Development

Nigeria’s new leadership raises hopes for science

On May 29, 2015, Muhammadu Buhari assumed office as the President of Nigeria. Although Nigeria is one of Africa’s most populous country with the largest economy, it publishes much fewer times than other African countries relative to the size of its economy.

Oye Ibidapo-Obe, vice-chancellor of the Federal University Ndufu-Alike Ikwo in Ebonyi State and former president of the Nigerian Academy of Science explains that Nigeria has suffered from leadership that has paid only lip service to the contribution of science to national development. The administration failed because they were not proactive in implementing a national science and innovation policy it introduced in 2012. Changes for the future of science in Nigeria could include implementing the 2012 policy and putting in place a NSF-like body to review grants and give funding. Mr. Ibidapo-Obe says, “I would like to see a country that is aware of the value of science. Our leaders know and profess that science can provide solutions to our contemporary challenges — poverty, education, good health provision, human security, clean and adequate energy, proper public infrastructure, food, climate-change adaptation, democracy and good governance. But they have not shown sufficient courage to invest massively in science and technology.” (Jackie Opara, Nature)

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June 9, 2015 at 9:00 am