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Posts Tagged ‘medicine

Science Policy Around the Web November 26th, 2019

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By: Andrew H. Beaven, PhD

Source: CDC

How Best To Use The Few New Drugs To Treat Antibiotic-Resistant Germs

Bacteria have existed for 3.5–4 billion years, and their survival demonstrates remarkable environmental plasticity. One consequence of their plasticity is that bacteria are able to rapidly become resistant to antibacterial drugs (drugs meant to help humans and animals stave off infection).

Combatting antibiotic drug resistant bacteria (so-called “superbugs”) has been pinpointed as a major, modern global health concern. A new Centers for Disease Control and Prevention (CDC) report published November 14, 2019 estimates that more than 2.8 million treatment-resistant infections and 35,000 annual deaths occur in the U.S. alone. Notably, the development of new antibiotics has lagged, therefore, scientists are recommending new methods to use old drugs. These include: limiting doses for healthy people and allow the body to do its work; flooding the body with multiple drug types at once; or sequentially changing drug types every 12 to 24 hours.

Given all we know about this global health concern, why is the market not being flooded with new antibiotics? Largely, because it is not lucrative for pharmaceutical companies to chase a moving target. Additionally, antibiotics are only used for short-term ailments (as opposed to those used for chronic illness) and many antibiotics remain unused in an effort to minimize new drug resistance. To help promote new antibiotic research, U.S. Senators Bob Casey (D-PA) and Johnny Isakson (R–GA) introduced the Developing an Innovative Strategy for Antimicrobial Resistant Microorganisms Act of 2019 (DISARM Act of 2019; S.1712) to the Senate on June 4, 2019. The goal of the act is to strengthen American antimicrobial research and improve the development pipeline. While the act was called “essential and timely” by the President of the Infectious Diseases Society of America, it has not passed the Senate at time of publication.

(Richard Harris, NPR)

As SpaceX Launches 60 Starlink Satellites, Scientists See Threat to ‘Astronomy Itself’

On November 11, 2019, the private American aerospace company SpaceX, founded by Elon Musk in 2002, launched its second Starlink satellite payload rocket into outer space. Starlink is a prodigious project that aims to provide “high speed internet access across the globe,” specifically allowing internet access to “locations where access has been unreliable, expensive, or completely unavailable.” The Starlink webpage states that they will provide near-global internet coverage by 2021 through their satellite constellation (a network in which satellites work together to provide continuous coverage).

Even with the relatively few Starlink satellites currently in place, astronomers have already noted significant impact on their work. The primary point of concern is that the satellites are very bright, and astronomers say that even if the satellites are darkened, they will have a profound effect on Earth-based astronomy. Additionally, astronomers worry that Starlink will pollute radio wavelengths used to probe deep space and permanently pollute low-orbit space with “space junk.” SpaceX says they are attempting to minimize the effects Starlink has on the scientific community and that the project is moving ahead. Indeed, Mr. Musk has requested the Federal Communications Commission to allow 30,000 more satellites than 12,000 that were already approved. If successful, SpaceX would have eight times more satellites in low-Earth orbit than currently in orbit.

Other companies, such as, Amazon, Telesat, and OneWeb, are following close behind launching similar satellite constellations. Using outer space for private gain yields an important, unanswered question – who can profit from outer space? Megan Donahue, president of the American Astronomical Society acknowledges that “international space law is pretty wide open,” and that it for now the public must trust corporate good will. Currently, a set of United Nations treaties and principles as well as resolutions have laid out guidelines on the peaceful usage of space, but few exact rules are in place.

(Shannon Hall, The New York Times

Written by sciencepolicyforall

November 26, 2019 at 3:14 pm

Should Vaccines be Mandatory?

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By: Juan I. Moliva, Ph.D.

Image by Angelo Esslinger from Pixabay 

The anti-vaccination movement has been gaining traction across the United States (U.S.) and the world. According to the Centers for Disease Control and Prevention (CDC), the number of measles cases in the first five months of 2019 surpassed the total number of cases per year for the past 25 years, with the number of children unvaccinated for preventable diseases quadrupling since 2001. The CDC also found an increase in the number of vaccine exemptions for kindergarteners across the U.S. While the numbers vary across the country mostly due to laws set in place by each state, in Mississippi, one of three states that do not grant exemptions due to religious or philosophical reasons, the rate of kindergartners with an exemption to one or more required vaccine was 0.1%. Compare that to Oregon, a state that grants exemptions to vaccines due to religious or philosophical reasons, where the rate of vaccine exemptions for kindergarteners is at 7.6%. As the number of individuals choosing not to get vaccinated or choosing not to vaccinate their children continues to increase, the debate as to whether vaccines should be mandatory has taken front stage.

Simply stated, a vaccine is a weakened or killed version of a pathogen that is delivered to the body with the sole goal of stimulating an immune response to protect you against the disease you are being vaccinated against. How is this accomplished? Your immune system has a subset of cells with “memory” that remember the pathogen and if you are later exposed to the pathogen these “memory” cells will eliminate the pathogen before it can make you sick. In the U.S. and most of the world, the general scientific consensus on vaccines is that they are safe and critical for maintaining healthy communities. The U.S. Senate Committee on Health, Education, Labor, and Pensions (HELP) strongly defended the case for vaccines. Chairman Lamar Alexander (R-TN) said, “The science is sound: Vaccines save lives – the lives of those who receive vaccines and the lives of those who are too young or vulnerable to be immunized.” Like any medication or medical procedure, vaccines do not come without risk, but the advantages vastly outnumber any disadvantages.

Not only do vaccines protect your body from dangerous pathogens, they also protect the community via a concept known as herd immunity. When a high enough percentage of the population is immune, pathogens have a harder time spreading. We rely on this principle to protect those who cannot receive a vaccine. However, vaccine opponents continue to argue that mandating vaccines is a slippery slope towards losing body autonomy. I have spent the last eight years studying vaccines, from basic research to pre-clinical testing through clinical trials and licensing. I have recently come to embrace the belief that the sole exemption for a vaccine that has been proven to be effective is a medical one – the case where you cannot receive a vaccine because your body responds negatively to the vaccine; this includes a weakened immune system that can be caused by chemotherapy or radiotherapy, a congenital condition that leads to an impaired immune system, or a history of serious adverse events related to vaccination such as an allergic reaction to one or more components in the vaccine.

The case against anti-vaxxers surrounds the choices they make on behalf of their children and how those choices affect their children and those in the community. If parents started claiming religious or philosophical exemptions against child safety seats or against buckling their children while driving, would we be arguing in their favor? Seatbelts and safety seats have been proven to save lives by preventing ejection from the vehicle after impact. Vaccines have been medically proven to save lives by preventing death or disability from infectious pathogens after exposure. While we expect not to be involved in a car accident, we rely on seatbelts in case we do. Vaccines work the same way; we do not want to be exposed to deadly pathogens but being vaccinated against these pathogens helps ensure we do not get sick if we do. Childrenare incapable of deciding if they want to be vaccinated as some vaccine regimens are initiated within the first year of life, thus their welfare is entrusted upon their parents. 

In the U.S. we have laws about providing proper nutrition, schooling, clothing, cleanliness, shelter, and many more that protect children’s welfare. Providing vaccines on time should be included as a basic necessity and be treated no different than other child welfare offenses. If an unvaccinated child contracts a preventable disease and is significantly affected to the point of disability or even death, the parent(s) should be directly responsible. A guardian refusing to vaccinate their medically eligible child should be treated like what it is: child endangerment. Take for example the recent case of a six-year-old in Oregon that nearly died of tetanus after sustaining a cut while playing outdoors. The child’s parents had refused to vaccinate the child with DTaP, a safe vaccine intended to protect against tetanus. The boy was held in an intensive care unit for 47 days, undergoing multiple intravenous infusions to alleviate the high fever, hypertension, and tachycardia caused by the infection. All at the cost of over $800,000 for a disease that could have been easily prevented with vaccination. Despite the episode, the boy’s parents continue to refuse the vaccine.

Although bipartisan support exists for vaccination at the federal level, it is unlikely federal law eliminating philosophical and/or religious exemptions would come to pass as public health policy is traditionally left to the states. State legislatures are beginning to introduce laws that will help limit the increase in numbers of preventable disease cases due to lack of vaccination. New York state recently passed legislation forbidding religious and non-medical exceptions to vaccines. After signing the bill, Governor Cuomo (D-NY) said, “While I understand and respect freedom of religion, our first job is to protect the public health.” Washington state, plagued by surging number of measles cases, also recently banned personal and philosophical exemptions to the MMR vaccine, which protects against measles, mumps, and rubella. Merely going to a public park or a grocery store can place unvaccinated children or immunocompromised individuals at risk of contracting a preventable disease. Such was the case of a six-year old boy with leukemia that died of measles after being exposed by his intentionally unvaccinated siblings. Senator Bill Cassidy (R-LA), a former physician, stressed: “If you are such a believer in liberty that you do not wish to be vaccinated, there should be a consequence and that should be that you cannot infect other people.” While I agree that autonomy over one’s body is important, the government has the right to require a reasonable level of vaccination for all citizens as a matter of public health. 

In the U.S. where freedom and personal liberty are valued above all else, mandatory vaccination would have a lengthy and challenging uphill battle. Additionally, data suggests that mandatory vaccination does not necessarily increase vaccination coverage. Instead, the U.S. could increase vaccinations via financial incentives similar to the system in place in Australia whereby parents receive monetary compensation when their children meet vaccination requirements. However, the most successful approach might be akin to the one the CDC implemented over sixty years ago to encourage stricter vaccination requirements by the states. By providing educational support, the CDC engaged parents, policymakers, and state and local health officials at every level to increase vaccination coverage. Education is one of the most effective ways to counter vaccination noncompliance. Specifically, educating people on the alternatives to vaccines – the morbidity and the mortality associated with lack of vaccination – was shown to be the most effective strategy. Educating why vaccines are so crucial is the most powerful weapon to combat vaccine non-compliance. 

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August 14, 2019 at 10:00 am

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Science Policy Around the Web August 9th, 2019

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By Ben Wolfson PhD

Image by OpenClipart-Vectors from Pixabay 

Scientists are making human-monkey hybrids in China

In a new report first published by Spanish newspaper El Pais, it was revealed that an international team of scientists lead by Dr. Juan Carlos Izpisúa have created human-monkey chimeras, with the end goal of growing human organs in monkeys for transplant.

Chimeras are organisms with cells coming from multiple genetically distinct individuals. Chimeras are commonly used for biomedical research, such as mice bred to express genes from other species, or immunocompromised animal models in which foreign tumors are introduced to facilitate cancer research.

While these uses are commonplace, the ethical ramifications of human-animal chimera creation are significant. If chimeras are created at early developmental stages, it is difficult to control the destiny of each individuals’ cells, meaning that human cells could end up in the nervous system or brain, potentially resulting in human-like behaviors. Due to these considerations, the National Institutes of Health have banned the use of federal funds to create human-monkey embryos, and the scientific community has instituted an unofficial “red-line” to cut off chimeric embryo growth at 14 days.

To avoid legal issues, Izpisúa’s team (based in the US and Spain) conducted research in China, where no legal limitations currently exist. In previous work producing human-pig and human-sheep chimeric embryos, Izpisúa reported that only 1 in 10,000 cells were human. While the results of his human-monkey research remain confidential as they await publication, a colleague stated that “the results are very promising“.

(Antonio Regalado, MIT Technology Review)

 

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August 9, 2019 at 3:38 pm

Disparity in the Global Supply and Demand for Vaccines Against Rotavirus

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By: Lawrence Wang

Image by Arek Socha from Pixabay 

Vaccines are extremely important for individual and global health. These life-saving biologics are especially important for children in developing countries, who suffer disproportionately from vaccine-preventable infectious diseases. One of these infectious diseases is rotavirus, which causes severe diarrhea and kills around 200,000 young children and babies per year1. Effective vaccines against rotavirus are responsible for saving countless lives and reducing suffering around the world.

In November 2018, the major pharmaceutical company Merck canceled a long-term commitment it made with GAVI (The Global Alliance for Vaccines and Immunization) to provide its rotavirus vaccine, RotaTeq, to four countries in West Africa (Burkina Faso, Cote d’Ivoire, Mali, and Sao Tome) for $3.50 a dose. Simultaneously, Merck increased RotaTeq shipments to China where a course sells for $40, nearly 12 times more than in West Africa2. Merck reported that the reason for this termination was “supply constraints” and an “unprecedented increase in global demand” for Rotateq3. Vaccines that were earmarked for West Africa were instead diverted to China, where they would protect millions of Chinese children while leaving millions of West African children vulnerable.

This situation exemplifies the disparity in the global supply of, and demand for, vaccines. Many vaccines are produced by large pharmaceutical companies like Merck that are based in wealthy nations (e.g., Merck’s headquarters are in New Jersey, USA). In general, developed countries have more agency in purchasing and providing vaccines for their citizens at higher prices. Developing countries, on the other hand, usually lack the money to pay for vaccines and are consequently reliant on free or subsidized vaccines. Profit-driven companies such as Merck are thus incentivized to sell their limited supply of vaccines to countries that can afford to pay more.

GAVI and UNICEF (United Nations Children’s Fund) are organizations that work to address disparities in vaccine access between industrialized and developing countries by buying and delivering vaccines to poor countries around the world4,5. However, they still rely on pharmaceutical companies to produce the vaccines that they distribute. When Merck notified GAVI and UNICEF that it would stop supplying discounted RotaTeq doses to the four West African nations, their only recourse to address the unexpected vaccine supply shortage was to help the affected countries switch to anot­­her rotavirus vaccine produced by a different company2.

This potentially problematic situation was avoided by a multilateral collaboration between aid organizations, governments, and manufacturers. Specifically, other pharmaceutical companies like GlaxoSmithKline (GSK), Serum Institute of India, and Bharat Biotech stepped in to fill the dearth left by Merck by pledging to supply their rotavirus vaccines (Rotarix, Rotavac, and Rotasiil) to the four West African countries. These countries are expected to switch to these alternative vaccines (which are actually cheaper than Merck’s) in 2020 after their supplies of RotaTeq run out6.

While this story ostensibly has a happy ending, it also highlights inherent problems in the way vaccines are supplied to the world, specifically poorer nations. These nations are reliant on aid organizations like GAVI and UNICEF to provide them with free or discounted vaccines, which are in turn dependent on profit-driven pharmaceutical companies to produce and supply the vaccines. Why can’t these multinational pharmaceutical companies, which have vast resources at their disposal, just produce more vaccines when there is a shortage?

The answer to this question is multifactorial. Firstly, vaccine production is complicated and only multinational pharmaceutical companies have the sufficient expertise, infrastructure, and manpower to produce high-quality vaccines that are safe and effective at scale. Thus, even these companies need advanced warning to produce millions of vaccine doses. Secondly, the prices for vaccines have been increasing in recent years, mostly due to high fixed overhead costs and exclusive licensing practices that preclude competition7. Thirdly, vaccines represent only about 2-3% of big pharma’s trillion-dollar production portfolio and are not the most profitable part of their portfolios8. Thus, pharmaceutical companies have little incentive to pick up the slack. How, then, can the costs of vaccines be reduced and who is supposed to step up to make vaccines when big pharmaceutical companies fall short of their obligations?

One potential solution to reduce the cost of vaccine production is for smaller pharmaceutical companies to produce generic vaccines. Generics are alternative versions of patented drugs and vaccines that are supposed to work just as well as the original formulation. In fact, India has become one of the world’s leading producers of generics and is a major source of vaccines and drugs for Africa. The issue with this solution is that the generics manufacturing industry is fraught with regulatory issues such as poor quality control and lack of oversight9. Africa is most starkly affected by these issues because manufacturers ship their lowest-quality drugs to the continent. Some of these products are completely counterfeit, with no active ingredients, while others have only a fraction of the active ingredients listed10. Without fixing quality control and oversight of generics manufacturing, this is not a viable long-term solution to provide vaccines to poor countries.

Another potential solution to increase vaccine coverage in Africa specifically would be to empower the continent to manufacture its own vaccines. Though Africa is home to nearly 17% of the world’s population, it produces less than 1% of the world’s vaccines and spends millions purchasing vaccines from foreign entities. For instance, African governments imported $900 million worth of basic vaccines for children in 201411. GAVI and UNICEF could partner with pharmaceutical companies to spearhead capacity building in specific African countries so they could produce their own vaccines, instead of making them dependent on vaccines from third parties. Apart from providing vaccines, this strategy would potentially build local biomedical capacity, provide jobs, and incentivize highly educated African expatriates to return to their home countries12.

Unfortunately, global vaccine stakeholders are hesitant to invest in building vaccine production capacity in Africa. For instance, profit-driven pharmaceutical companies tend to invest in healthy markets that will yield stable returns on their investments. Before investing in building a manufacturing facility, these companies must first ensure that the vaccines they produce will be bought for a certain price at a certain time12. Besides, It will be an uphill battle if Africa cannot produce vaccines more cheaply than other countries like India, which has already optimized infrastructure for producing vast quantities of vaccines cheaply13. Overall, there are many economic barriers that prevent Africa from developing its own vaccine manufacturing infrastructure.

In conclusion, the recent story revolving around providing rotavirus vaccine for West African countries exemplifies the complex challenges in supplying vaccines for the world, especially developing countries that contain some of the world’s most vulnerable populations. Despite the relatively vast resources that global organizations and companies like GAVI, Merck, GSK, and UNICEF have at their disposal, the demand for life-saving vaccines continues to outstrip the supply of vaccines. The bottom line is that vaccines are a vital component to the health of people and nations, and access to these medicines ought to be a universal human right.

  1. https://www.who.int/immunization/diseases/rotavirus/en/
  2. https://www.npr.org/sections/goatsandsoda/2018/11/01/655844287
  3. https://www.fiercepharma.com/pharma/amid-china-launch-merck-cites-supply-limits-plan-to-scale-down-rotateq-shipments-africa
  4. https://www.gavi.org/
  5. https://www.unicef.org/
  6. https://www.npr.org/sections/goatsandsoda/2019/05/31/726863111/it-looked-as-though-millions-of-babies-would-miss-out-on-a-lifesaving-vaccine
  7. https://www.who.int/immunization/programmes_systems/procurement/market/en/
  8. https://www.theatlantic.com/business/archive/2015/02/vaccines-are-profitable-so-what/385214/
  9. https://www.statnews.com/2019/07/22/indian-pharmaceutical-industry-drug-quality-charges/
  10. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4105729/
  11. https://www.dw.com/en/africa-wants-to-manufacture-its-own-vaccines/a-46151389
  12. https://www.frontiersin.org/articles/10.3389/fpubh.2019.00056/full
  13. https://www.thehindu.com/sci-tech/health/trusting-the-science/article25861183.ece

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August 7, 2019 at 2:45 pm

Science Policy Around the Web August 6th, 2019

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By Allison Dennis B.S.

Image by rawpixel from Pixabay 

Researchers weigh in on Trump’s $500 million plan to share childhood cancer data

Researchers are contemplating developing what they call The Childhood Cancer Data Initiative (CCDI). This approach comes in response to the 10-year $500 million research budget for childhood cancer proposed by President Donald Trump during his January State of the Union Address. Federal officials implementing the proposal have seized on an ongoing effort to transform “big data” into new medical discoveries by looking to expand the sharing of patients’ data to develop new approaches to treat childhood cancers. Currently, patient data can be found spread across state registries, tumor DNA databases, and clinical trial records, obscuring potential insights. 

A symposium held by the National Cancer Institute (NCI) in August 2019, gathered members of the research community to brainstorm the future of the CCDI. Experts made clear the need to first inventory what data already exists, including making efforts to digitally join the five largest existing pediatric cancer databases. Close attention and broad changes will be needed to unify the individual observations being made as children battling cancer make their way through the medical system. Yet despite these big ideas, it is not yet clear that Congress will follow through to appropriate the $50 million down payment needed to kick start CCDI. 

(Jocelyn Kaiser, Science)

 

‘Mosaic’ HIV vaccine to be tested in thousands of people across the world

The ‘mosaic” vaccine is the latest innovation in HIV prevention scheduled to start late-stage clinical trials in September. The experimental vaccine is designed to elicit an immune response to protect against more strains of HIV than any developed so far. The phase III trial (termed “Mosaico”) will be conducted in Europe and the Americas to follow its effectiveness at preventing the transmission of HIV in 3,800 participants, divided evenly into groups receiving four injections of vaccine or placebo. The innovative approach taken by researchers started with engineering a disabled common cold virus to carry pieces of DNA encoding synthetic copies of three HIV genes. The synthetic genes help the body to recognize several different global HIV strains. In addition to the DNA sequences, the vaccine is delivered with two synthetic proteins designed to match HIV strains common in Africa, the Americas, Europe, and Australasia.

Only four HIV vaccines have ever been tested for efficacy in humans. One which initially showed promise but whose efficacy waned over time resulted in a modest 31% difference in rates of infection between groups who received the vaccine compared with placebo. By combining DNA sequences and proteins reflecting a broad diversity of globally circulating HIV strains, the Mosaico team is hoping to give the body an immunological snapshot to prepare it to defend against any strain it might be exposed to. 

(Emiliano Rodriguez Mega, Nature)

 

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August 6, 2019 at 4:47 pm

Where to have a baby? Newborn screening is not created equal.

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By: Emily Wires, Ph.D.

Image by PublicDomainPictures from Pixabay 

In 2017, there were an estimated 3,855,500 live births in the United States, one of whom was my son. Within the first days of life, infants are tested for heritable disorders that can hinder physical and/or cognitive development. Unfortunately, the same set of potentially life-saving tests are not equal for all 3,855,500 infants. 

Since its conception in the 1960ss, newborn screening has been recognized as an essential preventative public health initiative. Serving as a frontline for early detection, conditions included on the screening panel are often considered rare and early intervention is essential to improved quality of life. The Newborn Screening Saves Lives Act was signed into law in 2008 and sought to bring public awareness among parents and health professionals as well as improve newborn screening programs.   

Currently, the panel of tested disorders varies state to state — yes, that means your baby’s health trajectory can be influenced by state lines. When considering birth location, most expecting parents grapple with the pros and cons of hospital versus birthing center versus home birth, but many are naïve to state regulations. Newborn screening standards, specimen collection, and follow-up procedures are implemented by state health departments. Contact information for newborn screening laboratories and follow-up program are available for individual states. Variations in statutes often extend beyond the panel of testable conditions, spilling over to funding for both screening programs and treatments, laboratory standards, and parents’ rights of refusal. To address discrepancies among states, the Department of Health and Human Services (HHS) has provided a Recommended Uniform Screening Panel (RUSP) that includes 34 core conditions and 26 secondary conditions and serves as a suggestive resource for state governments to incorporate as part of their screening programs. RUSP inclusion criteria stipulates a condition be identifiable 24-48 hours following birth, screening tests for the condition are available and validated, and the condition has been shown to benefit from early detection. Conditions included within RUSP are recommended by the Advisory Committee on Heritable Disorders in Newborns and Children along with the HHS Secretary. Despite these recommendations, not all states adhere to RUSP suggestions. 

Orphan drugs are intended to serve the rare disease community but are often commercially underdeveloped owing to their limited profitability. The Orphan Drug Act, passed in 1983, has since served as a legislative springboard set to improve the lives of individuals with rare diseases by incentivizing and accelerating drug discovery. Rare diseases are just that– rare, and because rare diseases are defined as disorders that affect less than 200,000 Americans at one time (with a sizeable proportion belonging to pediatric populations), pharmaceutical companies often expect to incur financial loss developing orphan drugs, which often means a treatment is not pursued. Collectively, however, rare diseases comprise a large healthcare need (an estimated 30 million people in the US are living with a rare disease) and have garnered increased public interest through the work of patient advocacy groupsresearch institutionsgrant funding opportunities, and public events such as Rare Disease Day

So, herein lies the dilemma. Are we essentially running a relay race but missing the handoff? Newborn screening assesses the presence of heritable conditions during a critical development period that otherwise may be missed and parallels an incentivized impetus for drug discovery, yet many states are dropping the proverbial baton regarding panel legislation. Consider, Spinal Muscular Atrophy (SMA) and its recent headlines in the rare disease community. SMA is a debilitating (and deadly), heritable disorder resulting in the loss of motor neurons and subsequent muscle atrophy. In 2016, the Food and Drug Administration approved Spinraza as the first-ever treatment of SMA. The American Academy of Neurology 2019 annual meeting recently reported data from 25 infants diagnosed with SMA at less than six weeks of age who received Spinraza prior to observable symptoms. By 14-34 months, none of these infants required permanent ventilation, all were able to sit and swallow without support, and most of them could walk on their own. However, despite this monumental advancement, not all states include SMA in their newborn screening panel. 

How do we become better for the rare disease community? Admittedly, a loaded question and likely multifaceted in approach. One potential approach would be to federally incentivize universal newborn screening across states. Screening implementation conditions typically vary due to financial constraints, frequency of disorder within state’s population, and treatment availability. California and Pennsylvania are currently the only states that with rare disease legislative caucuses. These bipartisan committees are comprised of individuals dedicated to bridging awareness among policy makers, scientists, patients, and the public. However, there are still discrepancies even among states with caucuses (California tests for 63 conditions, while Pennsylvania tests for 38), attesting to the critical necessity of a universal screening panel. 

Science is making vast strides in the detection and treatment of rare diseases. It is time for public health and policy makers follow suit in an appropriate manner, especially when time may be a crucial factor for our youngest rare disease patients

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August 2, 2019 at 2:38 pm

Science Policy Around the Web – July 18th, 2019

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By Silvia Preite

New therapeutic nutrition strategy corrects malnutrition by fostering a healthy gut

Malnutrition represents a major global health problem world-wide, causing nearly half of all deaths among children under 5 years of age. According to an estimate by UNICEF/WHO/World Bank, the number of children under 5, in March 2019, with wasting was more than 49 million. Moreover, impaired nutrition in the first 3 years of a child`s life can lead to failure to thrive, neurodevelopmental delay, and other long term health complications.

Commensal microbiota colonizing the gut is composed of a high number of bacteria, fungi and viruses, influences many normal body functions and is implicated in several health conditions, including obesity, autoimmunity and responses to cancer therapies. Two recent articles published in the journal Science report that specific food combinations promote the development of a mature gut microbiota that fights malnutrition and supports growth. As healthy children age from infants to toddlers, their gut microbiota composition progressively matures as well. In contrast, analysis of fecal samples from children in Bangladesh revealed that commensal bacteria remain immature in profoundly malnourished subjects. Animals reconstituted with immature microbiota showed impaired metabolism, less muscle formation, and weaker bones highlighting the importance of mature bacteria in the growth and development of children. 

Additionally, the authors found that a specific combination of food promotes healthy commensal bacterial that boosts body growth. Specifically, Microbiota-Directed Complementary Food (MDCF), containing chickpea, peanut and soy flour and raw banana, fostered microbiota maturation in mice and piglets transplanted with immature microbiota to a greater extent than a standard milk powder and rice-based Ready-to Use Supplementary Food (RUSF). The improved efficacy of MDCF compared to RUSF was further supported by a 1 month clinical trial conducted in 63 undernourished Bangladeshi children (12-18 months of age). Specifically, MDCF was more effective in promoting the engraftment of mature bacteria, resembling the ones in healthy children, and improved blood biomarkers associated with healthy development and immune function.

            Although the long-term consequences of these nutritional regimens remain to be assessed, these studies open opportunities for the use of new food supplements to help children recovering from malnutrition. Moreover, these discoveries can be used to improve the nutrition of well-fed children to support the development of healthy microbiota.

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July 18, 2019 at 9:59 am

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