Dr. Stanley Riddell’s team at the Seattle’s Fred Hutchinson Cancer Research Center earned themselves a place in medical history with the research they recently presented at the American Association for the Advancement for Science’s annual meeting. Riddell’s immunology team works with terminal patients. This new treatment engineers a patient’s own T-cells to target and fight back against the blood cancer cells that are attacking them–a major breakthrough in cancer research.

In the most recent trial, U.S. researchers used genetically modified T-cells in 35 terminally ill patients with leukemia, and 94 percent went into remission. Riddell’s T-cell research has only been applied to blood cancer but for the thousands of people in the United States alone suffering from blood cancer, this treatment could be the medical innovation they have waited years for. As with any new treatment, there is cause for caution–the data from this treatment is still being processed which means it still needs to be peer-reviewed and vetted by a host of evaluators. Furthermore, the risk involved in the treatment can be steep. For all the patients who witnessed major positive effects or full remission, there were several patients who were admitted to intensive care due to their treatment.

Cancer treatments are on the whole brutal and exhausting for the patient, but in this case, two of the participants in the study died because of adverse reactions to the treatment. It is important to remember that the patients for this trial were all terminal, which meant they were incredibly weak even as their newly trained T-cells tried to fight against the more aggressive cancer cells. However, members of the medical team believe they can minimize the dangers as time goes on by using lower doses of the therapy. This week, Dr Alan Worsley, from Cancer Research UK, told the BBC that while the field was incredibly exciting, “this is a baby step…the real challenge now is how do we get this to work for other cancers, how do we get it to work for what’s known as solid cancers, cancers in the tissue?”

Even though this research still has a long way to go before it becomes a typical cancer treatment, the attention that the stunning success rate has garnered will no doubt spark a wave of funding for similar T-cell therapy projects. There are dozens of top research institutions working around the clock to test experimental treatments and Riddell’s team is not the only one investigating the efficacy of engineering cells to fight off cancer attacks.

Nonprofits, corporate donors, and governments alike should all take note of this new T-cell trend and adjust cancer research funding accordingly. It can be difficult to divert funding into one particular branch of research when there are so many different forms of cancer that need cures, but if the results of T-cell training research continue to match the success rate of this study, the financial forces behind research hospitals may allocate more resources to this genetic branch of research.  Prioritizing treatment of one type of cancer over another may seem callous, but if this blood cancer treatment can be adapted to solid cancers then we may be looking at an actual cure for the disease as a whole–an opportunity it is difficult to ignore.

Jillian Sequeira

Jillian Sequeira was a member of the College of William and Mary Class of 2016, with a double major in Government and Italian. When she’s not blogging, she’s photographing graffiti around the world and worshiping at the altar of Elon Musk and all things Tesla. Contact Jillian at Staff@LawStreetMedia.com

The post Will the New T-Cell Treatment Change the Funding of Cancer Research? appeared first on Law Street.

Picture your Netflix homescreen. Besides some errant selections courtesy of your (ahem, tasteless) roommate, it’s pretty much a haven of your unique preferences. Like a doting butler, it recommends you watch “Breaking Bad” since you enthusiastically plowed through every episode of “Orange is the New Black.” Netflix knows you. Or think about Amazon. It’s your data-powered best friend. It recalls your purchase history and movie preferences better than you do. So what if this data-powered framework for knowing you is applied to healthcare? What if your doctor knows you as well as Netflix?

That’s what the Precision Medicine Initiative aims to do–unleash the full power of science and data to make our healthcare system better, more effective, and more specific to individuals and conditions. The new model proposes a system of health care that treats you like the complex human being you are. Just as Amazon cares deeply about your past purchase behavior, the new healthcare system would care about the science-based reasons you’re you: your genes, your lifestyle, and your environment. Instead of pushing purchases, it would use what it knows about you to determine what treatments and preventions work best for your health.

President Barack Obama announced the Precision Medicine Initiative during his 2015 State of the Union Address and since then people have been discussing the pros, cons, and implications. Here’s an overview of precision medicine and what it means for you.

What is precision medicine?

Take a look at the video below for a summary of precision medicine from Jo Handelsman, Associate Director for Science at the White House.

Precision medicine revolves around you. It uses your genes, environment, and lifestyle to determine what treatments keep you healthy.

The Precision Medicine Initiative may be new, but precision medicine has some history. Doctors already use it to treat conditions like cancer and Cystic Fibrosis. Examples of precision medicine in action include processes like blood typing and medications like Imatinib (Gleevec), a drug for Leukemia that inhibits an enzyme produced by certain genes. The new initiative plans to expand the reach of precision medicine to to tackle other diseases.

The plan stems from a  2011 report from the National Academy of Sciences. The report called out a major healthcare weakness: data suggests possible causes of deadly diseases, yet we don’t treat people until telltale signs and symptoms surface. You don’t wait until your friend’s liver is wrecked to stage an alcoholism intervention. Why wait for symptoms of a deadly disease when early risk factors might be available?

Great idea in theory, right? Of course, the execution promises far more complexity. Experts hope that precision medicine is within our grasp now because of recent scientific advances that make it easier to collect and analyze patient data.

Advances That Make Precision Medicine Possible

Advancement 1: New Methods of Uncovering Biological Data

It’s easier to understand patients and tumors on a cellular and genetic level more than ever before because of things like:

• The Human Genome Project, an initiative that aims to map the DNA sequence of the human genome to determine a sort of biological instruction manual for how humans function. The study of the genome is called genomics.
• Proteomics, a discipline that involves studying proteomes, the entire system of proteins in an organism. The goal is understanding changes, variations, and modifications in proteins over time to determine biomarkers for human diseases, especially cancer.
• Metabolomics, a field that leverages analytical tools to discover and quantify metabolites, which are substances produced by metabolism. Studying them provides experts with a glimpse of an organism’s physiological functioning as metabolism is a huge factor in overall health.

Advancement 2: New Tools For Biomedical Analysis

New analytic tools make it possible to decipher the intricate medical data collected by the disciplines above. Computers and programs help to collect, store, and study biological and medical information. Overall, the discipline is called bioinformatics.

Advancement 3: New Digital Health Tools That Make Large Datasets Manageable

I said large data sets. Sound familiar? Yes, we’re talking Big Data. You’ve probably heard enough about it, but it’s actually an amazing thing, especially when applied to healthcare. Take a look at the video below for more information.

From collecting to analyzing, sophisticated data management tools make the Precision Medicine Initiative possible.

Collectively, these advances create the right environment for the unified national effort that the Precision Medicine Initiative proposes.

How will it work?

The President’s 2016 Budget provides $215 million for the program. Four key agencies slated to do a bulk of the work each get a chunk of the budget.

National Institutes of Health (NIH)

Project Budget: $130 million.

Task: Recruit a volunteer research cohort and leverage existing data.

The National Institutes of Health must find 1 million American volunteers willing to provide medical records, gene profiles, lifestyle data, and more. While data drives the initiative, you need people to get the data. In addition to this, the NIH will find existing studies and research to build a foundation for the initiative. It’ll collaborate with stakeholders to determine approaches for collecting patient information.

National Cancer Institute (NCI)

Budget: $70 million.

Task: Find better cancer treatments.

The National Cancer Institute will explore precision treatments for cancer by increasing genetically based cancer trials, researching cancer biology, and establishing a “cancer knowledge network” to inform treatment decisions.

Food and Drug Administration (FDA)

Budget: $10 million.

Task: Develop safe, new DNA tests.

The Food and Drug Administration will seek technologies that rapidly sequence DNA and the human genome. Tests should make genetic data collection easier and more standardized.

Office of the National Coordinator for Health Information Technology (ONC)

Budget: $5 million.

Task: Manage the data.

The ONC has a tough job. It needs to figure out how to store, use, access, and exchange all of this medical data without any privacy concerns.

What Precision Medicine Could Mean For You

Here’s Notre Dame’s video on precision medicine in action:

Precision medicine could mean treatments more specific to you. For example, about 55-65 percent of women with mutations in the BRCA1 gene get Breast Cancer; only 12 percent of those without the gene get it. If the gene mutation is discovered, doctors can recommend enhanced prevention measures like increased cancer screenings or prophylactic surgery to remove at-risk tissue.

We hope more precise treatments lead to better outcomes. Using precision medicine, we hope to answer many questions, including:

• How can we treat this better?
• Is there a cure?
• Why does this disease happen in the first place?

The Downsides to Precision Medicine

Of course, the Precision Medicine Initiative has some drawbacks. The sheer amount of time it will take to collect and analyze all of this patient data leads the charge of negative comments. Below are some other downsides.

Interpretability

This article from the New Yorker calls out the problem of interpretability. To quote the author,l Cynthia Graber,

Many doctors are simply not qualified to make sense of genetic tests, or to communicate the results accurately to their patients.

Since doctors will be the sole executors of the initiative, more need to become fluent in the human genetic code. Programs like MedSeq have recognized this need and are already working to make genetic information translatable for practitioners.

The Budget Just Isn’t Enough

Experts say that even the $215 million proposed isn’t enough to meet the initiative’s lofty goals, like recruiting one million patient volunteers. One upside? Money can be saved by incorporating existing data, which the initiative plans to do.

Collecting the Data is Going to be Hard (This is an Understatement) 

If they do save money by integrating data from different studies, keeping the data clean will be hard considering the different time frames, constructs, and controls of various studies.

And as a practicing doctor writing for a New York Times blog points out, the lifestyle factors will be especially hard to study because of some uncooperative and intensely complex patients.

Insurance Companies May Not Pay For It

Precise matching of individuals to disease treatments sounds great, and extremely expensive, especially in the early days. Patients will need even more help determining what treatments suit them.

Hope For the Future

Sorry to bring up Netflix up again, but let’s face it, it’s very good at leveraging data to give you what you want. Consider any of its popular original series. Do you think Netflix just guessed what 50 million subscribers would like? Probably not. It used its massive stores of data to make informed decisions.

Early doctors and researchers puzzled over the symptoms of just a few patients, trying to find patterns, causes, and cures. While they did a fair job with the resources they had, trial and error medicine should be relegated to the less fortunate past. Today we have the power and knowledge to access data that helps doctors make more informed decisions on healthcare treatments.

Precision medicine will be complicated, difficult, time consuming, and who knows what else. But imagine what we can learn. We should be cautious, but we can also dare to hope.

Resources

Primary

White House: Infographic: The Precision Medicine Initiative

White House: FACT SHEET: President Obama’s Precision Medicine Initiative

White House: Precision Medicine is Already Working to Cure Americans: These Are Their Stories

National Cancer Institute: BRCA1 and BRCA2: Cancer Risk and Genetic Testing

National Institutes of Health: Precision Medicine Initiative

National Cancer Institute: What is Cancer Proteomics?

Additional

Nature: Obama to Seek $215 Million for Precision-Medicine Plan

New England Journal of Medicine: A New Initiative on Precision Medicine

National Academies: Toward Precision Medicine

National Institutes of Health: Precision Medicine Initiative

Nature: U.S. Precision-Medicine Proposal Sparks Questions

Brookings Institution: The Significance of President Obama’s Precision Medicine Initiative

New Yorker: The Problem With Precision Medicine

The New York Times: A Path For Precision Medicine

National Human Genome Research Institute: What is the Human Genome Project?

BioTechniques: What is Metabolomics All About?

Bioplanet: What is Bioinformatics?

Ashley Bell

Ashley Bell communicates about health and wellness every day as a non-profit Program Manager. She has a Bachelor’s degree in Business and Economics from the College of William and Mary, and loves to investigate what changes in healthy policy and research might mean for the future. Contact Ashley at staff@LawStreetMedia.com.

The post Precision Medicine: The Future of Health Care? appeared first on Law Street.

Is whether or not you survive Ebola all about your genetics? A new study on mice indicates that it might be. Scientists found that certain genetic factors determine if the disease manifests as mild or devastating.

To reach this conclusion, scientists injected mice with the same strain of Ebola that caused the 2014 West Africa Outbreak. The expressed severity of the disease among the mice was scattered although they were all injected with the same unaltered and unmutated strain. Why did some resist the disease while others surrendered?

One correlation posits a provocative answer. Scientists noticed a strong correlation between symptom expression and the genetic lines of the mice. Dr. Michael Katze, a researcher on the project, declared that their data suggest disease outcomes are largely dependent on genetic factors.

It seems that the genes of the mice determined their immune response. In some mice, the genes that promote blood vessel inflammation and cell death became agitated and ultimately these mice succumbed to the disease. In other mice, the white blood cells were more lively and the genes that promote blood vessel repair were activated. These mice were able to fight back. As they observed the mice over multiple generations, they found that the ability to survive was tied to genetic lines. The continuous correlation of immunity in genetic lines presents a puzzle. Did the mice pass specific immunity on to their offspring?

Immune responses to specific pathogens, like Ebola, only develop after exposure. Specific immunity is an acquired trait, and so far, science has told us that acquired traits cannot be passed on through DNA. Traits we acquire in our lifetimes are not written into DNA and therefore not built into genes. Acquired traits result from environmental influences, like memories or even tans. If you’re a bronze goddess while pregnant, you won’t have a baby with a gorgeous tan.

So it is intriguing to think that mice who were exposed to Ebola had somehow passed on their specific, acquired, immunity to offspring through their genes. Below we’ll explore the possibility of inheriting acquired immunity.

Your Two-Sided Immune System

We’re all born with an innate immune system. It’s responsible for the classic immune response that recognizes and eliminates foreign invaders with the help of killer cells and cytokines. Skin, mucus, cells, and molecules all present at birth innately protect your body from foreign pathogens. Think of any computer you buy. It comes with a built in operating system. But that doesn’t mean you can’t upgrade, right?

Environmental factors prompt us to make little upgrades to our basic innate immune system like we do to our computer’s operating system. This is called adaptive or acquired immunity. Adaptive immunity activates in response to a specific problem that the innate immune system isn’t able to overcome. As it works, it also forms memories, so it can remember how to fight a specific pathogen if it ever returns for vengeance. A classic example is the Chickenpox. It doesn’t take much for most people to catch it the first time, but after that, many are resistant for life.

Acquired immunity, like other acquired traits, is not inherited. Even though you might have had the chickenpox, your kid will probably still get it, just like they can’t inherit your amazing tan or stellar vocabulary. With that said, we return again to the mice in the study above. Is it possible that they passed on their acquired immunity to their offspring?

“Lamarck-y” malarkey! Or maybe not….

If you’re intrigued by the study above, one historical figure would be absolutely riveted. Jean Baptiste Lamarck had this idea a long time ago — in 1801 to be specific. He theorized that evolution takes place when species develop traits to adapt to their environment and then transmit those adaptations to their offspring. Per his theory, giraffes developed long necks to feed from the tallest trees and then passed the “long neck” trait to their offspring.

Somebody else thought that evolution occurred in a different way. Charles Darwin proposed that evolution occurs through random mutations that bestow a competitive advantage for survival over a long time. Per his theory, the giraffes didn’t develop long necks to feed. It was just that the giraffes that happened to have slightly longer necks were able to survive to make more offspring. Eventually, the long neck became a dominant feature of all giraffes.

Darwin’s theory eclipsed Lamarck’s as the favorite theory of evolution. But were there some nuggets of truth in Lamarck’s musings? A growing body of evidence is creating a whisper of renewed interest in Lamarckian evolution. Collectively, it’s a young field called epigenetics.

For example, observations of starving Dutch mothers during the famine of World War II revealed that they had offspring and grandchildren more susceptible to obesity. Experiments on rats have found that obesity in mice might be caused by the high fat diets of their fathers. And there’s more where that came from.

The proof is in…the roundworm?

Dr. Oliver Hobert was curious to find out if Lamarck might have been right about the heritability of acquired traits. He suspected that ribonucleic acid, or RNA, and its role in genetic expression might shed some light on the subject.

Hobert was specifically interested in RNA interference (RNAi). Cells use RNAi to turn down or suppress certain genes. Watch the video below to see how it works.

Hobert and his team of Columbia University Medical Center (CUMC) researchers turned to roundworms to study RNAi’s influence on immunity. Roundworms have a unique capacity to battle viruses using RNAi that made them ideal for the study. The team found that a RNA molecule memory of instructions on fighting off certain viruses could be passed on from one generation of roundworms to the next.

Here is a quote from Dr. Oded Rechavi, lead author of the study, courtesy of the CUMC newsroom:

In our study, roundworms that developed resistance to a virus were able to pass along that immunity to their progeny for many consecutive generations.The immunity was transferred in the form of small viral-silencing agents called viRNAs, working independently of the organism’s genome.

More Pieces in the Puzzle

Studies like this one give scientists pause on long standing notions about the heritability of acquired traits and what we know about our genes. While many more studies are needed to completely vindicate Lamarck and his ideas, some puzzling clues are coming together. Here are some highlights from other studies that tackle similar ideas:

SardiNIA Study of Aging: Researchers at the National Research Council’s Institute of Genetic and Biomedical Research in Italy found that genetics play a key role in our ability to fight off disease. According to the study, the immune system has evolved to reject certain pathogens and cancers. The basis of the study is that several adaptive immune cells are regulated by genetics. They found 89 gene variants with significant ties to the production of specific immune system cells.

Chief of NIA’s Laboratory of Genetics, David Schlessinger, Ph.D., sums it up nicely:

If your mother is rarely sick, for example, does that mean you don’t have to worry about the bug that’s going around? Is immunity in the genes? According to our findings, the answer is yes, at least in part.

Natural Environment Research Council UK: This study demonstrates that genetic variations in cytokines are a crucial component of individual variation in pathogen resistance and immune function. During both adaptive and innate immune responses, cytokines carry messages. They directly determine how an immune system will respond to a given challenger. So variations in the genes that control these cytokines, therefore, ultimately affect the immune system.

Analysis of Genetic Variation in Animals: A study of hemophiliac individuals infected with HCV showed that genetic factors determine the outcome of the disease. The researches studied siblings and found correlative rates of disease recovery among siblings was much higher than the pairs of randomly paired individuals, concluding that people who share genes might also share higher resistance to certain diseases.

Innate Immune Activity: Another study looked at the genome sequence that regulates expression of genes involved in the immune system. The study found that sometimes genes of interest reveal themselves when certain cells involved in fighting an infection are stimulated.

Back to the E-word…

Ebola usually depletes a person’s immune cells. Some immune systems stand up against the initial attack and their bodies are able to maintain some immune cells. These people are more likely to survive. We learned from the study on mice that it could be genetic factors that determine the disease outcome. What about people?

One study found that people with certain variations of the human leukocyte antigen-B  gene survived Ebola while those with another variation did not. Another finding deals with a mutation in the NPC1 gene. Cells taken from people with this gene are resistant to Ebola. The mutation is relatively common in certain populations in Europe and Nova Scotia.

More research is needed, but studying these genetic variances might reveal more secrets of why some survive Ebola and others do not.

Immuno Synergy

These findings do more than just play with our ideas of how traits can be inherited. If doctors were able to browse through your genetic catalog of specific pathogen resistance, they could administer therapies that create synergies among treatments. We might be able to predict what ailments you’re more susceptible to and take appropriate preventive actions. We might be able to study the genetic factors that make some people resistant to illnesses like Ebola, and synthesize them to construct even more effective treatments.

Is this science fiction? We don’t know yet, but no theories should be completely forgotten. As we’ve learned from Lamarck, even formerly discarded ideas can make a splash centuries after their inception.

 Resources

Primary

PLOS Genetics: Genetic Diversity in Cytokines Associated with Immune Variation and Resistance to Multiple Pathogens in a Natural Rodent Population

The Royal Society: Variation in Immune Defence as a Question of Evolutionary Ecology

NIH: Genetic Variability of Hosts

University of Western Australia: Genetic Variation of Host Immune  Response Genes and Their Effect on  Hepatitis C Infection and Treatment Outcome

Additional

Science Daily: Genetic Factors Behind Surviving or Dying From Ebola Shown in Mouse Study

Broad Institute: Scientists Make Connection Between Genetic Variation and Immune System in Risk for Neurodegenerative and Other Diseases

Wellcome Trust Centre for Human Genetics: Study Tracks Effects of Immune Activity Across the Genome

MNT: Immune Response Determined by Our Genes, Study Shows

History of Vaccines: Viruses and Evolution

LiveScience: How Do People Survive Ebola?

Research Gate: What is the Scientific Position on the Inheritance of Acquired Characteristics (Lamarckism)?

Ashley Bell

Ashley Bell communicates about health and wellness every day as a non-profit Program Manager. She has a Bachelor’s degree in Business and Economics from the College of William and Mary, and loves to investigate what changes in healthy policy and research might mean for the future. Contact Ashley at staff@LawStreetMedia.com.

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