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CRISPR and other new technologies open doors for drug development, but which diseases get prioritized? It comes down to money and science

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CRISPR and other new technologies open doors for drug development, but which diseases get prioritized? It comes down to money and science

So many diseases to treat, so little money and time.
Andriy Onufriyenko/Moment via Getty Images

C. Michael White, University of Connecticut

Prescription drugs and vaccines revolutionized health care, dramatically decreasing death from disease and improving quality of life across the globe. But how do researchers, universities and hospitals, and the pharmaceutical industry decide which diseases to pursue developing drugs for?

In my work as director of the Health Outcomes, Policy, and Evidence Synthesis group at the University of Connecticut School of Pharmacy, I assess the effectiveness and safety of different treatment options to help clinicians and patients make informed decisions. My colleagues and I study ways to create new drug molecules, deliver them into the body and improve their effectiveness while reducing their potential harms. Several factors determine which avenues of drug discovery that people in research and pharmaceutical companies focus on.

Funding drives research decisions

Research funding amplifies the pace of scientific discovery needed to create new treatments. Historically, major supporters of research like the National Institutes of Health, pharmaceutical industry and private foundations funded studies on the most common conditions, like heart disease, diabetes and mental health disorders. A breakthrough therapy would help millions of people, and a small markup per dose would generate hefty profits.

As a consequence, research on rare diseases was not well-funded for decades because it would help fewer people and the costs of each dose had to be very high to turn a profit. Of the more than 7,000 known rare diseases, defined as fewer than 200,000 people affected in the U.S., only 34 had a therapy approved by the Food and Drug Administration before 1983.

The passage of the Orphan Drug Act changed this trend by offering tax credits, research incentives and prolonged patent lives for companies actively developing drugs for rare diseases. From 1983 to 2019, 724 drugs were approved for rare diseases.

Person sluicing a bucket of ice water over another person's head
The viral ALS ice bucket challenge in 2014 was a fundraising success.
Elise Amendola/AP Photo

Emerging social issues or opportunities can significantly affect funding available to develop drugs for certain diseases. When COVID-19 raged across the world, funding from Operation Warp Speed led to vaccine development in record time. Public awareness campaigns such as the ALS ice bucket challenge can also directly raise money for research. This viral social media campaign provided 237 scientists nearly US$90 million in research funding from 2014 to 2018, which led to the discovery of five genes connected amyotrophic lateral sclerosis, commonly called Lou Gehrig’s disease, and new clinical trials.

How science approaches drug development

To create breakthrough treatments, researchers need a basic understanding of what disease processes they need to enhance or block. This requires developing cell and animal models that can simulate human biology.

It can take many years to vet potential treatments and develop the finished drug product ready for testing in people. Once scientists identify a potential biological target for a drug, they use high-throughput screening to rapidly assess hundreds of chemical compounds that may have a desired effect on the target. They then modify the most promising compounds to enhance their effects or reduce their toxicity.

When these compounds have lackluster results in the lab, companies are likely to halt development if the estimated potential revenue from the drug is less than the estimated cost to improve the treatments. Companies can charge more money for drugs that dramatically reduce deaths or disability than for those that only reduce symptoms. And researchers are more likely to continue working on drugs that have a greater potential to help patients. In order to obtain FDA approval, companies ultimately need to show that the drug causes more benefits for patients than harms.

YouTube video
Casgevy, a CRISPR-based treatment for sickle cell anemia, is considered a milestone in gene therapy.

Sometimes, researchers know a lot about a disease, but available technology is insufficient to produce a successful drug. For a long time, scientists knew that sickle cell disease results from a defective gene that leads cells in the bone marrow to produce poorly formed red blood cells, causing severe pain and blood clots. Scientists lacked a way to fix the issue or to work around it with existing methods.

However, in the early 1990s, basic scientists discovered that bacterial cells have a mechanism to identify and edit DNA. With that model, researchers began painstaking work developing a technology called CRISPR to identify and edit genetic sequences in human DNA.

The technology finally progressed to the point where scientists were able to successfully target the problematic gene in patients with sickle cell and edit it to produce normally functioning red blood cells. In December 2023, Casgevy became the first CRISPR-based drug approved by the FDA.

Sickle cell disease made a great target for this technology because it was caused by a single genetic issue. It was also an attractive disease to focus on because it affects around 100,000 people in the U.S. and is costly to society, causing many hospitalizations and lost days of work. It also disproportionately affects Black Americans, a population that has been underrepresented in medical research.

Real-world drug development

To put all these pieces of drug development into perspective, consider the leading cause of death in the U.S.: cardiovascular disease. Even though there are several drug options available for this condition, there is an ongoing need for more effective and less toxic drugs that reduce the risk of heart attacks and strokes.

In 1989, epidemiologists found that patients with higher levels of bad, or LDL, cholesterol had more heart attacks and strokes than those with lower levels. Currently, 86 million American adults have elevated cholesterol levels that can be treated with drugs, like the popular statins Lipitor (atorvastatin) or Crestor (rosuvastatin). However, statins alone cannot get everyone to their cholesterol goals, and many patients develop unwanted symptoms limiting the dose they can receive.

Two blister packs of burnt orange pills with days of the week listed on each dose
There are several statins on the market to treat high cholesterol levels.
Peter Dazeley/The Image Bank via Getty Images

So scientists developed models to understand how LDL cholesterol is created in and removed from the body. They found that LDL receptors in the liver removed bad cholesterol from the blood, but a protein called PCSK9 prematurely destroys them, boosting bad cholesterol levels in the blood. This led to the development of the drugs Repathy (evolocumab) and Praluent (alirocumab) that bind to PCSK9 and stop it from working. Another drug, Leqvio (inclisiran), blocks the genetic material coding for PCSK9.

Researchers are also developing a CRISPR-based method to more effectively treat the disease.

The future of drug development

Drug development is driven by the priorities of their funders, be it governments, foundations or the pharmaceutical industry.

Based on the market, companies and researchers tend to study highly prevalent diseases with devastating societal consequences, such as Alzheimer’s disease and opioid use disorder. But the work of advocacy groups and foundations can enhance research funding for other specific diseases and conditions. Policies like the Orphan Drug Act also create successful incentives to discover treatments for rare diseases.

However, in 2021, 51% of drug discovery spending in the U.S. was directed at only 2% of the population.. How to strike a balance between providing incentives to develop miracle drug therapies for a few people at the expense of the many is a question researchers and policymakers are still grappling with.The Conversation

C. Michael White, Distinguished Professor of Pharmacy Practice, University of Connecticut

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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Colors are objective, according to two philosophers − even though the blue you see doesn’t match what I see

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theconversation.com – Elay Shech, Professor of Philosophy, Auburn University – 2025-04-25 07:55:00

What appear to be blue and green spirals are actually the same color.
Akiyoshi Kitaoka

Elay Shech, Auburn University and Michael Watkins, Auburn University

Is your green my green? Probably not. What appears as pure green to me will likely look a bit yellowish or blueish to you. This is because visual systems vary from person to person. Moreover, an object’s color may appear differently against different backgrounds or under different lighting.

These facts might naturally lead you to think that colors are subjective. That, unlike features such as length and temperature, colors are not objective features. Either nothing has a true color, or colors are relative to observers and their viewing conditions.

But perceptual variation has misled you. We are philosophers who study colors, objectivity and science, and we argue in our book “The Metaphysics of Colors” that colors are as objective as length and temperature.

Perceptual variation

There is a surprising amount of variation in how people perceive the world. If you offer a group of people a spectrum of color chips ranging from chartreuse to purple and asked them to pick the unique green chip – the chip with no yellow or blue in it – their choices would vary considerably. Indeed, there wouldn’t be a single chip that most observers would agree is unique green.

Generally, an object’s background can result in dramatic changes in how you perceive its colors. If you place a gray object against a lighter background, it will appear darker than if you place it against a darker background. This variation in perception is perhaps most striking when viewing an object under different lighting, where a red apple could look green or blue.

Of course, that you experience something differently does not prove that what is experienced is not objective. Water that feels cold to one person may not feel cold to another. And although we do not know who is feeling the water “correctly,” or whether that question even makes sense, we can know the temperature of the water and presume that this temperature is independent of your experience.

Similarly, that you can change the appearance of something’s color is not the same as changing its color. You can make an apple look green or blue, but that is not evidence that the apple is not red.

Apple under a gradient of red to blue light
Under different lighting conditions, objects take on different colors.
Gyozo Vaczi/iStock via Getty Images Plus

For comparison, the Moon appears larger when it’s on the horizon than when it appears near its zenith. But the size of the Moon has not changed, only its appearance. Hence, that the appearance of an object’s color or size varies is, by itself, no reason to think that its color and size are not objective features of the object. In other words, the properties of an object are independent of how they appear to you.

That said, given that there is so much variation in how objects appear, how do you determine what color something actually is? Is there a way to determine the color of something despite the many different experiences you might have of it?

Matching colors

Perhaps determining the color of something is to determine whether it is red or blue. But we suggest a different approach. Notice that squares that appear to be the same shade of pink against different backgrounds look different against the same background.

Green, purple and orange squares with smaller squares in shades of pink placed at their centers and at the bottom of the image
The smaller squares may appear to be the same color, but if you compare them with the strip of squares at the bottom, they’re actually different shades.
Shobdohin/Wikimedia Commons, CC BY-SA

It’s easy to assume that to prove colors are objective would require knowing which observers, lighting conditions and backgrounds are the best, or “normal.” But determining the right observers and viewing conditions is not required for determining the very specific color of an object, regardless of its name. And it is not required to determine whether two objects have the same color.

To determine whether two objects have the same color, an observer would need to view the objects side by side against the same background and under various lighting conditions. If you painted part of a room and find that you don’t have enough paint, for instance, finding a match might be very tricky. A color match requires that no observer under any lighting condition will see a difference between the new paint and the old.

YouTube video
Is the dress yellow and white or black and blue?

That two people can determine whether two objects have the same color even if they don’t agree on exactly what that color is – just as a pool of water can have a particular temperature without feeling the same to me and you – seems like compelling evidence to us that colors are objective features of our world.

Colors, science and indispensability

Everyday interactions with colors – such as matching paint samples, determining whether your shirt and pants clash, and even your ability to interpret works of art – are hard to explain if colors are not objective features of objects. But if you turn to science and look at the many ways that researchers think about colors, it becomes harder still.

For example, in the field of color science, scientific laws are used to explain how objects and light affect perception and the colors of other objects. Such laws, for instance, predict what happens when you mix colored pigments, when you view contrasting colors simultaneously or successively, and when you look at colored objects in various lighting conditions.

The philosophers Hilary Putnam and Willard van Orman Quine made famous what is known as the indispensability argument. The basic idea is that if something is indispensable to science, then it must be real and objective – otherwise, science wouldn’t work as well as it does.

For example, you may wonder whether unobservable entities such as electrons and electromagnetic fields really exist. But, so the argument goes, the best scientific explanations assume the existence of such entities and so they must exist. Similarly, because mathematics is indispensable to contemporary science, some philosophers argue that this means mathematical objects are objective and exist independently of a person’s mind.

Blue damselfish, seeming iridescent against a black background
The color of an animal can exert evolutionary pressure.
Paul Starosta/Stone via Getty Images

Likewise, we suggest that color plays an indispensable role in evolutionary biology. For example, researchers have argued that aposematism – the use of colors to signal a warning for predators – also benefits an animal’s ability to gather resources. Here, an animal’s coloration works directly to expand its food-gathering niche insofar as it informs potential predators that the animal is poisonous or venomous.

In fact, animals can exploit the fact that the same color pattern can be perceived differently by different perceivers. For instance, some damselfish have ultraviolet face patterns that help them be recognized by other members of their species and communicate with potential mates while remaining largely hidden to predators unable to perceive ultraviolet colors.

In sum, our ability to determine whether objects are colored the same or differently and the indispensable roles they play in science suggest that colors are as real and objective as length and temperature.The Conversation

Elay Shech, Professor of Philosophy, Auburn University and Michael Watkins, Professor of Philosophy, Auburn University

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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Perfect brownies baked at high altitude are possible thanks to Colorado’s home economics pioneer Inga Allison

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theconversation.com – Tobi Jacobi, Professor of English, Colorado State University – 2025-04-22 07:47:00

Students work in the high-altitude baking laboratory.
Archives and Special Collections, Colorado State University

Tobi Jacobi, Colorado State University and Caitlin Clark, Colorado State University

Many bakers working at high altitudes have carefully followed a standard recipe only to reach into the oven to find a sunken cake, flat cookies or dry muffins.

Experienced mountain bakers know they need a few tricks to achieve the same results as their fellow artisans working at sea level.

These tricks are more than family lore, however. They originated in the early 20th century thanks to research on high-altitude baking done by Inga Allison, then a professor at Colorado State University. It was Allison’s scientific prowess and experimentation that brought us the possibility of perfect high-altitude brownies and other baked goods.

A recipe for brownies at high altitude.
Inga Allison’s high-altitude brownie recipe.
Archives and Special Collections, Colorado State University

We are two current academics at CSU whose work has been touched by Allison’s legacy.

One of us – Caitlin Clark – still relies on Allison’s lessons a century later in her work as a food scientist in Colorado. The other – Tobi Jacobi – is a scholar of women’s rhetoric and community writing, and an enthusiastic home baker in the Rocky Mountains, who learned about Allison while conducting archival research on women’s work and leadership at CSU.

That research developed into “Knowing Her,” an exhibition Jacobi developed with Suzanne Faris, a CSU sculpture professor. The exhibit highlights dozens of women across 100 years of women’s work and leadership at CSU and will be on display through mid-August 2025 in the CSU Fort Collins campus Morgan Library.

A pioneer in home economics

Inga Allison is one of the fascinating and accomplished women who is part of the exhibit.

Allison was born in 1876 in Illinois and attended the University of Chicago, where she completed the prestigious “science course” work that heavily influenced her career trajectory. Her studies and research also set the stage for her belief that women’s education was more than preparation for domestic life.

In 1908, Allison was hired as a faculty member in home economics at Colorado Agricultural College, which is now CSU. She joined a group of faculty who were beginning to study the effects of altitude on baking and crop growth. The department was located inside Guggenheim Hall, a building that was constructed for home economics education but lacked lab equipment or serious research materials.

A sepia-toned photograph of Inga Allison, a white woman in dark clothes with her hair pulled back.
Inga Allison was a professor of home economics at Colorado Agricultural College, where she developed recipes that worked in high altitudes.
Archives and Special Collections, Colorado State University

Allison took both the land grant mission of the university with its focus on teaching, research and extension and her particular charge to prepare women for the future seriously. She urged her students to move beyond simple conceptions of home economics as mere preparation for domestic life. She wanted them to engage with the physical, biological and social sciences to understand the larger context for home economics work.

Such thinking, according to CSU historian James E. Hansen, pushed women college students in the early 20th century to expand the reach of home economics to include “extension and welfare work, dietetics, institutional management, laboratory research work, child development and teaching.”

News articles from the early 1900s track Allison giving lectures like “The Economic Side of Natural Living” to the Colorado Health Club and talks on domestic science to ladies clubs and at schools across Colorado. One of her talks in 1910 focused on the art of dishwashing.

Allison became the home economics department chair in 1910 and eventually dean. In this leadership role, she urged then-CSU President Charles Lory to fund lab materials for the home economics department. It took 19 years for this dream to come to fruition.

In the meantime, Allison collaborated with Lory, who gave her access to lab equipment in the physics department. She pieced together equipment to conduct research on the relationship between cooking foods in water and atmospheric pressure, but systematic control of heat, temperature and pressure was difficult to achieve.

She sought other ways to conduct high-altitude experiments and traveled across Colorado where she worked with students to test baking recipes in varied conditions, including at 11,797 feet in a shelter house on Fall River Road near Estes Park.

Early 1900s car traveling in the Rocky Mountains.
Inga Allison tested her high-altitude baking recipes at 11,797 feet at the shelter house on Fall River Road, near Estes Park, Colorado.
Archives and Special Collections, Colorado State University

But Allison realized that recipes baked at 5,000 feet in Fort Collins and Denver simply didn’t work in higher altitudes. Little advancement in baking methods occurred until 1927, when the first altitude baking lab in the nation was constructed at CSU thanks to Allison’s research. The results were tangible — and tasty — as public dissemination of altitude-specific baking practices began.

A 1932 bulletin on baking at altitude offers hundreds of formulas for success at heights ranging from 4,000 feet to over 11,000 feet. Its author, Marjorie Peterson, a home economics staff person at the Colorado Experiment Station, credits Allison for her constructive suggestions and support in the development of the booklet.

Science of high-altitude baking

As a senior food scientist in a mountain state, one of us – Caitlin Clark – advises bakers on how to adjust their recipes to compensate for altitude. Thanks to Allison’s research, bakers at high altitude today can anticipate how the lower air pressure will affect their recipes and compensate by making small adjustments.

The first thing you have to understand before heading into the kitchen is that the higher the altitude, the lower the air pressure. This lower pressure has chemical and physical effects on baking.

Air pressure is a force that pushes back on all of the molecules in a system and prevents them from venturing off into the environment. Heat plays the opposite role – it adds energy and pushes molecules to escape.

When water is boiled, molecules escape by turning into steam. The less air pressure is pushing back, the less energy is required to make this happen. That’s why water boils at lower temperatures at higher altitudes – around 200 degrees Fahrenheit in Denver compared with 212 F at sea level.

So, when baking is done at high altitude, steam is produced at a lower temperature and earlier in the baking time. Carbon dioxide produced by leavening agents also expands more rapidly in the thinner air. This causes high-altitude baked goods to rise too early, before their structure has fully set, leading to collapsed cakes and flat muffins. Finally, the rapid evaporation of water leads to over-concentration of sugars and fats in the recipe, which can cause pastries to have a gummy, undesirable texture.

Allison learned that high-altitude bakers could adjust to their environment by reducing the amount of sugar or increasing liquids to prevent over-concentration, and using less of leavening agents like baking soda or baking powder to prevent dough from rising too quickly.

Allison was one of many groundbreaking women in the early 20th century who actively supported higher education for women and advanced research in science, politics, humanities and education in Colorado.

Others included Grace Espy-Patton, a professor of English and sociology at CSU from 1885 to 1896 who founded an early feminist journal and was the first woman to register to vote in Fort Collins. Miriam Palmer was an aphid specialist and master illustrator whose work crafting hyper-realistic wax apples in the early 1900s allowed farmers to confirm rediscovery of the lost Colorado Orange apple, a fruit that has been successfully propagated in recent years.

In 1945, Allison retired as both an emerita professor and emerita dean at CSU. She immediately stepped into the role of student and took classes in Russian and biochemistry.

In the fall of 1958, CSU opened a new dormitory for women that was named Allison Hall in her honor.

“I had supposed that such a thing happened only to the very rich or the very dead,” Allison told reporters at the dedication ceremony.

Read more of our stories about Colorado.The Conversation

Tobi Jacobi, Professor of English, Colorado State University and Caitlin Clark, Senior Food Scientist at the CSU Spur Food Innovation Center, Colorado State University

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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Why don’t humans have hair all over their bodies? A biologist explains our lack of fur

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theconversation.com – Maria Chikina, Assistant Professor of Computational and Systems Biology, University of Pittsburgh – 2025-04-21 07:33:00

Some mammals are super hairy, some are not.
Ed Jones/AFP via Getty Images

Maria Chikina, University of Pittsburgh

Curious Kids is a series for children of all ages. If you have a question you’d like an expert to answer, send it to CuriousKidsUS@theconversation.com.


Why don’t humans have hair all over their bodies like other animals? – Murilo, age 5, Brazil


Have you ever wondered why you don’t have thick hair covering your whole body like a dog, cat or gorilla does?

Humans aren’t the only mammals with sparse hair. Elephants, rhinos and naked mole rats also have very little hair. It’s true for some marine mammals, such as whales and dolphins, too.

Scientists think the earliest mammals, which lived at the time of the dinosaurs, were quite hairy. But over hundreds of millions of years, a small handful of mammals, including humans, evolved to have less hair. What’s the advantage of not growing your own fur coat?

I’m a biologist who studies the genes that control hairiness in mammals. Why humans and a small number of other mammals are relatively hairless is an interesting question. It all comes down to whether certain genes are turned on or off.

Hair benefits

Hair and fur have many important jobs. They keep animals warm, protect their skin from the sun and injuries and help them blend into their surroundings.

They even assist animals in sensing their environment. Ever felt a tickle when something almost touches you? That’s your hair helping you detect things nearby.

Humans do have hair all over their bodies, but it is generally sparser and finer than that of our hairier relatives. A notable exception is the hair on our heads, which likely serves to protect the scalp from the sun. In human adults, the thicker hair that develops under the arms and between the legs likely reduces skin friction and aids in cooling by dispersing sweat.

So hair can be pretty beneficial. There must have been a strong evolutionary reason for people to lose so much of it.

Why humans lost their hair

The story begins about 7 million years ago, when humans and chimpanzees took different evolutionary paths. Although scientists can’t be sure why humans became less hairy, we have some strong theories that involve sweat.

Humans have far more sweat glands than chimps and other mammals do. Sweating keeps you cool. As sweat evaporates from your skin, heat energy is carried away from your body. This cooling system was likely crucial for early human ancestors, who lived in the hot African savanna.

Of course, there are plenty of mammals living in hot climates right now that are covered with fur. Early humans were able to hunt those kinds of animals by tiring them out over long chases in the heat – a strategy known as persistence hunting.

Humans didn’t need to be faster than the animals they hunted. They just needed to keep going until their prey got too hot and tired to flee. Being able to sweat a lot, without a thick coat of hair, made this endurance possible.

Genes that control hairiness

To better understand hairiness in mammals, my research team compared the genetic information of 62 different mammals, from humans to armadillos to dogs and squirrels. By lining up the DNA of all these different species, we were able to zero in on the genes linked to keeping or losing body hair.

Among the many discoveries we made, we learned humans still carry all the genes needed for a full coat of hair – they are just muted or switched off.

In the story of “Beauty and the Beast,” the Beast is covered in thick fur, which might seem like pure fantasy. But in real life some rare conditions can cause people to grow a lot of hair all over their bodies. This condition, called hypertrichosis, is very unusual and has been called “werewolf syndrome” because of how people who have it look.

A detailed painting of a man and a woman standing next to one another in historical looking clothes. The man's face is covered in hair, while the woman's is not.
Petrus Gonsalvus and his wife, Catherine, painted by Joris Hoefnagel, circa 1575.
National Gallery of Art

In the 1500s, a Spanish man named Petrus Gonsalvus was born with hypertrichosis. As a child he was sent in an iron cage like an animal to Henry II of France as a gift. It wasn’t long before the king realized Petrus was like any other person and could be educated. In time, he married a lady, forming the inspiration for the “Beauty and the Beast” story.

While you will probably never meet someone with this rare trait, it shows how genes can lead to unique and surprising changes in hair growth.


Hello, curious kids! Do you have a question you’d like an expert to answer? Ask an adult to send your question to CuriousKidsUS@theconversation.com. Please tell us your name, age and the city where you live.

And since curiosity has no age limit – adults, let us know what you’re wondering, too. We won’t be able to answer every question, but we will do our best.The Conversation

Maria Chikina, Assistant Professor of Computational and Systems Biology, University of Pittsburgh

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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