Three scientists whose groundbreaking work redefined the future of cystic fibrosis (CF) care have been awarded one of the world’s most prestigious honors in medicine: the Lasker-DeBakey Clinical Medical Research Award. Often referred to as the “American Nobel,” the award recognizes contributions that radically improve human health. This year, it went to Dr. Michael Welsh of the University of Iowa, Paul Negulescu of Vertex Pharmaceuticals, and Jesús (Tito) González, now of Integro Theranostics.

Together, their decades of research led to the creation of Trikafta, a therapy that has extended the lifespan of cystic fibrosis patients by decades and fundamentally reshaped what it means to live with the disease.

When cystic fibrosis was first described in the 1930s, it was considered a fatal childhood condition. Patients rarely survived past elementary school. Even as late as the 2010s, before Trikafta’s approval in 2019, half of patients with CF died before the age of 40.

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Today, the outlook is dramatically different. Children born with CF between 2020 and 2024 who have access to Trikafta now face a median survival age of 65 years — nearly indistinguishable from the general population.

As Dr. Eric Sorscher of Emory University explained in The New England Journal of Medicine, “Available projections suggest that health and longevity may increase further as modulators begin to be administered at younger ages.”

This shift marks one of the most profound turnarounds in modern medicine.

The Role of the CFTR Gene

Cystic fibrosis is caused by mutations in a single gene: CFTR (cystic fibrosis transmembrane conductance regulator). The gene is critical for regulating the movement of ions across cell membranes, which in turn ensures proper water balance in tissues.

In healthy cells, CFTR forms channels that allow ions to flow freely. But in CF, the mutated gene produces faulty channels. The result is thick, sticky mucus that clogs the lungs and digestive system, fuels recurrent infections, and damages organs over time.

Dr. Michael Welsh, a pulmonologist and molecular biologist, helped illuminate the exact ways the most common CF mutation, delta-F508, disrupts cell function. He discovered two problems: the defective protein often gets “trapped” inside cells before reaching the surface, and even when it does reach the surface, it underperforms.

In a pivotal experiment, Welsh showed that simply lowering the temperature of cells allowed the trapped protein to move correctly. “That meant it was not totally broken,” he later recalled — a crucial realization that opened the door to correcting the defect.

Meanwhile, as a postdoctoral researcher in Nobel laureate Roger Tsien’s lab, Jesús (Tito) González developed a real-time system to track ion movement across membranes. Initially designed to study neurons, this tool proved invaluable for testing whether new drugs could restore CFTR function.

Negulescu and the Push to Find a Therapy

At Vertex Pharmaceuticals, Paul Negulescu helped drive the systematic search for compounds that could repair CFTR defects. Guided by Welsh’s molecular insights and González’s imaging system, the team screened thousands of molecules. The result was Trikafta, a triple-drug therapy that addressed the underlying cause of CF for most patients.

'Trikafta' Of Game-Changer in CF Treatment

Approved by the FDA in 2019, Trikafta combines three drugs — elexacaftor, tezacaftor, and ivacaftor — that work synergistically to help defective CFTR proteins fold correctly, reach the cell surface, and function effectively. The impact has been extraordinary. Since its introduction:

• Hospitalizations for lung infections have plummeted.
• The need for lung transplants has declined.
• Patients report stronger lung function, weight gain, and dramatically improved quality of life.
• For many families, the treatment has meant that milestones once unimaginable — graduating college, starting families, living into retirement — are now within reach.

Recognition at the “American Nobels”

The Lasker Awards, founded in 1945, celebrate biomedical achievements that shape the future of health. They are considered one of the highest honors in science, often predicting future Nobel Prizes.

The recognition of Welsh, González, and Negulescu underscores the profound impact of their work. The $250,000 prize, while symbolic compared to the billions Trikafta has generated, highlights the ethical and humanitarian dimension of their achievement: turning a once uniformly fatal disease into a chronic, manageable condition.

What CF Treatment Means for Medicine?

The CF breakthrough is not just about one disease. It represents a paradigm shift in genetic medicine. By targeting the root molecular defect rather than simply managing symptoms, Trikafta has become a model for other genetic conditions, from sickle cell disease to rare metabolic disorders.

It also illustrates the power of partnerships between academic researchers, biotech innovators, and patient foundations. The Cystic Fibrosis Foundation’s early investments in research were critical to advancing the work that ultimately led to Trikafta’s approval.

While Trikafta has transformed care in wealthy countries, challenges remain. The therapy is expensive — with an annual price tag of over $300,000 in the U.S. — putting it out of reach for many patients globally.

Furthermore, a subset of CF patients with rare genetic mutations still do not benefit from the drug, leaving an urgent need for alternative therapies. And as with all long-term treatments, researchers must continue monitoring for side effects and resistance.

Roughly 100,000 people worldwide live with cystic fibrosis. For decades, their lives were defined by daily medical regimens, frequent hospitalizations, and shortened lifespans. Today, thanks to the pioneering work of Welsh, González, and Negulescu, those same patients are looking toward futures filled with possibility.

It is no news that all appointments in the United States' Health Departments that have been made under RFK Jr are of the people who have been vaccine critiques. In a similar scenario, Dr Ralph Abraham, a state surgeon from Louisiana, who had ordered health officials to stop promoting mass vaccination will now serve as the US Centers for Disease Control and Prevention's (CDC) principal deputy director.

It was in February when Abraham instructed health department staff to stop promoting vaccines for preventable diseases.

He wrote in a February 13 internal memo that although patients should continue speaking with their providers about the risks and benefits of vaccination, the health department will stop promoting mass vaccination. The memo was issued on the same day Robert F. Kennedy Jr. was sworn in as secretary of the US Department of Health and Human Services. While his hiring has not been announced by HHS, but his new role has been confirmed. It was first reported by health newsletter Inside Medicine.

Who Are Part Of The CDC?

As of now the CDC does not have a permanent director. Kennedy ousted Dr Susan Monarez from the role in August. Jim O'Neill, who is serving as the deputy HHS secretary said this month that the agency has had "mission creep" and needs to focus on its original mandate.

“We want to … take the people we have and put them to their best use. And secondly, we are always recruiting. We are eager to hire wonderful scientists and data engineers and AI engineers and researchers and drug reviewers across the department, including CDC. If you are talented, you care about health or human services – please come work with us.”

What More Do We Know About Ralph Abraham?

The news of his appointment comes days after the CDC changed its guidelines on vaccines on its website. The CDC website now links vaccines with autism and states that "vaccines do not cause autism is not an evidence-based claim".

While Abraham has not outrightly been anti-vaccine, he had asked the health staff to not promote it for preventable diseases or infection and has long echoed Kennedy's 'Make America Healthy Again' (MAHA) rhetoric. He had questioned trust in the pharmaceutical industry and public health institutions.

“The solution to increased spending and declining outcomes in our country is unlikely to come in the form of a pill or a shot,” Abraham said in a February statement. “Much of the solution will likely come down to the usual hard work of improving diet, increasing exercise, and making better lifestyle choices.”

Changes In CDC

Apart from the guidelines being changes on autism and vaccine link, in August, Kennedy dismissed all members of the CDC's Advisory Committee on Immunization Practices (ACIP), the body whose recommendations form the basis of state immunization mandates.

Just days later, eight new members were named—though one withdrew amid ethics concerns. Unlike their predecessors, this panel includes only one epidemiologist and one infectious disease specialist, with no virologists or immunologists. Many appointees lack peer-reviewed vaccine research altogether, and those who do have published, on average, 78% fewer vaccine-related papers than former members.

The long-standing practice of adding fluoride to community drinking water in the United States is now under sharp review as questions rise about whether its advantages continue to outweigh possible risks. A fresh set of findings pushes back against recent warnings about fluoride in water and suggests that it might offer added benefits.

This renewed national discussion began after a government report from the National Toxicology Program stated that high fluoride exposure was tied to lower IQ in children. That report, however, assessed fluoride amounts that were at least twice higher than federal recommendations and had limited information about what happens at lower, commonly used levels, as per CNN.

However, the latest study examined fluoride quantities that match the usual suggested range in drinking water. Researchers found strong evidence showing that children who grew up with water containing these lower levels of fluoride performed better on cognitive assessments than those who had no fluoride exposure.

Fluoride in US Public Water Faces Renewed Questions

Dr. Rob Warren, lead author of the study released in Science Advances, said he was surprised by the National Toxicology Program’s earlier conclusions and felt the need to produce evidence more suitable for public policy. He explained that he pursued the work because it was a major question without a clear answer.

The national debate has also intensified as US Department of Health and Human Services Secretary Robert F. Kennedy Jr. has labeled fluoride an industrial waste and pointed to possible IQ loss while calling for a rollback of federal guidance. Utah and Florida have already moved to prohibit fluoridation of public water.

As per CNN, Warren compared the situation to testing a heart medication. If the advised dose is 100 milligrams, but a study measures reactions to nearly a million milligrams, the results do not reflect what happens at the normal dose. He said that this is how much of the fluoride research has been structured and that only extremely high doses have shown harmful effects, which is not helpful for policy decisions.

Warren directs a long-running program that began in the US Department of Education and has followed tens of thousands of Americans from their high school years in the 1980s through several decades.

Fluoride and Cognition: What The Study Found

For the current analysis, the team linked math, reading and vocabulary test scores from nearly 27,000 participants to the level of fluoride in their childhood drinking water. These measurements were based on older records from the US Geological Survey and the Department of Health and Human Services, as per CNN.

Researchers worked under the assumption that participants spent their entire upbringing near their high schools. They sorted people into three groups. One group had steady exposure to recommended fluoride levels either through natural sources or public water treatment. Another group never had fluoride in their water. A third group had mixed exposure because their community changed its water policy at some point.

Students who had fluoride for only part of their childhood scored higher on tests than peers who never had it. Those who grew up with fluoride throughout all their childhood years scored even better. Follow-up testing that continued up to 2021, when many had reached about 60 years of age, also showed no sign that fluoride contributed to cognitive decline.

Warren clarified that cognitive tests are not exact IQ scores, although they relate strongly. Test results reflect both mental ability and the learning opportunities a person receives. He is currently working on a follow-up project that will look more closely at fluoride and IQ with improved childhood location data.

Other studies this year suggested that removing fluoride from public water in the United States could lead to more than 25 million extra cavities among children and teenagers in five years along with nearly 10 billion dollars in dental treatment costs. Although the new study did not measure dental health, experts noted that pain from untreated cavities can interfere with a child’s ability to attend school or stay focused, which may influence academic scores.

Why Experts Still Differ on Fluoride Policy

Fluoride occurs naturally in some groundwater and foods. It protects teeth by strengthening enamel, which can be damaged by acids formed from bacteria, plaque and sugar. Communities in the United States started adding fluoride to water systems in 1945 to improve oral health in a cost-effective way.

The American Dental Association and numerous specialists continue to support community water fluoridation. The US Centers for Disease Control and Prevention has also kept its recommendations unchanged. The agency does not have authority to require fluoridation, but it considers 0.7 milligrams per liter the ideal amount.

Recently, the US Food and Drug Administration restricted the use of prescription fluoride supplements. The agency noted that unapproved fluoride products may alter the gut microbiome and that better options exist to protect teeth.

In a written response published with the new research, Dr. David Savitz from Brown University argued that before ending a decades-long public health practice, there must be clear proof of harm at commonly used fluoride levels. He wrote that there is still no convincing evidence that fluoridation lacks benefit or causes damage at recommended doses.

He quoted a well-known saying, noting that if something is not broken, there is no reason to fix it. He said the new study suggests that fluoridated water remains on the side of being effective and safe.

The Neuralink chip is a piece of technology that’s hoped will one day allow people to operate devices like phones and computers using their thoughts. Its creator, tech mogul Elon Musk, describes it as a “Fitbit in your skull with tiny wires.” Elon Musk's brain implant company Neuralink said recently that 12 people worldwide have received its chips. The device is meant to have several applications, from restoring motor functionality within people to enabling a brain-computer interface. The question now is whether it will be able to reverse paralysis in the future or not.

Neuralink: What Is Neuralink, And What Does It Do?

Neuralink is a neurotechnology company Musk set up in 2016. The device is roughly the size of a coin and is implanted into the skull, with hair-thin threads placed inside specific parts of the brain to form a working brain-computer interface.

The implant records brain signals and transfers them to an external device, such as a smartphone, through Bluetooth. Its first product, Telepathy, is designed to let a person operate their phone or computer through intention alone. By placing the chip in regions that manage movement, Neuralink believes it could help people living with neurological conditions. Musk has said that early users would likely be people who cannot use their limbs.

Neuralink: What Testing Has Neuralink Carried Out In The Past?

As per Reuters, the company has run trials using monkeys and pigs. Demonstrations have featured monkeys moving a cursor or playing simple on-screen games using the implant. Neuralink maintains that no monkey died because of the device itself, though reports have circulated describing complications in some animals, including paralysis, seizures and swelling in the brain.

Neuralink: Can Neuralink Cure Paralysis?

The human brain contains millions of neurons responsible for movement, emotion and thought. These neurons send electrical signals down pathways that run from the brain to the rest of the body. If a person tries to stand up, the brain sends electrical instructions to the legs, and the reaction is instant. Paralysis develops when pathways in the spinal cord are damaged or blocked, according to the description on Neuralink’s website.

Because the Neuralink implant reads the brain’s signals, it can forward those signals to the limbs even when the spinal cord no longer relays them. This would require one implant in the brain and another placed below the injured area of the spinal cord. With steady training, a person could regain movement, raising the possibility that Neuralink may one day help restore mobility.

Neuralink Ethical Concerns

Experts have questioned the company’s experiments on animals and the general risks linked to brain operations, which can include seizures or bleeding. They have also pointed to worries about data privacy and long-term surveillance, since there is limited detail on how much control users will have over their neural information.

Earlier this year, the US Department of Transportation fined the company for failing to register as a carrier of hazardous biological material, including implants removed from primates, according to agency records reviewed by Reuters.

Neuralink Is FDA Approved

In late May 2023, the FDA granted Neuralink permission to start human testing. The company said on Twitter that the approval marked the beginning of its first clinical study in people, calling it a vital step toward making the technology accessible in the future.

Neuralink has continued to share updates, and in July 2025, it announced that it had completed two brain-implant procedures on the same day. Both individuals are said to be recovering well. Musk responded soon after, expressing confidence in what the technology might achieve in the coming years.