The United States Food and Drug Administration has recently approved cabozantinib (Cabometyx) for patients with previously treated advanced neuroendocrine tumors. This is an oral tyrosine kinase inhibitor, which is offering a new standard for care of this patient group. This has been announced by the Dana-Farber Cancer Institute.

How Was The Drug Approved?

The approval came on the bases of the results from the phase 3 CABINET study. The study compared cabozantinib to a placebo in patients with advanced pancreatic neuroendocrine tumors.

As per the National Cancer Institute, neuroendocrine tumor grows from neuroendocrine cells. These cells receive and send messages through hormones to help the body function. These cells are found in organs throughout the body. The neuroendocrine tumor was called carcinoid tumor

These tumors grow very slowly and are mostly found in appendix, also known as appendiceal neuroendocrine tumors. When found in lungs, they are also called bronchial tumors. These areas are more common in children and young adults. Whereas for adults, it is mostly found in the digestive tract, called the GI neuroendocrine tumor. What makes it concerning is that it can also spread to other parts of the body but does so more in adults than in children.

They are rare in children and more common in adults, affecting up to 4 in 100,000 adults.

The drug which FDA has approved based on the phase 3 CABINER study's results have been published last September in the New England Journal of Medicine. Patients who were treated with cabozantinib survived significantly longer with no worsening of their disease compared with patients who received placebo. This also improved the efficacy as was observed in the interim analysis. The trial was in fact terminated early and unblinded in August 2023.

Are There Any Side Effects From The FDA Approved Drugs?

The reported side effects of cabozantinib were similar to those found in other studies of the drug. They include:

• Hypertension
• Fatigue
• Diarrhea

"Patients with neuroendocrine tumors often face a difficult journey," lead author of the CABINET study, Jennifer Chan, M.D., M.P.H., of the Dana-Farber Cancer Institute, said in a statement. "Despite advances in recent years, there has remained a critical need for new and effective therapies for patients whose cancer has grown or spread. Cabozantinib significantly improved outcomes in this patient population and this FDA approval provides new hope."

How Is Neuroendocrine tumor diagnosed?

While some people have symptoms, others may do not have any symptoms. These symptoms depends on where in your body the tumor has grown.

Patients who have appendiceal neuroendocrine tumors may have symptoms of appendicitis, such as pain in abdomen. They could also be later diagnosed with carcinoid tumor in case the doctor removes the appendix and finds a tumor. Patients with carcinoid tumor in other parts of the digestive tract may have symptoms such as:

• Pain in the abdomen
• Nausea or vomiting
• Diarrhea

Other ways to detect is by lab tests, imaging, and biopsy.

A new poll found that the number of American adults who are unable to afford necessary health care services and medications has reached its highest level since 2021. This is as per an analysis by Gallup and West Health. The findings show a growing gap in access to quality health care, especially between the high and the low-income Americans.

What Does The Data Say?

As per the numbers, 11% of adults in 2024, which makes it around 29 million people were classified as "cost desperate". This means that they lacked access to affordable care and were unable to pay for necessary medical treatments. This figure has increased from 8% in 2021. The survey was conducted form November 18 to December 27. This included responses from more than 6,200 adults.

What Is The Impact On Minority Communities?

The financial burden of health care is affecting certain demographic groups more than others. The percentage of Hispanic adults categorized as cost desperate increased from 10% in 2021 to 18% in 2024. Similarly, 14% of Black adults faced cost desperation in 2024, up from 9% in 2021. In contrast, the percentage of white adults in this category remained steady at 8%.

At the same time, the number of Americans classified as "cost secure"—those who can access and afford quality care—has fallen significantly. Only 51% of adults reported feeling financially secure about their health care costs in 2024, down from 61% in 2022. The decline was especially sharp for Hispanic and Black adults, with only 34% of Hispanic respondents and 41% of Black respondents feeling cost secure, compared to 51% and 54% in 2021, respectively.

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Is There A Gap Between Income Levels?

The data also highlight that there is a financial divide in health care access between high and low income households. Among individuals earning less than $24,000 annually, the percentage of those categorized as cost desperate rose sharply from 14% in 2021 to 25% in 2024. In contrast, for those earning $180,000 or more, cost desperation fell from 2% to just 1% over the same period.

Increasing Concerns Over Health Care Affordability

As financial strain increases, more Americans are worried about their ability to afford health care when needed. In 2024, 35% of adults said they would be unable to pay for medical services if required, compared to 29% in 2021. The data suggests that economic pressures are making it harder for people to access timely and necessary medical treatments.

Policy Changes and Their Potential Impact

The affordability crisis is unfolding at a time when key policy decisions could make access to health care even more challenging for many Americans. Medicaid, the largest source of health care coverage in the U.S., is at risk of severe funding cuts under a House-passed budget proposal that could slash up to $880 billion over the next decade. If enacted, such cuts would likely force millions of low-income individuals off Medicaid, exacerbating financial and health disparities.

Additionally, new tariffs proposed by the Trump administration could drive up drug prices. Analysts warn that if major exporters of generic medications, such as India, are affected by these levies, the cost of essential prescription drugs could increase, leading to further accessibility issues.

A Growing Crisis With No Clear Solution

The findings indicate that access to affordable health care is becoming more difficult for millions of Americans, particularly those in lower-income brackets and minority communities. With policy shifts and economic challenges on the horizon, health care affordability remains a pressing concern for many, with no immediate solutions in sight.

The United States Food and Drug Administration has recently approved Qfitila (fitusiran) for routine prophylaxis to prevent or to reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age and older with hemophilia. It works on those with hemophilia A or hemophilia B, with or without factor VII or IX inhibitors which neutralizes antibodies.

While subcutaneous Qfitilia does not replace the missing coagulation factor VII or IX, it reduces the amount of protein antithrombin, which leads to an increase in thrombin. It is an enzyme critical for blood clotting. The dosing starts at once every two months and it is also based on the FDA-cleared Innovance Antithrombin companion diagnostic test.

However, as of now, there is no fixed doses of Qfitila is not approved mainly due the risk excessive clotting. It has also a boxed warning for thrombotic events and gallbladder disease.

One of the most common side effects reported were viral infection, nasopharyngitis, and bacterial infection.

How Was This Approved?

The approval came on the basis of two randomized clinical trials that had 177 adult and pediatric male patients with either hemophilia A or hemophilia B enrolled. Among the participants with inhibitors who received the antithrombin-based dosing regimen of Qfitlia, there was a 73% reduction in the estimated annualized bleeding rate. This was seen in comparison with those who received on-demand treatment with bypassing agents.

Among the participants without inhibitors receiving the antithrombin-based dosing regimen of Afitlia, there was a 71% reduction in the estimated annualized bleeding rate versus that seen in those who received on-demand treatment with clotting factor concentrates.

"Today's approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options," Tanya Wroblewski, M.D., deputy director of the Division of Non-Malignant Hematology in the FDA Center for Drug Evaluation and Research, said in a statement.

What Is Hemophilia?

As per the Centers for Disease Control and Orevention (CDC) it is an inherited bleeding disorder in which blood does not clot properly. It can lead to spontaneous bleeding as well as bleeding following injuries or surgeries. Blood usually has proteins called clotting factors that can help one to stop bleeding. However, people with hemophilia have low levels of either factor VII or factor IX.

Hemophilia could also result in bleeding within joints which can then lead to chronic joint disease and pain. It could lead to bleeding in head and in brain, causing long term problems such as seizures or paralysis. In severe cases it can also lead to death if the bleeding cannot be stopped.

The most common types of hemophilia are inherited, thus it is a condition that is present from birth. There are rare cases where hemophilia happens later in life, which is known as acquired hemophilia.

Difference between Hemophilia A and Hemophilia B

As per the National Bleeding Disorders Foundation, hemophilia A, also called factor VIII (8) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII (FVIII), a clotting protein. Although it is passed down from parents to children, about 1/3 of cases found have no previous family history.

Hemophilia B, also called factor IX (FIX) deficiency or Christmas disease, is a genetic disorder caused by missing or defective factor IX, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a change in a gene.

Shingles, a painful viral infection due to the reactivation of the varicella-zoster virus (VZV), is primarily known for its blistering rash and nerve pain. New research indicates, however, that shingles vaccine can be significant in preventing dementia. This recent finding paves the way to new avenues to understand the association between viral illness and brain wellness, which might change preventive treatment against cognitive degeneration in aging individuals.

Once a person has had chickenpox, he or she carries the varicella-zoster virus for life. The virus lies dormant within nerve cells and may reappear years later as shingles, commonly because of compromised immunity with age, stress, or illness.

Shingles appears as a painful rash, typically on one side of the body, and may persist for weeks. Aside from the rash, the infection may cause severe complications like postherpetic neuralgia (PHN), a long-term nerve pain syndrome that lasts for months or even years. In rare instances, shingles may infect the eye, causing blindness.

The Centers for Disease Control and Prevention (CDC) estimates that approximately 1 in every 3 Americans will develop shingles at some time during their lifetime. Vaccines have been recommended to avoid the condition, but recent studies indicate they might also have an added bonus—prevention from dementia.

Shocking Link Between Shingles and Dementia

Dementia, such as Alzheimer's disease, is a rapidly increasing worldwide health issue, with millions of older adults at risk. Although the causes of dementia are still unknown, researchers have long suspected that some viruses, specifically those in the herpes family (which includes VZV), may play a role in cognitive decline.

A study recently published in Nature determined that getting the shingles shot lowered the risk of developing dementia dramatically. Scientists followed more than 280,000 elderly people in Wales who got the original shingles vaccine, Zostavax, and identified a 20% decrease in dementia incidence over the next seven years.

Lead author Dr. Pascal Geldsetzer of Stanford University said the results were "very robust" and added that women appeared to gain more benefit from the vaccine than men. Since women are at greater risk for dementia, these results could have important implications for future prevention efforts.

Can Shingles Cause Cognitive Decline?

Although no one knows the exact mechanisms, several theories discuss how the shingles virus might enhance the risk of dementia:

Inflammation and Brain Damage: The varicella-zoster virus has the ability to cause inflammation in the body, including the brain. Chronic inflammation is recognized to speed up cognitive decline and may play a role in neurodegenerative diseases such as Alzheimer's.

Vascular Effects: The virus has the ability to infect blood vessels in the brain, leading to clots or constricting blood flow. Decreased cerebral circulation is a well-documented risk factor for dementia and stroke.

Amyloid Protein Formation: A study by the University of Colorado Anschutz Medical Campus indicates that shingles can encourage the formation of amyloid plaques, a characteristic of Alzheimer's disease. These adhesive proteins clump together in the brain, interfering with normal function and speeding up cognitive decline.

Can Shingles Vaccination Protect Against Dementia?

The research suggests that shingles vaccination can lower the risk of dementia by inhibiting reactivation of the virus and triggering inflammation in the brain. It is unclear, however, whether the same protective mechanism works for the newer, more effective Shingrix vaccine, which supplanted Zostavax in 2020.

In contrast to Zostavax, which had a live but attenuated form of the virus, Shingrix is a genetically modified vaccine that consists of certain viral proteins to stimulate an immune response. Shingrix has been 97% effective in preventing shingles in clinical trials, versus 51% for Zostavax. Whether it can help prevent dementia, however, is an open question scientists are eager to investigate.

Who Should Receive the Shingles Vaccine?

The CDC advises Shingrix for people aged 50 and above, as well as younger adults with compromised immune systems. The vaccine is given in two doses, spaced a few months apart, and is very effective in preventing shingles and its complications.

With these advantages, however, vaccine acceptance continues to be low, with less than 40% of eligible Americans having gotten their shots. The most frequent side effects are mild flu-like symptoms and pain at the site of injection, but experts point out that the advantages far outweigh the risks.

Tips for Reducing Dementia Risk

Although shingles vaccination holds great potential for dementia prevention, it is not the only answer. Experts suggest an integrated approach to brain health that involves:

Living an Active Lifestyle: Physical exercise on a regular basis enhances circulation to the brain and averts cognitive loss.

Healthy Nutrition: A diet modelled on Mediterranean cuisine, featuring high intakes of fruits, vegetables, whole grains, and healthy fats, is supportive of brain function.

Social and Mental Stimulation: Mental activity with reading, puzzles, or social engagement may build cognitive reserve.

Control of Chronic Illnesses: Control of high blood pressure, diabetes, and cholesterol lowers the risk of dementia.

The association of shingles with dementia risk demonstrates the complex ways infectious illnesses affect overall well-being. Finding that shingles vaccination can decrease risk of dementia adds yet another persuasive argument to convince older people to be vaccinated. Although more study is necessary to validate the results, the study holds out hope that a straightforward vaccine has a key part to play in maintaining cognitive capacity and enhancing the quality of life among aging groups.

As scientists continue to investigate the link between viral infections and brain health, one thing is certain: guarding yourself against shingles might have advantages that reach far beyond the skin. With the increasing global burden of dementia, interventions such as vaccination could become a key part of the arsenal in the battle against cognitive decline.