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An experimental treatment happens to be the solution to delay Alzheimer's symptoms in some people. These people are the ones who are genetically destined to get the disease in their 40s or 50s. These new findings form ongoing research has now been caught up in Trump administration funding delas. The early results of the study has been published on Wednesday and the participants too are worried that politics could cut their access to a possible lifeline.
One of the participants had said, "It is still a study but it has given me an extension to my life that I never banked on having." The participant is named Jake Henrichs, form New York City, who is 50 years old. He is one of them to be treated in that study for more than a decade now and has remained symptom-free despite inheriting an Alzheimer's-causing gene that had killed his father and brother around the same age.
Two drugs which can modestly slow down early-stage Alzheimer's are sold in the United States. These drugs clear the brain of one of its hallmarks, a sticky gunk-like part called the amyloid. However, there have not been any hints that removing amyloid far earlier, way many years before the first symptoms appear, may postpone the disease.
The research is led by Washington University in St Louis, which involved families that passed down rare gene mutation as participants. This meant it was almost guaranteed that they will develop symptoms at the same age their affected relatives did.
The new findings is based on a subset of 22 participants who received amyloid-removing drugs the longest, on average eight years. Long-term amyloid removal cut in half their risk of symptom onset. The study is published in the journal Lancet Neurology.
Washington University's Dr Randall Bateman, who directs the Dominantly Inherited Alzheimer's Network of studies involving families with these rare genes says, "What we want to determine over the next five years is how strong is the protection. Will they ever get the symptoms of Alzheimer’s disease if we keep treating them?”
The researchers before though did not know what exactly caused Alzheimer's which affects nearly 7 million Americans, most of them in their later life. However, it is clear that these silent changes occur in the brain at least two decades before the first symptom shows up. The big contributor. At some point amyloid buildup can trigger a protein named tau that then starts to kill neurons, which can lead to cognitive decline.
Researchers are now thus studying the Tau-fighting drugs and are looking into other factors, like inflammation, brain's immune cells and certain virus.
The National Institute of Health (NIH) has expanded its focus as researchers have found more reasons for Alzheimer's. In 2013, the NIH's National Institute on Aging funded 14 trials of possible Alzheimer's drugs over a third targeting amyloid. By last fall, there were 68 drugs and 18% of them target amyloid. However, there are scientists too who think that amyloid is not everything and their is way more in the brain tissue, immune cells, and more which can be studied.
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The US Food and Drug Administration (FDA) has approved Mimrylo (rusfertide) for adults with polycythemia vera (PV), a chronic disorder in which the body produces too many red blood cells.
The drug is the first approved treatment for PV that mimics hepcidin, a naturally occurring hormone that regulates iron in the body.
The approval, announced on August 28, is particularly significant for patients whose disease remains inadequately controlled despite existing treatment and who need frequent blood-removal procedures.
Polycythemia vera belongs to a group of disorders in which the bone marrow produces blood cells excessively. In PV, the major problem is an overproduction of red blood cells.
More red blood cells mean a higher proportion of cells circulating in the blood. This can make blood thicker and increase the risk of blood clots, stroke and heart attack.
That is why doctors pay close attention to a patient's hematocrit, which represents the proportion of blood made up of red blood cells.
One of the major treatment goals is to keep hematocrit below 45%, which helps reduce cardiovascular risks.
Also read: Sweetener In Sugar-Free Gum Linked To 57% Higher Heart Attack And Stroke Risk: What Is Xylitol?
One of the traditional ways of controlling PV is removing some blood. The procedure, called phlebotomy, involves taking blood from a vein, reducing the number of circulating red blood cells and bringing hematocrit down.
But for some patients, this can become a recurring routine. They may need repeated blood draws even while receiving standard treatment. That burden is one reason the new drug has attracted attention.
“People living with polycythemia vera have long faced the challenge of managing a chronic blood disorder with frequent blood draws,” said Tanya Wroblewski, M.D., Director of the Division of Nonmalignant Hematology at the FDA's Center for Drug Evaluation and Research.
She added, “Today's approval of Mimrylo offers a new, first-in-class option that has the potential to meaningfully reduce patient burden.”
Also read: Norway’s King Harald V Dies At 89: What Is Hemolytic Anemia?
The FDA's approval was based on the VERIFY trial, a phase 3 study involving 293 adults with PV who continued to require frequent phlebotomies despite standard treatment. Participants received either Mimrylo or placebo for 32 weeks.
76.9% of patients receiving Mimrylo did not require a phlebotomy during weeks 20 to 32, compared with 32.9% of those receiving placebo, marking a substantial difference.
Mimrylo is administered once a week as an injection under the skin, with the dose adjusted to help maintain hematocrit below 45%.
The most common adverse reactions reported with Mimrylo were reactions at the injection site and anaemia, according to the FDA.
The drug was granted priority review and approved to Takeda Pharmaceuticals America.
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Melatonin is often opted to treat a bad night's sleep. But new research is raising an important question about what happens when the sleep supplement becomes a long-term habit.
A large preliminary study found that people with chronic insomnia who used melatonin for at least 12 months had a roughly 90% higher risk of developing heart failure over five years compared with similar people who did not use it.
The findings were presented at the American Heart Association's Scientific Sessions and are based on health-record data. Researchers have stressed that the study does not conclusively prove that melatonin causes heart failure.
The researchers observed adults with insomnia and compared those who used melatonin for 12 months or more with those who didn't.
Over a period of five years, 4.6% of long-term melatonin users developed heart failure, compared to 2.7% of non-users. That translated into approximately a 90% higher risk.
Researchers found a similar association when they used a stricter definition of long-term use, looking at people who had filled at least two melatonin prescriptions at least 90 days apart. In that analysis, the risk was 82% higher.
So, it does not mean that 90 out of 100 people taking melatonin will develop heart failure. It means the relative risk was about 90% higher in the long-term melatonin group than in the comparison group.
Melatonin is a hormone naturally produced by the body, mainly in response to darkness. It helps regulate the sleep-wake cycle, essentially telling the body that night time has arrived. Melatonin supplements are therefore used to help with sleep problems.
But chronic insomnia itself is not harmless. Poor sleep can be associated with high blood pressure, obesity, diabetes, and cardiovascular disease. And that creates a major problem in interpreting this study.
Also read: How To Read Your Blood Test Results: A Simple Guide To What Your Numbers Mean
People taking melatonin long term may have more severe or persistent insomnia than people who do not take it. That underlying sleep disorder could itself contribute to cardiovascular problems.
There may also be other differences between the two groups that a medical-record study cannot completely account for. This is why the researchers have described the findings as preliminary and say more research is needed to understand whether melatonin itself has a harmful cardiovascular effect.
Talking about heart failure, it simply does not mean that the heart suddenly stops working. It means the heart has become unable to pump blood efficiently enough to meet the body's needs. People may develop symptoms like:
The research is merely observational, so it cannot establish cause and effect. It also does not show that short-term melatonin use causes heart failure.
The American Heart Association described the findings as raising “new questions about the safety of taking melatonin for extended periods.”
If someone has been taking melatonin every night for months or years, particularly because of persistent insomnia, it may be worth discussing the underlying sleep problem with a doctor rather than simply continuing the supplement indefinitely.
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Statins have long had the job of lowering LDL cholesterol and protecting the heart. But new a study has emerged stating that these medicines may have another significant benefit for the brain.
A 15-year Danish study involving more than 132,000 people with type 2 diabetes found that those who started taking statins earlier had a 15% lower risk of developing dementia compared with those who started treatment later. Even later, statin use was associated with a 10% lower risk.
The researchers suggest that the timing of treatment is key as prolonged exposure to high cholesterol and other cardiovascular risk factors could contribute to damage that accumulate over many years.
The findings, presented at the European Society of Cardiology Congress and published in The Lancet Regional Health – Europe, come as researchers are constantly exploring the connection between cholesterol, blood vessels, and brain health.
The brain is an enormous delivery network in which billions of cells depend on a constant supply of oxygen and nutrients delivered through tiny blood vessels.
High levels of LDL cholesterol can contribute to atherosclerosis, where fatty deposits build up inside arteries. Over time, this can damage blood vessels and restrict blood flow.
Damage to the brain's blood vessels can contribute to vascular cognitive impairment and vascular dementia, while strokes can also increase the risk of subsequent cognitive decline.
Also read: Hormone Replacement Therapy May Lower Dementia Risk In Women, Shows Study
This study merely observed the correlation between statins and dementia risk, meaning it did not establish a definite cause-and-effect relationship. People who start statins earlier may also differ from those who start later in other ways, including their overall management of diabetes and cardiovascular risk.
The researchers therefore say more work is needed to determine whether statins have a direct protective effect on the brain.
Also read: Bruce Willis’ Daughter Rumer Undergoes ‘Terrifying’ Genetic Tests Amid Dad’s Dementia Diagnosis
It is important to note the difference between treating unhealthy LDL cholesterol levels and assuming that everyone should aggressively lower cholesterol to prevent dementia. Statins remain as the medication to reduce cardiovascular risk.
Their potential brain benefits are an additional area of research. So, people must not blindly start popping statins to lower the risk of neurodegenerative diseases like dementia.
There is another reassuring point for people already taking statins. The concerns that these medicines may result in the increase of dementia risk have not been supported by the strongest available evidence.
A large review of randomised trials earlier this year found that most reported statin side effects were no more common than with placebo, including reported memory problems.
The most interesting takeaway may not be that “statins prevent dementia.” The evidence isn't strong enough to make that claim yet. It is that heart health and brain health are deeply intertwined.
Managing cholesterol, blood pressure, diabetes and other cardiovascular risk factors, along with regular physical activity and a healthy diet, may help protect the blood vessels that keep the brain functioning.
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