Credits: Canva
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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Moderna has launched the first-in-human Phase 1 clinical trial of its investigational vaccine against the Bundibugyo ebolavirus (BDBV), the strain behind the ongoing Ebola outbreak in the Democratic Republic of the Congo.
The biotechnology company said Health Canada has authorized the study, which will evaluate its vaccine candidate, mRNA-1469, in healthy adult volunteers at three clinical sites across Canada.
The trial comes as the Bundibugyo Ebola outbreak continues to worsen, with 3,748 confirmed cases and 1,657 deaths reported across 49 health zones in five provinces of the Democratic Republic of the Congo, according to World Health Organization (WHO) data as of August 1.
The epidemic is now the second-largest and fastest-spreading Bundibugyo Ebola outbreak on record.
Also read: Russia's New Ebola Vaccine To Protect Against Rare Bundibugyo Strain, Says Health Minister
mRNA-1469 is an investigational vaccine developed using Moderna's messenger RNA (mRNA) platform—the same technology used in its COVID-19 vaccine.
The vaccine builds on the company's broader research into filoviruses, the family of viruses that includes Ebola.
"Vaccinating the first participants with mRNA-1469 marks an important milestone in advancing a vaccine candidate against Bundibugyo ebolavirus, for which no approved vaccine currently exists," said Stéphane Bancel, Chief Executive Officer of Moderna.
The Phase 1 study is being conducted at three clinical sites in Canada and is expected to enroll around 80 healthy adult volunteers.
Researchers will assess whether mRNA-1469 is safe and well tolerated, and whether it generates immune responses strong enough to justify further clinical development.
Read More:Uganda Declared Ebola-Free As Congo Outbreak Grows To 3,262 Cases, 1,437 Deaths
mRNA-1469 is one of four initial Bundibugyo vaccine candidates being supported by the Coalition for Epidemic Preparedness Innovations (CEPI). CEPI has committed up to US$50 million to fund preclinical testing and the Phase 1 trial.
The partnership also supports manufacturing additional clinical trial doses in parallel with early-stage testing, enabling Phase 2 and Phase 3 trials to begin rapidly if the Phase 1 results are positive.
If the vaccine is eventually approved, Moderna has committed to making at least 500,000 doses available at access pricing for low- and middle-income countries under its agreement with CEPI.
Moderna's study follows the launch of the University of Oxford–Serum Institute of India (SII) vaccine trial in July, making it the second human clinical trial targeting the Bundibugyo ebolavirus.
Unlike the Zaire strain of Ebola, there are currently no approved vaccines or antiviral treatments specifically for the Bundibugyo ebolavirus, making vaccine development a global public health priority.
Alongside vaccine development, WHO says clinical studies of experimental treatments and preventive medicines are progressing rapidly against the Bundibugyo strain.
The agency noted that research protocols prepared before the outbreak began have significantly accelerated the launch of clinical trials.
"If you compare this outbreak to previous Ebola outbreaks, we have been able to start trials more quickly," said Vasee Moorthy, acting head of WHO's R&D Blueprint Programme. He added that preclinical data for several candidates has shown encouraging results.
WHO-backed treatment trial: A WHO-sponsored clinical trial is underway at three Ebola treatment centers in Ituri province in partnership with medical charities ALIMA and Doctors Without Borders (MSF).
More than 50 patients enrolled: Over 50 confirmed Ebola patients have been enrolled and randomly assigned to receive experimental treatment options, according to WHO.
Preventive antiviral study: A separate prophylaxis study led by DR Congo's National Institute for Biomedical Research (INRB) and international partners has enrolled more than 25 high-risk contacts in Ituri province, Reuters reported.
Researchers are also evaluating whether a 10-day course of Gilead Sciences' oral antiviral drug Obeldesivir can prevent Ebola disease in people exposed to the virus.
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Cancer recurrence remains one of the biggest challenges in cancer treatment. Dormant tumour cells are capable of surviving treatment and therapy and can reactivate months or even years later.
Now, researchers say that a new generation of experimental drugs designed to target these dormant cancer cells could offer a promising strategy to prevent the disease from recurring.
The findings come as scientists have been increasingly focusing on tumour dormancy, a state in which cancer cells stop actively multiplying but remain hidden in the body. They become resistant to chemotherapy and other conventional cancer treatments.
The research suggests that targeting the biological pathways controlling dormancy may help stop these cells from getting reactivated and forming new tumours or metastases.
According to researchers, dormant cancer cells are one of the key reasons why some patients experience relapse long after completing treatment.
"Dormant tumour cells are a major driver of cancer recurrence and metastasis," the researchers noted, stating that therapies aimed at controlling or eliminating these cells could change how cancer is treated and managed in the future.
Also read: Bone Marrow Transplant: The Quiet Revolution Transforming India's Fight Against Blood Cancers
Unlike traditional cancer treatments that mainly attack rapidly dividing cells, these new experimental drugs target the molecular signals that allow dormant cancer cells to survive and go unnoticed in the body.
Researchers are investigating several approaches, including blocking pathways that trigger dormant cells to become active again, disrupting the cells' survival mechanisms, and making them more vulnerable to conventional cancer therapies.
Some experimental treatments are also being tested alongside immunotherapy to improve the body's ability to detect and destroy these hidden cells on time.
Scientists say this approach could be especially important for cancers known to recur years after treatment, including breast, lung and certain gastrointestinal cancers.
Cancer recurrence can happen when a small number of cells survive surgery, chemotherapy or radiation. These cells may remain inactive for long periods of time before starting to grow again, eventually leading to a relapse or metastatic diseas.
Researchers believe that the environment of the tumour, immune responses, inflammation and changes in cellular structure and metabolism all play a role in determining whether dormant cancer cells remain inactive or become aggressive again.
While the findings encourage advanced pathways for cancer treatments, experts caution that most drugs that target tumour dormancy are still in the experimental or early clinical trial stage.
Rather than only treating visible tumours, future therapies may also focus on preventing hidden cancer cells from ever becoming active again, potentially reducing the risk of relapse years after successful treatment.
More studies are needed to determine whether they can really reduce cancer recurrence and improve long-term survival in patients.
The promising research comes after a new study found that Viagra, best known as a treatment for erectile dysfunction, may also help stop cancer from spreading.
Researchers from the Weizmann Institute of Science in Israel found that sildenafil, the active ingredient in Viagra, may enhance a newly identified cholesterol-regulating mechanism that could be used to curb cancer metastasis.
The study, published in Cancer Research, showed that sildenafil limits cancer cells' ability to use cholesterol, an essential component of cell membranes.
Cholesterol is especially important for cancer cells that break away from the primary tumor, travel through the body, and invade distant organs. When their access to cholesterol is reduced, these cells have greater difficulty forming metastases.
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A common amino acid found in protein-rich foods, which is also available as an dietary supplement, may have surprising potential for strengthening the body's immune response against cancer and viral infections, according to a new study.
Published in the journal Cell, researchers from Rockefeller University found that arginine plays a key role in helping immune cells detect and attack abnormal cells.
Their findings suggest that restoring arginine levels could improve the effectiveness of the body's natural defences and may one day strengthen cancer or antiviral treatments.
"Our work reveals how a lack of arginine interferes with the immune system, and suggests that upping arginine intake could prove beneficial," said Qiushuang Wu, the study's first author and a postdoctoral researcher at Rockefeller University. "Perhaps that means it could be used in combination with other therapies to treat both cancer and viral infections."
The team investigated how low arginine levels affect gene expression in models of colon cancer, influenza and SARS-CoV-2 infection.
They found that arginine deficiency disrupted the production of major histocompatibility complex class I (MHC-I) proteins, molecules that help cells, allowing the immune system to detect and fight virus-infected or cancerous cells in the body.
The researchers discovered that when arginine was low, ribosomes, the cellular machinery responsible for making proteins, stalled while producing MHC-I proteins. As a result, infected or abnormal cells became harder for the immune system to identify.
However, after giving a moderate amount of arginine, roughly equivalent to a couple of over-the-counter tablets, restored the production of these stalled proteins.
Also read: Bone Marrow Transplant: The Quiet Revolution Transforming India's Fight Against Blood Cancers
Many cancers and viral infections are known to alter amino acid levels in the body. The new findings suggest that these changes may weaken immunity in an unexpected way by limiting the production of proteins essential for a stronger immune response.
Senior author Sohail Tavazoie, head of Rockefeller University's Laboratory of Systems Cancer Biology, believes the discovery could become an asset for future clinical research, especially in cancer treatment studies.
"Arginine supplementation could be readily tested in patients receiving immunotherapies or given to high-risk populations exposed to viral pathogens," Tavazoie said. "Considering that arginine is inexpensive and readily available, we hope that therapeutic and preventative studies could be undertaken soon."
Arginine is an essential amino acid, meaning the body usually produces. Additional amounts of of arginine may be needed during illness, injury or periods of physiological distress to the body.
It is naturally found in foods such as meat, poultry, fish, dairy products, nuts, seeds and legumes. It is also sold as a dietary supplement. It also serves as a building block for nitric oxide, a molecule involved in blood flow and immune function.
While the findings are promising, the researchers emphasise that the study does not prove that taking arginine supplements can prevent or treat cancer or viral infections in people.
Reliable human clinical trials are still needed to determine whether the supplementation is safe, effective and beneficial.
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