The FDA Considers Approving CRISPR Gene Editing Therapy for Sickle Cell Disease

The Food and Drug Administration (FDA) is on the verge of approving a groundbreaking therapy that uses the gene-editing technique called CRISPR to treat sickle cell disease. This would be the first time gene editing moves from the lab into clinical use, marking a significant milestone in medical history.

A committee of advisers to the FDA is meeting to review the scientific evidence for the treatment, known as “exa-cel,” including its long-term safety. The FDA scientists have concluded that exa-cel is highly effective at preventing episodes of excruciating pain in sickle cell disease patients. However, concerns have been raised about potential “off-target” effects of the treatment, which could cause long-term health problems.

During the meeting, the committee will hear presentations from Vertex Pharmaceuticals Inc., the company that led the development of exa-cel, as well as independent researchers. Additionally, the committee will have the opportunity to hear from Victoria Gray, a Mississippi woman who was the first sickle cell patient to receive the treatment as part of a clinical trial conducted by Vertex. Gray’s experience with the treatment has been life-changing, and she strongly advocates for its approval.

The potential approval of exa-cel represents a major breakthrough in the field of gene editing. CRISPR technology allows scientists to make precise changes in DNA, offering hope for the development of new medical treatments for various conditions, including muscular dystrophy, diabetes, cancer, Alzheimer’s, AIDS, and heart disease.

Sickle cell disease is a genetic illness that affects millions of people worldwide, particularly those of African, Middle Eastern, and Indian descent. The disease causes misshapen red blood cells that can lead to intense pain, organ damage, and other serious complications. Patients often require frequent hospital visits and face significant challenges in their daily lives. The CRISPR treatment aims to alleviate symptoms by restoring normal red blood cell function.

While the potential benefits of the therapy are promising, there are concerns about its cost and complexity. The treatment could cost up to $2 million per patient and requires a bone marrow transplant and lengthy hospitalization. This may limit access to the therapy for those who need it the most, both in the United States and in less affluent countries where the disease is prevalent.

Despite these challenges, experts believe that the potential long-term cost savings and improved quality of life for sickle cell patients make the therapy worthwhile. Efforts are underway to make the treatment more accessible through collaborations with insurance companies and the development of alternative payment options.

The FDA’s decision on exa-cel will have far-reaching implications for the future of gene editing and the treatment of genetic diseases. As we stand on the verge of this historic approval, it is a moment of hope and anticipation for scientists, industry professionals, and most importantly, the patients who may benefit from this groundbreaking therapy.

Sources:

• The CRISPR Revolution
• Sickle Cell Disease
• FDA Advisory Committee Meeting
• FDA Analysis
• Victoria Gray’s Experience

U.S. health regulators have issued a warning to consumers regarding the use of certain over-the-counter eyedrops that could potentially lead to blindness. The Food and Drug Administration (FDA) has advised against using more than two dozen varieties of lubricating drops sold by six companies, including CVS Health, Target, Rite Aid, and Cardinal Health. The FDA has identified unsanitary conditions and bacteria at the facility producing these drops, prompting the agency to request a recall of the products.

While no injuries related to these products have been reported, the FDA is urging consumers to stop using them immediately and avoid purchasing any remaining stock. The agency is also encouraging doctors and patients to report any cases through their online reporting system.

This recent advisory from the FDA highlights the importance of product safety and the potential risks associated with using certain medications. In a similar vein, a recent article from Paul Williams DDS explores the topic of over-the-counter availability of the drug Narcan, which is used to reverse opioid overdoses.

The article, titled “F.D.A. Advisers: Narcan Is Safe to Buy Over the Counter,” discusses the FDA’s decision to allow Narcan to be sold without a prescription. Narcan, also known as naloxone, is a life-saving medication that can rapidly reverse the effects of an opioid overdose. By making it available over the counter, the FDA aims to increase access to this critical drug and potentially save more lives.

Both the FDA’s warning about the eye drops and the availability of Narcan over the counter highlight the agency’s commitment to ensuring the safety and accessibility of medications. While the eye drops pose a risk to consumers due to unsanitary conditions and bacteria, Narcan offers a solution to a pressing public health crisis by providing easier access to a life-saving medication.

It is crucial for consumers to stay informed about product recalls and advisories issued by regulatory agencies like the FDA. By being aware of potential risks and taking appropriate action, individuals can protect their health and well-being. Additionally, healthcare professionals play a vital role in educating patients about the safe use of medications and reporting any adverse events.

As the FDA continues to monitor and regulate the pharmaceutical industry, it is essential for manufacturers to prioritize quality control and adhere to strict safety standards. The recent incidents involving the eye drops and the subsequent recall highlight the importance of maintaining sanitary conditions during the production process.

For more information on the FDA’s safety alerts and reporting system, visit their website: https://www.fda.gov/safety/medwatch-fda-safety-information-and-adverse-event-reporting-program.

A Cure for Herpes: Urgently Needed for Millions

A cure for Herpes Simplex Virus (HSV-1 and HSV-2) is urgently needed for the 6 billion people globally living with HSV. Herpes is not benign. We need a cure today because patients are waiting.

Despite herpes being highly common, it is a traumatic diagnosis. We believe that because Herpes Simplex is stigmatized so intensely, there have been no centralized advocacy efforts to date, and no cure.

Herpes Cure Advocacy (HCA), a grassroots membership-based international non-profit organization, is dedicated to the treatment, cure, and prevention of Herpes Simplex Virus types 1 and 2. Founded in 2020, HCA collaborates with patient advocates worldwide to create change and improve the quality of life for carriers of HSV.

While there is no cure for herpes currently, there is hope on the horizon. The clinical pipeline for Herpes Simplex Virus types 1 and 2, known as the Herpes Cure Pipeline 3.0, is continuously evolving. Researchers and scientists are working tirelessly to develop effective treatments and ultimately find a cure.

To stay updated on the latest news in clinical research and medical breakthroughs for the cure, treatment, and prevention of Herpes, visit the Herpes Cure Advocacy blog. The clinical pipeline is gaining momentum, and it is crucial to stay informed about the progress being made.

One of the significant challenges in the fight against herpes is the increased risk of HIV acquisition associated with herpes infection. According to a study, herpes infection increases the risk of HIV acquisition more than any other sexually transmitted infection. Additionally, herpes has been found to impact brain health, although the extent of this impact remains unknown.

Advocacy plays a crucial role in raising awareness and driving change. If you want to get involved and help rally for a herpes cure, consider joining Herpes Cure Advocacy. Together, we can make a difference and work towards a future where herpes is no longer a burden.

For more information about Herpes Cure Advocacy and our goals, visit our website and learn how you can contribute to the cause. There needs to be a greater emphasis on cure, treatment, and prevention, as no public health intervention or national strategy currently exists.

References:

• New HSV Advocates: A Starter’s Guide to Activism
• Living with Herpes
• Herpes Cure Advocacy Blog
• Herpes Simplex Virus 101: The Basics

Study shows antibodies against polyethylene glycol in 83 percent of the German population

It has long been known that people can form defenses and thus antibodies against viruses. But antibodies can also develop against polyethylene glycol (PEG), a substance used in cosmetics, food and medicine. These influence the effectiveness of drugs. A team of researchers from the Max Planck Institute for Polymer Research has now investigated how widespread these antibodies already are in German society and how they might influence medical therapies using nanocarriers.

A virus invades the body and the immune system begins to work: Antibodies develop that fight the infection. At the same time, an immune memory builds up so that antibodies can be quickly made available in the event of a new infection. Surprisingly, antibodies can also form against polyethylene glycol (PEG), a molecule with a fairly simple structure.

In addition to cosmetic products – from creams, perfumes and lotions to lipstick – polyethylene glycol is also used in medicine. Here, it serves as a kind of camouflage coat against the body’s own immune system, thus increasing the circulation time of an active ingredient in the blood.

“For us, PEG is interesting for coating nano-sized drug carriers with it,” says Svenja Morsbach, group leader in Katharina Landfester’s department at the MPI for Polymer Research. In this way, the researchers achieve a longer circulation time for the drug capsules, which are only nanometers in size and could be an important component in novel cancer therapies in the future, for example.

In their studies, the team led by Morsbach and Landfester examined more than 500 blood samples from patients taken in 2019. “The antibodies formed against PEG attach themselves to the coated nanocarriers, thus counteracting the effect that is actually desired: the nanocarrier becomes visible to the immune system and is removed before it can exert its effect,” explains Katharina Landfester, director of the department.

The researchers led by Morsbach and Landfester assume that therapies will have to be adapted in the future to respond to this behavior of the immune system. In their statistical studies of blood samples, they found that PEG antibodies were already detectable in 83% of the samples examined.

The concentration of PEG antibodies in the blood correlates antiproportionally with the age of the person examined: the older the person, the fewer PEG antibodies were present. “We currently assume that this is due to the increasing use of PEG in various areas of life only recently and the variation of the immune system in age,” says Morsbach.

In further studies, the researchers would now like to find out how future therapies could be adapted to compensate for the reduced camouflage of the nanocarriers. “Ideas would include whether PEG can be replaced or possibly dispensed with altogether,” Morsbach said. But determining the antibody concentration in a patient’s blood and individually adjusting the amount of active ingredient could also be an alternative.

They have published their current results in the renowned journal “Nanoscale Horizons“.

Reference: https://pubs.rsc.org/en/content/articlelanding/2021/nh/d1nh00368a#!divAbstract

Curbing carbon emissions and eating healthier may both start at the dinner table.

According to a new study co-authored by a Tulane University researcher and published in the journal Nature Food, making simple substitutions like switching from beef to chicken or drinking plant-based milk instead of cow’s milk could reduce the average American’s carbon footprint from food by 35%, while also boosting diet quality by between 4-10%, according to the study.

These findings highlight the potential of a “small changes” approach that researchers believe could encourage more consumers to adopt climate-friendly eating habits. Food production accounts for 25-33% of the nation’s greenhouse gas emissions with beef production being a primary contributor.

“This study shows that cutting dietary carbon emissions is accessible and doesn’t have to be a whole lifestyle change,” said Diego Rose, senior author and nutrition program director at Tulane University School of Public Health and Tropical Medicine. “It can be as simple as ordering a chicken burrito instead of a beef burrito when you go out to eat. When you’re at the grocery store, move your hand one foot over to grab soy or almond milk instead of cow’s milk. That one small change can have a significant impact.”

The study, which analyzed diet data from over 7,700 Americans, identified commonly eaten foods with the highest climate impact and simulated replacing them with nutritionally similar, lower-emission options.

“For us, substitutes included swapping a beef burger for a turkey burger, not replacing your steak with a tofu hotdog,” said Anna Grummon, lead author and assistant professor of pediatrics and health policy at Stanford University. “We looked for substitutes that were as similar as possible.”

The largest projected reductions in emissions were seen in mixed dishes: burritos, pastas and similar popular dishes where it’s easy to substitute a lower-impact protein instead of beef.

The study expanded on past research by including dietary data for children. Whereas it may be more effective for an adult to focus on protein swaps, Grummon said switching children to plant-based milk can have a “meaningful impact on the carbon footprint” and help start positive habits earlier.

Identifying healthy alternatives to high-carbon foods was not the intent of the study. And yet, swapping to lower carbon foods showed “sizable improvements in how healthy the diets were.”

While these substitutes are not intended as a cure-all for climate objectives or personal health goals, they are evidence that small changes can have a large impact.

“There is overlap between sustainable diets and healthy diets,” Grummon said. “Our study shows that changing just one ingredient, making one swap, can be a win-win, resulting in meaningful changes in both climate outcomes and how healthy our diets are.”

Reference: Grummon AH, Lee CJY, Robinson TN, Rimm EB, Rose D. Simple dietary substitutions can reduce carbon footprints and improve dietary quality across diverse segments of the US population. Nat Food. 2023. doi: 10.1038/s43016-023-00864-0

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Curbing carbon emissions and eating healthier may both start at the dinner table.

According to a new study co-authored by a Tulane University researcher and published in the journal Nature Food, making simple substitutions like switching from beef to chicken or drinking plant-based milk instead of cow’s milk could reduce the average American’s carbon footprint from food by 35%, while also boosting diet quality by between 4-10%, according to the study.

These findings highlight the potential of a “small changes” approach that researchers believe could encourage more consumers to adopt climate-friendly eating habits. Food production accounts for 25-33% of the nation’s greenhouse gas emissions with beef production being a primary contributor.

“This study shows that cutting dietary carbon emissions is accessible and doesn’t have to be a whole lifestyle change,” said Diego Rose, senior author and nutrition program director at Tulane University School of Public Health and Tropical Medicine. “It can be as simple as ordering a chicken burrito instead of a beef burrito when you go out to eat. When you’re at the grocery store, move your hand one foot over to grab soy or almond milk instead of cow’s milk. That one small change can have a significant impact.”

The study, which analyzed diet data from over 7,700 Americans, identified commonly eaten foods with the highest climate impact and simulated replacing them with nutritionally similar, lower-emission options.

“For us, substitutes included swapping a beef burger for a turkey burger, not replacing your steak with a tofu hotdog,” said Anna Grummon, lead author and assistant professor of pediatrics and health policy at Stanford University. “We looked for substitutes that were as similar as possible.”

The largest projected reductions in emissions were seen in mixed dishes: burritos, pastas and similar popular dishes where it’s easy to substitute a lower-impact protein instead of beef.

The study expanded on past research by including dietary data for children. Whereas it may be more effective for an adult to focus on protein swaps, Grummon said switching children to plant-based milk can have a “meaningful impact on the carbon footprint” and help start positive habits earlier.

Identifying healthy alternatives to high-carbon foods was not the intent of the study. And yet, swapping to lower carbon foods showed “sizable improvements in how healthy the diets were.”

While these substitutes are not intended as a cure-all for climate objectives or personal health goals, they are evidence that small changes can have a large impact.

“There is overlap between sustainable diets and healthy diets,” Grummon said. “Our study shows that changing just one ingredient, making one swap, can be a win-win, resulting in meaningful changes in both climate outcomes and how healthy our diets are.”

Reference: Grummon AH, Lee CJY, Robinson TN, Rimm EB, Rose D. Simple dietary substitutions can reduce carbon footprints and improve dietary quality across diverse segments of the US population. Nat Food. 2023. doi: 10.1038/s43016-023-00864-0

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Doctors in Taiwan recently made a surprising discovery when a 64-year-old woman visited their clinic complaining of strange sounds in her left ear. After four days of hearing beating, clicking, and rustling noises, the woman sought help from an ear, nose, and throat specialist. To her astonishment, doctors found a small spider and its moulted exoskeleton in her ear canal.

This unusual case was published in a short report in the New England Journal of Medicine, highlighting the importance of seeking medical attention for foreign bodies in the ear. While insects in the ear canal are not uncommon, they can lead to complications such as infection, hearing loss, and vestibular complaints if not properly addressed.

In a separate study published in the Journal of Family Medicine and Primary Care, it was found that emergency doctors and physicians often encounter patients with foreign bodies in their ear canals. These foreign bodies can range from cotton or seeds to live insects. Therefore, it is crucial to approach the removal of such objects with caution.

Experts recommend against attempting to remove bugs from the ear with fingers or cotton swabs, as this can potentially push the object further into the ear or cause damage. Instead, individuals should seek the assistance of an ear, nose, and throat specialist to ensure safe and effective removal.

According to the Mayo Clinic, pouring alcohol or oil into the ear can help dislodge the insect if there is no hole in the eardrum. The liquid creates an environment where the insect can float out. However, if there is a perforation in the eardrum, it is essential to consult a specialist to prevent complications.

While this case of a spider in the ear may seem unusual, it serves as a reminder of the potential risks associated with foreign bodies in the ear canal. Seeking professional medical help is crucial to ensure the safe removal of the object and prevent any further complications.

To learn more about the topic of foreign bodies in the ear, you can read the article “Foreign Bodies in Ear Canals: A Review of Cases in Emergency Department,” which provides insights into the experiences of emergency doctors and physicians in dealing with such cases.

MINNEAPOLIS (AP) — A poison specialist and former medical resident at Mayo Clinic in Minnesota is charged with fatally poisoning his wife, a 32-year-old pharmacist who died days after she went to a hospital in August with stomach distress.

Authorities say Connor Bowman, 30, tried to stop the autopsy on his wife, Betty Bowman — arguing she should be cremated immediately and claiming she had a rare illness, which hospital tests did not confirm. The medical examiner’s office halted the order for cremation, citing suspicious circumstances, according to a criminal complaint, and an autopsy showed Betty Bowman died from toxic effects of colchicine, a medicine used to treat gout.

Medical records indicate she was not diagnosed with gout and had not been prescribed the medicine, the complaint states, adding that Connor Bowman had been researching the drug prior to his wife’s death. Six days before she was hospitalized, he had also converted his wife’s weight to kilograms and multiplied that by 0.8 — with 0.8 mg/kg considered to be the lethal dosage rate for colchicine, according to the complaint.

Connor Bowman was charged Monday with second-degree murder. He was arrested Friday and was still in custody as of Tuesday. His attorney did not immediately respond to requests for comment from The Associated Press.

Mayo Clinic spokesperson Amanda Dyslin released a statement Tuesday that did not identify Bowman by name, but indicated he was a resident at the hospital.

“We are aware of the recent arrest of a former Mayo Clinic resident on charges unrelated to his Mayo Clinic responsibilities. The resident’s training at Mayo Clinic ended earlier this month,” the statement said. Dyslin did not say why Connor Bowman’s training at Mayo Clinic ended.

Betty Bowman was also a pharmacist at Mayo Clinic.

According to the criminal complaint, the Southeast Minnesota Medical Examiner’s Office alerted police to the “suspicious death” of Betty Bowman on Aug. 21, a day after she died.

She had been admitted to a hospital in Rochester on Aug. 16 with “severe gastrointestinal distress and dehydration where her condition deteriorated rapidly,” the complaint said. Her initial symptoms were similar to food poisoning and were treated that way, but they continued to worsen. She experienced cardiac issues, fluid in her lungs and organ failure.

While Betty Bowman was in the hospital, Connor Bowman suggested she was suffering from a rare illness called hemophagocytic lymphohistiocytosis, or HLH. Hospital tests came back inconclusive for HLH, but Connor Bowman told multiple people that she died from that disease, according to the complaint.

He also told the medical examiner’s office that Betty Bowman’s death was natural and that she “did not want to be a cadaver,” and therefore, the autopsy should be canceled, the complaint said. He asked an investigator at the medical examiner’s office if the toxicology analysis would be more thorough than the analysis done at the hospital.

The night before Betty Bowman went to the hospital, she told a man — identified as SS in the complaint — that she was drinking at home with Connor Bowman. The next morning, she told SS she was sick, possibly from a drink that was mixed into a large smoothie.

Connor Bowman was a poison specialist and answered calls about poisons, using devices from the University of Kansas for his work, according to the complaint. A woman from the University of Kansas told investigators that Connor Bowman had been researching colchicine, the drug used to treat gout, though he had not received any calls about colchicine, nor had any other employees.

Investigators found that Connor Bowman had searched “internet browsing history: can it be used in court?” and “delete amazon data police” on Aug. 5. He did calculations that match the lethal dosage rate for colchicine on Aug. 10.

The Minnesota Department of Health found colchicine in Betty Bowman’s blood and urine samples that were taken at the hospital, and the medical examiner determined the cause of death to be toxic effects of colchicine, the complaint says.

One woman told investigators that the Bowmans had been talking about divorce, and another told authorities that Connor Bowman said he was going to get $500,000 in life insurance from his wife’s death, the complaint says. Authorities found a receipt for a $450,000 bank deposit inside his home.

He is scheduled to appear in court on Nov. 1.

Trisha Ahmed is a corps member for the Associated Press/Report for America Statehouse News Initiative. Report for America is a nonprofit national service program that places journalists in local newsrooms to report on under-covered issues. Follow her on X, formerly known as Twitter: @TrishaAhmed15

References:

1. F.D.A. Advisers: Narcan Is Safe to Buy Over the Counter
2. A New Lease on Life: How NHS Is Saving Children with Gene Therapy for MLD
3. Uncovering the Connection: Exploring the Link Between Hearing Voices and Hearing Your Own Voice
4. Mississippi Misses Out: Why Millions in Pandemic Aid Remains Unspent Amidst COVID Surge
5. 30 Days to a Healthier Heart and Blood Sugar: Delicious Dinners That Won’t Break the Bank
6. Bernie Sanders’ Final Act in Washington Could Change the Course of History
7. The FDA’s Answer to the Age-Old Question: Is Chocolate Good for Your Heart?
8. Surprising Causes of Cognitive Decline: Dementia Isn’t the Only Player in the Game
9. COVID Tracker Shuts Its Doors as the Pandemic Starts to Fade Away
10. Charles Silverstein: A Life of Selfless Advocacy to End Discrimination of the LGBTQ+ Community
11. What Does the End of Public COVID Vaccinations in the U.S. Mean for You?
12. Miracle in the Morgue: Dead Patient Miraculously Gasping for Air
13. Eggs Prices Dropping, Avian Flu Risk Still Lingers
14. Deer Are Now Carrying Variants of COVID Unseen in Humans
15. Caution: Devastating Side Effects of India-Made Eye Drops Linked to Blindness and Death
16. No Compromise: Uncovering UnitedHealthcare’s Refusal to Provide Care for Chronically Ill Patients
17. Comfort and Security: Senior Housing That Seniors Love

Is Charcoal Toothpaste Safe and Effective? Here’s What You Need to Know

Charcoal toothpaste has been rising in popularity through recent years. Many people are intrigued by its potential benefits for teeth whitening. However, there are concerns about its safety and efficacy. In this article, we will explore the facts about charcoal toothpaste and provide insights from dental professionals.

1. Is Charcoal Toothpaste Safe?

Charcoal is a natural substance and is often marketed as natural and safe. However, its safety on enamel is dependent on usage. Charcoal is abrasive, and long-term use can lead to enamel erosion and sensitivity. Therefore, it is important not to use charcoal toothpaste daily. Before using charcoal toothpaste, it is recommended to read the ingredients or consult with your dentist to ensure it is suitable for you. (source)

2. What Stains Can Charcoal Toothpaste Remove?

Activated charcoal toothpaste can help remove surface staining caused by coffee, tea, wine, or other dark foods. However, it may not be effective for deeper stains that have developed due to natural aging or medication. If you are unsure if charcoal toothpaste is suitable for your specific staining, it is recommended to consult with your dental care provider for personalized guidance. (source)

3. Can Charcoal Toothpaste Whiten Teeth After One Use?

While charcoal toothpaste can help remove superficial staining, it is not a quick fix for teeth whitening. The effects vary from person to person, and it may take weeks or even months to notice visible improvements. For more significant and long-lasting teeth whitening results, it is recommended to consult with a dentist for professional treatments. (source)

In Conclusion

Charcoal toothpaste contains rough particles that aid in superficial stain removal. However, there are other teeth whitening methods available that may be safer and more effective. It is highly recommended to seek advice from a dentist to find a teeth whitening product or treatment that aligns with your goals and oral health needs. Remember, maintaining good oral hygiene practices, such as regular brushing and flossing, is essential for a healthy smile. (source)