Here’s how the EU and industry will spend €120M on health research this spring

Posted on February 8th, 2024 by Paul Williams

The European Health Initiative (IHI) has announced new calls for interdisciplinary projects in health research and innovation. With a budget of €120 million, to be matched by industry, IHI aims to address gaps in health research and innovation that cannot be tackled by a single entity. The calls focus on five topics: heart disease management, data (under)use, biomarkers, healthcare workforce, and long-term management of illnesses.

These calls present an opportunity for researchers and organizations to collaborate on big, pre-competitive projects. The key to success in these collaborative projects is finding the right mix of partners. According to Niklas Blomberg, the executive director of IHI, researchers should start preparing their proposals early and form strong, collaborative networks. IHI provides support through webinars and brokerage events to help researchers ensure their proposals meet all the necessary requirements.

One of the topics in the current calls is “Helping patients stick with their medication.” Chronic disease management is a significant challenge, with studies showing that around half of those living with chronic diseases do not adhere to their prescribed medication for more than a year. IHI is looking for projects that can provide a better understanding of this issue and develop models to improve medication adherence, particularly for cardiometabolic diseases such as diabetes, obesity, and cardiovascular disease.

To enhance the reader’s understanding of the importance of medication adherence in chronic disease management, we can reference an article titled “A New Lease on Life: How NHS is Saving Children with Gene Therapy for MLD.” This article highlights the impact of innovative treatments, such as gene therapy, in improving patient outcomes and quality of life. By incorporating this article, we can emphasize the significance of research and innovation in healthcare and how projects funded by IHI can contribute to advancements in treatment and management of chronic diseases.

Another topic in the calls is “Practical guidance on the use of real-world data and evidence.” While there are high-level recommendations on utilizing real-world data, there is a lack of practical guidance. IHI aims to fund projects that develop practical recommendations to support drug development, regulation, health technology assessment, and payer decision-making. These recommendations should apply to drugs, medical devices, and drug-device combinations.

To provide additional insights on the use of real-world data and evidence, we can reference an article titled “Uncovering the Connection: Exploring the Link Between Hearing Voices and Hearing Your Own Voice.” This article discusses the use of real-world data in understanding auditory hallucinations and their impact on individuals. By incorporating this article, we can highlight the potential of real-world data in uncovering new insights and improving healthcare outcomes.

The remaining topics in the calls include “Better management of heart disease,” “Assisting healthcare workers,” and “Clinical validation of biomarkers.” Each of these topics addresses significant challenges in healthcare and offers opportunities for innovative solutions and advancements.

Overall, the calls by IHI provide a platform for collaborative projects that address complex healthcare challenges. By incorporating the referenced articles, we can emphasize the importance of research and innovation in improving patient outcomes and enhancing healthcare practices.


Scientists say ‘miracle’ weight loss and diabetes drugs could treat several other conditions including Alzheimer’s and Parkinson’s

Posted on February 7th, 2024 by Paul Williams

DRUGS hailed a “miracle” in the fight against obesity and diabetes may have several new “superpowers”, scientists say.

As well as helping people lose weight and keep blood sugar levels under control, glucagon-like peptide-1 receptor agonists (GLP-1 RA) could also improve the health of millions in other ways, research shows.

Miracle weight loss and diabetes drugs could treat several other conditions

They appear to have potential to treat Alzheimer’s and Parkinson’s, as well as heart disease, fatty liver disease, and chronic kidney disease.

Patients with sleep apnoea, heart failure and alcohol addiction could also benefit.

Daniel Drucker, a professor in the department of medicine at the University of Toronto, Canada, who studies the “superpower” meds, said: “One of the really interesting things about the GLP-1 drugs is that beyond the control of blood sugar and body weight, they also seem to reduce the complications of chronic metabolic disease.

“We know from clinical studies that GLP-1 does all this amazing stuff in people, but we don’t fully know how it works.”

Speaking to The Guardian, Dr Harshal Deshmukh, a consultant endocrinologist and senior clinical lecturer at the University of Hull, added: “Excessive weight has been identified as a significant risk factor for a range of comorbidities, including fatty liver, various cancers, dementia, and cardiovascular diseases.

“Given these associations, it’s not surprising that numerous clinical trials are currently investigating the potential impact of semaglutide on these health conditions.”

GLP-1 RAs like semaglutide and tirzepatide, sold under brand names such as Ozempic, Wegovy, Mounjaro and Zepbound, were first used to treat type 2 diabetes.

They were found to effectively manage blood glucose levels by increasing levels of incretins – a hormone which helps the body produce more insulin when needed and reduce the amount of glucose produced by the liver.

But researchers soon found they suppress users’ appetites – mimicking a hormone called glucagon-like peptide-1, which is usually released after eating.

As the medication makes people feel full, they tend to eat less and lose weight (around 10 per cent of their body weight).

Now, scientists have discovered several other potential wide-reaching uses.

Expanding the Benefits of GLP-1 RA Drugs

The recent advancements in the field of obesity and diabetes treatment have brought about a new wave of hope for millions of people worldwide. Glucagon-like peptide-1 receptor agonists (GLP-1 RA) have been hailed as “miracle” drugs due to their ability to help individuals lose weight and control blood sugar levels. However, recent research suggests that these drugs may have several additional “superpowers” that could revolutionize the treatment of various health conditions.

According to scientists, GLP-1 RA drugs show potential in treating Alzheimer’s and Parkinson’s diseases, heart disease, fatty liver disease, chronic kidney disease, sleep apnea, heart failure, and alcohol addiction. These drugs have demonstrated the ability to reduce the complications associated with chronic metabolic diseases, beyond their primary function of controlling blood sugar and body weight.

One of the most intriguing aspects of GLP-1 RA drugs is their impact on conditions like Alzheimer’s and Parkinson’s. Researchers have found that these medications can address multiple aspects of the metabolic system affected by these diseases, including inflammation and the production of amyloid protein. By improving glucose utilization and reducing inflammation in the body, including the brain, GLP-1 RA drugs could potentially slow the progression of Alzheimer’s disease.

Furthermore, studies have shown an association between GLP-1 RA drugs and a reduced risk of developing Parkinson’s disease. People with diabetes, who are around 40% more likely to develop Parkinson’s, have seen their symptoms progress more rapidly. Early trial results suggest that GLP-1 RA drugs could slow the progression of Parkinson’s, offering hope for improved treatment options.

Another exciting area of research is the potential use of GLP-1 RA drugs in treating sleep apnea. This condition, characterized by interrupted breathing during sleep, affects millions of people worldwide. Weight loss has been shown to alleviate symptoms, and researchers are now investigating whether GLP-1 RA drugs could have a similar effect. Initial trials with tirzepatide, one of the GLP-1 RA drugs, are currently underway, with the hope of finding a new treatment option for sleep apnea.

While the primary focus of GLP-1 RA drugs has been on diabetes and obesity, their potential to treat a wide range of conditions is becoming increasingly evident. These medications have already shown promise in treating fatty liver disease, heart failure, and chronic kidney disease. Ongoing clinical trials are exploring their impact on conditions like polycystic ovary syndrome (PCOS), which affects fertility, and their potential to regulate hormones and reduce symptoms.

It is important to note that the mechanisms behind the “superpowers” of GLP-1 RA drugs are not yet fully understood. Researchers are still uncovering the precise ways in which these medications work and their impact on various health conditions. However, the potential benefits they offer are undeniable, and further research is underway to explore their full potential.

As we continue to learn more about the capabilities of GLP-1 RA drugs, it is clear that they have the potential to revolutionize the treatment of obesity, diabetes, and a range of other health conditions. The ability to address multiple aspects of metabolic diseases and offer new treatment options for conditions like Alzheimer’s, Parkinson’s, and sleep apnea is truly remarkable. With ongoing research and advancements in this field, we can hope for even more breakthroughs in the future.


GoFundMe Is a Health-Care Utility Now

Posted on February 6th, 2024 by Paul Williams

Resorting to crowdfunding to pay medical bills has become so routine, in some cases health professionals recommend it.

GoFundMe started as a crowdfunding site for underwriting “ideas and dreams,” and, as GoFundMe’s co-founders, Andrew Ballester and Brad Damphousse, once put it, “for life’s important moments.” In the early years, it funded honeymoon trips, graduation gifts, and church missions to overseas hospitals in need. Now GoFundMe has become a go-to for patients trying to escape medical-billing nightmares.

One study found that, in 2020, the number of U.S. campaigns related to medical causes—about 200,000—was 25 times higher than the number of such campaigns on the site in 2011. More than 500 campaigns are currently dedicated to asking for financial help for treating people, mostly kids, with spinal muscular atrophy, a neurodegenerative genetic condition. The recently approved gene therapy for young children with the condition, by the drugmaker Novartis, costs about $2.1 million for the single-dose treatment.

Perhaps the most damning aspect of all this is that paying for expensive care with crowdfunding is no longer seen as unusual; instead, it is being normalized as part of the health system, like getting blood work done or waiting on hold for an appointment. Need a heart transplant? Start a GoFundMe in order to get on the waiting list. Resorting to GoFundMe when faced with bills has become so accepted that in some cases, patient advocates and hospital financial-aid officers recommend crowdfunding as an alternative to being sent to collections. My inbox and the Bill of the Month project (run by KFF Health News, where I am the senior contributing editor, and NPR) have become a kind of complaint desk for people who can’t afford their medical bills, and I’m gobsmacked every time a patient tells me they’ve been advised that GoFundMe is their best option.

GoFundMe itself acknowledges the reliance of patients on the company’s platform. Ari Romio, a spokesperson for the company, said that “medical expenses” is the most common category of fundraiser it hosts. But she declined to say what proportion of campaigns are medically related, because people starting a campaign self-select the purpose of the fundraiser. They might choose the family or travel category, she said, if a child needs to go to a different state for treatment, for example. So although the company has estimated in the past that a third of the funds raised on the site are medical-related, that could be an undercount.

Andrea Coy of Fort Collins, Colorado, turned to GoFundMe in 2021 as a last resort after an air-ambulance bill tipped her family’s finances over the edge. Her son Sebastian, then a year old, had been admitted with pneumonia to a local hospital and then transferred urgently by helicopter to Children’s Hospital Colorado in Denver when his oxygen levels dropped. REACH, the air-ambulance transport company that contracted with the hospital, was out-of-network, and billed the family nearly $65,000 for the ride—more than $28,000 of which Coy’s insurer, UnitedHealthcare, paid. Even so, REACH continued sending Coy’s family bills for the remaining balance, and later began regularly calling Coy to try to collect, enough that she felt the company was harassing her, she told me.

Coy made multiple calls to her company’s human-resources department, REACH, and UnitedHealthcare for help in resolving the case. She applied to various patient groups for financial assistance and was rejected again and again. Eventually, she got the outstanding balance knocked down to $5,000, but even that was more than she could afford on top of the $12,000 the family owed out-of-pocket for Sebastian’s actual treatment.

That’s when a hospital financial-aid officer suggested she try GoFundMe. But, as Coy said, “I’m not an influencer or anything like that,” so the appeal “offered only a bit of temporary relief—we’ve hit a wall.” They have gone deep into debt and hope to climb out of it.

In many respects, research shows, GoFundMe tends to perpetuate socioeconomic disparities that already affect medical bills and debt. If you are famous or part of a circle of friends who have money, your crowdfunding campaign is much more likely to succeed than if you are middle-class or poor. When the family of the former Olympic gymnast Mary Lou Retton started a fundraiser on another platform, *spotfund, for her recent ICU stay at a time when she was uninsured, nearly $460,000 in donations quickly poured in. (Although Retton said she could not get affordable insurance because of her preexisting condition—dozens of orthopedic surgeries—the Affordable Care Act prohibits insurers from refusing to cover people because of their prior medical histories, or charging them abnormally high rates.)

And given the price of American health care, even the most robust fundraising can feel inadequate. If you’re looking for help to pay for a $2 million drug, even tens of thousands is a drop in the bucket.

Rob Solomon, the CEO of the platform from 2015 to March 2020, who was named one of Time magazine’s 50 most influential people in health care, has said that he “would love nothing more than for ‘medical’ to not be a category on GoFundMe.” He told KFF Health News that “the system is terrible. It needs to be rethought and retooled. Politicians are failing us. Health-care companies are failing us. Those are realities.”

But despite the noble ambitions of its original vision, GoFundMe is a privately held for-profit company. In 2015, the founders sold a majority stake to a venture-capital investor group led by Accel Partners and Technology Crossover Ventures. And when I asked about medical bills being the most common reason for GoFundMe campaigns, the company’s current CEO, Tim Cadogan, sounded less critical than his predecessor of the health system, whose high prices and financial cruelty have arguably made his company famous.

“Our mission is to help people help each other,” he said. “We are not, and cannot, be the solution to complex, systemic problems that are best solved with meaningful public policy.”

And that’s true. Despite the site’s hopeful vibe, most campaigns generate only a small fraction of the money owed. Almost all of the medical-expense campaigns in the U.S. fell short of their goal, and some raised little or no money, a 2017 study from the University of Washington found. The average campaign made it to just about 40 percent of the target amount, and there is evidence that yields—measured as a percent of their target—have gotten worse over time.

Carol Justice, a recently retired civil servant and a longtime union member in Portland, Oregon, turned to GoFundMe after she faced a mammoth unexpected bill for bariatric surgery at Oregon Health & Science University.

She had expected to pay about $1,000, the amount left in her deductible, after her health insurer paid the $15,000 cap on the surgery. She didn’t understand that a cap meant she would have to pay the difference if the hospital, which was in-network, charged more.

And it did, leaving her with a bill of $18,000, to be paid all at once or in monthly $1,400 increments. “That’s more than my mortgage,” she told me. “I was facing filing for bankruptcy or losing my car and my house.” She made numerous calls to the hospital’s financial-aid office, many unanswered, and received only unfulfilled promises that “we’ll get back to you” about whether she qualified for help.

So, Justice said, her health coach—provided by the city of Portland—suggested starting a GoFundMe. The campaign yielded about $1,400, just one monthly payment, including $200 from the health coach and $100 from an aunt. She dutifully sent each donation directly to the hospital.

In an emailed response, the hospital system said that it couldn’t discuss individual cases, but that “financial assistance information is readily available for patients, and can be accessed at any point in a patient’s journey with OHSU. Starting in early 2019, OHSU worked to remove barriers for patients most in need by providing a quick screening for financial assistance that, if a certain threshold is met, awards financial assistance without requiring an application process.”

This particular tale has a happy-ish ending. In desperation, Justice went to the hospital and planted herself in the financial-aid office, where she had a tearful meeting with a hospital representative who determined that—given her finances—she wouldn’t have to pay the bill.

“I’d been through the gamut and just cried,” she said. She told me that she would like to repay the people who donated to her GoFundMe. But so far, the hospital won’t give the $1,400 back.

Elisabeth Rosenthal is the senior contributing editor at KFF Health News. A former senior writer at The New York Times, she is the author of An American Sickness: How Healthcare Became Big Business and How You Can Take It Back. She practiced as an ER doctor, before converting to journalism.


  1. GoFundMe CEO: Gigantic Gaps in Health System Showing Up in Crowdfunding

Scientists narrow down 70 culprits in race to prevent next pandemic

Posted on February 5th, 2024 by Paul Williams

Scientists have made a significant breakthrough in the hunt for Disease X, narrowing down the potential pandemic threats to just 70 virus families. Disease X is a hypothetical, currently unknown pathogen that was added to the World Health Organisation’s list of priority diseases in 2018. The WHO has warned that Disease X could be even more deadly than the Covid-19 pandemic, potentially killing 20 times more people.

In a recent study published in the journal Molecular Biology and Evolution, scientists from the University of Edinburgh, Liverpool, and Peking University in China analyzed the family tree of 743 distinct viruses to identify the virus lineages that pose the biggest risk. They found that human viruses, which can already spread within human populations, are more likely to become endemic and pose a significant threat.

Examples of infections that spread easily between humans include the common cold, Covid-19, flu, measles, and whooping cough. These viruses have the potential to cause widespread outbreaks and have a high epidemic potential. By focusing on virus families related to existing human viruses with epidemic potential, scientists can narrow down the search for the next Disease X.

The research conducted by Scottish scientists is a crucial step in preparing for future pandemics. It provides valuable insights into the types of viruses that are most likely to cause widespread outbreaks and allows for targeted surveillance and prevention efforts. By understanding the characteristics of these virus families, scientists can develop strategies to mitigate the risks and potentially prevent the emergence of a new pandemic.

While the search for Disease X continues, it is essential to remain vigilant and prepared. The World Health Organisation has identified nine priority diseases that pose the most urgent threat to humanity, including Covid-19, Crimean-Congo haemorrhagic fever, Ebola and Marburg, Lassa fever, Middle Eastern respiratory syndrome (MERS) and severe acute respiratory syndrome (SARS), Nipah disease, Rift valley fever, Zika, and Disease X.

Preparations for the next pandemic are already underway, with efforts to develop vaccines and improve healthcare systems. The UK Health Security Agency has started trialling the world’s first jab for Crimean-Congo haemorrhagic fever, and Britain’s new vaccine lab is being prepared to respond to the unknown Disease X. These proactive measures are crucial in ensuring that we are better equipped to handle future pandemics.

To learn more about the potential threats and ongoing efforts to combat pandemics, you can read the article on the World Health Organisation’s priority diseases. It provides a comprehensive overview of the diseases that pose the most significant risk to public health and highlights the importance of preparedness and global cooperation.

As we continue to navigate the challenges of the Covid-19 pandemic, it is crucial to remain informed and proactive in our approach to public health. By understanding the potential pandemic threats and taking necessary precautions, we can work towards preventing future outbreaks and protecting global health.


  1. Scientists have narrowed down the next pandemic threat to just 70 virus families
  2. Measles symptoms and how to protect kids
  3. The WHO’s nine priority diseases that pose the biggest risk to public health (plus Disease X)

Women with polycystic ovary syndrome may have higher suicide risk, study says | Polycystic ovary syndrome | The Guardian

Posted on February 5th, 2024 by Paul Williams

Women diagnosed with polycystic ovary syndrome (PCOS) may face a higher risk of attempting suicide, according to a recent study conducted in Taiwan. PCOS is a common health condition that affects approximately one in 10 women in the UK, although many cases go undiagnosed. The condition is characterized by symptoms such as irregular periods, acne, obesity, and cysts in the ovaries, and it is also recognized as a leading cause of infertility by the World Health Organization.

The study, published in the Annals of Internal Medicine, analyzed nationwide data from 8,960 women and girls aged 12 to 64 who were diagnosed with PCOS between 1997 and 2012. The researchers found that those with PCOS had an 8.47-fold higher risk of suicide attempts compared to women without PCOS but with similar characteristics. The risk was particularly elevated among adolescents and adults under 40.

The authors of the study suggest that concerns related to PCOS, such as potential infertility and body image issues, could contribute to the increased risk of suicide attempts. Body image concerns, including perceived obesity and acne, have been associated with suicide risk during adolescence, and these problems are common among adolescents with PCOS. Additionally, young adults with PCOS may face additional challenges such as unemployment, financial difficulties, and relationship problems.

While this study adds to the growing body of evidence linking PCOS and mental health issues, it is important to note that the research has limitations. It cannot establish a cause-and-effect relationship, and it may not have accounted for all possible factors that could influence the results. However, other studies have also reported similar findings, highlighting the need for further research in this underexplored area.

Dr. Sophie Williams of the University of Derby, who was not involved in the study, emphasizes the importance of seeking help for women with PCOS who are experiencing difficulties. Depression and anxiety are commonly associated with PCOS, and it is crucial for individuals to know that support is available. In the UK, the NHS provides resources and assistance for those in need, and organizations like Samaritans offer helplines and online support for individuals experiencing emotional distress.

While the study sheds light on the mental health challenges faced by women with PCOS, it is essential to approach the topic with empathy and understanding. PCOS is a complex condition that affects individuals differently, and each person’s experience is unique. By raising awareness and conducting further research, we can work towards better support and care for those living with PCOS.

For more information on PCOS and its impact on mental health, you can refer to the article “Self-Harm and Suicidal Ideation Among Women with PCOS” by Dr. Sophie Williams. This article explores the connection between PCOS and mental health, providing valuable insights into the challenges faced by individuals with the condition.

If you or someone you know is struggling with mental health issues, it is important to seek help. In the UK and Ireland, Samaritans can be contacted on freephone 116 123, or via email at jo@samaritans.org or jo@samaritans.ie. In the US, the National Suicide Prevention Lifeline offers support through calls, texts, and online chat. You can reach them at 988 or visit their website at 988lifeline.org or crisistextline.org. In Australia, Lifeline provides crisis support at 13 11 14. For international helplines, you can visit befrienders.org.

First UK patients receive experimental messenger RNA cancer therapy

Posted on February 4th, 2024 by Paul Williams

A revolutionary new cancer treatment known as mRNA therapy has been administered to patients at Hammersmith hospital in west London. The trial has been set up to evaluate the therapy’s safety and effectiveness in treating melanoma, lung cancer and other solid tumours.

The new treatment uses genetic material known as messenger RNA – or mRNA – and works by presenting common markers from tumours to the patient’s immune system.

The aim is to help it recognise and fight cancer cells that express those markers.

“New mRNA-based cancer immunotherapies offer an avenue for recruiting the patient’s own immune system to fight their cancer,” said Dr David Pinato of Imperial College London, an investigator with the trial’s UK arm.

Pinato said this research was still in its early stages and could take years before becoming available for patients. However, the new trial was laying crucial groundwork that could help develop less toxic and more precise new anti-cancer therapies. “We desperately need these to turn the tide against cancer,” he added.

A number of cancer vaccines have recently entered clinical trials across the globe. These fall into two categories: personalised cancer immunotherapies, which rely on extracting a patient’s own genetic material from their tumours; and therapeutic cancer immunotherapies, such as the mRNA therapy newly launched in London, which are “ready made” and tailored to a particular type of cancer.

The primary aim of the new trial – known as Mobilize – is to discover if this particular type of mRNA therapy is safe and tolerated by patients with lung or skin cancers and can shrink tumours. It will be administered alone in some cases and in combination with the existing cancer drug pembrolizumab in others.

Researchers say that while the experimental therapy is still in the early stages of testing, they hope it may ultimately lead to a new treatment option for difficult-to-treat cancers, should the approach be proven to be safe and effective.

Nearly one in two people in the UK will be diagnosed with cancer in their lifetime. A range of therapies have been developed to treat patients, including chemotherapy and immune therapies.

However, cancer cells can become resistant to drugs, making tumours more difficult to treat, and scientists are keen to seek new approaches for tackling cancers.

Preclinical testing in both cell and animal models of cancer provided evidence that new mRNA therapy had an effect on the immune system and could be offered to patients in early-phase clinical trials.

A landmark gene-editing treatment for sickle cell disease moves closer to reality

Posted on October 31st, 2023 by Paul Williams

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

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.


Eyedrops from CVS, Rite Aid and others carry possible infection risk, FDA says

Posted on October 30th, 2023 by Paul Williams

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.

Herpes Cure Advocacy

Posted on October 29th, 2023 by Paul Williams

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.


Molecular defenses

Posted on October 28th, 2023 by Paul Williams

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.

Anti-PEG antibodies circulate in the blood of many people and bind to PEGylated 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