The US Food and Drug Administration has approved the first continuous glucose monitor (CGM) people can buy without a prescription. Dexcom's Stelo Glucose Biosensor System has a sensor users are meant to insert into their upper arm, similar to the company's other CGMs that need a doctor's prescription for purchase. It pairs with a smartphone application that can show the user's blood glucose measurements and trends every 15 minutes.
The company designed the device specifically for adults 18 and up who are not using insulin, such as those managing their diabetes with oral medications and non-diabetics making a conscious effort to control their sugar intake. It could be a great tool for people with insulin resistance, including individuals with PCOS and other metabolic issues that heighten their probability of developing diabetes in the future. In general, it could give users the insight to be able to better understand how the food they eat and the movements they make impact their overall health.
While CGMs aren't anything new, they've become a wellness trend on social media last year, and even non-diabetics started using them. By clearing Stelo, the FDA is making the monitors more accessible than before. "CGMs can be a powerful tool to help monitor blood glucose," said Jeff Shuren, MD, director of the FDA's Center for Devices and Radiological Health. "Today's clearance expands access to these devices by allowing individuals to purchase a CGM without the involvement of a health care provide. Giving more individuals valuable information about their health, regardless of their access to a doctor or health insurance, is an important step forward in advancing health equity for U.S. patients."
Stelo will be available starting this summer. Each patch is meant to last for 15 days before users will need to replace it. Dexcom has yet to reveal how much it would cost, but it said Stelo will "provide an option for those who do not have insurance coverage for CGM."
This article originally appeared on Engadget at https://www.engadget.com/fda-approves-the-first-over-the-counter-continuous-glucose-monitor-130008629.html?src=rss
There’s a reason smartwatches haven’t replaced clinically-validated gear when you visit the hospital — accuracy and reliability are paramount when the data informs medical procedures. Even so, researchers are looking for ways in which these devices can be meaningfully used in a clinical setting. One project in the UK has explored if a Garmin Venu 2 and dedicated companion app could be used to free up doctors and nurses, six minutes at a time.
The Six Minute Walk Test (6MWT) is used to diagnose and monitor a number of cardiovascular maladies. This includes conditions like Pulmonary Hypertension that, if left untreated, are eventually fatal. “[The test has been] a cornerstone of hospital practice and clinical trials for decades all around the world as [...] a marker of how well the heart and lungs are working,” explained project leader Dr. Joseph Newman. While a change in a blood test marker might be clinically relevant, Newman said “it’s probably more important to someone that they can walk to the shop and back.” The test requires a patient walk on a flat, hard surface for six minutes straight, which stresses the heart enough to measure its capacity. A professional tests the patient’s heart rate and blood oxygen levels at the start and end, and while it’s simple and reliable, "it’s not perfect,” according to Newman. “This is why we’ve looked to change it in two important ways," he said, "can we make it shorter [...] and digitize it for remote use?"
After all, six minutes is a lifetime in a clinical setting, and patients dislike having to schlep all the way to their hospital just to walk up and down a corridor. It’s why Newman and Lucy Robertson — both researchers at the Royal Papworth Hospital in Cambridge — began looking for ways to revolutionize the test. They wanted to see if the test could be shortened to a single minute, and also if it could be carried out by a patient at home using a Venu 2. The watch was connected to a secure and dedicated clinical trial platform built by Aparito – a Wrexham-based developer – for testing. This was then sent out to patients who were instructed to wear the watch and walk outdoors to complete their own tests. “They’re asked to walk on flat, even, dry, relatively straight roads rather than in laps or circuits,” said Dr. Newman, with patients walking at their own natural pace.
“We carried out a product appraisal early on in the research process and were open-minded as to the brand or model,” said Newman. “Garmin came out on top for a few reasons; we can access raw data as well as Garmin’s algorithmically-derived variables,” he said. Because the research was being funded by a charity, the British Heart Foundation, the watch had to offer good value for money. It helped that Garmin, because of its existing health research division, gave the team “confidence in the accuracy of the sensors,” not to mention the fact that Aparito feels that “the Garmin SDK is relatively easy to work with,” he said. But while Garmin is in use right now, there’s no reason this setup couldn’t eventually work with a number of other brands. “As long as the technology works, it’s accurate, reliable and patients accept it, then we’re not tied to any brand.”
There are several benefits in giving patients the ability to run the tests at home: it’s more representative of the demands of their actual life, and patients can retake the test at regular intervals, making it easier to track that person’s health over time. “We can see real value in providing patients with pulmonary hypertension with an app and smartwatch to monitor their progress,”said Dr. Newman. “It’s unlikely to ever fully replace the need for in-person hospital reviews, but it will likely reduce their frequency.”
The results of the study right now suggest cutting the test to one minute has no detrimental effect on its outcome or accuracy,and that patients are far more likely to run the test regularly if they’re able to do so at home. “It’s likely that the upfront costs of wearables [to a hospital] may be offset by the longer term reduction in hospital visits,” said Newman. If that turns out to be right, then it means clinicians can better focus their time and efforts where their expertise is more valuable.
This article originally appeared on Engadget at https://www.engadget.com/dr-garmin-will-see-you-now-160013340.html?src=rss
The “P” in HIPAA doesn’t stand for privacy. It’s one of the first things a lot of experts will say when asked to clear up any misconceptions about the health data law. Instead, it stands for portability — it’s called the Health Insurance Portability and Accountability Act —and describes how information can be transferred between providers. With misinterpretations of HIPAA starting with just its name, misunderstandings of what the law actually does greatly impact our ability to recognize how the kinds of data do and don't fall under its scope. That’s especially true as a growing number of consumer tech devices and services gather troves of information related to our health.
We often consider HIPAA a piece of consumer data privacy legislation because it did direct the Department of Health and Human Services to come up with certain security provisions, like breach notification regulations and a health privacy rule for protecting individually identifiable information. But when HIPAA went into effect in the 1990s, its primary aim was improving how providers worked with insurance companies. Put simply, “people think HIPAA covers more than it actually does,” said Daniel Solove, professor at George Washington University and CEO of privacy training firm TeachPrivacy.
HIPAA has two big restrictions in scope: a limited set of covered entities, and limited set of covered data, according to Cobun Zweifel-Keegan, DC managing director of the International Association of Privacy Professionals. Covered entities include healthcare providers like doctors and health plans like health insurance companies. The covered data refers to medical records and other individually identifiable health information used by those covered entities. Under HIPAA, your general practitioner can't sell data related to your vaccination status to an ad firm, but a fitness app (which wouldn't be a covered entity) that tracks your steps and heart rate (which aren't considered covered data) absolutely can.
“What HIPAA covers, is information that relates to health care or payment for health care, and sort of any piece of identifiable information that’s in that file,” Solove said. It doesn’t cover any health information shared with your employer or school, like if you turn in a sick note, but it does protect your doctor from sharing more details about your diagnosis if they call to verify.
A lot has changed in the nearly 30 years since HIPAA went into effect, though. The legislators behind HIPAA didn’t anticipate how much data we would be sharing about ourselves today, much of which can be considered personally identifiable. So, that information doesn’t fall under its scope. “When HIPAA was designed, nobody really anticipated what the world was going to look like,” Lee Tien, senior staff attorney at the Electronic Frontier Foundation said. It’s not badly designed, HIPAA just can’t keep up with the state we’re in today. “You're sharing data all the time with other people who are not doctors or who are not the insurance company,” said Tien.
Think of all the data collected about us on the daily that could provide insight into our health. Noom tracks your diet. Peloton knows your activity levels. Calm sees you when you’re sleeping. Medisafe knows your pill schedule. Betterhelp knows what mental health conditions you might have, and less than a year ago was banned by the FTC from disclosing that information to advertisers. The list goes on, and much of it can be used to sell dietary supplements or sleep aids or whatever else. “Health data could be almost limitless,” so if HIPAA didn’t have a limited scope of covered entities, the law would be limitless, too, Solove said.
Not to mention the amount of inferences that firms can make about our health based on other data. An infamous 2012 New York Times investigation detailed how just by someone’s online searches and purchases, Target can figure out that they’re pregnant. HIPAA may not protect your medical information from being viewed by law enforcement officers. Even without a warrant, cops can get your records just by saying that you’re a suspect (or victim) of a crime. Police have used pharmacies to gather medical data about suspects, but other types of data like location information can provide sensitive details, too. For example, it can show that you went to a specific clinic to receive care. Because of these inferences, laws like HIPAA won’t necessarily stop law enforcement from prosecuting someone based on their healthcare decision.
Today, state-specific laws crop up across the US to help target some of the health data privacy gaps that HIPAA doesn’t cover. This means going beyond just medical files and healthcare providers to encompass more of people’s health data footprint. It varies between states, like in California which provides options to charge anyone who negligently discloses medical information or some additional breach protections for consumers based in Pennsylvania, but Washington state recently passed a law specifically targeting HIPAA’s gaps.
Washington State’s My Health My Data Act, passed last year, aims to “protect personal health data that falls outside the ambit of the Health Insurance Portability and Accountability Act,” according to a press release from Washington’s Office of the Attorney General. Any entity that conducts business in the state of Washington and deals with personal information that identifies a consumer’s past, present or future physical or mental health status must comply with the act’s privacy protections. Those provisions include the right not to have your health data sold without your permission and having health data deleted via written request. Under this law, unlike HIPAA, an app tracking someone’s drug dosage and schedule or the inferences made by Target about pregnancy would be covered.
My Health My Data is still rolling out, so we’ll have to wait and see how the law impacts national health data privacy protections. Still, it’s already sparking copycat laws in states like Vermont.
This article originally appeared on Engadget at https://www.engadget.com/hipaa-protects-health-data-privacy-but-not-in-the-ways-most-people-think-184026402.html?src=rss
If there’s one thing we can all agree upon, it’s that the 21st century’s captains of industry are trying to shoehorn AI into every corner of our world. But for all of the ways in which AI will be shoved into our faces and not prove very successful, it might actually have at least one useful purpose. For instance, by dramatically speeding up the often decades-long process of designing, finding and testing new drugs.
Risk mitigation isn’t a sexy notion but it’s worth understanding how common it is for a new drug project to fail. To set the scene, consider that each drug project takes between three and five years to form a hypothesis strong enough to start tests in a laboratory. A 2022 study from Professor Duxin Sun found that 90 percent of clinical drug development fails, with each project costing more than $2 billion. And that number doesn’t even include compounds found to be unworkable at the preclinical stage. Put simply, every successful drug has to prop up at least $18 billion waste generated by its unsuccessful siblings, which all but guarantees that less lucrative cures for rarer conditions aren’t given as much focus as they may need.
Dr. Nicola Richmond is VP of AI at Benevolent, a biotech company using AI in its drug discovery process. She explained the classical system tasks researchers to find, for example, a misbehaving protein – the cause of disease – and then find a molecule that could make it behave. Once they've found one, they need to get that molecule into a form a patient can take, and then test if it’s both safe and effective. The journey to clinical trials on a living human patient takes years, and it’s often only then researchers find out that what worked in theory does not work in practice.
The current process takes “more than a decade and multiple billions of dollars of research investment for every drug approved,” said Dr. Chris Gibson, co-founder of Recursion, another company in the AI drug discovery space. He says AI’s great skill may be to dodge the misses and help avoid researchers spending too long running down blind alleys. A software platform that can churn through hundreds of options at a time can, in Gibson’s words, “fail faster and earlier so you can move on to other targets.”
Dr. Anne E. Carpenter is the founder of the Carpenter-Singh laboratory at the Broad Institute of MIT and Harvard. She has spent more than a decade developing techniques in Cell Painting, a way to highlight elements in cells, with dyes, to make them readable by a computer. She is also the co-developer of Cell Profiler, a platform enabling researchers to use AI to scrub through vast troves of images of those dyed cells. Combined, this work makes it easy for a machine to see how cells change when they are impacted by the presence of disease or a treatment. And by looking at every part of the cell holistically – a discipline known as “omics” – there are greater opportunities for making the sort of connections that AI systems excel at.
Using pictures as a way of identifying potential cures seems a little left-field, since how things look don’t always represent how things actually are, right? Carpenter said humans have always made subconscious assumptions about medical status from sight alone. She explained most people may conclude someone may have a chromosomal issue just by looking at their face. And professional clinicians can identify a number of disorders by sight alone purely as a consequence of their experience. She added that if you took a picture of everyone’s face in a given population, a computer would be able to identify patterns and sort them based on common features.
This logic applies to the pictures of cells, where it’s possible for a digital pathologist to compare images from healthy and diseased samples. If a human can do it, then it should be faster and easier to employ a computer to spot these differences in scale so long as it’s accurate. “You allow this data to self-assemble into groups and now [you’re] starting to see patterns,” she explained, “when we treat [cells] with 100,000 different compounds, one by one, we can say ‘here’s two chemicals that look really similar to each other.’” And this looking really similar to each other isn’t just coincidence, but seems to be indicative of how they behave.
In one example, Carpenter cited that two different compounds could produce similar effects in a cell, and by extension could be used to treat the same condition. If so, then it may be that one of the two – which may not have been intended for this purpose – has fewer harmful side effects. Then there’s the potential benefit of being able to identify something that we didn’t know was affected by disease. “It allows us to say, ‘hey, there’s this cluster of six genes, five of which are really well known to be part of this pathway, but the sixth one, we didn’t know what it did, but now we have a strong clue it’s involved in the same biological process.” “Maybe those other five genes, for whatever reason, aren’t great direct targets themselves, maybe the chemicals don’t bind,” she said, “but the sixth one [could be] really great for that.”
In this context, the startups using AI in their drug discovery processes are hoping that they can find the diamonds hiding in plain sight. Dr. Richmond said that Benevolent’s approach is for the team to pick a disease of interest and then formulate a biological question around it. So, at the start of one project, the team might wonder if there are ways to treat ALS by enhancing, or fixing, the way a cell’s own housekeeping system works. (To be clear, this is a purely hypothetical example supplied by Dr. Richmond.)
That question is then run through Benevolent’s AI models, which pull together data from a wide variety of sources. They then produce a ranked list of potential answers to the question, which can include novel compounds, or existing drugs that could be adapted to suit. The data then goes to a researcher, who can examine what, if any, weight to give to its findings. Dr. Richmond added that the model has to provide evidence from existing literature or sources to support its findings even if its picks are out of left-field. And that, at all times, a human has the final say on what of its results should be pursued and how vigorously.
It’s a similar situation at Recursion, with Dr. Gibson claiming that its model is now capable of predicting “how any drug will interact with any disease without having to physically test it.” The model has now formed around three trillion predictions connecting potential problems to their potential solutions based on the data it has already absorbed and simulated. Gibson said that the process at the company now resembles a web search: Researchers sit down at a terminal, “type in a gene associated with breast cancer and [the system] populates all the other genes and compounds that [it believes are] related.”
“What gets exciting,” said Dr. Gibson, “is when [we] see a gene nobody has ever heard of in the list, which feels like novel biology because the world has no idea it exists.” Once a target has been identified and the findings checked by a human, the data will be passed to Recursion’s in-house scientific laboratory. Here, researchers will run initial experiments to see if what was found in the simulation can be replicated in the real world. Dr. Gibson said that Recursion’s wet lab, which uses large-scale automation, is capable of running more than two million experiments in a working week.
“About six weeks later, with very little human intervention, we’ll get the results,” said Dr. Gibson and, if successful, it’s then the team will “really start investing.” Because, until this point, the short period of validation work has cost the company “very little money and time to get.” The promise is that, rather than a three-year preclinical phase, that whole process can be crunched down to a few database searches, some oversight and then a few weeks of ex vivo testing to confirm if the system’s hunches are worth making a real effort to interrogate. Dr. Gibson said that it believes it has taken a “year’s worth of animal model work and [compressed] it, in many cases, to two months.”
Of course, there is not yet a concrete success story, no wonder cure that any company in this space can point to as a validation of the approach. But Recursion can cite one real-world example of how close its platform came to matching the success of a critical study. In April 2020, Recursion ran the COVID-19 sequence through its system to look at potential treatments. It examined both FDA-approved drugs and candidates in late-stage clinical trials. The system produced a list of nine potential candidates which would need further analysis, eight of which it would later be proved to be correct. It also said that Hydroxychloroquine and Ivermectin, both much-ballyhooed in the earliest days of the pandemic, would flop.
And there are AI-informed drugs that are currently undergoing real-world clinical trials right now. Recursion is pointing to five projects currently finishing their stage one (tests in healthy patients), or entering stage two (trials in people with the rare diseases in question) clinical testing right now. Benevolent has started a stage one trial of BEN-8744, a treatment for ulcerative colitis that may help with other inflammatory bowel disorders. And BEN-8744 is targeting an inhibitor that has no prior associations in the existing research which, if successful, will add weight to the idea that AIs can spot the connections humans have missed. Of course, we can’t make any conclusions until at least early next year when the results of those initial tests will be released.
There are plenty of unanswered questions, including how much we should rely upon AI as the sole arbiter of the drug discovery pipeline. There are also questions around the quality of the training data and the biases in the wider sources more generally. Dr. Richmond highlighted the issues around biases in genetic data sources both in terms of the homogeneity of cell cultures and how those tests are carried out. Similarly, Dr. Carpenter said the results of her most recent project, the publicly available JUMP-Cell Painting project, were based on cells from a single participant. “We picked it with good reason, but it’s still one human and one cell type from that one human.” In an ideal world, she’d have a far broader range of participants and cell types, but the issues right now center on funding and time, or more appropriately, their absence.
But, for now, all we can do is await the results of these early trials and hope that they bear fruit. Like every other potential application of AI, its value will rest largely in its ability to improve the quality of the work – or, more likely, improve the bottom line for the business in question. If AI can make the savings attractive enough, however, then maybe those diseases which are not likely to make back the investment demands under the current system may stand a chance. It could all collapse in a puff of hype, or it may offer real hope to families struggling for help while dealing with a rare disorder.
This article originally appeared on Engadget at https://www.engadget.com/ai-is-coming-for-big-pharma-150045224.html?src=rss
The FDA has provided clearance for a medical device called Osteoboost, a vibrating belt that improves bone density in patients with osteopenia. The device, which was developed by California-based startup Bone Health Technologies and in part with NASA, is the first medical device of its kind to get regulatory approval as a treatment option for postmenopausal women.
One in two older women who have experienced menopause gets osteoporosis (the disease that comes after prolonged and untreated osteopenia), which is characterized by porous bones that can easily fracture. The Osteoboost belt is designed to prevent bone density from reaching that stage through early intervention. It works by mechanically stimulating the strength of the bones in the hips and spine of a wearer and prevents the further progression of bone density disintegration. The blueprint for the technology comes from NASA research that was investigating ways to prevent bone density from weakening in astronauts that work in mostly zero gravity environments where deterioration becomes a concern.
The belt should be worn for 30 minutes every day or at least five times a week for it to fully take effect. It delivers a gentle vibration that makes it easy to be worn pretty much anywhere or at any time, such as during dog walks or while washing dishes. During clinical trials, CT scans showed that following the integration of the belt into a patient’s care plan, bone density visually improved over time. In a study backed by the NIH, women aged 50 to 60 lost 3.4 percent of their bone density by the end of 12 months without any intervention, while patients who wore the belt lost only 0.5 percent of their bone strength.
Current standards of care for preventing osteoporosis during the osteopenia stage are mostly lifestyle suggestions that can be hard to adhere to, such as a well-balanced and calcium-rich diet, frequent weight-bearing exercises and reducing the risk of falls. “Although lifestyle interventions such as exercise and diet are beneficial to bone, the effect is small. The Osteoboost shows promise in slowing the loss of bone density and strength and may fill the treatment gap,” Laura Bilek, a researcher who has studied the belt’s effectiveness said.
Osteoboost is still not yet available for sale, but you can sign up to get notified when the device is released. A company representative said they will begin shipping later this year and will accept pre-orders in the next few months. While the price is also still not disclosed, the representative told Engadget that the belt will be “affordable and accessible to the millions of patients who need it.” To get the device, you will need a prescription from your doctor — so pricing may vary depending on insurers and co-pays. Bone Health Technology said it is currently in talks with insurers regarding coverage for the medical device. While the price projection could have drastically changed, three years ago the CEO Laura Yecies told NS Medical Devices she believed the device could debut for about $800.
This article originally appeared on Engadget at https://www.engadget.com/vibrating-belt-that-treats-low-bone-density-gets-fda-approval-181552362.html?src=rss
The US government has reportedly approved AI-based memory loss prediction software for the first time. Darmiyan, a San Francisco-based brain imaging analytics company, says the FDA has granted De Novo approval for its product BrainSee. The software platform assigns “an objective score that predicts the likelihood of progression from aMCI to Alzheimer’s dementia within 5 years,” according to the medical company. Fierce Biotech first reported the announcement.
Darmiyan says BrainSee can predict memory loss progression using clinical brain MRIs and cognitive tests, which are already standard for patients worried about early signs of decline. After the program analyzes the imaging and cognitive assessments, it assigns a predictive score indicating the patient’s odds of memory deterioration within the following five years. At least in theory, that would lead to early treatment for some and peace of mind for others.
“This shifts the patient experience from prolonged anxiety to proactive management, which is crucial in an era of emerging Alzheimer’s treatments where accurate prognosis can help determine suitable treatment candidates,” Darmiyan wrote in a press release announcing the FDA approval. “The economic impact of BrainSee will be significant for all stakeholders in healthcare, promising to reduce the billions of dollars annually spent on Alzheimer’s care, through more effective management and treatment.”
The FDA’s “De Novo” designation means the product has no clear market predecessors but has proven its effectiveness and safety in clinical trials. BrainSee first received FDA “breakthrough” designation in 2021, an earlier stage of the approval path for a first-of-its-kind treatment.
Darmiyan says BrainSee is fully automated and provides results on the same day the scans and cognitive test scores are entered. The company views the tech as shifting the treatment of mild / early cognitive decline from biomarker-based methods to “non-invasive and actionable forecasts of future improvement or progression.”
This article originally appeared on Engadget at https://www.engadget.com/the-fda-has-reportedly-approved-an-ai-product-that-predicts-cognitive-decline-184534034.html?src=rss
Following last year's smart toilet which debuted at CES 2023, Vivoo is at it again for CES 2024 with another urine analysis product. The company has unveiled an at-home digital urinary tract infection (UTI) testing kit that provides what it calls "gold standard accuracy results" via a two-minute test.
To use it, just pee on the provided UTI test strip and scan it to obtain results via Vivoo's app in "seconds," the company says. If the result is positive, customers can then connect with a doctor to obtain a prescription if required. The company says the product "saves customers time, prevents confusion in readings, and digitalizes the data so customers can share results with healthcare providers via the app, if instant treatment is desired." From the looks of it, the results are obtained via the strip, then deciphered by the app.
Vivoo notes that UTIs are the most common type of outpatient infection, with six in ten women experiencing them in their lifetimes. Normally, you'd send your urine off to a lab for analysis, or use an existing at-home test kit. The company says that the new product spares users the bureaucracy of lab testing while also keeping the relevant data for users who might need that, unlike regular testing kits.
In fact, many women experience recurrent UTIs, which have become resistant to at least one or even multiple types of antibiotics. By keeping a record of past infections, Vivoo's app could help patients and medical professionals track the problem and treat it appropriately.
Last year, the company unveiled a smart toilet device that clips onto existing toilets and provides data like your body's water, magnesium, PH, protein and sodium levels. Later on, it released strips for vaginal PH levels. The new home UTI test will come to market in Q2 2024, but pricing isn't yet available.
We're reporting live from CES 2024 in Las Vegas from January 6-12. Keep up with all the latest news from the show here.
This article originally appeared on Engadget at https://www.engadget.com/vivoos-new-at-home-uti-test-kit-and-app-can-tell-you-if-you-have-a-urinary-tract-infection-030021462.html?src=rss
2023 was an important year for patients with sickle cell disease. Prior to CRISPR, the only cure for the life-long ailment was a bone marrow transplant, which is notoriously dangerous and costly. This month, the FDA approved Vertex’s “Casgevy,” a CRISPR-based therapy for the treatment of sickle cell disease in patients 12 and older. The landmark approval made the therapeutic the first genetically edited therapy to reach the general market.
Casgevy, which also received the greenlight from regulators in the UK for another blood disorder called beta thalassemia, works by being administered in a single-infusion of genetically modified stem cells to a patient. Clinical study participants that took Casgevy were free from symptoms associated with sickle cell disease, like periodic episodes of extreme pain due to blocked blood flow through vessels, for up to a year.
CRISPR, which modifies precise regions of a human’s DNA strands, was once thought to be a far off scientific innovation. Human cells were first modified using CRISPR in clinical trials in China back in 2016. Less than a decade later, these landmark approvals have set the stage for future nods by regulators for other CRISPR-based therapies that can treat things like HIV, cancers and high blood pressure. “Gene therapy holds the promise of delivering more targeted and effective treatments,” Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research said in a recent press release.
CRISPR-based gene editing can be designed as a therapeutic for a number of diseases. A scientist can either delete, disrupt or insert segments of DNA to treat conditions by either targeting specific genes or engineering new cell therapies. The editing process can occur ex vivo (outside the body), in the same way Casgevy does, or in vivo (inside the body). Using CRISPR, sickle cell patients’ blood stem cells are modified in a lab before they are re-infused via a single-dose infusion as part of a hematopoietic transplant.
Neville Sanjana, a core faculty member at the New York Genome Center and associate professor in the Department of Biology at New York University, runs the Sanjana lab, which develops gene therapies for complex diseases like autism and cancer. “One of the really fundamental characteristics of CRISPR is its programmability,” Sanjana told Engadget. While working at the Zhang lab at the Broad Institute of MIT and Harvard, Sanjana says he helped design the “guide RNA” that became the blueprint for Vertex’s Casgevy. “CRISPR screens can be powerful tools for understanding any disease or genetic trait,” Sanjana said. Right now, he said biomedical folks are focused on applying CRISPR-based therapies for really serious inheritable diseases.
While it does “set a precedent” to have these first CRISPR-based gene therapies out there, it could also mean that regulators and the general public will regard future innovations in the space as “less novel,” Katie Hasson, a researcher with the Center for Genetics and Society (CGS) told Engadget. The CGS is a public interest and social justice organization that is focused on making sure gene editing is developed and distributed for good. Hasson explained, it doesn't mean that because one got approved that all other innovative therapies to come after it will not get as much scrutiny.
LIVE NOW: The media call on the approval of the first gene therapies to treat sickle cell disease is happening now!
Beyond therapeutics, gene editing has very broad applications for the discovery and understanding of diseases. Scientists can use CRISPR to explore the origins of things like cancer and pave paths for therapeutics and incurable diagnoses, but that's not all there is to it. Scientists still need to conduct “considerable experimental research” when it comes to bringing an actual therapeutic to fruition, Sanjana said. “When we focus on therapeutic activity at a particular site in the genome, we need to make sure that there will not be any unintended consequences in other parts of the genome.”
Still, the spotlight will always shine a brighter light on the flashy developments of CRISPR from a therapeutic standpoint. Currently, a new gene editing method is being developed to target specific cells in a process called “cancer shredding“ for difficult-to-treat brain cancer. Scientists have even discovered a pathway to engineer bacteria to discover tumorous cells. However, there are barriers to using CRISPR in clinical practice due to the lack of “safe delivery systems to target the tissues and cells.”
“Maybe by curing one disease, you might give them a different disease — especially if you think of cancer. We call that a secondary malignancy,” Sanjana said. While there is strong reason for concern, one cure creating a pathway for other diseases or cancers is not unique to CRISPR. For example, CAR T cell therapy, which uses an entirely different approach to cell-based gene therapy and is not reflective of CRISPR, is a lifesaving cancer treatment that the FDA discovered can, in certain situations, cause cancer.
“We definitely don't want any unintended consequences. There are bits of the genome that if you edit them by mistake, it's probably no big deal but then there are other genes that are vitally important,” Sanjana said. Direct assessment of “off-target effects” or events in which a gene edit incorrectly edits another point on a DNA strand in vivo is challenging.
The FDA recommends that after a clinical trials’ period of investigatory study looking at the efficacy of a gene editing-based therapy, there needs to be a 15-year long term follow up after product administration. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said that the agency’s approval of Casgevy follows “rigorous evaluations of the scientific and clinical data.” Right now, researchers are focused on improving the precision and accuracy of gene editing and having the proper follow up is absolutely well merited, Sanjana explained. “The process right now is a careful one.”
Hasson believes that the 15-year recommendation is a good start. “I know that there is a big problem overall with pharmaceutical companies actually following through and doing those long term post-market studies.”
That’s where new approaches come into play. Base editing, a CRISPR-derived genome editing method that makes targeted changes to DNA sequences, has been around since 2016. Drugs that use base editing have already made headway in the scientific community. Verve Therapeutics developed a gene edited therapy that can lower cholesterol in patients with a single infusion. At higher doses, Verve said the treatment has the potential to reduce proteins associated with bad cholesterol for 2.5 years. Base editing, like CRISPR, has many potential applications for treatment and discovery. For example, base editing could repair a gene mutation that causes childhood blindness. Researchers at Weill Cornell Medicine also found base editing could help understand what genetic changes influence a patient’s response to cancer therapies.
Base editors use CRISPR to bring another functional element to a specific place in the genome. “But it doesn't matter whether it's CRISPR cutting or base editing… any time you're modifying DNA…you would want to know what the off target effects are and you can bet that the FDA wants to know that too. You're going to need to collect data using standard models like cell culture, or animal models to show there are zero or near zero off-target impacts,” Sanjana said.
CRISPR-based therapies already show high therapeutic potential for conditions beyond sickle cell disease. From blood based treatments, to edited allogeneic immune cells for cancers, there are a number of human clinical trials underway or expected to start next year. Trials for gene-edited therapies that target certain cells for cancer and autoimmune diseases are expected to begin in 2024.
It won't be until 2025 before we get a better understanding of how Excision BioTherapeutics’ CRISPR-based therapy works to treat HIV. The application of gene editing as a therapeutic for Alzhiemer’s is still in the early stages, with mice at the forefront of research. Similarly, University College London researchers proved that CRISPR has promise as a potential therapeutic for treatment-resistant forms of childhood epilepsy. In a recent study, a gene edited therapy developed in the lab was shown to reduce seizures in mice.
But the clinical process of getting CRISPR to safely and effectively work as it's intended isn’t the only hurdle. The pricing of CRISPR and related therapies in general will be a huge barrier to access. The Innovative Genomics Institute (IGI), a research group that hopes to advance ethical use of these gene editing in medicine, estimates that the average CRISPR-based therapy can cost between $500,000 and $2 million per patient. The IGI has built out an “Affordability Task Force” to tackle the issue of expanding access to these novel therapies. Vertex’s sickle cell treatment costs a cool $2.2 million per treatment, before hospital costs. David Altshuler, the chief scientific officer at Vertex, told MIT Tech Review that wants to innovate the delivery of the therapeutic and make it more accessible to patients. “I think the goal will be achieved sooner by finding another modality, like a pill that can be distributed much more effectively,” Altshuler said.
“Access is a huge issue and it's a huge equity issue,” the CGS’ Hasson told Engadget. “I think we would also like to look at equity here even more broadly. It's not just about who gets access to the medication once it comes on the market but really how can we prioritize equity in the research that's leading to these treatments.” The US already does a poor job of providing equitable healthcare access as it is, Hasson explained, which is why it's important for organizations like CGS to pose roundtable discussions about implementing guardrails that value ethical considerations. “If you support people having access to healthcare, it should encompass these cutting edge treatments as well.”
This article originally appeared on Engadget at https://www.engadget.com/2023-was-a-big-year-for-crispr-based-gene-editing-but-challenges-remain-160009074.html?src=rss
A Senate Finance Committee inquiry revealed on Tuesday that police departments can get access to private medical information from pharmacies, no warrant needed. While HIPAA may protect some access to personally identifiable health data, it doesn't stop cops, according to a letter from Senator Ron Wyden, Representative Pramila Jayapal and Representative Sara Jacobs to the Department of Health and Human Services. None of the major US pharmacies are doing anything about it, either, the members of Congress say.
"All of the pharmacies surveyed stated that they do not require a warrant prior to sharing pharmacy records with law enforcement agents, unless there is a state law that dictates otherwise," the letter said. "Those pharmacies will turn medical records over in response to a mere subpoena, which often do not have to be reviewed or signed by a judge prior to being issued."
The committee reached out to Amazon, Cigna, CVS Health, The Kroger Company, Optum Rx, Rite Aid Corporation, Walgreens Boots Alliance and Walmart about their practices for sharing medical data with police. While Amazon, Cigna, Optum, Walmart and Walgreen said they have law enforcement requests reviewed by legal professionals before complying, CVS Health, The Kroger Company and Rite Aid Corporation said they ask in-store staff to process the request immediately. Engadget reached out to the pharmacies mentioned in the letter about the claims. CVS said its pharmacy staff are trained to handle these inquiries and its following all applicable laws around the issue. Walgreens said it has a process in place to assess law enforcement requests compliant with those laws, too, and Amazon said while the law enforcement requests are rare, it does notify patients and comply with court orders when applicable. The others either haven't responded or refuse to comment.
The pharmacies mostly blamed the current lack of legislative protections for patient data for their willingness to comply with cop requests. Most of them told the committee that current HIPAA law and other policies let them disclose medical records in response to certain legal requests. That's why the Senate Finance Committee is targeting HHS to strengthen these protections, especially since the 2023 Dobbs decision let states criminalize certain reproductive health decisions.
Under current HIPAA law, patients have the right to know who is accessing their health information. But individuals have to request the medical record disclosure data, instead of health care professionals being required to share it proactively. "Consequently, few people ever request such information, even though many would obviously be concerned to learn about disclosures of their private medical records to law enforcement agencies," the letter states. The letter also urges pharmacies to change their policies to require a warrant, and publish transparency reports about how data is shared.
This article originally appeared on Engadget at https://www.engadget.com/police-are-using-pharmacies-to-secretly-access-medical-information-about-members-of-the-public-182009044.html?src=rss
In a landmark decision, the FDA greenlit two new drugs for the treatment of sickle cell disease in patients 12 and older, one of which —Vertex’s drug Casgevy — is the first approved use of genome editing technology CRISPR in the US. Bluebird Bio’s Lyfgenia also is a cell-based gene therapy, however, it uses a different gene modification technique to deliver tweaked stem cells to the patient.
Both approvals cultivate new pathways for the treatment of sickle cell disease, which is an inherited blood disorder that is characterized by red blood cells that can’t properly carry oxygen, which leads to painful vaso-occlusive crises (VOCs) and organ damage. The disease is particularly common among African Americans and, to a lesser extent, among Hispanic Americans. Bone marrow transplants are currently the only cure for sickle cell disease, but they require well-matched donors and often involve complications.
While both drug approvals use gene editing techniques, Casgevy’s CRISPR/Cas9 genome editing works by cutting out or splicing in DNA in select areas. Patients first have blood drawn so that their own stem cells can be isolated and edited with CRISPR. They then undergo a form of chemotherapy to remove some bone marrow cells, so the edited stem cells can be transplanted back in a single infusion.
Both drug approvals are based on studies that evaluated the effectiveness and safety of the novel therapies in clinical patients. With Casgevy, study participants reported that they did not experience “severe VOCs” for at least 12 consecutive months during the 24-month follow-up. Similarly, patients on Lyfgenia did not experience a “pain crisis” for six to 18 months after the therapy.
The FDA's decision comes shortly after UK regulators, as well as the National Health Regulatory Authority in Bahrain both approved Vertex’s Casgevy. The approval for a CRISPR-based treatment creates opportunity for further innovation in the gene editing space — for treatments ranging from cancers to heart diseases to Alzheimer’s. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited,” Nicole Verdun, director of the Office of Therapeutic Products at the FDA’s Center for Biologics Evaluation and Research said. Casgevy is still currently under review by the European Medicines Agency.
This article originally appeared on Engadget at https://www.engadget.com/crispr-based-gene-editing-therapy-approved-by-the-fda-for-the-first-time-200726474.html?src=rss