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.”

CellProfiler / Carpenter-Singh laboratory at the Broad Institute

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.”

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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.

Yuichiro Chino via Getty Images

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.

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.

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.”

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.

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.

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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.

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.

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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.”

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. 

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.

Amazon Prime subscribers now have access to a deeply discounted One Medical membership for primary care services. The company announced today that Prime members can sign up for just $99 a year, or $9 per month. A One Medical membership typically costs $199 annually. In addition to the individual plan, Prime members who sign up for One Medical can add up to five other people for $6 each.

Amazon announced its acquisition of One Medical in 2022 and finalized the $3.9 billion deal in February, when it began offering a temporary discount for One Medical memberships at $144 per year. It's now slashed that even further. One Medical has offices in major cities across the US, all of which will be available to Prime members who sign up. It also offers round-the-clock virtual care and drop-in lab services. The membership doesn’t cover the cost of visits though; patients will still have to bill through their insurance or pay out of pocket. 

Amazon has been pushing heavily into healthcare in recent years, with prescription services and even its own video- and text-based virtual clinic now among the perks of a Prime subscription. The company made its Amazon Clinic available to all states in the US over the summer, and began offering generic prescriptions for $5 a month with its RxPass at the beginning of the year. Amazon has also started offering drone deliveries for prescriptions in College Station, Texas.

A Parkinson’s patient can now walk 6km (3.7 miles) thanks to an implant targeting the spinal cord. The Guardian reports that the man — 62-year-old “Marc” from Bordeaux, France — developed severe mobility impairments from the degenerative disease. “I practically could not walk anymore without falling frequently, several times a day,” he said in a press release announcing the breakthrough. “In some situations, such as entering a lift, I’d trample on the spot, as though I was frozen there, you might say.” Wearing the spinal implant allows him to walk “almost normally” as the research team eyes a full clinical trial.

Marc underwent a “precision neurosurgical procedure” two years ago at Lausanne University Hospital (CHUV), which helped facilitate the research. The surgery fitted him with an electrode field placed against his spinal cord and an electrical impulse generator under the skin of his abdomen. Although conventional Parkinson’s treatments often target brain regions affected by the loss of dopamine-producing neurons, this approach instead focuses on the spinal area associated with activating leg muscles for walking.

The procedure used a personalized map of Marc’s spinal cord, identifying the specific locations signaling leg movements. He wears a movement sensor on each leg that tells the implant he’s trying to walk; it then switches on and sends electrical impulses to the targeted spinal neurons, adapting to his movement in real-time.

GABRIEL MONNET via Getty Images

“In response to precise stimulation of the lumbar spinal cord, I’ve observed for the first time remarkable improvements of gait deficits due to Parkinson’s disease,” project supervisor Jocelyne Bloch, professor and neurosurgeon at CHUV Lausanne University hospital, said in a webinar discussing the patient’s success. “I really believe that these results open realistic perspectives to develop a treatment.”

The patient says he could walk practically normally with the stimulation after several weeks of rehab. He now wears it for around eight hours daily, only turning it off when sleeping or lying down for a while. “I turn on the stimulation in the morning and I turn off in the evening,” he said. “This allows me to walk better and to stabilise. Right now, I’m not even afraid of the stairs anymore. Every Sunday I go to the lake, and I walk around 6 kilometres. It’s incredible.”

The researchers caution that there’s still a vast chasm between tailoring the approach to one person vs. optimizing it for wide-scale use. Co-leads Grégoire Courtine and Bloch are working on a commercial version of the neuroprosthetic in conjunction with Onward Medical. “Our ambition is to provide general access to this innovative technology to improve the quality of life of Parkinson’s patients significantly, all over the world,” they said.

Michael J. Fox Foundation

In the meantime, research on six new patients will continue in 2024. The team says a “generous donation” of $1 million from the Michael J. Fox Foundation for Parkinson’s Research is funding the upcoming work. In 2021, the actor’s organization announced it had contributed over $1.5 billion to Parkinson’s research.

The ARC nerve stimulation therapy system from startup Onward Medical passed another developmental milestone on Wednesday, as the company announced the first successful installation of its brainwave-driven implantable electrode array to restore function and feeling to a patient’s hands and arms. The news comes just five months after the researchers implanted a similar system in a different patient to help them regain a more natural walking gait.

The ARC system used differs depending on how what issue it's being applied to. The ARC-EX is an external, non-invasive stimulator array that sits on the patient’s lower back and helps regulate their bladder control and blood pressure, as well as improving limb function and control. Onward’s lower limb study from May employed the EX along with a BCI controller from CEA-Clinatec to create a “digital bridge” spanning the gap in the patient’s spinal column.

The study published Wednesday instead utilized the ARC-IM, an implantable version of the company’s stimulator array which is installed near the spinal cord and is controlled through wearable components and a smartwatch. Onward had previously used the IM system to enable paralyzed patients to stand and walk short distances without assistance, for which it was awarded an FDA Breakthrough Device Designation in 2020.

Medical professionals led by by neurosurgeon Dr. Jocelyne Bloch, implanted the ARC-IM and the Clinatec BCI into a 46-year-old patient suffering from a C4 spinal injury, in mid-August. The BCI’s hair-thin leads pick up electrical signals in the patient’s brain, convert those analog signals into digital ones that machines can understand, and then transmits them to a nearby computing device where a machine learning AI interprets the patient’s electrical signals and issues commands to the implanted stimulator array. The patient thinks about what they want to do and these two devices work to translate that intent into computer-controlled movement.

How well that translation occurs remains to be seen while the patient learns and adapts to the new system. “The implant procedures involving the Onward ARC-IM and Clinatec BCI went smoothly,” Dr. Bloch said in an press release. “We are now working with the patient to use this cutting-edge innovation to recover movement of his arms, hands, and fingers. We look forward to sharing more information in due course.”

“If the therapy continues to show promise, it is possible it could reach patients by the end of the decade,” Onward CEO Dave Marver said in a statement to Engadget. “It is important to note that we do not expect people with spinal cord injury to wait that long for Onward to commercialize an impactful therapy - we hope to commercialize our external spinal cord stimulation solution, ARC-EX Therapy, to restore hand and arm function in the second half of 2024.”

Onward Medical among a quickly expanding field of BCI-based startups working to apply the fledgling technology to a variety of medical maladies. Those applications include loss of limb and self-regulatory function due to stroke, traumatic brain or spinal cord injury, physical rehabilitation from those same injuries, as well as a critical means of communication for people living with Locked-In Syndrome.