This study examines how medical students and clinicians report experiencing patient medication requests associated with prescription drug direct-to-consumer advertising on social media; survey data from 98 respondents indicate that those providing both in-person and virtual care encounter more frequent requests for medications advertised online, particularly branded glucagon-like peptide-1 (GLP-1) weight loss drugs.
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STAT+: Trump celebrates closing first round of drug pricing deals, promises more ahead
WASHINGTON — President Trump heralded a drug pricing agreement with Regeneron on Thursday, closing the last of 17 deals initially sought by the White House last year.
Regeneron, as part of the private deal, will reduce prices on drugs to Medicaid, provide cholesterol medicine Praluent on TrumpRx for $225, and invest $27 billion in drug development in the United States.
On the same day, Regeneron also announced Food and Drug Administration approval of Otarmeni, the first gene therapy to be greenlit under the agency’s new National Priority Voucher program. In early trials, the drug provided modest hearing gains for people with a rare type of hearing loss, though its development has received pushback from parts of the Deaf community. Regeneron plans to offer the drug at no cost to American patients.
How to De-Escalate an Autistic Meltdown
A common misconception about tantrums and meltdowns is that they’re interchangeable. But while they share some similarities in their initial expression — crying, screaming, door slamming, harsh words — they’re actually quite different. Dealing with a meltdown requires a more specialized approach, especially with kids on the autism spectrum.
What is a tantrum vs a meltdown?
The two events happen for different reasons. A child throws a tantrum when they’re angry or frustrated, acting out because they feel an injustice has been done to them. They are aware of what they’re doing and still have some sense of control. And if a child’s tantrum is ignored by their parent or caregiver, it will likely subside quickly.
Meltdowns, on the other hand, happen involuntarily and seemingly out of nowhere. They also tend to become much more intense than a typical tantrum and may involve violent behavior such as head banging, hitting others, and damaging property. Once a meltdown has started, intervention is needed to stop it, whether it’s self-imposed (e.g., removing oneself from the trigger) or external (e.g., support from the parent or caregiver). The event can last between a few minutes and several hours.
Tantrums are common among all children, but kids with autism are more likely to experience meltdowns of varying degrees, says Conner Black, PhD, associate director of the Autism Center at the Child Mind Institute.
What are the stages of an autistic meltdown?
For a child with autism, a meltdown is triggered when they become overwhelmed, whether it’s by stress, powerful emotions, sensory input, change, or something else. Their sympathetic nervous system — the network in the body responsible for our “fight-or-flight” response — goes into overdrive and they lose control.
There are several stages to an autistic meltdown and understanding them can help you know how to respond effectively. The duration and intensity of the meltdown depend on whether intervention, including learned coping skills, can stop the child from reaching a crisis point, Dr. Black explains. “Certain skills may not work every time, and that’s really no one’s fault,” he says, but once a child reaches that crisis stage, intervention is no longer useful. He describes the course of a meltdown via the phases of the behavior escalation cycle:
- Calm: “This is basically the valley or plain on the side of mountain, which is considered the baseline, when the child is happy, relaxed, and at their best,” Dr. Black says. For instance, in a classroom setting, a student’s behavior might be described as cooperative and responsive to instruction. These behaviors are specific to the individual, so it helps to recognize what that looks like in your child.
- Trigger: While triggers can vary, Dr. Black says, there are some common ones that he typically encounters in kids with autism. “They’re often related to the misunderstanding of social situations, a lack of time to engage with their preferred interests, a sudden change in their schedule, or a transition that was unexpected,” he says. “It could also be certain sensory aversion, so things like loud noises or loud conversations. It could even sometimes be as simple as how food is presented on someone’s plate.” The child’s response to that trigger can vary depending on their current internal state or outside environmental factors. But if the trigger isn’t removed or is strong enough to dysregulate the child, they’re going to enter the next phase: agitation.
- Agitation: At this point, the child will begin to display behaviors that indicate they are no longer in their calm phase. They might start fidgeting, darting their eyes back and forth, or tapping their hands. For other kids, it could look like total disengagement or staring into space. While removing the trigger might still work at the start of this phase, attempts at problem-solving may backfire and push the child to escalate their behaviors.
- Acceleration/Escalation: “This is really when you start seeing a ramping up of behaviors,” Dr. Black says. “Anything from screaming to throwing toys to aggressing toward the caregiver or whoever’s in the room. Or they could turn that aggression on themselves, whether that’s head banging or hitting themselves repeatedly.” The child may become resistant to intervention and argumentative.
- Peak/Crisis: At this phase, the child hasn’t responded to attempts to de-escalate and will continue to engage in potentially dangerous behaviors. “When thinking about the crisis point, I think about behaviors that are often going to require a higher level of care. So that could be violence, self-injurious behaviors, or even intense suicidal ideation,” says Dr. Black. To be able to distinguish between escalation and crisis, he adds, it’s important to know what the top level of your child’s behaviors look like. “Throwing things could be the escalation stage, and then the next stage is actually when they’re destroying property,” Dr. Black explains.
- De-escalation: Finally, the intensity of the behavior begins to subside. The child may appear disoriented, confused, and tired. They will gradually become calmer.
- Recovery: The child is officially in this phase when they’re fully back at their baseline, Dr. Black says. The behaviors you’ll see at this phase are the same ones you see when they’re in their calm phase.
How to prevent meltdown escalation
Once a child has started to experience a meltdown, it’s hard to get them back to baseline. Depending on the phase, certain interventions may help while others might make things worse.
First, you want to avoid triggers, Dr. Black advises. “Autistic individuals can have a lot of difficulty talking about or even understanding what their emotions are. So, it’s typically up to the parents or caregivers to identify what things can trigger them in a certain way,” he says.
For instance, some kids with autism really thrive with routine and can become agitated when there are unexpected changes. Having a visual schedule of exactly what’s going to happen during the day can help prevent that, says Dr. Black. “If you know there’s going to be a change, you can pick a time, maybe a couple of days in advance, where you talk to them about what that difference is going to be.”
And if your child is known to have meltdowns in public spaces, says Dr. Black, think about what those outside triggers are and how to prepare ahead of time. If they tend to get upset by loud noises, for example, a pair of headphones can be an item — along with phone, wallet, keys! — that you never leave the house without. If possible, work with a mental health professional to identify triggers and develop an escalation plan.
What to do in the agitation phase
If your child has reached the agitation phase, says Dr. Black, you can try to intervene with coping skills that you’ve learned in therapy, whether it’s something as simple as removing a trigger or giving them a preferred activity in that moment to help prevent their behaviors from escalating.
Sometimes kids encounter an environment, like school, that is beyond your control but contains a wide range of potential triggers and pushes them into the agitation phase. Because their house is a more comfortable environment, kids with autism may keep themselves together at school and then quickly melt down once they get home.
“In that case, for that first hour, let them have their alone time where they can just chill,” Dr. Black suggests. “It could be eating snacks, watching a TV show, or even just sitting quietly in their room. Maybe it’s engaging in some sort of self-stimming behavior.” This can give them the space to cool down and take some time away from any sort of outside stimuli that could push them to move from the agitation phase into the escalation point of a meltdown.
What to do in the acceleration/escalation phase
It can be hard to anticipate every possible trigger, especially when there might be multiple at once on any given day. And sometimes coping strategies aren’t enough to keep a child from escalating or the trigger is too strong. Still, there are some things that Dr. Black suggests you can do to try to keep them from reaching that crisis point.
Keep communication short and concrete
Too much talking can be overwhelming for the child at this stage and might push them to crisis, Dr. Black explains, so the less communication the better. “A simple instruction looks like using just a short sentence. Say there’s a loud noise, for example. You can just say, ‘Go get your headphones,’” he says.
Use visual prompts
Instead of trying to communicate verbally, you can hold up a visual prompt. “If your child has already been working with a therapist or if they’ve learned some coping skills, it would be helpful to have a laminated sheet readily available with their name and pictures of four different coping skill options — like headphones, deep breathing, coloring, sitting alone in their room.”
Dr. Black advises only giving a few options, as it’s already difficult for the child to focus while they’re upset. Additionally, if they don’t choose one right away and you want to try again, he recommends that you “let there be silence for 60 seconds at minimum between prompts, because you don’t want to over-prompt and exacerbate the situation even more.” But providing these choices allows them to maintain their autonomy, which is important during escalation.
What to do in the peak/crisis phase
“Once they get to that apex, they’ve reached the point of no return and just need to go through the process,” says Dr. Black. He stresses that at this point, communication needs to be very minimal or nonexistent.
When maintaining safety is the focus
“The goal switches to really being able to maintain safety for both the individual as well as the family members in the area,” Dr. Black explains. “If they’re harming themselves, such as head banging, move them to their bed so at least it’s on something that’s softer and not going to potentially cause significant injury.”
Efforts to make sure the child is as safe as possible can put you in harm’s way. “If there’s aggression, you can be watching and making sure they’re safe but not getting too close where you could get aggressed upon,” says Dr. Black.
If there are other children in the house, Dr. Black advises that you make plans for how to keep them safe. “Maybe they can go to their room and lock the door while it’s happening,” he says. “Some families have the other kids go to the car and sit and wait until their parents come out to get them.”
When you need emergency services
If the crisis phase goes on for a long period of time, says Dr. Black, “this is when you’d have to think about calling 911. And as kids become adolescents, the response is going to look a lot different. Because of size alone, it’s a little bit easier to manage the situation in a 5-year-old than it would be in a 15-year-old.”
Dr. Black advises that you get in touch with your local police department or EMT service in advance to let them know you have a child with autism in the home, so if you call during an emergency, they are already familiar with your family.
What to do in the de-escalation and recovery phases
Watch for signs that the child is beginning to de-escalate, Dr. Black says. “All you’re doing at this point is maintaining safety until you’re really able to see a lessening of the intensity of the behavior or the frequency decreases a little bit.” Then, he says, you can start to slowly communicate with them again. You really need to be careful here, because it may look like they’re calming down, but if they’re pushed too hard and they’re not ready to talk, they might go right back into crisis phase.
At the recovery phase, “the whole family is recovering,” Dr. Black says. It’s at this point where you can all debrief and work through what may have triggered this escalation and how to possibly prevent it in the future.
“Make sure you’re also debriefing separately with the other siblings in the home after it happens,” Dr. Black adds. “They’ve just witnessed something that may have been traumatic and really stressful for them. There’s often so much focus given to the child with the big behaviors in the moment.”
Medication treatment
Sometimes, a child or teen may suffer from frequent meltdowns to the point that it’s interfering with their quality of life and their ability to attend school. At that time, a mental health professional may recommend working with a psychiatrist to add medication to their treatment.
The type of medication depends on the underlying mechanisms contributing to the behaviors, Dr. Black says. “For instance, if it’s coming from significant anxiety, psychiatrists may prescribe an SSRI like Prozac or Zoloft. If a child has co-occurring ADHD, which is very common, stimulant or non-stimulant ADHD medication might be recommended. And if the behavior stems from irritability or some kind of rigidity, antipsychotic medications like Abilify or risperidone can be useful.”
Improvement is possible
Dr. Black notes that when kids receive the support they need, their quality of life really improves. “I’ve seen that when families work with therapists to come up with different behavioral plans and figure out a proper medication regimen, there’s a lot of improvement in behavior challenges,” he says. “The duration, frequency, and intensity of the meltdowns decrease as the child learns how to handle strong emotions and parents learn how to respond to them. And the medication can help to increase their likelihood of being able to use coping skills or regulation techniques to calm back down when they start to get really frustrated.”
The post How to De-Escalate an Autistic Meltdown appeared first on Child Mind Institute.
CRISPR Base Editing Repairs Hard-to-Treat Cystic Fibrosis Mutation in Cell Models
Affecting an estimated 100,000 people globally, cystic fibrosis (CF) cases stem from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In the past several decades, scientists have successfully engineered various small-molecule therapies that lessen the severity of the disease. However there are still treatment challenges. Now, data from a new study in a cell model demonstrates that a gene therapy can successfully repair an “untreatable” mutation associated with a particularly severe form of the disease. Details of the potential therapy are published in a new Science Translational Medicine paper titled “Functional correction of the untreatable CFTR 1717-IG>A mutation through mRNA- and sgRNA-optimized base editing.”
Many current therapies benefit patients with the most common disease-associated mutation, F508del. However they often have little effect on patients who harbor other types of mutations. For example, some patients have a mutation named 1717-1G>A, which is relatively common but doesn’t have any approved therapies due to being a splicing mutation that results in little to no protein production. In fact, “about 10% of people with CF do not qualify for any of the available CFTR modulator therapies, particularly those people with severe splicing mutations that result in frameshifts and the formation of premature termination codons.”
The 1717-1G>A mutation is the target of the therapy described in the paper, which was written by scientists from the University of Trento and their collaborators elsewhere. Specifically, the team developed an “adenine base editing strategy to efficiently correct the 1717-1G>A mutation,” they wrote in Science Translational Medicine. “By harnessing the SpRY- ABE9 system, which we delivered as optimized RNAs for both the base editor and single guide RNA (sgRNA), we achieved functional correction in patient-derived models.”
Furthermore, the scientists note that they opted to use base editing rather than strategies like base editing because it has the advantage of “typically higher nucleotide modification efficiencies and a streamlined system requiring only the editor and an sgRNA” and because it has been used in other CF studies.
Using their ABE9 base editor and modified CRISPR-Cas9 tool, the scientists report successfully editing up to 30% of target DNA in human embryonic kidney cell lines and patient-derived airway epithelial cells with minimal off-target effects. It also corrected the mutation in intestinal organoids derived from CF patients as evidenced by restored CFTR activity.
Additional studies are needed, especially in animals, to fully assess the effectiveness of potential therapy but early results are promising. Overall, the approach achieved an editing efficiency of 13%. Prior studies showed that 10% efficiency may be enough for functional recovery. The results suggest that the therapy could benefit the subset of patients whose disease is caused by 1717-1G>A.
The post CRISPR Base Editing Repairs Hard-to-Treat Cystic Fibrosis Mutation in Cell Models appeared first on GEN – Genetic Engineering and Biotechnology News.
New Markers of Diabetes and Heart Disease Revealed via Genetic Study in Indians
A study in 3,000 Punjabi Sikhs has identified previously unreported molecular pathways that contribute to cardiometabolic disease. Published today in PLOS Medicine, these findings highlight the benefits of including diverse participants in these types of studies, which have historically centered on individuals of European ancestry.
“Genetic mechanisms that predispose people to type 2 diabetes and cardiovascular disease remain poorly understood, partly because of a lack of sufficient data on non-European ethnic groups,” write the authors of the study, who were led by Dharambir K. Sanghera, PhD, director of the Genetic Epidemiology Laboratory at the University of Oklahoma Health Sciences Center. “Extending these evaluations to diverse cohorts is essential for gaining insights into the molecular pathways involved in disease.”
Sanghera and colleagues conducted a metabolite genome-wide association study to look for links between the human lipidome and cardiometabolic disorders in a Punjabi population originating from Northern India. Epidemiological studies have repeatedly shown that South Asians living abroad experience a higher incidence of type 2 diabetes and are more susceptible to cardiovascular disease compared to other ethnic groups. However, the exact mechanism responsible for this increased risk remains unknown and lipidomic and genome-wide data is lacking for Indian populations.
“Genome-wide studies have shown that genes influencing blood lipid metabolites are often linked to different diseases,” write the study authors. “However, most of this research has been done on people of European ancestry. Studying more diverse populations is important to better understand how these genetic pathways contribute to disease in different ethnic groups.”
The study looked at genetic influences on 516 lipids in 3,000 Punjabi Sikh individuals and then validated the results in larger cohorts, with both European and non-European ancestry, using data from UK Biobank, GeneRISK, DIAMANT, PROMIS, and other studies. After multiple rounds of testing and correction, results showed strong associations in 36 pairs of lipid metabolites and single nucleotide polymorphisms (SNPs). Among them, 33 had not been reported before, and three were confirmed to be ancestry-specific.
Further investigation identified a causal association between type 2 diabetes and the metabolite LPC O-16:0, which was paired with a genetic variant in the gene encoding for CD45, a key regulator of immune signaling. Another possible causal relationship was found with PC 38:4, a metabolite shown to protect against coronary artery disease in Indian populations that was paired with a genetic variant in an untranslated region of the FADS1/2 genes.
“Our study has discovered new metabolite markers and genes that intersect with pathways of inflammation and immuno-vascular diseases, which have not been reported in previous European studies, specifically emphasizing how immune system signaling affects metabolic health,” state the authors. “By identifying unique genetic signatures in Asian Indians, the research advocates for ancestry-specific medical approaches to address chronic immuno-vascular conditions in cardiometabolic disease. These advances could be beneficial in clinical practice, enabling effective personalized therapies and preventive strategies.”
The post New Markers of Diabetes and Heart Disease Revealed via Genetic Study in Indians appeared first on Inside Precision Medicine.
Vitamin D Linked to Lower Diabetes Risk in People with VDR Gene Variant
A genetic analysis of a large U.S. clinical trial suggests that vitamin D supplementation may reduce the risk of progression from prediabetes to type 2 diabetes, but only for those people who harbor specific variants of the vitamin D receptor gene. The study, led by researchers at Tufts University and published in JAMA Network Open, found that daily high-dose vitamin D lowered diabetes risk by 19% in participants with certain genotypes, opening the possibility of using vitamin D as a diabetes prevention strategy.
The new findings build on data from the Vitamin D and Type 2 Diabetes (D2d) clinical trial, a multi-site randomized study that enrolled more than 2,000 U.S. adults with prediabetes. Study participants were assigned to receive either 4,000 IU of vitamin D3 daily or a placebo. The subjects were then followed for a median of 2.5 years to assess progression to diabetes. The original trial did not show a statistically significant reduction in diabetes risk across all participants.
“But the D2d results raised an important question: Could vitamin D still benefit some people?” said lead author Bess Dawson-Hughes, MD, a senior scientist at the Jean Mayer USDA Human Nutrition Research Center on Aging at Tufts University. “Diabetes has so many serious complications that develop slowly over years. If we can delay the time period that an individual will spend living with diabetes, we can stop some of those harmful side effects or lessen their severity.”
In their follow-on research, the Tufts noted that subsequent analysis of the D2d trial data showed that outcomes varied based on achieved blood levels of vitamin D in participants. The new study also found a genetic link to those who had improved outcomes.
To explore the role genetics might play, the investigators conducted a post hoc analysis of 2,098 D2d participants who consented to genetic testing. They focused on three common polymorphisms in the vitamin D receptor (VDR) gene: ApaI, BsmI, and FokI. The researchers first examined how vitamin D levels correlated with diabetes risk across genotypes, then evaluated how genetic variants influenced response to supplementation.
The data showed that the ApaI polymorphism is a key determinant of response. Participants with the AA genotype, which was about 30% of the cohort, did not experience a reduction in diabetes risk with vitamin D supplementation. By comparison, those with the AC or CC genotypes, the remaining 70% of participants, showed a 19% lower risk of developing diabetes when treated with vitamin D compared with placebo.
The biological basis for this effect is linked to the role the VDR gene plays in pancreatic β cells, where it influences insulin secretion and glucose regulation. Variations in the receptor may alter how effectively vitamin D exerts these effects, explaining why some individuals benefit from supplementation while others do not.
Earlier research has suggested there is a connection between vitamin D and diabetes risk. In earlier analyses of the D2d trial, participants who maintained higher blood levels of vitamin D experienced substantial reductions in diabetes incidence. These findings were supported by meta-analyses and observational studies, including research from the UK Biobank, which found that genetic variation in VDR could modify its activity.
“We hypothesized that VDR gene variants modify the association between achieved intratrial 25-hydroxyvitamin D (25(OH)D) level and diabetes risk and may modify the effect of vitamin D3 supplementation on the risk of developing diabetes,” the researchers wrote. 25(OH)D is the main form of vitamin D circulating in the blood.
The current study broadens knowledge on the role vitamin D can play in diabetes prevention by identifying the specific polymorphisms at play. The overlap between ApaI and BsmI variants provides further evidence of the role of VDR genetics, although the researchers noted that ApaI alone may be sufficient to identify likely responders.
“This genetic association analysis of the D2d study suggests that genetic variation in the VDR, specifically the ApaI polymorphism, is associated with diabetes risk at higher intratrial 25(OH)D levels and is associated with response to 4000 IU/d of vitamin D3 supplementation among adults with prediabetes,” the researchers wrote.
The implications for clinical care include the potential use of genetic testing to guide preventive treatment. A single test for the ApaI polymorphism could help identify patients with prediabetes who are most likely to benefit from higher-dose vitamin D supplementation.
While the results have established a link between variations in the VDR gene and diabetes development, the research noted that the study was not designed to assess the mechanisms underlying the genetic effects. Further, its sample size limited subgroup analyses by race and ethnicity.
“Our findings suggest we may eventually be able to identify which patients with prediabetes are most likely to benefit from additional vitamin D supplementation,” Dawson-Hughes said. “In principle, this could involve a single, relatively inexpensive genetic test.”
Next steps in this line of research include replicating the findings in independent cohorts and conducting prospective trials designed to test genotype-guided supplementation strategies.
The post Vitamin D Linked to Lower Diabetes Risk in People with <i>VDR</i> Gene Variant appeared first on Inside Precision Medicine.
AI Tool Creates Designer Antibiotics
A generative AI tool for molecular design has created a new antibiotic that has shown promising preclinical results against methicillin-resistant Staphylococcus aureus (MRSA).
The SyntheMol-RL generative model, described in Molecular Systems Biology, could speed drug discovery and help in the fight against antibiotic resistance.
The algorithm uses reinforcement learning to rapidly design easily synthesizable small-molecule drug candidates from a massive chemical space of 46 billion compounds.
It created a compound that the researchers named synthecin, which was effective against MRSA wound infection in a mouse model, showing its utility for real-world drug discovery.
“We used our model to design new antibiotics, but it’s capable of so much more,” said researcher Jon Stokes, PhD, from McMaster University.
“We built it to be disease agnostic, meaning it could just as easily generate novel drug candidates for diabetes or cancer or other indications.”
The rapid spread of antibiotic resistance is a critical challenge facing modern medicine. In 2019, just under five million deaths were linked with drug-resistant bacteria and this number is expected to more than double by 2050 if the emergence of antimicrobial resistance continues to outpace the creation of new antibiotics.
Stokes and team examined whether SyntheMol-RL could identify potential antibiotics for MRSA, an infection listed by the World Health Organization as a high priority for new antibiotics.
It replaces SyntheMol, a previous incarnation that was not as effective for exploring the chemical space and was not able to optimize more than one molecular property, which is a necessity in real-world drug discovery.
The second-generation model uses reinforcement learning, which enables it to rapidly explore massive combinatorial chemical spaces with tens of billions of molecules for promising compounds that are easy to synthesize.
The researchers deployed SyntheMol-RL to identify compounds that simultaneously possessed the multiple drug-like properties of antibacterial activity against MRSA and aqueous solubility.
Next, they synthesized and experimentally tested 79 compounds designed by two variants of SyntheMol-RL and found a corresponding two and 11 potent hits.
One of these compounds, which they named synthecin, was able to fully arrest the growth of MRSA in a murine wound infection model.
“These results demonstrate that SyntheMol-RL is an effective and flexible framework for drug design applications,” the authors maintained.
They added: “SyntheMol-RL is compatible with any property predictor and combinatorial chemical space, it can be readily extended to a wide variety of drug discovery and molecular design problems.”
The post AI Tool Creates Designer Antibiotics appeared first on Inside Precision Medicine.
Regenerative Medicine: Promise, Hype, and What Actually Works
From stem cells to platelet-rich plasma, regenerative medicine is often positioned as the future of healthcare. But not all approaches deliver on that promise. As interest grows, so do questions regarding what actually works. GEN’s Editor in Chief John Sterling spoke with Thomas Buchheit, MD, founder and medical director of the Triangle Regen Medicine and Biologics Center in Chapel Hill, NC, in relation to the science, the hype, and the realities shaping the field today.
GEN: How do you define regenerative medicine?
Buchheit: Many people think of regenerative medicine as growing new organs, but I define it more broadly as any therapy that improves tissue health or function. With that definition, we can include platelet-rich plasma (PRP), stem cells, and autologous conditioned serum (ACS). These approaches aim to enhance tissue health and improve function.
GEN: The field is promising, but also sometimes criticized as overhyped. Which areas deserve that criticism, and which have gained credibility through clinical validation?
Buchheit: Some criticism is valid, especially around stem cells. We’ve all seen claims over “miracle” stem cells that regrow cartilage. In reality, while these cells can be therapeutic, they typically don’t survive long after injection. Instead, they work by activating the body’s immune-based healing mechanisms. They can improve tissue health, but they’re not the miracle cures they were once portrayed to be.
On the other hand, therapies like PRP and ACS have gained credibility when properly applied and studied, particularly in musculoskeletal conditions.
GEN: How do you incorporate regenerative medicine into your practice?
Buchheit: I focus on patient function—what people can do now and what they want to achieve. Then tailor therapies accordingly. I prioritize treatments with strong evidence. One example is ACS, also known as the Regenokine* program. It’s highly standardized and supported by over 20 years of research in osteoarthritis, sciatica, and radiculopathy.
I also use PRP, which can be effective, but only when properly dosed. That’s been a major challenge since there are many ways to prepare PRP. We now know that dose matters. For example, treating knee osteoarthritis typically requires close to 10 billion platelets. At our clinic, we measure platelet counts before and after preparation to ensure accuracy, something often not done enough or at all.
GEN: Where did these approaches originate, and how widely are they used?
Buchheit: ACS originated in Germany in the 1990s with Dr. Peter Wehling. It was initially developed as an alternative to steroids for treating sciatica. The process involves incubating whole blood under controlled conditions, which stimulates the release of anti-inflammatory proteins, growth factors, and exosomes.
It became popular as patients, including athletes, traveled to Germany for treatment. Today, it’s available in the United States, though still more common in Europe. We now better understand how it works. Our research shows that exosomes play a key role in long-term benefits. If you remove them, effectiveness drops significantly.
GEN: Your new book Healing Joints and Nerves—who is it for?
Buchheit: It’s written for patients and a broad audience. I focused on authoring a book on regenerative medicine based on scientific accuracy and depth. I wanted to create a resource that explains these therapies clearly and truthfully—what they can and cannot do. It took over six years to complete. The book covers the history of stem cells and concludes with ACS, including both research and my personal experience with it as an avid runner and bicycle rider.
GEN: You often mention “good” vs. “bad” inflammation. What’s the difference?
Buchheit: Chronic inflammation is harmful. It damages tissue, drives pain, and contributes to diseases like osteoarthritis. But acute, controlled inflammation is essential for healing. It triggers the body’s repair processes. Exercise is a good example. It creates cycles of inflammation and recovery that make us stronger. Regenerative therapies aim to harness this same mechanism.
Interestingly, suppressing inflammation too aggressively can backfire. Studies show that patients who take anti-inflammatories after acute injuries may have a higher risk of chronic pain. Repeated steroid injections can also worsen joint damage over time.
GEN: Does all PRP work for osteoarthritis?
Buchheit: No. PRP must contain a sufficient platelet dose to be effective. Research shows that below approximately three billion platelets, it’s unlikely to work. Above four billion, effectiveness improves, and near 10 billion provides optimal results.
A practical tip: patients should ask how much blood is drawn. If only 10 mL is used to produce PRP, it’s mathematically impossible to achieve a high dose. Proper preparation typically requires 60–120 mL. Patients should also ask whether platelet counts are measured.
GEN: Please talk a bit more about Regenokine.
Buchheit: The program is based on ACS, enhanced through a controlled incubation process. This stimulates cells to release anti-inflammatory proteins, growth factors, and exosomes. Treatment typically takes roughly a week. Patients often come to the clinic for that duration. We’ve seen strong results in osteoarthritis and spine conditions, especially in patients who haven’t responded to other treatments, including stem cells.
GEN: What about safety, efficacy, and durability of results?
Buchheit: Outcomes vary by patient, but the primary goal is restoring function—whether that’s walking a dog or running a marathon. My approach is to stay as evidence-based as possible. That’s critical in a field where there is some overpromise or poorly validated treatments.
There are real concerns regarding product quality, sourcing, and transparency in some parts of the market. We need to know exactly what we’re using, how it works, and what evidence supports it. That’s how regenerative medicine will continue to advance responsibly.
Thomas Buchheit, MD, founded the Triangle Regen Medicine and Biologics Center in Chapel Hill, NC, to bring a range of regenerative therapies to patients. He now serves as an adjunct associate professor at Duke and continues to work with scientists at the Center for Translational Pain Medicine.
Buchheit began studying nerve injury pain and served as chief of pain medicine at Duke University Medical Center. He investigated the immune basis of pain relief following injury and the mechanisms behind regenerative therapies, including platelet-rich plasma, stem cells, and autologous conditioned serum. He has led several studies funded by the NIH and the Department of Defense.
*Regenokine was developed by Peter Wehling, MD, in Germany, originally in the 1990s. It utilizes a patient’s own blood to create a serum rich in anti-inflammatory proteins, particularly the interleukin 1 receptor antagonist (IL-1Ra), which helps reduce inflammation and promote healing in joints and tendons. The treatment is used for conditions like osteoarthritis and has gained popularity among athletes seeking pain relief. While it has shown promise in small studies, it is not yet FDA-approved and is not covered by insurance in the United States.
The post Regenerative Medicine: Promise, Hype, and What Actually Works appeared first on GEN – Genetic Engineering and Biotechnology News.
STAT+: Legislatures in Colorado and Virginia resist moves to constrain drug affordability boards
Legislators in two states have resisted efforts to restrict prescription drug affordability boards, the controversial panels that are designed to function as rate-setting authorities and place limits on the cost of prescription medicines.
In Virginia, the General Assembly unanimously rejected a move by Gov. Abigail Spanberger (D) to delay a key provision of two bills that would create a board and allow it to place price caps that mirror the negotiated prices paid by Medicare. Spanberger must now either accept or veto the legislation as originally intended.
In Colorado, the House Health and Human Services Committee postponed consideration of a bill that would exempt orphan drugs, which are used to treat rare diseases, from pricing caps that might be pursued by the state board. By delaying action until the end of the legislative session, the bill is effectively dead.
How Geospatial Science is Reshaping Cancer Epidemiology: Three Perspectives from the Front Lines
The intersection of geography and oncology is no longer a speculative frontier—it is rapidly becoming the new standard in understanding who gets cancer, who survives it, and why. At a recent session at AACR 2026 bringing together leading researchers from the Fred Hutch Cancer Center, Harvard, and UCSF, the consensus was clear: geospatial methods are fundamentally altering how epidemiologists interrogate the cancer continuum, from incidence to mortality, from prevention to palliative care.
Trang VoPham, PhD, from Fred Hutch opened proceedings with a sweeping overview of how location data is being weaponized against cancer disparities. Her team’s work exemplifies the field’s evolution beyond crude ecological fallacies toward granular, individual-level exposure assessment. “We linked geospatial data on agricultural pesticide operations with all death certificates in the U.S. from 1989 to 2023,” she explained, detailing their findings that higher linuron use correlated with a 16% increased risk of colorectal cancer mortality among under-50s—higher than the 11% seen in older populations. The precision matters: “It is absolutely critical… can you access or generate residential address histories, not just baseline, not just at diagnosis, to consider life course exposures, timing of exposures, during relevant and critical time periods?”
VoPham’s lab is already translating these insights into population health interventions. Their GeoXMap web application—developed with community advisory boards across Washington State—enables neighborhood-level mapping of over 175 health variables, each paired with actionable mitigation strategies. “When you map radon, you can click on the tips button and see strategies for exposure mitigation, like where to get free radon test kits,” she noted. During 2023’s wildfire season, her team used Epic electronic health records to identify and contact 64,000 high-risk patients, resulting in over 4,000 same-day virtual primary care appointments. “This approach could absolutely be scaled to target other populations… to empower high-risk patients with information to help protect themselves from environmental hazards.”
Jaime Hart, ScD, from Harvard, shifted focus to the atmospheric dimensions of cancer risk, tracing how air pollution research has matured since IARC’s 2013 carcinogen declaration. She noted that evidence at the time was largely restricted to lung cancer data. Today, the picture has broadened considerably. Hart highlighted recent consortium work linking traffic-related nitrogen dioxide with premenopausal and Black women’s breast cancer risk—”mostly being driven by premenopausal breast cancer and breast cancer among Black women and non-Hispanic white women”—while PM2.5 associations remain more equivocal for this site.
The mechanistic sophistication has advanced in parallel. Hart detailed how particulate matter can “translocate across your lungs, get into your circulation and deposit in every tissue in your body,” even ascending the nasal pathway to breach the blood-brain barrier. Her collaboration with VoPham on wildfire-specific PM2.5 revealed that “even for the same increase in air pollution exposure… if that PM2.5 is coming more from wildfires than not, you saw an elevated risk,” suggesting source-specific toxicity profiles that carry profound regulatory implications.
Iona Cheng, PhD, from University of California, San Francisco, anchored the session in the structural determinants that underlie these spatial patterns. Her work on redlining—historical mortgage discrimination encoded into contemporary health disparities—demonstrates how geospatial tools can excavate systemic injustice. “In Detroit… about 70% [of non-Hispanic white men with prostate cancer] do live in an area that has not been redlined, in contrast to about 30%,” she reported, whereas “almost 60%” of African American patients resided in the most heavily denied neighborhoods. The mortality gradient was stark: “Higher prostate cancer mortality, or lower survival, associated with living in neighborhoods with more redlining, for both Black and white men.”
Cheng emphasized that these are not proxy measures for individual behavior but independent contextual effects. “We do see that the neighborhood itself has contributions,” she said, describing how her team is developing racially-ethnic-specific composite indices of structural racism across housing, education, employment, and judicial domains. The community-engaged methodology proved essential: working with Hawaiian advisory boards revealed that non-Hispanic white reference groups made little demographic sense for the islands, prompting recalibration.
The session closed with a palpable sense of acceleration. From Google’s nascent geospatial reasoning AI to wearable sensor validation of satellite models; from street-view imagery classifying 350 million U.S. locations to ecological momentary assessment tracking real-time exposures, the toolkit is expanding exponentially. As Hart observed: “The places where we live, work, and play influence our risk of cancer, impose diagnostic outcomes. There are robust biological mechanisms that underlie this.” The geospatial revolution in cancer epidemiology, it seems, is only getting started.
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