Latest news with #AAN
Medscape
27-06-2025
- Health
- Medscape
New Guidance on the Use of Unproven Neurologic Therapeutics
A new position statement outlining guiding principles for navigating the use of unproven therapies for neurologic conditions that lack approval from the FDA or robust scientific evidence has been released by the American Academy of Neurology (AAN). The statement is intended to support informed conversations between clinicians, patients, and policymakers about emerging or unproven treatments. While it includes examples of such therapies, the document stops short of offering clinical recommendations. The use of emerging neurologic therapies not yet supported by science has been 'an ongoing concern,' with patients often asking providers about these therapies, the statement's lead author Larry B. Goldstein, MD, professor and chair of the Department of Neurology and associate dean for Clinical Research, University of Kentucky, Lexington, Kentucky, told Medscape Medical News. Developed in response to requests from AAN members seeking guidance on emerging therapeutics, the statement outlines guiding principles to support clinical and policy discussions. It was published online on June 25 in Neurology. Lack of FDA-Approved Options Many neurologic diseases have no available FDA-approved therapeutic options and no, or limited, evidence-based treatments. However, many potential treatments, both synthetic and naturally occurring, may be in varying stages of expert evaluation for neurologic conditions. Psychedelics have emerged as potential therapies for pain disorders such as cluster headache, as well as psychiatric conditions including major depression, posttraumatic stress disorder (PTSD), generalized anxiety, and substance use disorders. However, data on psychedelics' risks and benefits for these indications are limited. The AAN committee cites the example of midomafetamine combined with psychotherapy for the treatment of PTSD. Although this approach has generated interest, an FDA advisory committee recently recommended against its approval, citing significant methodological flaws in the supporting studies and advising the FDA to reject the Investigational New Drug Application. 'This illustrative example highlights the need for rigorous, placebo-controlled, double-blind trials to adequately test the effectiveness of these therapies while exhaustively documenting adverse events to characterize patient safety issues,' the statement's authors noted. The AAN supports the FDA-accelerated review of novel therapeutics for neurologic conditions 'when this process is appropriate,' said Goldstein. Off-Label Use The statement also addresses the expanded use of therapies beyond their original FDA-approved indications — for example, the off-label use of alteplase for thrombolysis in ischemic stroke patients treated 3-4.5 hours after they were last known to be well, as well as the use of tenecteplase in select cases of acute ischemic stroke. Goldstein noted that these examples reflect issues commonly encountered in hospitals nationwide. 'The AAN supports the off-label use of FDA-approved therapies in settings in which the high-quality evidence indicates that the benefit of the therapy outweighs the risks with shared decision-making between the patient and physician in the model of informed consent,' the statement authors noted. Another example is natalizumab, a humanized IgG4κ monoclonal antibody approved for the treatment of relapsing-remitting multiple sclerosis (MS). The drug was initially withdrawn from the market following reports of progressive multifocal leukoencephalopathy. However, it was later reintroduced, as some patients with MS have no alternative therapies to effectively manage their disease. Physician-patient discussions about unproven therapies might include situations where patients are considering their 'Right to Try.' Signed into law in 2018, the Right to Try Act allows individuals with a terminal illness who have tried all approved treatments and who are unable to participate in a clinical trial to receive an experimental treatment. The statement also addresses the issue of adverse events reported after FDA approval. For example, the statin cerivastatin was approved for cholesterol reduction but was withdrawn from clinical use following reports of deaths and hospitalizations. These cases, said Goldstein, illustrate the complexity of this issue. 'It's particularly challenging because healthcare providers must be constantly aware of new data that may become available as therapeutics enter general use after FDA approval,' said Goldstein. In cases where a therapy is approved but carries significant risks or an incomplete adverse event profile, the statement advises that the AAN should generally refrain from taking a definitive position until further review by the FDA is completed. Unproven 'Treatments' The authors also address the use of therapeutics with limited or no supporting data, many of which have been popularized on social media, to treat or prevent conditions such as dementia. Such use not only exposes patients to unknown risks but may also discourage them from pursuing evidence-based treatments or participating in clinical trials that could offer potential benefits, Goldstein noted. When seeing patients, healthcare providers should discuss potential participation in a relevant trial and ask more detailed questions about the use of unproven therapies, he said. 'Physicians should routinely not only confirm their patient's prescribed medications but also ask about any other substances they may be using. Some, including certain supplements, may have potential toxicities or interactions with prescribed medications.' Discussions between neurologists and patients about unproven therapies are becoming increasingly relevant. 'In the current climate of unfiltered, at times incorrect or misinterpreted information, having a trustworthy source of fact-based advice is critically important,' said Goldstein. 'The neurologist brings particular expertise and training related to neurological disorders and what is known about the risks and benefits of potential treatments to help inform patient decisions,' he added. The AAN policy statement offers 'a framework' to guide neurologists in their role as patient advocates, Goldstein added. Although it does not address specific treatments, it does provide a structure for conversations with patients, said Goldstein.
Fox News
20-05-2025
- Health
- Fox News
Mini-strokes may cause surprising long-term health issue, experts warn
Prolonged fatigue could indicate poor sleep habits — but it could also be a lingering effect of mini-strokes. That's according to a new study from Aalborg University Hospital in Denmark, which was published this week in Neurology, the medical journal of the American Academy of Neurology (AAN). A mini-stroke — medically known as a transient ischemic attack (TIA) — is a temporary blockage of blood flow to the brain that causes a "short period of symptoms," according to Mayo Clinic. The study found that people who experience a TIA are more likely to report prolonged fatigue lasting up to one year. "Patients with a presumed transient event reported fatigue at levels comparable to a stroke," lead study author Birgitte Hede Ebbesen, PT, PhD, a physiotherapist at Aalborg University Hospital, told Fox News Digital. The researchers followed 354 people averaging 70 years of age who had experienced a mini-stroke. Over a 12-month period, the participants reported their level of fatigue in five areas: overall tiredness, physical tiredness, reduced activity, reduced motivation and mental fatigue, according to a university press release. On a scale ranging from 4 to 20 — with 20 being the most fatigued — the participants reported an average score of 12.3 in the two weeks after the mini-stroke, 11.9 at three months, 11.4 at six months and 11.1 at the one-year mark. Two weeks after the mini-stroke, 61% reported high levels of fatigue. At three, six and 12 months, 54% said they experienced fatigue. Those who reported prolonged fatigue were twice as likely to have experienced anxiety and/or depression, the study found. Some also reported "We had encountered fatigue among patients with TIA in clinical settings, so we knew it was there — but the frequency still surprised us," Modrau told Fox News Digital. "Long-term fatigue was common in our group of study participants, and we found that if people experience fatigue within two weeks of leaving the hospital, it is likely they will continue to have fatigue for up to a year." Based on these findings, Modrau suggests that people diagnosed with a transient ischemic attack should be monitored for lingering fatigue in the ensuing weeks and months. "This could help us better understand who might struggle with fatigue long-term and require further care." The more commonly known symptoms of stroke include face drooping, arm weakness or slurred speech, which usually resolve within a day, according to Modrau. Some patients also reported long-term cognitive issues. Bradley Serwer, an interventional cardiologist and chief medical officer at VitalSolution, an Ingenovis Health company that offers cardiovascular and anesthesiology services to hospitals nationwide, confirmed that fatigue is very common — and sometimes "debilitating" — following a stroke. "Fatigue is multifactorial and can rarely be attributed to a single cause," Serwer, who was not involved in the study, told Fox News Digital. "Fatigue is multifactorial and can rarely be attributed to a single cause." The Maryland-based cardiologist shared the following potential reasons for fatigue following a mini-stroke. Brain healing: "After a stroke, the brain tries to heal itself," Serwer said. "This process causes the brain to work harder to 'rewire' itself, which results in a higher demand for energy. This often leaves patients feeling drained or fatigued." Increased inflammation: This can occur due to the immune response following a TIA. Lower levels of neurotransmitters in the brain: Reduced levels of chemicals like serotonin, dopamine and norepinephrine can result in depression, fatigue or lack of motivation, according to Serwer. Sleep disturbances: "These are very common after a stroke and can lead to significant sleep deprivation," the cardiologist said. Medications: Drugs used to treat strokes may have adverse side effects, including fatigue. "Betablockers are excellent blood pressure medications and are often prescribed after a stroke or heart attack, but they may cause notable fatigue," Serwer noted. Other factors: "Simple tasks may require more mental effort than before the injury," the cardiologist said. "Depression or anxiety after a stroke can also be a confounding predictor of fatigue." The study did have some limitations, the researchers noted. "It is an observational study and therefore we cannot determine causality," Modrau said. "Results are based on self-reported questionnaires, and we cannot be certain that relatives didn't help fill them out or influence results." The researchers also did not have information on pre-TIA fatigue levels, although previous studies suggested that it was "much more frequent" after mini-strokes. Looking ahead, Modrau said she hopes healthcare providers begin to acknowledge lasting fatigue after TIA and provide care pathways for these patients. For more Health articles, visit "Up until now, patients with lasting challenges after TIA have been left alone in many cases," she told Fox News Digital. "We as a society should start to acknowledge their difficulties instead of viewing them as 'the lucky ones,'" she continued. "My aim with this study has been to give these patients a voice – and to start to listen to their struggles."
Yahoo
14-04-2025
- Business
- Yahoo
AAN 2025: Sanofi's tolebrutinib likely to be first BTK inhibitor for the treatment of MS
At the American Academy of Neurology (AAN) 2025 Annual Meeting Clinical Trials Plenary Session, Sanofi presented data on tolebrutinib in patients with non-relapsing secondary progressive multiple sclerosis (nrSPMS) from the HERCULES trial and in patients with relapsing multiple sclerosis (RMS) from GEMINI 1 and GEMINI 2. The presentation highlighted tolebrutinib's potential as the first Bruton's tyrosine kinase (BTK) inhibitor to receive regulatory approval for the treatment of progressive MS. During the Clinical Trials Plenary Session, Sanofi announced that tolebrutinib delayed the time to onset of six-month confirmed disability progression (CDP) by 31% compared to placebo in patients with nrSPMS, meeting the primary endpoint of the HERCULES trial. In addition, tolebrutinib met some of its secondary endpoints where it delayed the time to three-month CDP by 24% compared to placebo, and significantly lowered the annualised rate of new or enlarging T2 lesions versus placebo by 38%. While a lower proportion of participants in the tolebrutinib group showed progression in the Timed 25-Foot Walk, tolebrutinib did not differentiate from placebo in the 9-hole peg test. During the Plenary Session, Sanofi also reported that tolebrutinib failed to meet the primary endpoint of reducing annualised relapse rate over teriflunomide in patients with RMS in the GEMINI studies. A pooled analysis of GEMINI 1 and GEMINI 2 for a key secondary endpoint, not controlled for multiplicity, showed that tolebrutinib delayed the time to onset of six-month confirmed disability worsening by 29% versus teriflunomide. But the number of new Gd-enhancing T1 lesions was higher in the tolebrutinib arm and the number of new or enlarging T2 lesions was similar between both treatment arms. The results of the GEMINI trials follow Sanofi's decision, in January 2025, to remove its tolebrutinib RMS programme from its pipeline, focusing on progressive forms of MS only. In addition to development in nrSPMS per the HERCULES trial, there is an ongoing Phase III study to investigate the efficacy of tolebrutinib compared to placebo in delaying disease progression in patients with primary progressive MS (PERSEUS). Tolebrutinib was generally well-tolerated in the HERCULES, GEMINI 1, and GEMINI 2 trials. Liver enzyme elevations, defined by greater than three times the upper limit of normal, were observed in 4.0% of tolebrutinib patients in the HERCULES trial and 5.6% of tolebrutinib patients in the GEMINI studies. However, safety concerns have been previously raised. In June 2022, the US Food and Drug Administration (FDA) placed a partial clinical hold on tolebrutinib due to reported cases of drug-liver induced injury in patients who received the drug during clinical trials. Key opinion leaders (KOLs) previously interviewed by leading data and analytics company GlobalData stated that should tolebrutinib receive approval, it is likely to be accompanied by a boxed warning and risk evaluation and mitigation strategies. The majority of marketed disease-modifying therapies for MS target peripheral inflammation for reducing relapses, but few possess neuroprotective effects and, as such, have insufficient impact on the underlying neurologic deterioration caused by MS. The MS therapeutics market is heavily skewed towards reducing relapses in patients with RMS. KOLs previously interviewed by GlobalData stressed the need for a new product that could prevent disease progression. The gravity of this unmet need is highlighted by the number of BTK inhibitors in clinical development for MS. According to GlobalData's drug database, there are three BTK inhibitors in Phase III development. Tolebrutinib will compete with Genentech/Roche's fenebrutinib, Novartis' remibrutinib, and InnoCare Pharma's orelabrutinib. Last month, Sanofi announced tolebrutinib's US regulatory submission was accepted for priority review, and the target action date for the FDA decision for tolebrutinib is 28 September 2025. If approved, tolebrutinib would be the first and only BTK inhibitor to treat both non-relapsing SPMS and slow disability accumulation. "AAN 2025: Sanofi's tolebrutinib likely to be first BTK inhibitor for the treatment of MS" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Yahoo
10-04-2025
- Health
- Yahoo
AAN 2025: Lundbeck highlights sustained efficacy of Vyepti for migraine prevention
On 9 April, at the American Academy of Neurology (AAN) 2025 annual meeting, three posters highlighting the sustained long-term efficacy and real-world impact of Lundbeck's anti-calcitonin gene-related peptide (CGRP) monoclonal antibody (mAb) Vyepti (eptinezumab) for migraine prevention were presented. Vyepti was approved by the FDA for the prevention of migraine in adults in 2020. As the last of the anti-CGRP mAbs to enter the migraine market, Vyepti has seen moderate uptake since its approval, with the majority of key opinion leaders (KOLs) previously interviewed by GlobalData indicating that one of the subcutaneously administered anti-CGRP mAbs would be their first choice, highlighting the intravenous administration of Vyepti as a logistical challenge. The posters show that early response to Vyepti can be predictive of sustained treatment response, as well as highlighting meaningful benefits of Vyepti treatment. Post hoc analysis of the long-term, open-label Phase III PREVAIL (NCT02985398) study evaluating two-year safety and patient-reported outcomes with Vyepti in adults with chronic migraine was presented in a poster at AAN 2025. The analysis showed that the majority of the participants who achieved 50% or greater headache response or 75% or greater headache response in the first 12 weeks after receiving their first dose of Vyepti were able to maintain that level of response up to Week 84. Sustained treatment response was further demonstrated in another poster presenting post hoc analysis of data from the randomized, placebo-controlled Phase III DELIVER (NCT04418765) trial. In this study, patients had either chronic migraine or high-frequency episodic migraine, and they had failed to respond to two to four prior migraine preventive treatments. Up to 83% of patients who responded to Vyepti treatment within the first one to 24 weeks of the trial (i.e., having received the first two doses of Vyepti) were able to maintain that response over the 72-week trial. These analyses highlight that early treatment response with Vyepti may be able to predict long-term sustained treatment response. A third poster focused on patient-reported impact of Vyepti treatment in adults with chronic migraine. It presented a post hoc analysis of the impact of a 75% or greater increase in good days/month in patients treated with Vyepti from a real-world, observational, US-based study. Approximately two-thirds of the patients in the study reported a 75% or greater increase in good days/month, and this was correlated with higher satisfaction regarding the effects of Vyepti on migraine symptom severity, frequency, and duration; daily activities, such as being able to participate in social/family life, being productive, and ability to plan; and overall wellbeing when compared with patient with less than 75% increase in good days/month. Prior to treatment, the prevalence of brain fog was similar across the treatment response subgroups; however, following Vyepti treatment, 76.6% of the patients with a 75% or greater increase in good days/month reported moderate-to-complete improvement in brain fog, compared with 42.3% of patients with less than 75% increase in good days/month. These results highlight the meaningful benefits that patients experience following Vyepti treatment and will help Lundbeck with its focus on 'raising the bar around preventive treatment expectations in migraine.' Historically, migraine prevention has been managed with a wide variety of drug classes, such as beta-adrenergic receptor blockers, calcium antagonists, antidepressants, and anti-epileptics. However, many of these oral preventive treatments have been associated with poor side-effect profiles, and KOLs previously interviewed by GlobalData reported that low efficacy of oral preventive treatment was common among patients, meaning that patients often cycle through the different drug classes of oral preventive treatments. The first preventive therapies developed specifically for the treatment of migraine were the anti-CGRP mAbs, followed by the oral gepants; however, they are typically prescribed as second- or third-line options for many patients due to reimbursement restrictions requiring failure with the oral preventives before they can be prescribed. In the US, a 2024 statement was published by the American Headache Society recommending that the anti-CGRP mAbs and gepants should be used as first-line preventive options due to their superior efficacy and tolerability compared with the oral preventive options. Long-term sustained efficacy data and demonstration of meaningful benefit for patients, such as was presented for Vyepti at AAN 2025, are likely to further cement the case for first-line adoption of the anti-CGRP mAbs; however, given the comparative costs of the oral preventives and the mAbs, whether this recommendation will be implemented by insurance companies remains to be seen. Vyepti faces significant competition from three other anti-CGRP mAbs: Amgen's Aimovig (erenumab), Eli Lilly's Emgality (galcanezumab), and Teva's Ajovy (fremanezumab). Additional competition also comes from two orally administered gepants: Pfizer's Nurtec (rimegepant) and AbbVie's Qulipta (atogepant). KOLs agreed that the CGRP mAbs had similar efficacy profiles, with treatment choice often based on side effects, dosing schedule, or patient preference. As the only intravenously administered mAb, Vyepti may be at a disadvantage among patients who prefer to self-administer one of the other mAbs, and therefore highlighting the long-term efficacy, particularly with a focus on some of the patient-reported meaningful benefits, will be crucial in helping Vyepti to remain competitive in the migraine prevention market. "AAN 2025: Lundbeck highlights sustained efficacy of Vyepti for migraine prevention" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Yahoo
10-04-2025
- Health
- Yahoo
AAN 2025: J&J reveals data for nipocalimab, showing long-term disease control in MG
On 8 April, at the 2025 American Academy of Neurology (AAN) meeting, Johnson & Johnson (J&J) announced results from additional analyses of the Phase III Vivacity-MG3 double-blind study and the ongoing open-label extension (OLE) (NCT04951622), evaluating the long-term efficacy and safety of investigational nipocalimab in a broad population of acetylcholine receptor antibody–positive (AChR+), muscle-specific tyrosine kinase antibody–positive (MuSK+), and low-density lipoprotein receptor-related protein 4 antibody–positive (LRP4+) adults with generalised myasthenia gravis (gMG). The additional analysis confirmed that patients treated with nipocalimab plus standard of care (SOC) maintained improvements in their Myasthenia Gravis Activities of Daily Living (MG-ADL) score and quantitative myasthenia gravis (QMG) scores over 84 weeks with sustained reductions in total IgG. Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block the neonatal Fc receptor (FcRn) and reduce levels of circulating immunoglobulin G (IgG) antibodies, potentially without impact on other immune functions. Vivacity-MG3 is a Phase III, randomised, double-blind, placebo-controlled study conducted at 81 outpatient centres with expertise in myasthenia gravis in 17 countries in Asia–Pacific, Europe, and North America. The Vivacity trial included adults aged 18 or over with gMG inadequately controlled with SOC therapy. The participants were randomly assigned to receive either nipocalimab at a loading dose of 30mg/kg, followed by a maintenance dose of 15mg/kg every two weeks, or placebo infusions every two weeks, added to SOC therapy in both groups, for 24 weeks. Patients treated with nipocalimab plus SOC maintained improvements in their MG-ADL and QMG scores over 84 weeks with sustained reductions in total IgG. J&J exhibited additional findings from the Vivacity-MG3 study, displaying that nipocalimab met its key secondary endpoint, a change in QMG score. Patients treated with nipocalimab plus SOC achieved statistically significant improvements in their QMG score by negative 4.9 versus placebo plus SOC over seeks 22 and 24. Patients in the nipocalimab plus SOC treatment group were four times more likely to sustain symptom improvement at 20 weeks compared to the placebo plus SOC group, as measured by a three or greater point improvement on the QMG score. J&J also showed that nipocalimab demonstrated a mean change in MG-ADL of negative 5.64 from the double-blind baseline after 60 weeks in the OLE for study participants receiving nipocalimab and SOC, and a mean change of negative 6.01 for study participants who transitioned from placebo and SOC to nipocalimab and SOC. As part of the trial, nipocalimab also exhibited a steroid-sparing effect. In the antibody-positive population, 45% of patients receiving steroids at the OLE baseline were able to decrease or discontinue steroids at 84 weeks by more than half of their baseline dose. Among these patients, the mean dose of prednisone decreased from 23mg to 10mg per day. Finally, nipocalimab had a consistent and tolerable safety profile throughout the OLE phase. The additional results of the trial are exciting. Given that MG is a chronic condition and there is a significant disease burden on patients living with this condition, investigational therapies must be able to demonstrate long-term disease control. Furthermore, the Vivacity-MG3 trial showed improvements in disease control as measured by MG-ADL and QMG scores across a broad population seropositive for AChR, MuSK, or LRP4 autoantibodies. This is essential, as currently, most marketed therapies are indicated for AChR+ patients with gMG. Key opinion leaders (KOLs) previously interviewed by GlobalData have noted that there is a significant unmet need for effective treatments for patients who are MuSK+ or LRP4+. These patients are usually prescribed standard treatments such as acetylcholinesterase inhibitors, steroids, and immunosuppressants, and although effective in non-refractory MG patients, refractory MG patients are left with few additional treatment options. Therefore, the results of the Vivacity-MG3 trial are a welcome development for MuSK+ and LRP4+ MG patients, as they indicate that an exciting new treatment option may be on the horizon. The steroid-sparing effect displayed in the Vivacity-MG3 trial is also noteworthy. KOLs have noted that despite their effectiveness in treating MG, the prolonged use of corticosteroids is associated with severe side effects and that their use must be carefully managed to attain a therapeutic effect without impacting a patient's quality of life. Nipocalimab may be able to assist in helping patients transition off corticosteroid treatment, improving both their treatment outcomes and quality of life. The findings from the Vivacity-MG3 trial underscore the potential of nipocalimab as a transformative treatment for gMG. By demonstrating sustained improvements in MG-ADL and QMG scores, alongside reductions in IgG levels, nipocalimab addresses critical unmet needs for patients with AChR+, MuSK+, and LRP4+ antibodies. Its steroid-sparing effect further enhances its appeal, offering a pathway to improved quality of life by reducing reliance on corticosteroids and their associated side effects. With a consistent safety profile and promising long-term efficacy, nipocalimab represents a promising advancement in gMG therapy, paving the way for broader disease management and better patient outcomes. "AAN 2025: J&J reveals data for nipocalimab, showing long-term disease control in MG" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
