Latest news with #clinicalTrials
Reuters
22-07-2025
- Business
- Reuters
Sarepta to pause Elevidys gene therapy shipments in US
July 21 (Reuters) - Sarepta Therapeutics (SRPT.O), opens new tab said on Monday it will pause all shipments of Elevidys in the United States after two teenage boys with a rare condition called Duchenne muscular dystrophy, who had received the company's gene therapy, died of liver failure this year. The U.S. Food and Drug Administration on Friday asked Sarepta to voluntarily halt shipments of its gene therapy. The company responded that it would continue providing the treatment to ambulatory patients — while maintaining a halt it implemented June 15 for non-ambulatory patients. Sarepta's shares are down 8.6% to $12.17 after the bell. The stock plummeted, opens new tab on Friday after it disclosed the dead of a third patient who had received an experimental gene therapy, deepening investor concerns over the company's treatments. While the 51-year-old man with limb girdle muscular dystrophy who died most recently was not taking Elevidys, the experimental therapy he took was based on similar gene technology, the FDA said. The FDA put Sarepta's clinical trials for limb girdle muscular dystrophy on hold due to safety concerns. All shipments of Elevidys for Duchenne muscular dystrophy will be temporarily paused by close of business on Tuesday, Sarepta said Monday. The company said that the pause will allow it the "time to respond to any requests for information and allow Sarepta and FDA to complete the Elevidys safety labeling supplement process." Sarepta said it anticipates a review and dialogue with the FDA. The FDA did not immediately respond to a Reuters request for comment. After the first two deaths, Sarepta in June suspended the use of Elevidys for patients whose disease had progressed to the point where they need a wheelchair. Elevidys has full FDA approval for children with DMD who are still able to walk, and conditional approval for non-ambulatory patients. Last week, Sarepta said it was adding a warning on Elevidys' label for the risk of acute liver injury and liver failure in DMD patients who can walk. It also announced 500 job cuts and halted development of several gene therapies for limb girdle muscular dystrophy. Separately, the Children's Hospital Los Angeles, said on Monday it has paused usage of Elevidys in all patients with muscular dystrophy.
Medscape
10-07-2025
- Health
- Medscape
Which Brain Stim Offers the Best Outcomes With Depression?
TOPLINE: Transcranial electrical stimulation (tES) was associated with reduced depressive symptoms in a new meta-analysis – but individual types of tES were linked to different outcomes. Transcranial direct current stimulation (tDCS) was associated with greater improvements in patients with psychiatric or medical comorbidities compared to those with major depressive disorder (MDD) alone, while transcranial alternating current stimulation (tACS) was associated with reduced MDD symptoms and increased response rates. METHODOLOGY: Researchers conducted a systematic review and meta-analysis of 88 randomized clinical trials. The data included more than 5500 participants (mean age, 43 years; 60% women) with MDD, depression with medical comorbidities, or depression with psychiatric comorbidities. The investigators evaluated tES modalities, including tDCS (79 trials), tACS (six trials), and transcranial random noise stimulation, or tRNS (three trials), compared with sham or other treatment modalities. Primary outcomes included depression severity, rates of response and remission, and adverse events (AEs). TAKEAWAY: tES overall vs sham or no treatment was associated with a reduction in depressive symptoms (standardized mean difference [SMD], -0.59) and a greater improvement in patients with medical (SMD, -1.05) or physical comorbidities (SMD, -0.78). Mild to moderate AEs were more frequent with tES vs sham treatment. tDCS was associated with significantly reduced depressive symptoms in patients with medical (SMD, -1.05) or physical comorbidities (SMD, -0.88), but not with MDD only. Active tDCS plus antidepressant use vs sham tDCS plus the medication was linked to a reduction in MDD symptoms (SMD, -0.51) and an increase in response rates (odds ratio [OR], 2.25). Compared with sham treatment, tACS was linked to a significant reduction in MDD symptoms (SMD, -0.58) and to a greater likelihood of increased response rates (OR, 2.07). Anodal left dorsolateral prefrontal cortex stimulation was associated with improved outcomes. Transcranial random noise stimulation was not associated with significant improvements in any type of depressive symptoms or response rates. IN PRACTICE: "These findings suggest that tES is well-tolerated, associated with mild to moderate AEs, and poses a minimal risk of serious AEs. Future research should study ideal stimulation parameters and individualize tES interventions," the investigators wrote. SOURCE: The study was led by Caili Ren, MD, Mayo Clinic, Rochester, Minnesota. It was published online on June 18 in JAMA Network Open. LIMITATIONS: Limited data on tACS and tRNS, with most of the studies' findings reflecting tDCS, led to an inability to perform robust subgroup analyses. Other limitations included potential confounding in categorizing tDCS monotherapy vs combined therapy when medication use was unspecified; a lack of stratification by depression severity; incomplete safety profiling because more than 40% of the studies did not report AEs; and potential publication bias in tDCS vs sham comparisons, especially in depression with medical comorbidities. DISCLOSURES: The study was funded by the Mayo Clinic and the National Center for Advancing Translational Sciences. One investigator reported numerous disclosures, including being a paid member of corporate scientific advisory boards and a co-founder and/or chief medical officer of another company. He also reported receiving grants from several foundations and holding patents on real-time integration of transcranial magnetic stimulation with electroencephalography and MRI. Additional disclosures are fully listed in the original article. This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.
Bloomberg
09-07-2025
- Health
- Bloomberg
Antidepressant Pullback Symptoms Fewer Than Thought, Study Shows
Stopping antidepressants doesn't lead to as many withdrawal symptoms as previously thought, according to a new study. In a large trial examining the crucial weeks after people end treatment, scientists found that the symptoms reported after one week weren't even enough register as clinically significant. The study, published in Jama Psychiatry on Wednesday, ruled out mood change as a symptom, while dizziness was the effect most commonly reported.
Health Line
04-07-2025
- Health
- Health Line
Treatment Options for Brain Cancer
Key takeaways Surgery, radiation therapy, and chemotherapy are common treatments for brain cancer, and they can be used alone or in combination. The specific approach depends on the cancer's characteristics and location. Targeted and electric field therapies are additional options for managing brain cancer. Clinical trials, like those researching immunotherapy, also offer promising new treatments that could improve the outlook for people with this condition. A brain tumor happens when cells in your brain begin to grow and divide out of control. While some brain tumors are benign (noncancerous), others are malignant (cancerous). There are a few different treatments that may be used for brain cancer, either alone or together. And clinical trials are currently testing new treatments for brain cancer that can help to improve your outlook. Keep reading to learn more about each treatment, how it's used, and the side effects associated with it. Can you survive brain cancer? It's possible to survive brain cancer. But survival can vary greatly based on many factors, such as the type of brain cancer that you have and your age. Surgery for brain cancer Surgery is a part of treatment for many brain cancers. During surgery, a neurosurgeon will try to remove as much of the tumor as possible without affecting the function of your brain. Surgery is often done by craniotomy. This is where a small opening is made in your skull, allowing for access to your brain. You may be under general or local anesthesia during surgery. While operating, a neurosurgeon can use various techniques to help them safely remove your tumor without damaging the surrounding tissue. These can include: Using surgery in combination with other brain cancer treatments like radiation therapy (RT) may be able to eliminate smaller, less aggressive cancers. But this can be more challenging for cancers that are larger or more aggressive. Surgery can also be used for other purposes. These include inserting a shunt or drain to reduce intracranial pressure or placing an Ommaya reservoir to help deliver chemotherapy (chemo). Side effects of surgery for brain cancer Some of the possible side effects of surgery for brain cancer may include: a reaction to the anesthetic infections bleeding swelling of the brain seizures loss of brain function Radiation therapy for brain cancer RT uses high-energy radiation to destroy cancer cells. It's often given for brain cancer using a radiation source located outside of your body. This is called external beam RT. Radiation has the potential to damage healthy brain tissue, so several methods have been developed to help lower this risk. An example of one is conformational RT, which makes a 3D image of your tumor and shapes the radiation to fit to it. You may receive RT after surgery to help kill any remaining cancer cells. RT may also be one of the main treatment options if your cancer can't be operated on. This may be the case if your cancer is: very extensive located deep within your brain found at or around an area that's vital for brain function Side effects of radiation therapy for brain cancer Some of the potential side effects of RT for brain cancer are: fatigue nausea or vomiting headache hair loss cognitive changes, which can include issues with memory, difficulties with concentration, or changes in personality increased risk of a second cancer Chemotherapy for brain cancer Chemo uses drugs that disrupt the ability of cancer cells to grow and divide. It can be used along with other treatment types, such as surgery or RT, or alone when other treatments haven't been effective. While many chemo drugs are given directly into your bloodstream or taken orally, this isn't always possible with brain cancer. The reason is that many chemo drugs can't cross the blood-brain barrier. As such, some chemo may be given directly into your cerebrospinal fluid. Other types of chemo may also be given as a wafer that's placed in your brain during surgery. A few examples of chemo drugs used for brain cancer, either alone or in combination, are: carmustine lomustine procarbazine temozolomide vincristine Side effects of chemotherapy for brain cancer The possible side effects of chemo include: nausea or vomiting diarrhea mouth sores loss of appetite hair loss low blood counts, which can cause: anemia an increased risk of infections easy bleeding Targeted therapy for brain cancer Targeted therapy uses drugs that target specific markers on or in cancer cells. Currently, there are only a handful of targeted therapy drugs used for brain cancer. Bevacizumab (Avastin) inhibits a protein that promotes the growth of blood vessels around tumors. It's given by an intravenous (IV) line and may be used to treat glioblastoma. Everolimus (Afinitor) blocks the activity of a protein involved in cell growth and division. It's taken as a pill and is used for some types of astrocytomas. Side effects of targeted therapy for brain cancer Specific side effects can vary based off of the targeted therapy drug used. But some of the more general side effects of targeted therapy drugs may include: fatigue loss of appetite nausea diarrhea mouth sores headache an increased risk of infections Alternating electric field therapy for brain cancer Alternating electric field therapy exposes a tumor to electric fields that affect its ability to grow. This type of therapy involves the use of a wearable device called the Optune system that generates those electric fields. The Optune system is used for people who recently received a diagnosis of glioblastoma or those who have recurrent glioblastoma. Side effects of alternating electric field therapy for brain cancer Some of the side effects that you may have while using alternating electric field therapy include: skin irritation where the device and its electrodes are placed on your scalp headache seizures low blood counts and digestive side effects when used with chemo Clinical trials for brain cancer Clinical trials evaluate potentially new or improved ways to treat a disease or disorder. They're essential for testing the safety and effectiveness of new treatments before they're made more widely available. One type of treatment that's being heavily researched for brain cancer is immunotherapy. This is a type of cancer treatment that helps your immune system respond to cancer. It's already used for many other cancer types. For some people with brain cancer, receiving treatment through a clinical trial may be recommended. This is particularly true if you have a cancer that: is rare or very aggressive has a limited number of approved treatment options hasn't responded to conventional treatments has come back after treatment If you're interested in a clinical trial for brain cancer, talk with your medical care team. They can recommend clinical trials you may qualify for. You can also find clinical trials through the website of the National Brain Tumor Society or by searching What's the outlook for a person with brain cancer? The outlook for people with brain cancer depends on many factors. These include: the type of brain cancer you have the grade of the cancer, which estimates how quickly the tumor may grow where the tumor is in your brain how large the tumor is whether or not the tumor can be removed using surgery and, if so, how much of the tumor can be removed the presence of certain genetic changes in the tumor cells your age and overall health For example, the 5-year relative survival rate for adults ages 20–44 years old with diffuse astrocytoma, a slow-growing cancer, is 73%. In contrast, the 5-year relative survival rate is 22% in the same age group for glioblastoma, an aggressive cancer. If you've recently received a diagnosis of brain cancer, your medical care team will consider all of the factors above to give you a better idea of your individual outlook. Relative survival rate vs. survival rate A relative survival rate suggests how long someone with a condition may live after receiving a diagnosis compared with someone without the condition of the same race, sex, and age over a specific time. This is different from overall survival rate, which is a percentage of people still alive for a specific time after receiving a diagnosis of a condition.
Medscape
03-07-2025
- Health
- Medscape
Meta-Analysis Compares Hidradenitis Suppurativa Treatments
TOPLINE: In a network meta-analysis of 25 randomized trials, sonelokimab was ranked highest for treating patients with moderate-to-severe hidradenitis suppurativa (HS). Adalimumab, lutikizumab, and bimekizumab were also ranked high. METHODOLOGY: Researchers conducted a systematic review and network meta-analysis of 25 phase 2 and 3 randomized clinical trials of medical treatments for adults with moderate-to-severe HS (up to June 28, 2024), with primary efficacy assessments performed between 12 and 16 weeks. The trials included 5767 patients with moderate-to-severe HS and 39 unique treatments, including the three approved treatments in the US and Europe: adalimumab, secukinumab, and bimekizumab. Primary outcomes were the HS Clinical Response of at least 50% (HiSCR-50), serious adverse events, and discontinuation due to adverse events. Researchers also reported HiSCR-75. TAKEAWAY: Compared with placebo, significantly higher HiSCR-50 responses were noted for sonelokimab, 120 mg, every 4 weeks (odds ratio [OR], 4.44; 95% CI, 2.29-8.61) and 240 mg (OR, 2.62; 95% CI, 1.37-5.00); lutikizumab, 300 mg, every 2 weeks (OR, 2.72; 95% CI, 1.08-6.86); adalimumab, 40 mg, once weekly (OR, 2.63; 95% CI, 2.06-3.36); bimekizumab, 320 mg, every 2 weeks (OR, 2.63; 95% CI, 2.06-3.36) and every 4 weeks (OR, 2.27; 95% CI, 1.52-3.39); povorcitinib, 15 mg, once per day (OR, 2.28; 95% CI, 1.02-5.13); and secukinumab, 300 mg, every 2 weeks (OR, 1.60; 95% CI, 1.18-2.16) and every 4 weeks (OR, 1.62; 95% CI, 1.20-2.20). Compared with placebo, higher HiSCR-75 responses (a secondary endpoint) were noted for sonelokimab, 120 mg, every 4 weeks (OR, 4.12; 95% CI, 2.00-8.51); lutikizumab, 300 mg, every 2 weeks (OR, 4.01; 95% CI, 1.40-11.47) and once weekly (OR, 2.95; 95% CI, 1.03-8.42); bimekizumab, 320 mg, every 2 weeks (OR, 2.91; 95% CI, 1.89-4.49); sonelokimab, 240 mg, every 4 weeks (OR, 2.89; 1.38-6.06); adalimumab, 40 mg, once per week (OR, 2.85; 95% CI, 1.89-4.30); bimekizumab, 320 mg, every 4 weeks (OR, 2.26; 95% CI, 1.39-3.66); and secukinumab, 300 mg, every 4 weeks (OR, 2.04; 95% CI, 1.39-3.00) and every 2 weeks (OR, 1.85; 95% CI, 1.26-2.73). Sonelokimab, 120 mg, every 4 weeks was the top treatment for both HiSCR-50 and HiSCR-75. Other high-rated treatments were adalimumab, 40 mg, once per week; sonelokimab, 240 mg, every 4 weeks; lutikizumab, 300 mg, every 2 weeks; and bimekizumab, 320 mg, every 2 weeks. Serious adverse event rates ranged from 0% to 10% for placebo, 0% to 8% for adalimumab (40 mg once per week), and 0% to 6% for other active treatment groups. Discontinuation rates due to adverse events were 0%-10%, 0%-4%, and 0%-15%, respectively. IN PRACTICE: The network meta-analysis 'provides evidence for the comparative efficacy and safety of currently approved cytokine inhibitors for moderate to severe HS in the absence of head-to-head trials,' the authors of the study wrote. 'Phase 2 results for several cytokine and small-molecule treatments are promising and require confirmation in larger phase 3 trials,' they added. SOURCE: The study was led by Amit Garg, MD, Department of Dermatology, Zucker School of Medicine at Hofstra/Northwell, New Hyde Park, New York, and was published online on July 2 in JAMA Dermatology. LIMITATIONS: Limitations were reliance on indirect evidence for most comparisons, small sample size for individual treatment groups, and short follow-up. DISCLOSURES: The authors reported no funding information. Garg declared receiving grants and personal fees from AbbVie, Almirall, Boehringer Ingelheim, Engitix, Immunitas Therapeutics, Incyte, Insmed, Novartis, Pfizer, Priovant Therapeutics, Sonoma Biotherapeutics, Sun Pharma, UCB, UNION Therapeutics, and Zura Bio, and having a patent for an HS Investigator Global Assessment and a licensed patent for HS quality of life. This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.
