Rigosertib Shows Promise in EB-Associated Skin Cancer
METHODOLOGY:
Researchers conducted an open-label, single-arm phase 2 study in Austria and the United States of five patients with advanced, treatment-refractory RDEB-SCC who had failed prior standard of care and were then treated with oral or intravenous (IV) rigosertib.
Participants (median age, 23 years) received either oral rigosertib (560 mg twice daily on days 1-21 of each 28-day cycle for 13 cycles) or IV rigosertib (1800 mg/24 h as a 72-h continuous infusion on days 1-3 of each 14-day cycle for eight cycles, then on days 1-3 of 28-day cycles until 1 year of treatment).
Two patients received IV treatment, two received oral treatment, and one received a combination of both.
The primary outcome was the objective response rate and safety. Quality of life was a secondary outcome.
TAKEAWAY:
Two patients achieved complete a compete response, and two others had partial responses. The fifth patient discontinued treatment early because of unrelated complications.
Rigosertib was generally well tolerated. Most adverse events were urinary-related (cystitis, hematuria) and manageable with dose adjustments or symptomatic treatment.
IN PRACTICE:
'These initial results indicate rigosertib as a promising drug therapy for RDEB-SCC where there is a substantial unmet need and no approved therapies,' the study authors wrote. The study, they noted, was the 'first clinical trial of any experimental cancer therapeutic in this rare and complicated monogenic disease.'
SOURCE:
The study was led by Martin Laimer, Department of Dermatology and Allergology and EB House Austria, University Hospital of the Paracelsus Medical University, Salzburg, Austria, and was published online on May 29 in British Journal of Dermatology .
LIMITATIONS:
Usefulness of imaging techniques in this study was limited, particularly because of the chronic inflammatory response in lesional skin that is inherent to RDEB.
DISCLOSURES:
The study was funded by Debra International through Debra Austria, Debra of America, Cure EB, and Debra UK. Laimer and three other authors reported receiving consulting fees and grants from Onconova Therapeutics, Inc. Two authors declared being employees of Onconova, which provided the study drug.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
31 minutes ago
- Yahoo
CGTN: How China's economy remains vibrant despite U.S. tariff war
BEIJING, July 25, 2025 /PRNewswire/ -- With Chinese Vice Premier He Lifeng to hold economic and trade talks with the United States in Sweden later this month, the strength and resilience of the Chinese economy is in the limelight once again. This round of talks, mutually agreed upon by both nations, is not only a diplomatic engagement but also a testament to China's enduring economic vitality in a complex international environment. Recent statistics underscore the robustness of China's economy. In the first half of 2025, the GDP grew by an impressive 5.3 percent year on year, surpassing market expectations despite global headwinds. This number reflects more than temporary growth; it exhibits the structural resilience and adaptability of an economy that continues to evolve and upgrade. Domestic demand emerged as the cornerstone of growth, contributing 68.8 percent to GDP expansion in this period. Initiatives such as large-scale equipment upgrades and consumer goods trade-in programs have effectively stimulated spending, cushioning China's economy from external shocks. In the first five months of 2025 alone, China's consumer goods trade-in program generated 1.1 trillion yuan ($153.1 billion) in sales, surpassing the figure for entire 2024. Boosted by the program, China's retail sales of consumer goods grew 5 percent year on year in the past six months – 0.4 percentage point faster than the growth recorded in the first quarter. While external uncertainties have introduced some pressure, China's trade diversification and the steady output of high-tech manufacturing and service industries have provided strong support to the economy. Though China's trade with some Western countries declined, its trade with Belt and Road partners, ASEAN countries, the European Union, and African nations saw respective increases of 4.7 percent, 9.6 percent, 3.5 percent, and 14.4 percent in the first half of 2025. This expansion of trade relationships has helped China reduce its reliance on any one market, lessening the impact of some Western economies' protectionist policies. China's resilience reverberates beyond its borders. As a crucial driver of global growth, China's steady economic performance boosts international market confidence and provides a stabilizing influence amid global uncertainties. Through continued focus on quality growth and opening up, China offers the international community a reliable engine for shared prosperity. A recent report from the U.S.-China Business Council indicates that 82 percent of American companies operating in China turned a profit in 2024. Though many cited the uncertainties in China-U.S. relations and tariffs as their main worry, the Chinese market continues to be crucial for them. Trade tensions pose obstacles, yet they have not crippled the resilience in the Chinese economy. The upcoming Beijing-Washington talks in Sweden demonstrate China's willingness to tackle differences via negotiations. While obstacles remain, China's ability to sustain growth, adapt to changing global landscapes, and engage constructively with international partners signals a future of shared opportunities and mutual advancement. View original content to download multimedia: SOURCE CGTN
Yahoo
2 hours ago
- Yahoo
Fossils unearthed in Grand Canyon reveal new details of evolutionary explosion of life
Paleontologists have discovered remarkable fossils in the Grand Canyon that reveal fresh details about the emergence of complex life half a billion years ago. The newfound remains of fauna from the region suggest that it offered ideal conditions for life to flourish and diversify, in a 'Goldilocks zone' between harsh extremes elsewhere. This evolutionary opportunity produced a multitude of early animals, including oddballs with peculiar adaptations for survival, according to new research. During the Cambrian explosion, which played out in the coastal waters of Earth's oceans about 540 million years ago, most animal body types that exist today emerged in a relatively short time span, scientists believe. Back then, the Grand Canyon was closer to the equator, and the region was covered by a warm, shallow sea teeming with burgeoning life — aquatic creatures resembling modern-day shrimp, pill bugs and slugs — all developing new ways to exploit the abundant resources. Researchers turned to the Grand Canyon's layers of sedimentary rock to unlock secrets of this pivotal moment in the history of life, digging into the flaky, claylike shale of the Bright Angel Formation where most of the canyon's Cambrian-era fossils have been found. The study team expected to recover mostly the fossilized remains of hard-shelled invertebrates typical of the region. Instead, the team unearthed something unusual: rocks containing well-preserved internal fragments of tiny soft-bodied mollusks, crustaceans, and priapulids, also known as penis worms. 'With these kinds of fossils, we can better study their morphology, their appearance, and their lifestyle in much greater resolution, which is not possible with the shelly parts,' said Giovanni Mussini, the first author of the study published Wednesday in the journal Science Advances. 'It's a new kind of window on Cambrian life in the Grand Canyon.' Using high-powered microscopes, the team was able to investigate innovations such as miniature chains of teeth from rock-scraping mollusks and the hairy limbs and molars of filter-feeding crustaceans, providing a rare look into the biologically complex ways Cambrian animals adapted to capture and eat prey. The 'Goldilocks zone' for innovation For most of the planet's 4 billion-year history, simplicity reigned. Single-celled microbes remained stationary on the ocean floor, thriving on chemical compounds such as carbon dioxide and sulfur molecules to break down food. What changed? Scientists still debate what drove the Cambrian explosion, but the most popular theory is that oxygen in the Earth's atmosphere slowly began to increase about 550 million years ago, said Erik Sperling, an associate professor of Earth and planetary sciences at Stanford University. Oxygen provided a much more efficient way to metabolize food, giving animals more energy to mobilize and hunt for prey, suggested Sperling, who was not involved in the new study. 'The (emergence of) predators kicked off these escalatory arms races, and then we basically got the explosion of different ways of doing business,' Sperling said. During the Cambrian, the shallow sea covering the Grand Canyon was especially oxygen-rich thanks to its perfect, 'Goldilocks' depth, said Mussini, a doctoral student in Earth sciences at the University of Cambridge in the United Kingdom. Ranging from 40 to 50 meters (about 130 to 165 feet) in depth, the ecosystem was undisturbed by the shoreline's constant waves shifting around sediments, and sunlight was still able to reach photosynthesizing plants on the seafloor that could provide oxygen. The abundance of food and favorable environmental conditions meant that animals could take more evolutionary risks to stay ahead of their competition, Mussini said. 'In a more resource-starved environment, animals can't afford to make that sort of physiological investment,' Mussini said in a news release from the University of Cambridge. 'It's got certain parallels with economics: invest and take risks in times of abundance; save and be conservative in times of scarcity.' Many soft-bodied fossil finds before this one have come from regions with harsh environments such as Canada's Burgess Shale formation and China's Maotianshan Shales, noted Susannah Porter, a professor of Earth science at the University of California in Santa Barbara who was not involved in the study. 'It's not unlike if paleontologists far in the future only had great fossil records from Antarctica, where harsh cold environments forced people to adapt. … But then found great human fossils in New York City, where people flourished,' Porter explained. 'We have an opportunity to see different sorts of evolutionary pressures that aren't like, it's really cold, it's really hot, there's not a lot of water.' Weird adaptations of Cambrian animals While some of the feeding mechanisms uncovered in the Grand Canyon fossils are still around today, others are much more alien. Among the most freakish: penis worms that turned their mouths inside out, revealing a throat lined with hairy teeth. The worms, also known as cactus worms, are mostly extinct today, but were widespread during the Cambrian. The fossilized worm found in the Grand Canyon represents a previously unknown species. Due to its relatively large size — about 3.9 inches (10 centimeters) — and distinct teeth, it was named Kraytdraco spectatus, after the fictional krayt dragon from the Star Wars universe, Mussini said. This particular penis worm appears to have had a gradient of hundreds of branching teeth used to sweep food into an extendable mouth. 'It's a bit hard to understand how exactly it was feeding,' Mussini said. 'But it was probably eating debris on the seafloor, scraping it away with some of the most robust teeth that it had, and then using these other, more delicate teeth to filter and retain it within this long, tube-like mouth.' Rows of tiny molars, sternal parts and comblike limbs that once belonged to crustaceans were also among the findings, which all date back 507 million to 502 million years. Similar to today's brine shrimp, the crustaceans used these fine-haired limbs to capture floating food from the water and bring it to the mouth, where molars would then grind down the particles, Mussini explained. Nestled among the molars, researchers even found a few unlucky plankton. Other creatures resembling their modern counterparts included sluglike mollusks. The fossils revealed chains of teeth that likely helped them scrape algae or bacteria from along the seafloor. 'For each of these animals, there's different components, but most of what we found directly relates to the way these animals were processing their food, which is one of the most exciting parts, because it tells us a lot about their lifestyle, and as a consequence, their ecological implications,' Mussini said. Sign up for CNN's Wonder Theory science newsletter. Explore the universe with news on fascinating discoveries, scientific advancements and more. Solve the daily Crossword
Yahoo
3 hours ago
- Yahoo
Scientists Just Linked This Diet to a Lower Lung Cancer Risk
Reviewed by Dietitian Jessica Ball, M.S., RD At this point, you've likely heard about the Mediterranean diet, the DASH diet and the MIND diet. But there's one diet that might be new to you: the planetary health diet (PHD). The PHD was developed in 2019 by the EAT-Lancet Commission and focuses on promoting human health and environmental sustainability. The planetary health diet is primarily plant-based, focusing on fruits, vegetables, nuts, seeds, legumes, whole grains and unsaturated fats. It includes small amounts of animal-based proteins, including seafood, poultry and dairy, but these are eaten in much smaller quantities, as most protein comes from plant protein. Added sugars, refined grains and processed meats are limited or avoided. Research regarding the planetary health diet has been mixed, so researchers from Austria and Switzerland wanted to see what they could find out about the relationship between the planetary health diet and mortality, cancer and heart disease. They published their results in the American Journal of Clinical Nutrition. How Was This Study Conducted? Researchers drew their data from the UK Biobank, a long-running study that collects demographic, health and medical data from over 500,000 UK citizens. Baseline data were gathered between 2006 and 2010, with the average age at baseline ranging from 40 to 69. Assessments were repeated every few years and collected data on lifestyle, health status and diet. Researchers also had access to medical records and death registries. For this current study, researchers used varying numbers of participants for three different analyses: almost 205,000 were used in the mortality risk analyses, over 200,000 for the cancer risk analyses, and over 196,000 for the cardiovascular disease risk analyses. Adherence to the planetary health diet eating pattern was scored based on the inclusion of 14 primary food components of the planetary health diet. Participants scored one point for each food or food group they included, with a possible high score of 14. Scores for this study ranged from 1 to 11 points, which represents low to medium diet adherence. What Did This Study Find? After running statistical analyses, including adjusting for potential confounding factors (factors that might influence or change results), researchers found that: Every 1-point increase in the PHD score was related to a 3% decrease in all-cause mortality (risk of dying from anything). Every 1-point increase in the PHD score was associated with a 9% lower lung cancer risk. When all factors were fully adjusted for, no relationship was found in this study between the PHD and cardiovascular disease risk. There were several limitations of this study. Because comparatively few people in the Biobank were following a planetary health diet eating pattern, the confidence of these results is lower than it would have been had there been more people following it. Also, out of the five possible Biobank 24-hour dietary recalls, about 40% of the participants completed just one of them. Again, less data means less confidence in the results. And since the Biobank includes mostly white adults from Europe, it is unknown if the results can be generalized to other groups. How Does This Apply to Real Life? The term 'plant-based diet' can mean different things to different people. While some equate it to a vegan, all-plant diet, others see it more as making plants the foundation of your diet, while still including small amounts of animal-based foods, like eggs, dairy, fish and poultry. The latter is what the planetary health diet is about. It also limits added sugar, refined grains and processed meats. Eating more plants has many benefits, including reduced disease risk. This is in part thanks to plants' antioxidants and their ability to create a healthy gut microbiome by providing food for your gut's beneficial bacteria. A healthy gut can help lower chronic inflammation, which helps reduce disease risk. And while you can add helpful bacteria to your gut with fermented plants—like sauerkraut or kimchi—many people find it easier to get them in yogurt or kefir, which is where the animal protein can come in. It's easy to get stuck in a rut with fruits and vegetables, or to always turn to bread for whole grains. But there are lots of creative ways to use whole grains—like rice, quinoa, farro and oats—that don't include bread. Grain bowls are a delicious way to eat more plants, and they can be tailored to your taste and preferences. They're also easy to meal-prep for lunches throughout the week. We also love salads as meals. You can pack every food group into a salad if you want to. You'll love our filling, satisfying High-Protein Pasta Salad, Massaged Kale Salad with Roasted Sweet Potato & Black Beans or Salmon Salad with Crispy White Beans. If you want more guidance and are ready to go all in on increasing your plant intake, check out our 7-Day Plant-Based High-Protein Meal Plan for Beginners. It's loaded with a variety of fruits, vegetables, whole grains and plant-based proteins, like peanut butter, edamame and beans, to keep you full and satisfied. Whether you use the plan as is or use it as inspiration, you'll find it fun and easy to get more plants into your meals and snacks. Our Expert Take This study suggests that eating more plants may reduce your risk of all-cause mortality and lung disease. While this study didn't show an association between eating a plant-based diet and reduced risk of heart disease, other studies have. Just remember that eating more plants doesn't have to be all or nothing. Start where you're at and be intentional with choosing plant-based foods more often. Snacks can be an easy way to start. Instead of reaching for your typical snack, try a handful of nuts and a piece of fruit, or dip fruit slices into nut butter or veggie sticks into guacamole or hummus. Read the original article on EATINGWELL
