13, Mar 2026
Researchers break decades-old bottleneck in chemotherapy drug manufacturing
Researchers break decades-old bottleneck in chemotherapy drug manufacturing
An international team of researchers has achieved a breakthrough in the production of doxorubicin, a vital chemotherapy agent. The study identifies and resolves molecular “bottlenecks” that have limited the natural production of this drug for over 50 years.
Doxorubicin is a chemotherapy drug that was first approved for medical use in the 1970s. It is a cornerstone in treating various cancers, including breast cancer, bladder cancer, lymphomas and carcinomas, with over one million patients receiving the treatment annually. However, bacteria naturally produce this important drug very inefficiently. Consequently, the pharmaceutical industry has relied on expensive, multi-step semi-synthetic processes.
“We have uncovered several independent factors that limit the formation of doxorubicin,” says Researcher, Keith Yamada, PhD, from the University of Turku in Finland, a lead scientist on the study. “By addressing these bottlenecks, we have harnessed rational strain engineering to pave the way for cost-effective manufacturing that can meet growing global demand”.
Researchers develop new strain of bacteria that enhances drug production
The study was the result of an extensive international collaboration involving a total of six research laboratories: the University of Turku in Finland, three laboratories in the United States, and two in Leiden, the Netherlands.
Together, the teams identified three primary constraints that prevent the high-yield production of doxorubicin.
Firstly, the team identified the specific natural “biological power supply” — redox partners named Fdx4 and FdR3 — that provide the necessary electron flow to power the drug-producing enzyme.
Secondly, they discovered that a protein called DnrV acts as a drug-binding “molecular sponge”. It sequesters (binds and holds) doxorubicin so the drug does not shut down the enzyme’s own production machinery.
Last, using X-ray crystallography, the team visualized the enzyme for the first time, revealing that the drug molecule sits in an unfavorable position within the enzyme, explaining the slow reaction rate.
By combining these discoveries, the researchers engineered a new strain of bacteria that produces 180% more doxorubicin than current industrial standards.
To bring these findings to the real world, the spin-out company Meta-Cells Oy was formed last year at the University of Turku. The company aims to commercialize these advanced technologies for the sustainable manufacturing of essential antibiotics and anti-cancer agents. This shift toward fully biosynthetic production promises a cleaner, more reliable supply of life-saving medicines.
The study was published in the renowned journal Nature Communications.
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- By Neel Achary
13, Mar 2026
Lurie Autism Institute Announces Dr. Huda Zoghbi as the Inaugural Recipient of the Nancy Lurie Marks Prize for Autism Research
Mar 13 – The Lurie Autism Institute, a joint initiative of Children’s Hospital of Philadelphia (CHOP) and Penn Medicine created to drive discovery, develop new treatments, and improve the lives of individuals and families affected by autism, is proud to announce that geneticist Huda Y. Zoghbi, MD, has been selected as the inaugural recipient of the Nancy Lurie Marks Prize for Autism Research, the Institute’s highest honor recognizing transformative contributions to autism research.
Zoghbi is a Distinguished Service Professor in the Departments of Molecular and Human Genetics, Pediatrics, Neuroscience, and Neurology at Baylor College of Medicine, and Director of Texas Children’s Duncan Neurological Research Institute (Duncan NRI). She is also an Investigator at the Howard Hughes Medical Institute. A pioneering pediatric neurologist and physician–scientist, Zoghbi has fundamentally reshaped understanding of the genetic and molecular basis of neurological disease – including autism spectrum disorder – by integrating human genetics, animal models, and systems-level neuroscience to define how disruptions in gene regulation, neuronal maturation, and circuit function drive disease.
“When thinking of an appropriate inaugural recipient of the Nancy Lurie Marks Prize for Autism Research, we wanted to consider the pre-eminent minds whose long history of incredible work in autism research continues to have a lasting effect,” said prize committee chair Frances E. Jensen, MD, Chair of the Department of Neurology and Professor of Neurology at the Perelman School of Medicine at the University of Pennsylvania and co-director of the Penn Translational Neuroscience Center. “Dr. Zoghbi’s record speaks for itself, and we couldn’t think of a more deserving inaugural recipient who exemplifies the pioneering work in autism research that the Lurie Autism Institute wants to make possible.”
The Prize Selection Committee recognized Zoghbi for her landmark discovery that mutations in the MECP2 gene cause Rett syndrome, an autism-related neurodevelopmental disorder. This breakthrough transformed Rett syndrome from an enigmatic clinical condition into a foundational model for understanding autism genetics and neurobiology. Her work established core principles that have guided modern autism research; these principles now underpin contemporary large-scale genomic studies of autism and have shaped how investigators conceptualize risk, penetrance, and phenotypic variability across neurodevelopmental disorders.
“I am deeply honored to receive the inaugural Nancy Lurie Marks Prize for Autism Research,” said Zoghbi. “Nancy’s dedication to autism research and to the families she so passionately championed has left an indelible mark on our field. The Lurie Autism Institute’s continued commitment to advancing impactful autism research benefits us all. I share this recognition with the patients and families who inspire our work every day, and with the remarkable trainees and collaborators whose insight, creativity, and dedication have advanced our understanding of how genetic disruptions alter brain function.” She continued, “I hope that continued progress in this field will lead to better insights and treatments, ultimately improving the lives of individuals with autism and their families. This honor serves as a powerful reminder of the promise rigorous science holds for truly transforming lives.”
By demonstrating that de novo mutations underlie Rett syndrome, Dr. Zoghbi helped catalyze study designs that enrich for de novo variation. These studies served as an important basis for projects such as the Simons Simplex Collection, a core project and resource of the Simons Foundation Autism Research Initiative (SFARI) that establishes a permanent repository of genetic samples of families of children with autism. This framework was then later adopted by the Simons Foundation Powering Autism Research for Knowledge (SPARK) and the Autism Sequencing Consortium. This paradigm led to the discovery of dozens of de novo variants and hundreds of autism-related genes, many of which encode chromatin regulators, firmly establishing epigenetic and chromatin-mediated mechanisms as central pathways in autism.
“Dr. Zoghbi’s incredible discoveries of some key biological mechanisms underlying autism are important steps in the journey we’re on with the Lurie Autism Institute to provide patients with more answers,” said Daniel Rader, MD, Interim Director, Lurie Autism Institute and Chief of Translational Medicine and Human Genetics, Penn Medicine and CHOP. “Her central role in advancing our understanding of neurobiology and translating that basic science into clinical progress makes her an extremely deserving recipient of the inaugural Nancy Lurie Marks Prize for Autism Research.”
The Nancy Lurie Marks Prize for Autism Research honors the legacy of Nancy Lurie Marks, whose visionary philanthropy has played a pivotal role in advancing autism research and improving the lives of individuals with autism. The Prize includes a $100,000 award and recognizes a single individual whose work has made a profound and lasting impact on the field.
“The Lurie Autism Institute was established to usher in a new era of scientific discovery in autism, and the work of extraordinary talents like Dr. Zoghbi exemplifies the kind of breakthrough discoveries we hope to make possible,” said Jeffrey Lurie, Chairman and CEO of the Philadelphia Eagles and founder of the Eagles Autism Foundation.
Zoghbi will be formally honored at the 2026 Lurie Autism Institute Symposium, to be held on May 7, 2026, in Philadelphia, where she will deliver a featured lecture.
The Lurie Autism Institute, which reflects the combined strength of Children’s Hospital of Philadelphia and Penn Medicine, was made possible through the generosity of the Lurie Family Foundation and the Nancy Lurie Marks Family Foundation. Launched in June, 2025, the Institute is dedicated to advancing autism science and care, while aiming to accelerate discovery, deepen understanding of autism’s complexities and improve outcomes for individuals and families.
12, Mar 2026
Miror Therapeutics Empowers Bengaluru’s “Ground Heroes” with Landmark Hormonal Health Initiative at Magadi Road Maternity Hospital
Bengaluru, Mar 12: Miror Therapeutics Pvt Ltd successfully hosted “Women’s Midlife & Beyond,” a high-impact hormonal health awareness program for frontline women workers in Bengaluru. Held at the Magadi Road Maternity Hospital, the event provided critical medical education and nutritional support to 62 women, including Pourakarmikas, ASHA workers, and staff from the government hospital.
The initiative targeted a significant but often overlooked demographic: the women who serve as the backbone of the city’s infrastructure. Through clinical screening of 54 participants, Miror identified a staggering need for early intervention, discovering that 84% of attendees were in the perimenopause stage, while 16% had reached menopause.
“This data underscores an urgent need for localised advocacy and early-stage hormonal support within these professional sectors,” stated Mr Sanjith Shetty, Founder & CEO, Miror Therapeutics.
The program moved beyond theory by providing actionable health solutions. Under the guidance of the Indian Menopause Society (IMS) and Miror Representatives, attendees discussed Perimenopause, Menopause, and Hormone Replacement Therapy (HRT).
While the event provided foundational dietary consultations, Miror has announced a strategic pivot for future outreach. Moving forward, the company will prioritise Gynaecologist-led advocacy to provide the “medical seal of authority” required for complex symptom management and HRT eligibility.
Mr Shetty also stated that “Our mission is to ensure every woman leaves with clinical clarity,” the report concludes. “By shifting toward clinical gynaecology, we aim to foster higher medical literacy and a more confident adoption of evidence-based treatments”.
12, Mar 2026
Guwahati family shares experience of hospital-based palliative care during advanced cancer treatment
Guwahati, Mar 12: A Guwahati family has shared its experience of hospital–based palliative care after their mother, diagnosed with advanced-stage liver cancer, was shifted to comfort-focused treatment during a recent admission at Peerless Hospital Guwahati.
The 62-year-old patient had initially been admitted for what the family expected would be a short hospital stay. During the admission, doctors informed them that immunotherapy had not been effective and the disease had progressed, making palliative care the most appropriate course of treatment.
While home-based care was discussed, the family felt her condition required continuous medical supervision.
“We wanted her to remain in a setting where immediate medical support was available if needed. Continuing care at home did not seem medically advisable in her condition,” a family member said.
The family explored several palliative care options in Guwahati before opting for Peerless Hospital’s structured palliative care package, which was arranged within 24 hours of discussion with the medical team.
The patient was shifted to a single cabin where a family member could remain round the clock. She was placed under continuous vital monitoring, with nurses conducting regular manual checks of blood pressure, temperature and oxygen saturation. Preventive measures such as an air mattress were used to reduce the risk of bedsores, while medications were administered on schedule and her positioning was adjusted regularly for comfort.
A dietician reviewed her meals and adjusted food choices based on medical requirements and personal preferences. Mobility support was also encouraged, with wheelchair outings and assisted short walks when possible. Daily physiotherapy sessions were provided to help maintain strength and movement.
The patient’s primary physician reviewed her condition twice a day, and specialists were consulted whenever required.
Over time, the patient was able to resume oral intake after previously requiring assisted feeding, and both her feeding tube and catheter were removed.
“After months without appetite, being able to eat normally again meant a great deal to her,” the family member said.
Dr. Gautam Kumar Das, CEO at Peerless Hospital Guwahati, said palliative care focuses on improving comfort and quality of life when curative treatment is no longer effective.
“Palliative care is often misunderstood as only end-of-life care. In reality, it is about managing symptoms, maintaining dignity, and supporting both patients and families during serious illness. Many families want to remain closely involved in caring for their loved ones but also need the reassurance of medical supervision. Structured palliative care within a hospital environment can provide that balance of clinical support and family presence,” he said.
The family said the structured care plan helped them remain close to the patient while ensuring consistent medical supervision during a difficult phase of treatment.
10, Mar 2026
Park Hospital Launches Panchkula Facility, Expands Mohali Campus; Tricity Capacity to Reach 850 Beds
Panchkula/Mohali, Mar 10: Park Medi World Limited has approved the launch of an advanced multi-super specialty hospital in Panchkula along with the expansion of its existing facility in Mohali, further strengthening the presence of Park Group of Hospitals across the Tricity healthcare ecosystem.
The newly launched hospital in Panchkula will be expandable up to 350 beds and is scheduled to become operational from March 29, 2026. This development aims to expand access to advanced healthcare services for patients across Haryana, Punjab, Himachal Pradesh, and Chandigarh.
Alongside the Panchkula launch, the company will also expand its 350-bed Grecian Super Specialty Hospital in Mohali with an additional 150 beds, significantly enhancing its clinical capacity and specialty care services.
With these developments, Park Group of Hospitals will reach a combined capacity of 850 beds across the Tricity region, positioning it as the largest private healthcare network in the area. The Tricity cluster will comprise 350 existing beds at Mohali, a capacity of up to 350 beds at the newly launched Panchkula facility, and the upcoming 150-bed super specialty expansion at Mohali.
Advanced Healthcare Infrastructure
The Panchkula hospital has been designed to deliver comprehensive tertiary and quaternary care, supported by advanced diagnostics, modern operation theatres, and robust critical care infrastructure. The facility will provide specialized treatments across multiple medical disciplines to address the increasing demand for advanced healthcare services in the region.
Meanwhile, the Mohali expansion will focus on strengthening high-acuity specialties such as Oncology, Neurosciences, Gastro Sciences, and Robotic Surgeries. The facility will also introduce robotic joint replacement capabilities, enabling cutting-edge minimally invasive procedures and enhancing treatment outcomes for patients.
Leadership Perspective
Commenting on the development, Dr. Ankit Gupta, Managing Director of Park Medi World Limited, said:
“The launch of our Panchkula hospital and the expansion of our Mohali facility mark a significant milestone in Park Hospital’s journey to strengthen advanced healthcare access across the Tricity region. With a combined capacity of 850 beds across our Mohali and Panchkula campuses, we are building one of the most comprehensive private healthcare networks in the region.”
He further added, “The new Panchkula hospital, along with the upcoming 150-bed super specialty expansion in Mohali, reflects our long-term commitment to developing centres of excellence in high-acuity care. Our focus is on strengthening capabilities in areas such as oncology, neurosciences, gastro sciences, and robotic surgeries while introducing advanced technologies that enable minimally invasive and patient-centric treatment.”
Expanding Healthcare Access in North India
With the launch of the Panchkula hospital and the expansion of its Mohali facility, Park Group of Hospitals continues to expand its footprint in North India, reinforcing its commitment to delivering high-quality, specialized, and technology-enabled healthcare at scale.
The initiative aligns with the group’s broader vision of building an integrated healthcare ecosystem that combines clinical expertise, advanced technology, and large-scale infrastructure to provide world-class care closer to communities across the region.
9, Mar 2026
kyron.bio Announces Strategic Partnership with Servier to Advance Precision Glycosylation in Antibody Therapeutics
Paris, 9 March, 2026 – kyron.bio, a biotechnology company pioneering precision glycoengineering for antibody therapeutic development, today announced a strategic partnership with Servier, an international pharmaceutical group governed by a Foundation.
Under the terms of the agreement, kyron.bio will use its technology to glycoengineer an antibody selected by Servier, who will fund the associated research activities. Servier will have the option to further explore antibody engineering and development opportunities based on the outcomes. Financial details are not disclosed.
kyron.bio’s proprietary glycoengineering platform can enhance therapeutic performance of antibodies by enabling precise control of the glycan structures to improve efficacy, safety, and scalability. In this partnership kyron.bio will seek to demonstrate clear glycan control on the Servier antibody of interest for a specific pre-determined N-glycoform.
To date, engineering of glycans have been under-exploited, due to technical challenges, limiting the use of glycan engineering in drug design. kyron.bio is changing that. The company has developed a scalable, proprietary method to achieve comprehensive control over glycosylation, unlocking the possibility to use precision glycosylation in next generation drug design.
Dr. Emilia McLaughlin, founder and Chief Executive Officer of kyron.bio said,
“We are delighted that Servier has chosen to explore the potential of our glycoengineering platform. Servier has deep expertise in therapeutic development and combined with our precision glycosylation technology, this partnership provides a powerful opportunity to unlock new levels of antibody performance and deliver better outcomes for patients.
“Precision glycosylation represents a transformative approach in biologics development. By engineering defined glycan profiles, therapeutic antibodies can be optimized for improved immune engagement, pharmacokinetics, and reduced variability.”
In 2024, kyron.bio was the winner of the Servier Golden Ticket award which has provided invaluable support and mentorship through the company’s early translational phase and has developed a foundation for understanding the potential of kyron.bio’s technology.
Dr. Emmanuel Nony, Director of External Innovation Europe at Servier said,
“Meeting kyron.bio as a winner of Servier’s Golden Ticket award has enabled our scientists to develop an understanding of the kyron.bio glycan engineering technology and its exciting possibilities in antibody drug design. This collaboration is opening new frontiers for antibody derivatives as well. Together, we are exploring innovative pathways to optimize drug design and production, with a shared commitment to bringing safer and more effective therapies to patients.”
kyron.bio’s strategy is to form strategic drug design partnerships with pharmaceutical and biotech companies working on next-generation antibody therapeutics, alongside in house therapeutic development programs.
A successful company creation from the French Entrepreneur First Scheme, in 2025 kyron.bio raised €5.5m in a seed round from an experienced syndicate of venture investors including HCVC, Verve Ventures, Entrepreneurs First and Saras Capital, as well as private angel investors and the European Innovation Council. It has established an R&D base at the biotech hub Paris Biotech Santé in the Cochin Hospital.
9, Mar 2026
kyron.bio Announces Strategic Partnership with Servier to Advance Precision Glycosylation in Antibody Therapeutics
Innovative glycobiology platform aimed at enhancing the efficacy, safety, and scalability of next-generation antibody therapeutics across multiple disease areas
Paris, Mar 09 – kyron.bio, a biotechnology company pioneering precision glycoengineering for antibody therapeutic development, today announced a strategic partnership with Servier, an international pharmaceutical group governed by a Foundation.
Under the terms of the agreement, kyron.bio will use its technology to glycoengineer an antibody selected by Servier, who will fund the associated research activities. Servier will have the option to further explore antibody engineering and development opportunities based on the outcomes. Financial details are not disclosed.
kyron.bio’s proprietary glycoengineering platform can enhance therapeutic performance of antibodies by enabling precise control of the glycan structures to improve efficacy, safety, and scalability. In this partnership kyron.bio will seek to demonstrate clear glycan control on the Servier antibody of interest for a specific pre-determined N-glycoform.
To date, engineering of glycans have been under-exploited, due to technical challenges, limiting the use of glycan engineering in drug design. kyron.bio is changing that. The company has developed a scalable, proprietary method to achieve comprehensive control over glycosylation, unlocking the possibility to use precision glycosylation in next generation drug design.
Dr. Emilia McLaughlin, founder and Chief Executive Officer of kyron.bio said,
“We are delighted that Servier has chosen to explore the potential of our glycoengineering platform. Servier has deep expertise in therapeutic development and combined with our precision glycosylation technology, this partnership provides a powerful opportunity to unlock new levels of antibody performance and deliver better outcomes for patients.
“Precision glycosylation represents a transformative approach in biologics development. By engineering defined glycan profiles, therapeutic antibodies can be optimized for improved immune engagement, pharmacokinetics, and reduced variability.”
In 2024, kyron.bio was the winner of the Servier Golden Ticket award which has provided invaluable support and mentorship through the company’s early translational phase and has developed a foundation for understanding the potential of kyron.bio’s technology.
Dr. Emmanuel Nony, Director of External Innovation Europe at Servier said,
“Meeting kyron.bio as a winner of Servier’s Golden Ticket award has enabled our scientists to develop an understanding of the kyron.bio glycan engineering technology and its exciting possibilities in antibody drug design. This collaboration is opening new frontiers for antibody derivatives as well. Together, we are exploring innovative pathways to optimize drug design and production, with a shared commitment to bringing safer and more effective therapies to patients.”
kyron.bio’s strategy is to form strategic drug design partnerships with pharmaceutical and biotech companies working on next-generation antibody therapeutics, alongside in house therapeutic development programs.
A successful company creation from the French Entrepreneur First Scheme, in 2025 kyron.bio raised €5.5m in a seed round from an experienced syndicate of venture investors including HCVC, Verve Ventures, Entrepreneurs First and Saras Capital, as well as private angel investors and the European Innovation Council. It has established an R&D base at the biotech hub Paris Biotech Santé in the Cochin Hospital.
9, Mar 2026
Akshar Yoga Kendraa Launches PURANTHA, a Global Centre for Advanced Yogic Sciences in Bengaluru
The campus aims to train 100,000 yoga teachers annually through structured yogic education
Bengaluru, India | Mar 09— Akshar Yoga Kendraa has announced the launch of PURANTHA, a Himalayan-origin centre dedicated to advanced yoga and spiritual science. Conceptualised under the vision of Himalayan Siddhaa Akshar, the new campus has been developed near Kempegowda International Airport in Bengaluru. Spread across about 165,000 square feet, the facility has been designed to train 100,000 yoga teachers each year, responding to the rising global demand for structured and authentic yoga education.
PURANTHA has been established as a space for deeper engagement with Himalayan yogic sciences and Siddha traditions, combining practice, study, and research within one environment. The campus brings together several aspects of yogic learning, including advanced practice modules, teacher education programs, and applied study of traditional knowledge systems.
The design of the centre allows different disciplines to function alongside one another. Facilities have been created for yogic training, meditation, prāṇāyāma, Ayurveda, research and development, and residential learning. With its scale and layout, the campus is intended to support sustained practice and long-term study without interruption between different activities.
All training and educational programs at PURANTHA are conducted under the guidance of Himalayan Siddhaa Akshar through Akshar Yoga Kendraa, an institution recognised by the Ministry of AYUSH under the Yoga Institution category. The centre has been developed with an emphasis on disciplined learning, methodological clarity, safety, and continuity of practice.
Speaking on the launch, Himalayan Siddhaa Akshar said: “PURANTHA has been envisioned as a space where the depth of Himalayan yogic traditions can be preserved, studied and practiced through a structured institutional framework. The intention is to support practitioners and teachers from across the world who wish to explore yoga as a science of inner evolution.”
Prataprao Ganpatrao Jadhav, Union Minister of State (Independent Charge), Ministry of AYUSH; Minister of State, Ministry of Health & Family Welfare, Government of India, said on the launch: “I extend my heartfelt congratulations and best wishes to Akshar Yoga Kendraa on the launch of PURANTHA, a Himalayan-origin centre dedicated to advanced yoga and spiritual science. I commend Himalayan Siddhaa Akshar and the entire Akshar Yoga community for this inspiring initiative, which will further strengthen India’s leadership in yoga and holistic wellness across the world.”
The campus is also intended to serve as a global platform for the structured transmission of Himalayan yogic and spiritual knowledge. Through residential training programs, institutional collaborations, and international participation, PURANTHA aims to contribute to the continued study and dissemination of yogic sciences across diverse cultural and educational contexts.
7, Mar 2026
Taiwan Researchers Develop Fluorescent Imaging That Could Make Tumor-Guided Surgery Easier to Adopt
During cancer surgery, one of the most challenging decisions surgeons face is identifying exactly where a tumor ends and healthy tissue begins. To the naked eye, cancer tissue often looks very similar to surrounding tissue, making it challenging to resect tumors completely without damaging healthy areas. As a result, studies across multiple cancer types suggest that 20–30% of patients may require additional treatment or repeat surgery due to unclear tumor margins.
Researchers led by Professor Li-Feng Liu from the School of Medicine at I-Shou University are developing a new imaging approach designed to make tumor boundaries visibly distinguishable during surgery, giving surgeons clearer guidance at the moment it matters most.
The method builds on a simple biological insight: cancer cells consume far more sugar than normal cells because they grow rapidly. Professor Liu’s team developed a fluorescent sugar-based imaging agent that exploits this behavior. When administered before surgery, the agent is taken up more readily by tumor tissue than by surrounding healthy cells.
During the operation, surgeons illuminate the surgical area using a simple ultraviolet light. Tumor tissue then emits visible fluorescence, creating a clear visual contrast that helps surgeons identify tumor boundaries in real time as they remove the cancer while preserving healthy tissue.
What sets this approach apart is its ease of adoption. Many existing fluorescence-guided systems rely on complex imaging platforms and specialized equipment that are not available in every operating room. In contrast, this method works with widely available light sources, integrates smoothly into existing surgical workflows, and is designed to be non-toxic and safe for intraoperative use.
By lowering technical and operational barriers, the technology could help extend tumor-guided surgery beyond major medical centers. Potential applications include regional hospitals, minimally invasive procedures, and veterinary oncology, where access to advanced imaging infrastructure may be limited.
As Professor Liu and his team continue to evaluate the technology across different tumor types and surgical settings, their goal is to make precision-guided cancer surgery easier to adopt, more widely accessible, and safer for patients. Readers interested in the science behind the innovation and its real-world potential can learn more by exploring the full press release and watching the accompanying video, which illustrates how the technology works in practice.
6, Mar 2026
First Gene Regulation Clinical Trials for Epilepsy Show Promising Results
Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children’s Hospital of Chicago, show that the first gene regulation treatment for epilepsy is safe and well tolerated by patients with Dravet syndrome for whom antiseizure medications are not effective. Results, published in the New England Journal of Medicine, include significant seizure reduction and improvement in other symptoms of Dravet syndrome, such as language, motor and behavior issues. Researchers also report sustained treatment benefits in ongoing open-label extension studies.
“Our results are highly promising, especially since currently there are no approved treatments that address the underlying cause of Dravet syndrome,” said Dr. Laux, Head of the Epilepsy Center and Associate Division Head of Neurology at Lurie Children’s, as well as Associate Professor of Pediatrics at Northwestern University Feinberg School of Medicine. “Since this gene regulation product targets the actual root cause of Dravet syndrome, we observed improvements in other developmental and cognitive symptoms, in addition to seizure control. This is unprecedented.”
Dravet syndrome includes a spectrum of symptoms that emerge in infancy and evolve. Most patients experience cognitive deficits, communication and behavioral impairments, motor dysfunction, growth delays and autistic traits. Difficulties with feeding, poor appetite, and weight loss are also common.
As Dr. Laux explained, patients with Dravet syndrome have a mutation on one SCN1A (sodium channel receptor) gene with one normal SCN1A gene. The mutation causes a haploinsufficiency (only half of the amount of the alpha 1 sodium receptor subunit is made). This causes seizures, as well as cognitive and motor issues. The study medication (zorevunersen) acts on the normal SCN1A gene to make it work harder and overcome the deficit caused by the mutated SCN1A gene. Zorevunersen is injected into the spinal fluid by a lumber puncture.
Owen – a Lurie Children’s patient who participated in the clinical trial and now continues in the open label extension study – is a 12-year-old boy with Dravet syndrome, whose seizures were not controlled by medications. He also had intellectual disability and gait abnormalities. With zorevunersen, Owen’s seizures are significantly reduced and he has had marked improvement in language and behavior, according to Dr. Laux.
“He is able to make friends, which is kind of a new development,” said Owen’s mother Austin. “His quality of life has increased substantially so that he’s able to enjoy more activities with neurotypical peers.”
The two Phase 1/2a, open-label, multicenter studies (one in the U.S. and the other in the U.K.) enrolled 81 patients with Dravet syndrome aged 2–18 years on standard antiseizure medications.
Patients who received two to three doses of 70 mg zorevunersen had reduction of motor seizures of nearly 85% at three months and 73% at six months from dosing.
Eligible patients, like Owen, rolled over to the open-label extension studies. These patients were given 45 mg of zorevunersen every four months, and they continued to have significant seizure reduction ranging from 58% to 90% over the first 20 months. For patients in the extension studies for more than 36 months, expressive and receptive communication were significantly improved.
While nearly all patients had a treatment-emergent adverse event (TEAE), most of these were mild to moderate. The most common TEAE in the Phase 1/2a trials was post-lumbar puncture syndrome (nearly 25%) while the most common event in the extension studies was cerebrospinal fluid (CSF) protein increase (45%). However, none of the patients with CSF protein increase had increased intracranial pressure or hydrocephalus. Of the serious TEAE, only one was considered treatment related.
“Our data support zorevunersen safety and tolerability, as well as improvement in overall clinical status, quality of life and adaptive behavior following continued dosing in the extension studies,” said Dr. Laux.
A Phase 3, double-blind, placebo-controlled trial of zorevunersen for Dravet syndrome is currently underway.
Funding was provided by Stoke Therapeutics.
Dr. Laux holds the Lorna S. and James P. Langdon Chair in Pediatric Neurology at Lurie Children’s.
Ann & Robert H. Lurie Children’s Hospital of Chicago is a nonprofit organization committed to providing access to exceptional care for every child. It is the only independent, research-driven children’s hospital in Illinois and one of less than 35 nationally. This is where the top doctors go to train, practice pediatric medicine, teach, advocate, research and stay up to date on the latest treatments. Exclusively focused on children, all Lurie Children’s resources are devoted to serving their needs. Research at Lurie Children’s is conducted through Stanley Manne Children’s Research Institute, which is focused on improving child health, transforming pediatric medicine and ensuring healthier futures through the relentless pursuit of knowledge. Lurie Children’s is the pediatric training ground for Northwestern University Feinberg School of Medicine. It is ranked as one of the nation’s top children’s hospitals by U.S. News & World Report.
