In research published in Developmental Medicine & Child Neurology, investigators developed a brief, reliable, and valid screening tool to help identify individuals with Duchenne muscular dystrophy (a neuromuscular disorder) who are at increased risk of brain-related comorbidities, such as language disorders, attention-deficit/hyperactivity disorder, and anxiety.
The research team developed the questionnaire-based screening tool, called the BIND (Brain Involvement iN Dystrophinopathies) screener, by reviewing the medical literature and incorporating expert consensus, and translated it into 11 languages. The questionnaire asks parents, caregivers, or patients to rate the impact of 18 cognitive, behavioural, and emotional items.
The BIND screener demonstrated strong accuracy in identifying individuals with Duchenne muscular dystrophy who had previously been diagnosed with neurodevelopmental or psychiatric conditions in an international sample of 835 participants. Additional validation was conducted in a subsample of 90 children and adolescents who underwent in-depth cognitive and clinical assessments.
“Families often tell us that cognitive and behavioural difficulties can be as challenging as the physical symptoms of Duchenne. This screener is designed to help identify those concerns earlier, so that children and adults can be referred for appropriate support,” said corresponding author Rubén Miranda, PhD, of Universidad Complutense de Madrid, Spain.
A new Cochrane review has found no clear evidence for cannabis-based medicines work for chronic neuropathic pain
Photo by Kindel Media on Unsplash
There is no clear evidence that cannabis-based medicines provide pain relief for chronic neuropathic pain, an updated Cochrane review finds.
Chronic neuropathic pain is caused by nerve damage. Existing medications help only a minority of patients, driving interest in alternatives, such as cannabis-based medicines. These can include herbal cannabis or isolated ingredients of the cannabis plant such as tetrahydrocannabinol (THC) by inhalation, mouth sprays, tablets, creams, and patches placed on the skin.
Researchers reviewed 21 clinical trials involving more than 2100 adults, comparing cannabis-based medicines with placebo over periods of two to 26 weeks.
Cannabis-based medicines were grouped into three types: products which contain mostly THC, the psychoactive component of cannabis; products which contain mostly cannabidiol (CBD), a non-intoxicating compound; and balanced THC/CBD products, which contain similar amounts of both.
The review found no high-quality evidence that cannabis-based medicines reduce neuropathic pain more than placebo across the three types of medicines. While some small improvements were reported by patients using products with both THC and CBD, these changes were not large enough to be considered clinically meaningful.
Reporting of adverse events was not consistent across the included trials, so certainty around side-effects was low to very low across all types of cannabis-based medicines. Products containing THC were associated with increases in symptoms such as dizziness and drowsiness, with a potential increase in the number of people withdrawing from trials due to side effects.
Clinician and review author from Technische Universität München and Medical Center Pain Medicine and Mental Health Saarbrücken, Winfried Häuser, emphasized the need for better quality studies:
We need larger, well-designed studies with a treatment duration of at least 12 weeks that include people with comorbid physical illnesses and mental health conditions to fully understand the benefits and harms of cannabis-based medicines. At present, the quality of most of the trials is too poor to draw firm conclusions.
— Winfried Häuser, clinician and author
The authors conclude that the evidence remains weak and uncertain, underscoring the need for higher-quality research before cannabis-based medicines can be recommended for chronic neuropathic pain.
A drug mimicking the hormone progesterone has anti-cancer activity when used together with conventional anti-oestrogen treatment for women with breast cancer, a new Cambridge-led trial has found.
In the two-week window that we looked at, adding a progestin made the anti-oestrogen treatment more effective at slowing tumour growth. What was particularly pleasing to see was that even the lower dose had the desired effectRebecca Burrell
A low dose of megestrol acetate (a synthetic version of progesterone) has already been proven as a treatment to help patients manage hot flushes associated with anti-oestrogen breast cancer therapies, and so could help them continue taking their treatment. The PIONEER trial has now shown that the addition of low dose megestrol to such treatment may also have a direct anti-cancer effect.
Around three-quarters of all breast cancers are ER-positive. This means the tumours are abundant in a molecule known as an oestrogen receptor, ‘feeding’ on the oestrogen circulating in the body. These women are usually offered anti-oestrogens, medication that reduces level of oestrogen and hence deprives the cancer of oestrogen and inhibits its growth. However, reducing oestrogen levels can bring on menopause-like symptoms, including hot flushes, joint and muscle pain, and potential bone loss.
In the PIONEER trial, post-menopausal women with ER-positive cancers were treated with an anti-oestrogen with or without the progesterone mimic, megestrol. After two weeks of treatment, those that received the combination saw a greater decrease in tumour growth rates compared to those treated with an anti-oestrogen only.
Although further work is required in larger patient cohorts and over a longer period of time to confirm the findings, researchers at the University of Cambridge say the trial suggests that megestrol could help improve the lives of thousands of women for whom anti-oestrogen medication causes uncomfortable side-effects and can lead to some women stopping taking the medication.
PIONEER was led by Dr Richard Baird from the Department of Oncology at the University of Cambridge and Honorary Consultant Medical Oncologist at Cambridge University Hospitals NHS Foundation Trust (CUH). He said: “On the whole, anti-oestrogens are very good treatments compared to some chemotherapies. They’re gentler and are well tolerated, so patients often take them for many years. But some patients experience side effects that affect their quality of life. If you’re taking something long term, even seemingly relatively minor side effects can have a big impact.”
Some ER-positive breast cancer patients also have high levels of another molecule, known as progesterone receptor (PR). This group of patients also respond better to the anti-oestrogen hormone therapy.
To explain why, Professor Jason Carroll and colleagues at the Cancer Research UK Cambridge Institute used cell cultures and mouse models to show that the hormone progesterone stops ER-positive cancer cells from dividing by indirectly blocking ER. This results in slower growth of the tumour. When mice treated with anti-oestrogen hormone therapy were also given progesterone, the tumours grew even more slowly.
Professor Carroll, who co-leads the Precision Breast Cancer Institute and is a Fellow of Clare College, Cambridge, said: “These were very promising lab-based results, but we needed to show that this was also the case in patients. There’s been concern that taking hormone replacement therapy – which primarily consists of oestrogen and synthetic versions of progesterone (called progestins) – might encourage tumour growth. Although we no longer think this is the case, there’s still been residual concern around the use of progesterone and progestins in breast cancer.”
To see whether targeting the progesterone receptor in combination with an anti-oestrogen could slow tumour growth in patients, Dr Baird and Professor Carroll designed the PIONEER trial, which tested adding megestrol, a progestin, to the standard anti-oestrogen treatment letrozole.
A total of 198 patients were recruited at ten UK hospitals, including Addenbrooke’s Hospital in Cambridge, and randomised into one of three groups: one group received only letrozole; one group received letrozole alongside 40mg of megestrol daily; and the third group received letrozole plus a much higher daily dose of megestrol, 160mg. In this ‘window of opportunity’ trial, treatment was given for two weeks prior to surgery to remove the tumour. The percentage of actively growing tumour cells was assessed at the start of the trial and then again before surgery.
In findings published today in Nature Cancer, the team showed that adding megestrol boosted the ability of letrozole to block tumour growth, with comparable effects at both the 40mg and 160mg doses.
Joint first author Dr Rebecca Burrell from the Cancer Research UK Cambridge Institute and CUH said: “In the two-week window that we looked at, adding a progestin made the anti-oestrogen treatment more effective at slowing tumour growth. What was particularly pleasing to see was that even the lower dose had the desired effect.
“Although the higher dose of progesterone is licenced as an anti-cancer treatment, over the long term it can have side effects including weight gain and high blood pressure. But just a quarter of the dose was as effective, and this would come with fewer side effects. We know from previous trials that a low dose of progesterone is effective at treating hot flushes for patients on anti-oestrogen therapy. This could reduce the likelihood of patients stopping their medication, and so help improve breast cancer outcomes. Megestrol – the drug we used – is off-patent, making it a cost-effective option.”
Because women in the trial were only given megestrol for a short period of time, follow-up studies will be needed to confirm whether the drug would have the same beneficial effects with reduced side-effects over a longer period of time.
The research was funded by Anticancer Fund, with additional support from Cancer Research UK, Addenbrooke’s Charitable Trust and the National Institute for Health and Care Research Cambridge Biomedical Research Centre.
Personalised and precise cancer treatments underpin the focus of care at the future Cambridge Cancer Research Hospital. The specialist facility planned for the Cambridge Biomedical Campus will bring together world-leading researchers from the University of Cambridge and its Cancer Research UK Cambridge Centre and clinical excellence from Addenbrooke’s Hospital under one roof in a brand-new NHS hospital.
Cipla Limited (BSE: 500087; NSE: CIPLA; and hereafter referred to as “Cipla”), through its subsidiary Medpro Pharmaceutica, has entered into an exclusive license and supply agreement with Immunoadoptive Cell Therapy Private Limited (ImmunoACT). Under this partnership, Cipla will commercialise talicabtagene autoleucel, India’s first indigenously developed CAR-T cell therapy, in the Republic of South Africa, Algeria, and Morocco.
Talicabtagene autoleucel (the product) is an autologous (of a patient’s own blood sample) anti-CD19 CAR-T indicated for the treatment of patients with relapsed or refractory B-cell Non-Hodgkin’s Lymphoma (B-NHL) and B-cell Acute Lymphoblastic Leukaemia (B-ALL) who have failed standard lines of therapy. Administered to over 500 patients in India, the therapy has demonstrated high efficacy, durable responses, and a well‑tolerated safety profile, leading to reduced ancillary healthcare costs.
As part of this collaboration, ImmunoACT will manufacture the product and Cipla will commercialise in the licensed African territories, thereby expanding access of this revolutionary new treatment to markets currently with unmet needs.
Commenting on the partnership, Achin Gupta, Managing Director and Global CEO Designate, Cipla Limited, said, “Our collaboration with ImmunoACT reinforces Cipla’s vision of leveraging cutting-edge science to deliver transformative and affordable treatments, especially for patients with critical healthcare needs. By introducing CAR-T therapy in Africa, we aim to bring world-class innovation closer to patients and strengthen our commitment to accessible healthcare in the region.”
Adding on, Paul Miller, Chief Executive Officer of Cipla Africa, said, “We are proud to be at the forefront of efforts to bring CAR-T cell therapy to Africa. This collaboration not only advances our oncology portfolio but also reinforces Cipla’s mission of making next-generation therapies accessible to patients worldwide.”
Dr. Rahul Purwar, ImmunoACT’s Founder & Chairmanand a professor of the Indian Institute of Technology (IIT), Bombay, said,“Our mission has always been to innovate and make cell & gene therapies accessible and affordable, addressing the significant unmet medical needs across the globe. This strategic partnership with Cipla seeks to accelerate our endeavours; ensuring that patients with B-cell cancers have a fighting chance at a durable remission, with our CAR-T platform.”
About CAR-T cell therapy:
CAR T-cell therapy is a groundbreaking form of immunotherapy that uses a patient’s own immune cells to fight the disease. Doctors collect immune cells (T cells) from the patient, reprogram them to identify and destroy cancer cells, and then return them to the body, enabling a targeted and personalized approach to treatment.
About Cipla
Established in 1935, Cipla is a global pharmaceutical company focused on agile and sustainable growth, complex generics, and deepening portfolio in our home markets of India, South Africa, North America, and key regulated and emerging markets. Our strengths in the respiratory, antiretroviral, urology, cardiology, anti-infective and CNS segments are well-known. Our 46 manufacturing sites around the world produce 50+ dosage forms and 1500+ products using cutting-edge technology platforms to cater to our 80+ markets. Cipla is ranked 3rd largest in pharma in India (IQVIA MAT Sep’25), 2nd Largest in the pharma prescription market in South Africa (IQVIA MAT Aug’25), and 4th largest by prescription in the US Gx (Repulses + MDI) products (IQVIA MAT Aug’25). For over nine decades, making a difference to patients has inspired every aspect of Cipla’s work. Our paradigm-changing offer of a triple anti-retroviral therapy in HIV/AIDS at less than a dollar a day in Africa in 2001 is widely acknowledged as having contributed to bringing inclusiveness, accessibility and affordability to the centre of the HIV movement. A responsible corporate citizen, Cipla’s humanitarian approach to healthcare in pursuit of its purpose of ‘Caring for Life’ and deep-rooted community links wherever it is present make it a partner of choice to global health bodies, peers and all stakeholders. For more, please visit www.cipla.com, or click on Twitter, Facebook, LinkedIn.
About ImmunoACT
As pioneers of India’s first fully integrated CAR-T cell therapy platform, ImmunoACT (Immunoadoptive Cell Therapy Private Limited), develops and manufactures accessible, affordable cutting-edge gene-modified cell therapies for blood cancers and solid tumours. With NexCAR19™, India’s first CAR_T cell therapy (developed in collaboration with the Indian Institute of Technology, Bombay and Tata Memorial Centre) commercially approved in India having unprecedentedly transformed the treatment landscape in refractory/relapsed B-cell malignancies, ImmunoACT also has a robust pipeline including a clinical-stage BCMA-directed CAR-T for multiple myeloma, and solid tumour CAR-Ts under development. The company is accelerating its mission to expand global access to life-saving cell and gene therapies through strategic partnerships.
Researchers from University of Maryland Schools of Public Health and Engineering in College Park and the School of Medicine in Baltimore wanted to find out how the flu spreads, so they put college students already sick with the flu into a hotel room with healthy middle-aged adult volunteers. The result? No one caught the flu.
“At this time of year, it seems like everyone is catching the flu virus. And yet our study showed no transmission – what does this say about how flu spreads and how to stop outbreaks?” said Dr Donald Milton, professor at SPH’s Department of Global, Environmental and Occupational Health and a global infectious disease aerobiology expert who was among the first to identify how to stop the spread of COVID-19.
The study, out in PLOS Pathogens, is the first clinical trial in a controlled environment to investigate exactly how the flu spreads through the air between naturally infected people (rather than people deliberately infected in a lab) and uninfected people. Milton and his colleague Dr Jianyu Lai have some ideas about why none of the healthy volunteers contracted the flu.
“Our data suggests key things that increase the likelihood of flu transmission – coughing is a major one,” said Lai, post-doctoral research scientist, who led data analysis and report writing for the team.
The students with the flu had a lot of virus in their noses, says Lai, but they did not cough much at all, so only small amounts of virus got expelled into the air.
“The other important factor is ventilation and air movement. The air in our study room was continually mixed rapidly by a heater and dehumidifier and so the small amounts of virus in the air were diluted,” Lai said.
Lai adds that middle-aged adults are usually less susceptible to influenza than younger adults, another likely factor in the lack of any flu cases.
Most researchers think airborne transmission is a major factor in the spread of this common disease. But Milton notes that updating international infection-control guidelines requires evidence from randomised clinical trials such as this one. The team’s ongoing research aims to show the extent of flu transmission by airborne inhalation and exactly how that airborne transmission happens.
The lack of transmission in this study offers important clues to how we can protect ourselves from the flu this year.
“Being up close, face-to-face with other people indoors where the air isn’t moving much seems to be the most risky thing – and it’s something we all tend to do a lot. Our results suggest that portable air purifiers that stir up the air as well as clean it could be a big help. But if you are really close and someone is coughing, the best way to stay safe is to wear a mask, especially the N95,” said Milton.
The team used a quarantined floor of a Baltimore-area hotel to measure airborne transmission between five people with confirmed influenza virus with symptoms and a group of 11 healthy volunteers across two cohorts in 2023 and 2024. A similar quarantine set-up was used in an earlier study and exhaled breath testing was used in several pioneering studies by Milton and colleagues on influenza transmission.
During the most recent flu study, participants lived for two weeks on an isolated floor of the hotel, and did daily activities simulating different ways that people gather and interact – including conversational ice-breakers, physical activities like yoga, stretching or dancing. Infected people handled objects such as a pen, tablet computer and a microphone, before passing the objects among the whole group.
Researchers measured a wide range of parameters throughout the experiment, including participant symptom monitoring, daily nasal swabs and saliva samples and blood collection to test for antibodies. The study measured the viral exposure in volunteers’ breathing area as well as the ambient air of the activity room. Participant exhaled breath was also measured daily in the Gesundheit II machine, invented by Milton and colleagues at Harvard T.H. Chan School of Public Health.
Finding ways to control flu outbreaks is a public health priority, says Milton. Flu is responsible for a considerable burden of disease in the United States and globally – up to 1 billion people across the planet catch seasonal influenza every year and this season has seen at least 7.5 million flu cases so far in the United States alone, including 81 000 hospitalisations and over 3000 deaths.
