Latest news with #osteoarthritis
Yahoo
25-05-2025
- Health
- Yahoo
Warrington woman's life turned around thanks to robotic knee replacement
A WARRINGTON woman's life has been turned around thanks to robotic knee replacement surgery. Amanda Burns, 56, suffered from severe osteoarthritis pain in both knees. This was significantly impacting her life and her mobility had become so reduced that she had no option but to seek advice. Amanda said: 'Even everyday activities became difficult. "I struggled to drive any distance and found it challenging to do much beyond managing my full-time office-based job. 'The constant discomfort and limitation on movement affected not just my physical wellbeing, but also my overall lifestyle and independence.' Desperate to find relief and a chance to reclaim her independence, Amanda sought out a specialist who could offer a solution to her ongoing pain. She chose Spire Cheshire Hospital's Consultant Orthopaedic Surgeon, Mr Gareth Stables, who advised she would need a total knee replacement. After discussing all options with Mr Stables, Amanda had decided on the ROSA robotic knee replacement procedure. She said: 'It mattered that I could fully trust the surgeon performing such a major procedure. 'Mr Stables explained the potential benefits, including enhanced precision and alignment through the technology working alongside the surgeon, as well as the possibility of smaller incisions. "These advantages made the robotic approach feel like the right decision for me.' In February, 2025 Amanda was admitted for four days in the inpatient ward and underwent a total knee replacement procedure. She praised the 'professional, reassuring and kind' team there and said this level of care continued following surgery. 'The nursing staff were brilliant in managing my pain medication and ensuring my overall wellbeing, always checking in and ready to help with anything I needed," Amanda said. "The kind ladies who cleaned my room and brought me cups of tea were always friendly and took the time for a welcome chat, which really helped keep spirits high. 'The physiotherapist was also incredibly supportive in helping me to mobilise and build confidence step by step. "A mention also must go to the aftercare physio Matthew Gouldthorpe, who was pivotal in my progression. "A special mention goes to the lovely pharmacist, Danielle, who was so organised and kind while guiding me through the pain management plan for when I went home. "And I can't leave out the amazing catering team—the food was of exceptional quality, with a fantastic range of choices. "Overall, the entire team showed so much care, professionalism, and positivity — it made such a difference to my recovery experience and I'm incredibly grateful to them all.' Following the surgery, an 'incredible' difference has been made to Amanda's daily life. 'I'm currently eight weeks post-operation, and one of the biggest changes is that I'm sleeping really well now— something that had been difficult before," Amanda explained. "Interestingly, I'm now experiencing more discomfort from my other knee, which still has osteoarthritis, compared to the operated one, especially during the night. It really highlights just how successful the surgery has been.' When asked about the difference the surgery has made, Amanda said: 'The most significant benefit so far is that the awful osteoarthritis pain that I lived with for so long is now completely gone in the operated knee. It's incredible to feel that difference, especially when I compare it to the ongoing pain in my other knee. 'My knee feels stronger than before, and the wound has healed really well. 'The progress has given me the confidence to start looking ahead again — I've even planned a holiday later in the summer. "My family have commented that I seem more like my old self, which means a lot. "My experience has given me the confidence in having my other knee replaced. Once I've had this done, I'm really looking forward to putting the pain and mobility limitations behind me and getting back to enjoying life without those constant restrictions."
The National
19-05-2025
- Health
- The National
Osteoarthritis pain could be treated with camel placenta, scientists say
Camel placenta could be the latest biomaterial used in medicine to regenerate damaged tissue, as scientists in Saudi Arabia explore how effective it may be in treating osteoarthritis. Cartilage is a critical component in healthy joints but damage caused by old age, obesity and injury can result in painful, life-limiting conditions. Most existing therapies focus on management of symptoms and pain. But scientists hope new research into the use of camel placenta extract in animals with osteoarthritis − the most common chronic joint condition in Saudi Arabia − could help regenerate damaged human tissue. Regenerative therapies using stem cells or platelet-rich plasma extracted from human fat are currently used to treat chronic patients, but this can cost up to Dh30,000 ($8,160). The process involves removing stomach fat through liposuction, spinning the material to extract stem cells, then injecting it back into the patient's damaged joint. Using, camel placenta − material discarded after birth as waste but loaded with essential amino acids − could be a more affordable alternative, scientists believe. 'Camels are highly valued in our culture here in the kingdom, and that deep interest helped spark a new idea for us,' Nouf bin Awad, a health policy specialist and pharmacist at the Saudi Food and Drug Authority, who led the research at King Saud University, told The National. 'The benefits of camel milk have been researched, but the placenta was a completely different idea. It offered a great alternative to human placenta, which often comes with ethical concerns when used in medical or cosmetic products. Potentially, this is a good option for those patients who can't go for surgery, which can be a costly approach.' A recent cross-sectional study of adults over 50 in the country found between 30 and 40 per cent were affected by knee arthritis, significantly more than elsewhere. The World Health Organisation estimates that worldwide, around 10 per cent of men and 18 per cent of women over 60 have the condition. According to the kingdom's Ministry of Environment, Water, and Agriculture, there are 1.8 million camels in the country − providing plenty scope to utilise natural biowaste for research and potential medical treatments. Researchers found an injectable treatment using camel placenta reduced inflammation and swelling in rats, while promoting cartilage regeneration. It was also seen to suppress enzymes that break down joint tissue and had a similar effect on pain management as diclofenac − a commonly prescribed arthritis medication − but without the side effects of gastrointestinal problems. The treatment was presented at the Geneva International Exhibition of Inventions 2025 in April, and was awarded a gold innovation award. 'At this early stage, the initial cost is low as the camel placenta is the primary component, and a natural by-product that is disposed with after the camel gives birth,' said Ms Awad. 'This gives us access to a natural rich resource that in the future we can manage and study further to develop.' Clinical trials with humans are now planned in partnership with the Ministry of Health in Saudi Arabia, to further develop the novel treatment as a commercially available injectable. Dr Hesham Al Khateeb, an orthopaedic surgeon at Fakeeh University Hospital in Dubai, said the research had potential for wide use. 'Placentas have a lot of growth factors and stem cells, while from an abundance perspective you've got a lot of camels in this region, so this should be explored,' he said. 'Stem cells are used a lot in arthritis, but usually we take the stem cells from the individual, then inject them. Current treatments for arthritis vary, it's an extensive field. You can start very basic with cortisone injections, which is the gold standard. But it's not a one size fits all category, as it depends on the degree of arthritis. If it's early stages, this is where this regenerative, biological treatment can be very effective.' The cartilage regeneration market is a significant growth area of medical research, and investment. Globally, the market accounted for $1.16 billion in 2023 and is forecast to be worth $5,62 billion by 2034, analysts at Fortune Business Insights predict. Rising rates of joint disorders and sporting injuries, as well as more people living longer into old age make it an area of healthcare primed for growth. 'Osteoarthritis is basically wear and tear of joint cartilage,' said Dr Amrut Borade, an orthopaedic surgeon at International Modern Hospital, Dubai. 'The most common factor is old age and other contributing factors like obesity and muscle inactivity. The first line of treatment is lifestyle modification and weight reduction.' While some patients in early stages of osteoarthritis may benefit from supplements, such as fish oils, calcium, glucosamine and chondroitin, doctors can also inject damaged areas with a cortisone steroid or hyaluronic acid to lubricate the joint. 'It's actually quite difficult to regenerate completely damaged cartilage, but we want to focus in early stages of osteoarthritis to prevent the progression and protect the cartilage,' said Dr Borade. 'Placenta treatments are clearly in experimental stages but do have some promise. There still needs a lot of research, especially when being used in the human body.' The Ministry of Health in Saudi Arabia is exploring the potential solution to directly address the need for accessible, regenerative osteoarthritis therapies. 'We have these promising results and we hope to see this treatment in reality and used by the Ministry of Health and also, by the world,' said Dr Meznah Alturaiki, project and international relations manager at the innovation centre, at the Saudi Ministry of Health.
Medscape
16-05-2025
- Health
- Medscape
Trial Tests Carbon Fibre Inserts for Osteoarthritis Relief
BIRMINGHAM — A commonly used method by podiatrists and physiotherapists to manage osteoarthritis (OA) of the first metatarsophalangeal joint (MTPJ) is undergoing formal testing in a randomised clinical trial, a UK researcher said on the first day of The Primary Care Show 2025. Michael Backhouse The Big Toe OstEoarthritis (BigTOE) Inserts Trial compares the use of a carbon fibre insert to stiffen shoes against a sham insole. The goal is to determine if this approach can alleviate the pain associated with toe OA, said the trial's chief investigator Michael Backhouse. Backhouse, who is a podiatrist and associate professor of clinical trials at Warwick University told Medscape News UK, 'The truth is we don't know if this approach works, which is why we are doing the trial'. Lack of Evidence Although carbon fibre inserts have been available and used within the NHS for years, there is very little evidence to show that these, or indeed any other insoles, actually do anything to help people with foot OA, according to Backhouse. As such, the National Institute for Health and Care Excellence (NICE) guidance on diagnosis and management of OA does not currently recommend their routine use. A survey of UK and Australian podiatrists and physiotherapists found over 50 different treatment approaches were being used to manage OA of the first MTPJ, which 'reflects the lack of evidence', Backhouse said. Helen Branthwaite, the newly appointed chief clinical advisor for the Royal College Podiatry, told Medscape News UK : 'What's really unique about this trial is that they are going to have a multicentred population that will be randomised, so you don't know which treatment you're getting, which would address the psychological impact of the intervention.' Branthwaite added: 'Osteoarthritis of the first MP joint, or any joint, is progressive', and the amount of pain experienced, or how long that lasts, depends on where a patient sits on a scale. 'When the joint completely fuses, you'll have no pain at all. Clinically, we know that because we see that when we see patients and change the stiffness of their shoe', added Branthwaite, who also works in private musculoskeletal podiatry practice in Macclesfield. Carbon fibre insoles are used to alter impact forces and improve joint mechanics, Branthwaite explained, highlighting that it was not just the quantity but the quality of data that was lacking on their use. The BigTOE Inserts Trial The BigTOE Inserts Trial aims to provide quality evidence to support podiatrists' individualised approaches. The target is to recruit 438 participants who have been screened at 25-30 general practice sites. Eligible participants must have a diagnosis of OA in the first MTPJ in one or both feet, experience activity-related pain in that joint that has been ongoing for at least 3 months, and be aged 18 years or older. Pain will be assessed on a numerical rating scale (NRS), where 0 is no pain and 10 indicates the worst pain. Participants have an activity-related pain NRS score of four or higher for inclusion. A comprehensive list of exclusion criteria has been put in place to ensure that patients with other musculoskeletal or inflammatory causes for their activity-related MTPJ pain are not included. This includes those experiencing pain predominantly on the plantar aspect of the joint, suggestive of sesamoid pathology; morning stiffness lasting for 30 minutes or more, or a clinical suspicion or previous diagnosis of inflammatory arthritis, connective tissue disease, septic arthritis, or crystal arthropathy. Also excluded are people with a history of surgery or clinically important trauma to the foot and ankle, or planned foot and ankle surgery within the next 12 months. Anyone who has had a corticosteroid injection in the affected foot within the last 3 months will be excluded, as will anyone who is identified as having moderate or severe grade 3 or 4 hallux valgus. Finally, anyone who has used an insole prescribed by a healthcare professional in the last 3 months cannot be recruited. Pragmatic Approach Backhouse described the trial design as 'pragmatic'. 'It reflects the patients you see in the clinic', he said. As such, all participants will be given usual care, which involves tailored therapeutic exercise, advice on weight management, and provision of patient information and support as per NICE guidance. Additionally, they will be randomly allocated to receive either a carbon fibre insert or a sham insole. 'We're working with one supplier; the sham and the active insert appear the same,' Backhouse said. To ensure that patients do not know which device they are using, the carbon fibre component has been sandwiched between other layers of the insole. 'We don't tell them that we're looking at carbon fibre inserts, and we don't tell them that we're looking at stiff inserts – we tell you we're looking at inserts, maybe different materials,' said Backhouse. The primary outcome will be the change from baseline in the Manchester Oxford Foot Questionnaire walking/standing subscale at 3 months, with additional assessments at 1, 6, and 12 months. This means that after the baseline assessment, no further clinic visits are required, and all questionnaires will be organised by the University of Warwick team. 'OA is common and disabling,' Backhouse noted. While generally diagnosed clinically without imaging, when imaging is used it suggests that around 17% of people over 50 years of age have foot OA. Moreover, three quarters of people with symptomatic radiographic OA reported disabling foot symptoms, 'that impacted on their daily lives'. With 'very limited evidence for treating OA', the BigTOE Inserts Trial was one step towards changing that, Backhouse said. The BigTOE trial is funded by the National Institute for Health Care and Research. Backhouse and Branthwaite reported no relevant financial relationships.
Telegraph
13-05-2025
- Health
- Telegraph
The best treatments for arthritic hips if you want to avoid a hip replacement
Arthritic hips are no walk in the park. The first signs of trouble can creep up subtly with morning pain and stiffness. Before you know it, even bending down to put on your socks is a monumental pain. You may even notice a popping sound, a clicking or crunching when moving a troublesome hip. Or gradually realise that you've developed a change in your gait and are limping. Studies suggest that 10-25 per cent of adults experience hip pain at some point in their lives, with higher rates among older individuals. Estimates suggest that osteoarthritis (OA), one of the most common hip disorders, affects about 10-15 per cent of adults aged over 60 years worldwide. In the UK alone, approximately 1.4 million people are estimated to have hip OA. Osteoarthritis involves the wear-and-tear degeneration of cartilage, leading to pain and stiffness, whereas rheumatoid arthritis is an autoimmune condition causing joint inflammation and can affect younger people. Around 7 per cent of men and 11 per cent of women will eventually need surgery. But thankfully, if your symptoms aren't sufficiently controlled by anti-inflammatories, gels and supplements such as turmeric or cod liver oil, there are many treatments that can alleviate pain without resorting to a full-blown hip replacement. Non-invasive treatments Red light, laser therapy or a combination of both £150-£500 Also known as photo-biomodulation, this uses specific wavelengths of light to stimulate the body's natural healing processes, potentially reducing pain and inflammation, and promoting tissue regeneration. Red light therapy uses a broad range of light to cover larger areas, while laser therapy focusses a narrow beam of light with a single wavelength for more targeted treatment. Kineon is one such company selling devices, their Move+ Pro's red light and laser therapy. Kineon report positive outcomes with 60-80 per cent of patients experiencing less pain and improved function. Risks are minimal, including mild skin irritation, redness, or temporary discomfort at the treatment site. Worth trying? Rarely available on the NHS, devices cost between £150-£500. Having tried the Kineon MOVE+ Pro it was effective in expediting recovery time following sporting activity. Shockwave therapy £100-£250 Shockwave therapy ses sound waves administered with a type of gun to stimulate healing in damaged tissues. As the name implies, it's uncomfortable but not excruciating and can reduce inflammation and pain in joints affected by arthritis, particularly osteoarthritis. The therapy has demonstrated success rates ranging from 60-87 per cent for hip pain conditions. Worth trying? GPs can refer you for this on the NHS when chronic symptoms persist for at least three to six months. Privately, shockwave therapy sessions for arthritis can cost between £100 to £250. Injectable treatments Corticosteroid (steroids) £120-£200 Anti-inflammatory medicine is injected directly into the hip joint to reduce pain and inflammation. Studies indicate approximately 50 per cent of patients experience immediate pain relief lasting up to two weeks, while relief extends beyond two weeks for 30 per cent. However, about 20 per cent may not respond at all while after three months any beneficial effect is likely to decline. The least expensive injectable, privately costing £120-£200 per injection, its side effects include potential bruising and facial flushes, but are generally minimal. Injections are widely available but expect waiting times of four months and upwards if opting for the NHS route. Dale Naylor of DJN Physios states: 'I see the efficacy of steroids when combined with rehab to alleviate pressure across the hips, by suppressing inflammation and relieving pain.' Steroids represent a good option for patients with moderate to severe osteoarthritis, bursitis or labral tears. If the hip joint is infected, steroids are considered dangerous and not advised (also to be avoided in prosthetic joints due to infection risk); steroids can also spike blood glucose levels significantly, so can be problematic for diabetics. Worth trying? It has minimal side effects and cheap when obtained privately, steroids can relieve pain for up to several months. Injections should be spread out every few months and used no more than three to four times per year, according to the Mayo Clinic. Hyaluronic acid £250-£500 A gel-like substance also used on skin, hyaluronic acid acts as a shock absorber when injected into the hip joint providing lubrication and cushioning, aiming to reduce pain and improve mobility. Only available privately, costing between £250-£500 per hip and quickly administered, 60-70 per cent of patients experience improvement that may last several months. Mr Rama Mohan, a UK-based hip and knee surgeon says: 'Hyaluronic acid is a naturally occurring gel-like substance that lubricates your hip joint. As the joint wears away, the arthritic hip joints lose this lubricant. You can think of the injection like WD-40 for your joints.' Generally, patients receive a series of injections (usually two to five) over a few weeks. Severe reactions are rare, though temporary pain or swelling may occur. In the UK the NHS doesn't offer this treatment, mainly because of cost-effectiveness considerations and limited evidence of long-term benefits. Worth trying? More commonly used in knees but more beneficial than steroid injections alone with the added benefit of a cushioning effect on the affected hip. Affordable and helpful for those wishing to get back to sporting activities. Prolozone therapy £200 to £500 This is a regenerative therapy which involves injecting a concentrated form of oxygen into joints to stimulate the body's healing process. It is often used to treat hip labral tears and other hip conditions. Pain levels for treatment are minimal and – having recently had the treatment carried out on my own hips at the ProHealth Clinic, one of the UK's leading Prolotherapy & Prolozone Therapy clinics – I can attest to a reduction in pain and soreness in less than a week. 'One of the benefits is it's such a thin substance so spreads easily to surrounding areas,' says Mr Eaton. Costs per session vary depending on the clinic and specific treatment protocol, typically ranging from £200 to £500. After a couple of sets of injections over the course of a month, for arthritis Mr Eaton recommends waiting 12 months for a single top-up, while for anything soft tissue-related the treatment should last longer as the patient's blood supply takes over. Worth trying? Prolozone achieves results in a faster timeframe than many options. It's not available on the NHS. Platelet-rich plasma (PRP) therapy £250-£600 This treatment uses the patient's own blood (platelets) to reduce inflammation and promote tissue regeneration. As well as managing pain, it can make moving easier. It's best suited to those with early or moderate hip osteoarthritis in otherwise good health, who are looking to delay surgery. Dr David Porter, a sports medicine physician, says: 'PRP is administered with the aim of reducing pain, restoring the function of the hips, and preventing further cartilage damage.' Recent studies show over 70 per cent of patients experience positive outcomes, particularly in the short to medium term (up to 12 months), though two to three injections of PRP may maximise benefits for patients. PRP involves a viscous substance which might not spread as well as newer treatments like Prolozone therapy. A 2021 analysis found PRP was more effective than hyaluronic acid, especially in early to moderate stages. Available at certain NHS Trusts with a wait time or privately cost between £250-£600 in the UK. Worth trying? Beneficial for promoting healing and reducing pain if other methods haven't worked for those under 70, and available on the NHS. While private treatment is affordable, often multiple injections are required. PRP is generally not recommended for older patients with advanced joint degeneration, those with severe cartilage loss, active infection, cancer or severe diabetes. Stem cell therapy £4,000-£7,000 Stem cells are extracted from bone marrow or fat tissue then processed in a laboratory and injected into the afflicted joint. While regarded as something of an alternative therapy in the UK and not covered by the NHS, the practice is gaining in popularity. Jorja Healthcare, a UK-based regenerative medicine clinic, states: 'Between 65 per cent and 75 per cent of all patients treated have had a positive outcome with improvements in both pain scores as well as functional scores.' Beyond the budgets of many, and not covered widely by insurance, costs range from £4,000 to £7,000 per hip treated although cheaper clinics can be found abroad in India, and Mexico. Worth trying? Involves significant cost beyond the means of many, from £4,000-£7,000. While anecdotal evidence is promising, controlled trials are needed and patients should conduct extensive research on the clinic's results. Five alternatives to full hip replacement surgery Beyond injectable solutions, more radical forms of minimally invasive surgeries could present the next logical step in improving symptoms and hip mobility while avoiding a total replacement. Hip resurfacing £8,000-£15,000 This is a surgical procedure that involves removing the damaged surface of the femoral head (the ball part of the hip joint) and capping it with a metal prosthesis instead of replacing the entire hip joint. Resurfacing preserves more of the natural bone and full recovery can be achieved in just three months. This is available privately or offered on the NHS for suitable candidates, particularly younger, active patients or privately between £8,000 to £15,000. Success rates are roughly 90-95 per cent up to five years and still 85-90 per cent after a decade. Worth trying? It's generally available on the NHS but often with a lengthy waiting time. Success rates are excellent. Arthroscopy £5,000-£10,000 A form of 'keyhole' surgery that allows surgeons to address problems within a joint. Studies report largely positive outcomes and success rates while, but costs are significant, between £5,000 to £10,000. Risks are minor but include infection, nerve injury or even reoperation. Mr Giles Stafford, a consultant orthopaedic hip surgeon in London, says: 'One year after surgery, approximately 80 per cent of patients are satisfied that the procedure has improved their symptoms. Unfortunately, 15 per cent may feel no change in their condition and 5 per cent feel that their symptoms have in fact become worse.' Patients need to take care with physio and rehabilitation afterwards. Worth trying? It's good for certain conditions and available privately or on the NHS (expect a lengthy waiting time), risks are minor while success rates are strong. However, patients over 40 years old or those with significant OA may experience less favourable results. Autologous chondrocyte implantation (ACI) £10,000-£20,000 This uses healthy cartilage from the patient's own body by harvesting cartilage cells, culturing them, and implanting them back into the damaged area to promote new cartilage growth. ACI has reported success rates of around 70-80 per cent at five years, with many patients experiencing significant pain relief and improved joint function, while after a decade studies suggest that about 50-60 per cent of patients maintain good results. The British Editorial Society of Bone & Joint Surgery suggests ACI could be a viable option for the treatment of cartilage defects in the hip. ACI is available on the NHS for treating knee cartilage defects, but is not typically used for hip joints, while private costs range from £10,000 to £20,000, depending on procedure specifics. The ideal candidate should be less than 50 years old and not suffering from advanced osteoarthritis. Worth trying? Particularly effective in younger individuals with early-stage cartilage defects but costs can be prohibitive for most. Generally safe, risks include infection, graft failure, and complications related to the surgery. Osteochondral grafting £8,000-£16,000 Autograft transplantation involves harvesting healthy cartilage and bone from a non-weight-bearing area of the patient's own joint and transplanting it to the damaged area of the hip. Typically, it's considered particularly for younger patients where conservative treatment options haven't worked. Alternatively, allograft transplantation uses donated cartilage tissue to reconstruct large joint cartilage lesions in the hip joint. Both can improve joint function and reduce pain, while delaying or even preventing the need for a hip replacement and can be performed on arthritic hips, particularly when there aren't widespread degenerative changes. Limitations exist with the amount of available tissue when taken from your own body, making this approach more suitable for smaller defects. Dr Benjamin Domb, an orthopaedic surgeon specialising in hip preservation believes, 'appropriate patient selection is crucial. Factors such as defect size, patient age, activity level, and the presence of osteoarthritis significantly influence the procedure's effectiveness'. Availability on the NHS is based on an evaluation of the patient's age, activity level, size and location of the cartilage defect, and overall joint health. Privately, autograft and allograft costs in the region of £8,000 to £16,000. Success rates of approximately 70-90 per cent at five years, diminishing at 10 years. Worth trying? Particularly effective in younger individuals with early-stage cartilage defects but costs can be prohibitive if unavailable on the NHS. Ideal for patients with hip fractures and no arthritis in the socket. Partial hip replacement £8,000-£13,000 This surgical option replaces the ball of the hip joint (the top of the thigh bones) while leaving the socket intact. Most commonly used for patients who have a hip fracture, limited mobility or aren't good candidates for full hip replacement, the damaged femoral head is removed and replaced with a prosthetic implant. A partial hip replacement is often used in patients with osteoporosis who have fractured a hip. While short to mid-term outcomes are good (implant survival rates are 85 per cent at five years, long-term success is variable and may not last as long as total hip replacements in more active patients. Treatment is available on the NHS especially for elderly patients or emergency treatment following hip fractures, while private costs range from £8,000-£13,000. Worth trying? Ideal for patients with hip fractures and no arthritis in the socket; minor risks include infection, blood clots, implant loosening, or leg length difference. The hospital stay is usually two to five days depending on recovery and patient health.
Associated Press
12-05-2025
- Business
- Associated Press
Genascence Phase 1b DONATELLO Trial Evaluating Potential First-in-Class Gene Therapy for Knee Osteoarthritis (OA) Meets Primary Endpoint Showing GNSC-001 Was Safe and Well Tolerated Across Multiple Dosing Arms
PALO ALTO, Calif.--(BUSINESS WIRE)--May 12, 2025-- Genascence Corporation ('Genascence'), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced positive 12-month safety and biomarker results from the Phase 1b DONATELLO clinical trial evaluating GNSC-001, a potential first-in-class gene therapy blocking interleukin 1 (IL-1) for the treatment of knee osteoarthritis (OA). Results from the 12-month analysis showed the study met the primary endpoint, demonstrating continued safety and tolerability across all doses tested, as well as the key secondary endpoint showing sustained IL-1Ra expression in synovial fluid, building on data reported through the six-month visit. GNSC-001 is a genetic medicine – a recombinant adeno-associated viral vector expressing an optimized human interleukin-1 receptor antagonist (IL-1Ra), a naturally occurring protein that blocks IL-1 signaling. IL-1 is considered one of the key mediators involved in the pathogenesis of OA, causing inflammation, joint pain, and cartilage destruction. GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single intra-articular injection into the affected joint. The U.S. Food and Drug Administration (FDA) granted GNSC-001 Fast Track designation in the fourth quarter of 2024. Genascence recently completed a successful meeting with the FDA on the design of the Phase 2b/3 clinical trial of GNSC-001 focused on clinical efficacy and plans to initiate the Phase 2b/3 study in 2026. 'Osteoarthritis is incapacitating, causing years of pain and disability for people living with the disease. Current treatment options are limited to managing pain and do not treat the underlying disease itself,' said Thomas Chalberg, Ph.D., founder and CEO of Genascence. 'The 12-month safety and sustained IL-1Ra expression data affirms the promise of GNSC-001 to potentially transform the treatment paradigm for OA. We are pleased by the successful meeting with the FDA, and look forward to initiating the study, transitioning GNSC-001 to late-stage clinical development so we can bring a new treatment option to people suffering from this disabling disease.' 'GNSC-001 is the first IL-1 inhibitor that has been shown to generate IL-1Ra expression levels that reach and maintain therapeutic thresholds long-term following a single administration,' said Annahita Keravala, Ph.D., chief scientific officer (CSO) of Genascence. 'Results from the DONATELLO clinical trial suggest that our novel therapeutic approach, a local gene therapy can potentially have therapeutic benefit in knee OA, a disease for which there are no treatments beyond management of symptoms. This would be transformative for people suffering from this debilitating condition, and thus warrants further development.' 'These results from the DONATELLO trial reflect the kind of innovation CIRM was created to support,' said Lisa Kadyk, Ph.D., CIRM Fellow, Clinical Development at the California Institute for Regenerative Medicine (CIRM), which supported the DONATELLO clinical trial with a $12 million award. 'By harnessing the power of gene therapy, GNSC-001 represents a novel and potentially disease-modifying approach to treating osteoarthritis. We are encouraged by the 12-month safety and biomarker data and proud to have supported this important step toward a more effective, long-term treatment for people living with knee OA.' Dr. Keravala will present data from the six-month interim analysis of the DONATELLO clinical trial at the 28 th Annual American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting, being held May 13-17, 2025 in New Orleans, LA and virtually. The poster presentation details are provided below. Title: A Randomized, Double-Blind, Placebo-Controlled Dose-Ranging Phase 1b Study Evaluating Safety, Tolerability, and Pharmacodynamics of a Local AAV-Mediated Anti-Interluekin-1 Gene Therapy in Subjects with Knee Osteoarthritis: 6-Month Interim Results Date/Time: Thursday, May 15, 2025, 5:30-7:00 pm CT Location: Poster Hall Abstract Number: AMA616 Poster Number: 1849 Abstracts can be found at About the DONATELLO Clinical Trial The DONATELLO Phase 1b clinical trial (NCT05835895) is a double-blind, placebo-controlled dose-ranging study designed to evaluate the safety, tolerability, and pharmacodynamics of a single intra-articular injection of GNSC-001 in patients with OA of the knee. The study enrolled 67 participants with OA at 10 centers across the U.S. The first five groups were randomized to receive GNSC-001 at doses of 110 12 vg or 110 13 vg, with or without a short course of oral steroids for immune-conditioning, or a placebo (saline) injection. The trial was expanded to enroll an additional, non-randomized arm. In this arm, pre-treatment synovial fluid sampling was required for entry, and subjects received 110 13 vg GNSC-001 with an abbreviated three-day course of oral steroids plus a local, intra-articular steroid injection. Data and Safety Summary The primary endpoints of the DONATELLO clinical trial are safety and tolerability. Through 12 months of follow-up, data show that GNSC-001 was well tolerated, with no treatment-emergent or treatment-related deaths, serious adverse events (SAEs), or adverse event (AE)-related withdrawals reported. The most common target knee AEs included arthralgia, joint swelling, and joint effusion. The study's secondary endpoints include expression levels of interleukin-1 receptor antagonist (IL-1Ra) in synovial fluid at Month 12, as well as change from baseline to Month 12. Results revealed that mean expression of IL-1Ra reached target therapeutic levels in multiple arms of the study and remained above the target threshold throughout the 12-month follow up period. Immune-conditioning with a short course of steroids generally supported higher levels of prolonged IL-1Ra expression. The DONATELLO clinical trial was supported by a $12 million award from the California Institute for Regenerative Medicine (CLIN2-14265). About Osteoarthritis (OA) of the Knee Osteoarthritis (OA) is a progressive joint disease that is a leading cause of disability. It is characterized by destruction of cartilage and structural changes in bone within the joint, which contribute to pain and loss of joint function. Osteoarthritis affects more than 30 million Americans and is increasing as a result of the aging population and increasing prevalence of obesity. Osteoarthritis represents a major economic burden, owing to direct medical costs and loss of productivity. Each year, millions of patients are treated for knee OA with NSAIDs, opioids, and steroid injections into the knee to manage their knee pain. There are no currently available therapies known to alter or slow down OA progression. About Genascence Corporation Genascence, a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, is developing life-changing treatments for highly prevalent conditions affecting millions of people. The company was founded in 2017 with technology licensed from three leading U.S. research institutions: Mayo Clinic, University of Florida, and NYU Langone Health. Headquartered in Palo Alto, California, Genascence's founders and leadership team have deep experience in the design, development, and manufacturing of successful gene therapies and biological medicines. For more information, please visit View source version on CONTACT: Media Contact: Kathy Vincent [email protected] KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA INDUSTRY KEYWORD: BIOTECHNOLOGY HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS SOURCE: Genascence Corporation Copyright Business Wire 2025. PUB: 05/12/2025 07:45 AM/DISC: 05/12/2025 07:46 AM
