QurAlis’ Post

Kv7.2/7.3 has become a hot target recently, with data emerging in epilepsy, pain, bipolar and major depressive disorder. But #didyouknow that Kv7.2 is mis-spliced in sporadic ALS and that this contributes to motor neuronal loss through hyper-excitability? Pleased to announce the initiation of a multiple ascending dose trial of QRL-101, our highly selective Kv7.2/7.3 potential precision therapy for ALS.

In rare disease clinical trials, improvement in patients’ daily function is a key indicator that an investigational therapy is effective. However, designing endpoints that capture the evolution of daily function in the context of rare disease trials, which often span years and face various methodological constraints, is not an easy task. Home-based video assessments may be an answer to that challenge because they can capture incremental change in daily function over time by evaluating patients in their real-world environments, thus tracking the real-world impact of novel treatments. From an equity perspective, such assessments – which are a form of RWE – can also help expand access to trials as they are well-suited for decentralized trial modalities. Have a read at the article below to learn how one company that has developed a home-based video assessment tool for patients with Duchenne muscular dystrophy envisions its use as a primary endpoint in Duchenne and possibly other rare disease trials. #realworlddata #rwd #realworldevidence #rwe

Celine Dion’s recent appearance at the Olympics and a new documentary on Amazon Prime has brought widespread attention to Stiff Person Syndrome (SPS), a rare autoimmune disorder that has profoundly impacted her life. This debilitating condition, characterized by severe muscle stiffness and spasms, is now in the spotlight thanks to her resilience and bravery. 🔍 Market Research in Rare Disease Treatment: The quest for effective treatment options for SPS has been significantly influenced by market research, which plays a crucial role in identifying promising therapies and guiding clinical development. Kyverna Therapeutics is at the forefront of this effort, pioneering innovative CAR T-cell therapy to treat SPS. Their preclinical trial, presented at the EULAR 2024 conference, provides valuable insights into the potential of CAR T-cell therapy in modulating the immune system to address the root causes of SPS. This study is a key example of how early-stage research informs the path to clinical advancements. Key insights from this research include: Targeted Therapy Development: Kyverna's CAR T-cell therapy is specifically designed to combat the autoimmune components of SPS, providing a more precise and potentially effective treatment. Clinical Trial Advancements: Ongoing clinical trials, informed by thorough market research, have shown promising early results in alleviating severe SPS symptoms. Hope for the Future: For patients like Celine Dion, these advancements offer a new hope for managing symptoms and improving quality of life. Celine Dion’s courage in sharing her journey not only inspires millions but also underscores the critical need for continued research, supported by market insights, in the fight against rare diseases like SPS. #SPS #StiffPersonSyndrome #CelineDion #AutoimmuneDisease #CAR_TCellTherapy #KyvernaTherapeutics #HealthcareInnovation #PharmaResearch

Tofacitinib or ustekinumab in patients with ulcerative colitis failing two therapeutic lines? Here are the interesting results from a multicentre international case-control real-world study. Thanks to all friends and colleague who joined this project. https://lnkd.in/dudXaRvs

💡 An international panel of physicians with expertise in myelofibrosis (MF) together with a diverse extended faculty developed new recommendations addressing the critical gaps in the management of MF. There were 5 consensus themes encompassing 25 key clinical questions. These were defining the thresholds for anemia, and when to initiate or modify treatment, defining the threshold for thrombocytopenia and when to begin or adjust therapy, characterizing Janus kinase (JAK) inhibitor failure and what would call for switching therapy, when and how to evaluate prognosis in patients with MF, and unmet needs in MF clinical trials. Read more: https://lnkd.in/efQyMSDF #RareDiseaseResearch

I’m excited to share that Brain Trust Bio has finally received approval to run Phase I clinical trials targeting ALS with a continuous intrathecal drug delivery method! After years of engaging with regulatory agencies globally, our team succeeded in Australia, where we will launch the clinical trials. This milestone propels us to enhance treatment efficacy and opens doors for direct progression to Phase II trials in the U.S. without repetition. I want to take a moment to thank the incredible team at Brain Trust Bio Nicholas Boulis and Chen Benkler. Your dedication, expertise, and tireless effort to develop this therapy for ALS patients have been instrumental in reaching this milestone. Brain Trust Bio’s innovative approach aims to revolutionize central nervous system disorder management by delivering therapeutic agents directly to the cerebrospinal fluid. This approach offers promising potential for neuroprotection while minimizing systemic side effects, especially compared to the side effects of oral cns drugs. Learn more about Brain Trust Bio’s groundbreaking work at braintrustbio.com. Let's redefine the future of CNS treatment!  #BrainTrustBio #Innovation #CNSDiseases #ClinicalTrials #ALSResearch

🚀 Mesoblast's pioneering MCL technology platform leverages mesenchymal precursor cells to develop tailored therapies for various diseases, promising a paradigm shift in treatment approaches. Mesoblast's allogenic therapy is a strong candidateas treatment for both advanced and end-stage chronic heart failure. Despite challenges, Mesoblast's commitment to innovation extends to inflammatory diseases, exemplified by Ryoncil. Strategic partnerships highlight its relentless pursuit of medical breakthroughs. By modulating the immune system with anti-inflammatory factors, Mesoblast's candidates tackle diseases at their roots, promising transformative outcomes. This approach holds the potential to revolutionize treatment paradigms for conditions like chronic low back pain and beyond. Mesoblast's bold strides toward medical innovation herald a brighter future for patients worldwide. #Mesoblast #MedicalInnovation #newmedicine👩⚕️👨⚕️

We conduct an open-label, single-arm, phase I/II study of BC19 CAR T cells in 50 patients with R/R MM (ChiCTR2000033567). The primary endpoint was safety. BC19 CAR T cells are well tolerated with grade 3 or higher cytokine release syndrome in 8% of patients and grade 1 neurotoxic events in 4% of patients, which meet the pre-specified primary endpoint. Secondary endpoints include overall response rate (92%), median progression-free survival (19.7 months), median overall survival (19.7 months) and median duration of response (not reached). Our study demonstrates that bispecific BC19 CAR T cells are feasible, safe and effective in treating patients with R/R MM.

The final published version of our comprehensive review of interventions for Alzheimer’s disease is now online: https://lnkd.in/grkjAGnm In this review, we discuss the challenges for Alzheimer’s Disease (AD) patients, who experience diverse symptoms, including memory loss, cognitive impairment, behavioral abnormalities, mood changes, and mental issues. Treatment strategies are currently evolving, as interventions we discuss include approved drugs and an extensive list of current clinical trials underway. In summary, we have tried to cover all the therapeutic interventions which are available for the treatment and management of AD under sections including approved drugs, clinical phase evaluations, pre-clinical phase or futuristic interventions, off-labelled drugs, and non-pharmacological interventions for AD, offering positive findings and well as challenges that remain. Where possible, we have provided likely hypothesized mechanisms driving the thought behind different treatments, and referenced the latest developments that have provided positive and negative results thus far. Thanks go out to the whole co-author team who made the review possible, and for bringing the hopes and challenges available to the literature. 

The advancements in #CNS therapeutics offer hope for conditions like #Alzheimer's and #Huntington's disease, with innovative treatments targeting disease progression and symptom management, paving the way for improved patient outcomes and quality of life. #AMR #CNStherapeutics #AlzheimersResearch #HuntingtonsTreatment #NeuroscienceAdvancements #MedicalInnovations #HopeForPatients https://lnkd.in/dVWcAnUr