es/iode’s Post

CAR T cell therapy for refractory pediatric systemic lupus erythematosus: a new era of hope? CAR T-cell therapy, which involves collecting T cells, genetically re-engineering them to target specific antigens, and reinfusing them into patients, has shown promise beyond its initial focus on hematological cancers like B-cell non-Hodgkin lymphoma and multiple myeloma. Researchers are now exploring its potential in various other indications, including autoimmune diseases such as Systemic Lupus Erythematosus (SLE). While investigation of CAR T cells in human subjects with SLE has been limited, early data are encouraging. A recent study examining CAR T-cell infusion in patients with three autoimmune diseases, including SLE, demonstrated that the therapy was feasible, safe, and effective. This study, which included five young adults (aged 18-24) with SLE, reported sustained depletion of circulating B cells, control of Lupus Nephritis (LN), and a decrease in SLE Disease Activity Index scores, suggesting potential therapeutic benefits for SLE patients. Pubmed article: https://lnkd.in/dadHzGiJ

Historic Day for Pediatric Cancer Treatment! Today marks a groundbreaking moment in the fight against pediatric low-grade glioma (#pLGG). The U.S. FDA has granted accelerated approval for the first targeted therapy, OJEMDA (tovorafenib), heralding a breakthrough for young patients with BRAF fusion or rearrangement, or BRAF V600 mutation. With an ORR of 51%, this innovative medicine offers new hope, especially for those who've faced relapse or resistance to previous treatments. Thanks to Day One Biopharmaceuticals' relentless endeavour, OJEMDA (tovorafenib) emerges with a transformative era for young pLGG patients. #OJEMDA #pLGG #Pediatrics #PediatricOncology #FDAApproval #DayoneBiopharmaceuticals #CancerResearch #FDAAcceleratedApproval #ElixirAssociates

📃Scientific paper: Non-Wilms' renal tumors in children: experience with 139 cases treated at a single center Abstract: Background Pediatric non-Wilms renal tumors (NWRTs), which comprise a small proportion of renal tumors, are a heterogeneous group of neoplasms with variable malignant potential, mortality, and response to treatment. We performed this study to determine the clinical characteristics, management and prognosis of children with Pediatric NWRTs. Methods Medical records of all patients (n = 139) treated for NWRTs over a 12-year period (2008.01–2019.10) at a single center were reviewed retrospectively. Results The histopathological groups of NWRTs included malignant rhabdoid tumor of the kidney (MRTK) (n: 30, 21.6%), renal cell cancer (RCC) (n: 26,18.7%), clear cell sarcoma of the kidney (CCSK) (n: 24,17.3%), congenital mesoblastic nephroma (CMN) (n: 21,15.1%), cystic nephroma (CN) (n: 16,11.5%), metanephric tumors (n: 12, 8.6%), renal angiomyoliporma (RAML) (n: 3, 2.2%), renal primitive neuroectodermal tumor (n: 2, 1.4%), renal hemangioma (n: 2, 1.4%), inflammatory myofibroblastic tumor (n: 2, 1.4%), ossifying renal tumor of infancy (ORTI) (n: 1, 0.7%). The distribution of all malignant NWRTs, including MRTK, CCSK, RCC and PNET, according to stage was as follows: stages I (n = 26), II (n = 16), III (n = 29), and IV (n = 11). The summary table shows the treatment offered to children with NWRTs. A total of 123 children were followed up for an average of 42 months. Sixteen children were lost to follow-up. Tumor-free survival was observed in 94 children. One patient who suffe... Continued on ES/IODE ➡️ https://etcse.fr/6I ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Persistent dyskinesias in patients with fetal tissue transplantation for Parkinson disease Abstract: Cell transplants are being developed for patients with Parkinson disease (PD) who have insufficient benefit with standard medical treatment. We describe the clinical features of five patients who developed persistent dyskinesias after fetal dopaminergic tissue transplantation. All had levodopa-induced dyskinesias preoperatively. We implanted fetal mesencephalic dopaminergic tissue into the putamina bilaterally in 34 patients with advanced PD. They were not immunosuppressed. Five of 34 patients (15%) developed troublesome choreic or dystonic dyskinesias that persisted despite lowering or discontinuing medications. Attempts to treat the involuntary movements with amantadine, clozapine, anticholinergics, dopamine depletors and other medicines had limited success. Metyrosine eliminated dyskinesias but led to the parkinsonian “off” state. Increasing the dose of levodopa worsened the dyskinesias. Three patients required placement of pallidal stimulators, bilaterally in two and unilaterally in one patient who had only contralateral dyskinesias. The two with the bilateral stimulators had improvement in dyskinesias. The patient with the unilateral pallidal stimulator had a substantial reduction of the dyskinesias, but attempts to treat residual “off” symptoms with levodopa were limited by worsening dyskinesias. Although the number of patients developing these persistent dyskinesias was small, these five patients had dramatic improvement after transplant. As a group, the... Continued on ES/IODE ➡️ https://etcse.fr/wAdK ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

First ever drug approved for NHS patients with life-threatening genetic, tumour-causing disease NHS patients in England with a rare genetic disease that causes tumours in major organs, including the brain and spine, can now benefit from the first-ever therapy which could shrink their tumours to help them avoid high-risk surgery. In clinical trials of belzutifan, 95% of patients did not experience any growth in their tumours in two years of taking the treatment, and 56% of patients’ tumours shrunk. Belzutifan, a take-at-home pill, is available for those with von Hippel-Lindau disease on the NHS in England from today (Thursday 5 September), following a positive recommendation from the National Institute for Health and Care Excellence (NICE) enabled by an NHS England commercial deal with manufacturer MSD UK. Around 800 people in England live with von Hippel-Lindau (VHL) disease, a rare, life-long and incurable disorder which causes multiple, malignant and benign tumours in major organs around the body. Currently, the only treatment available is invasive surgery or radiotherapy, and people with VHL usually need multiple surgeries throughout their lives. Because of the location of tumours in the brain, spinal cord, eyes, pancreas and kidneys, procedures to remove tumours are often high-risk, and can have life-changing consequences, including paralysis, loss of vision, diabetes, or the need for life-long dialysis. The new, oral drug is another treatment option for patients which could help people avoid high-risk surgeries. It is estimated around 100 people could benefit from belzutifan in the first year, and around 50 people per year thereafter, with this ground-breaking drug being available with managed access through the NHS England’s Cancer Drugs Fund, while further evidence is gathered. https://lnkd.in/eECJUnA4

📃Scientific paper: Persistent dyskinesias in patients with fetal tissue transplantation for Parkinson disease Abstract: Cell transplants are being developed for patients with Parkinson disease (PD) who have insufficient benefit with standard medical treatment. We describe the clinical features of five patients who developed persistent dyskinesias after fetal dopaminergic tissue transplantation. All had levodopa-induced dyskinesias preoperatively. We implanted fetal mesencephalic dopaminergic tissue into the putamina bilaterally in 34 patients with advanced PD. They were not immunosuppressed. Five of 34 patients (15%) developed troublesome choreic or dystonic dyskinesias that persisted despite lowering or discontinuing medications. Attempts to treat the involuntary movements with amantadine, clozapine, anticholinergics, dopamine depletors and other medicines had limited success. Metyrosine eliminated dyskinesias but led to the parkinsonian “off” state. Increasing the dose of levodopa worsened the dyskinesias. Three patients required placement of pallidal stimulators, bilaterally in two and unilaterally in one patient who had only contralateral dyskinesias. The two with the bilateral stimulators had improvement in dyskinesias. The patient with the unilateral pallidal stimulator had a substantial reduction of the dyskinesias, but attempts to treat residual “off” symptoms with levodopa were limited by worsening dyskinesias. Although the number of patients developing these persistent dyskinesias was small, these five patients had dramatic improvement after transplant. As a group, the... Continued on ES/IODE ➡️ https://etcse.fr/wAdK ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Persistent dyskinesias in patients with fetal tissue transplantation for Parkinson disease Abstract: Cell transplants are being developed for patients with Parkinson disease (PD) who have insufficient benefit with standard medical treatment. We describe the clinical features of five patients who developed persistent dyskinesias after fetal dopaminergic tissue transplantation. All had levodopa-induced dyskinesias preoperatively. We implanted fetal mesencephalic dopaminergic tissue into the putamina bilaterally in 34 patients with advanced PD. They were not immunosuppressed. Five of 34 patients (15%) developed troublesome choreic or dystonic dyskinesias that persisted despite lowering or discontinuing medications. Attempts to treat the involuntary movements with amantadine, clozapine, anticholinergics, dopamine depletors and other medicines had limited success. Metyrosine eliminated dyskinesias but led to the parkinsonian “off” state. Increasing the dose of levodopa worsened the dyskinesias. Three patients required placement of pallidal stimulators, bilaterally in two and unilaterally in one patient who had only contralateral dyskinesias. The two with the bilateral stimulators had improvement in dyskinesias. The patient with the unilateral pallidal stimulator had a substantial reduction of the dyskinesias, but attempts to treat residual “off” symptoms with levodopa were limited by worsening dyskinesias. Although the number of patients developing these persistent dyskinesias was small, these five patients had dramatic improvement after transplant. As a group, the... Continued on ES/IODE ➡️ https://etcse.fr/wAdK ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

FDA granted accelerated approval to selpercatinib (Retevmo, Eli Lilly and Company) for pediatric patients two years of age and older with the following: 1. advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy; 2. advanced or metastatic thyroid cancer with a RET gene fusion, as detected by an FDA-approved test, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate); and 3. locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options. This is the first FDA approval of a targeted therapy for pediatric patients < 12 years of age with RET alterations. #cancer #pediatrics #oncology #FDA #drugapproval

📃Scientific paper: Does kidney biopsy in pediatric lupus patients “complement” the management and outcomes of silent lupus nephritis? Lessons learned from a pediatric cohort Abstract: Background Silent lupus nephritis (SLN) is systemic lupus erythematosus (SLE) without clinical and laboratory features of kidney involvement but with biopsy-proven nephritis. This study aims to describe and compare the baseline characteristics and outcomes of pediatric SLN with overt LN (OLN) and to identify associated risk factors and biochemical markers. Methods In this retrospective, observational study, multivariate logistic regression and receiver operating characteristic (ROC) analyses studied age, sex, race, serum complements, anti-double-stranded-DNA antibody, anti-Smith antibody, eGFR, and proliferative nephritis. Results In our cohort of 69 patients, 47 were OLN, and 22 were SLN. OLN ( OR  = 4.9, p  = 0.03) and non-African Americans (AA) ( OR  = 13.0, p  < 0.01) had higher odds, and increasing C3 and C4 were associated with lower odds of proliferative nephritis ( OR 0.95 and 0.65 per one unit increase in C3 and C4, respectively, p  < 0.01). They demonstrated a good discriminative ability to detect proliferative nephritis as assessed by the area under the ROC curve (C3 = 0.78, C4 = 0.78). C3 and C4 in proliferative SLN and OLN were comparable and significantly lower than their non-proliferative counterparts. No association was observed between age, sex, anti-double-stranded-DNA antibody, anti-Smith antibody, eGFR, and proliferative nephritis. Proliferative SLN and OLN patients received similar treatments. Adverse events were identified in the proliferative... Continued on ES/IODE ➡️ https://etcse.fr/HhI ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Persistent dyskinesias in patients with fetal tissue transplantation for Parkinson disease Abstract: Cell transplants are being developed for patients with Parkinson disease (PD) who have insufficient benefit with standard medical treatment. We describe the clinical features of five patients who developed persistent dyskinesias after fetal dopaminergic tissue transplantation. All had levodopa-induced dyskinesias preoperatively. We implanted fetal mesencephalic dopaminergic tissue into the putamina bilaterally in 34 patients with advanced PD. They were not immunosuppressed. Five of 34 patients (15%) developed troublesome choreic or dystonic dyskinesias that persisted despite lowering or discontinuing medications. Attempts to treat the involuntary movements with amantadine, clozapine, anticholinergics, dopamine depletors and other medicines had limited success. Metyrosine eliminated dyskinesias but led to the parkinsonian “off” state. Increasing the dose of levodopa worsened the dyskinesias. Three patients required placement of pallidal stimulators, bilaterally in two and unilaterally in one patient who had only contralateral dyskinesias. The two with the bilateral stimulators had improvement in dyskinesias. The patient with the unilateral pallidal stimulator had a substantial reduction of the dyskinesias, but attempts to treat residual “off” symptoms with levodopa were limited by worsening dyskinesias. Although the number of patients developing these persistent dyskinesias was small, these five patients had dramatic improvement after transplant. As a group, the... Continued on ES/IODE ➡️ https://etcse.fr/wAdK ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.