Innovative Therapies in Nail Disorders

2021 ◽  
pp. 67-69
Author(s):  
Smail Hadj-Rabia
Keyword(s):  
Innovative Therapies

scholarly journals ETMR-14. TREATMENT OF EMBRYONAL TUMOURS WITH MULTILAYERED ROSETTES (ETMR) WITH CARBOPLATIN-ETOPOSIDE INDUCTION AND TANDEM HIGH-DOSE CHEMOTHERAPY WITHIN THE PROSPECTIVE HIT-TRIALS AND REGISTRIES

2020 ◽  
Vol 22 (Supplement_3) ◽  
pp. iii325-iii326
Author(s):  
Björn-Ole Juhnke ◽  
Marco Gessi ◽  
Nicolas Ulrich Gerber ◽  
Carsten Friedrich ◽  
Christine Haberler ◽  
...  
Keyword(s):  
Progression Free Survival ◽  
High Dose Chemotherapy ◽  
High Dose ◽  
Disease Relapse ◽  
Total Resection ◽  
Free Survival ◽  
Entire Cohort ◽  
Innovative Therapies ◽  
Embryonal Tumours ◽  
Aggressive Tumors

Abstract BACKGROUND Embryonal tumours with multilayered rosettes (ETMR) are highly aggressive tumors, mostly occurring in infants. Published clinical data refer to retrospective cohorts of inhomogeneously treated patients. Here, we describe the outcome of patients, who were prospectively treated within the P-HIT2000-trial, the subsequent HIT2000-interim-registry and earlier HIT-trials. PATIENTS AND METHODS Nineteen patients from the P-HIT2000-trial (2001–2011), 12 patients from the subsequent HIT2000-interim-registry (2012–2014) and 4 patients from earlier HIT-trials with centrally reviewed neuropathological and molecularly-confirmed diagnosis of ETMR were included. Outcome of 18 patients treated with carboplatin-etoposide-induction followed by tandem-high-dose chemotherapy (“CARBO-ETO+HDCT”) with stage-stratified radiotherapy administered in case of persistant disease, relapse or progression were compared to patients treated with HIT-SKK chemotherapy ± radiotherapy (n=9) or other regimens (n=8). RESULTS Median age at diagnosis was 2.9(1.0–5.3) years. Metastases at diagnosis were detected in 9 patients (26%). For the entire cohort of n=35, 5-year overall survival (OS) was 26.7%, and progression-free survival (PFS) was 18.5%. Five-year OS for patients with CARBO-ETO+HDCT, SKK chemotherapy or other regimens was 44.4%, 13.0% and 0%, respectively (p=0.006). Five-year PFS was 33.3%, 0% and 0%, respectively (p=0.119). Of 10 survivors, n=8 were treated with CARBO-ETO+HDCT; n=4 had craniospinal, n=2 local and n=4 no radiotherapy. Impact of initial gross-total-resection (p=0.231) and non-metastatic disease (p=0.097) was limited. CONCLUSIONS We show improved survival with carboplatin-etoposide-induction followed by tandem-high-dose chemotherapy, indicating that a cure is possible for some patients. However, despite intensive treatment, outcome is unsatisfactory and innovative therapies urgently need to be included in an upfront setting.

scholarly journals Toward a Personalized Therapy in Soft-Tissue Sarcomas: State of the Art and Future Directions

Cancers ◽  
2021 ◽  
Vol 13 (10) ◽  
pp. 2359
Author(s):  
Liliana Montella ◽  
Lucia Altucci ◽  
Federica Sarno ◽  
Carlo Buonerba ◽  
Stefano De Simone ◽  
...  
Keyword(s):  
Soft Tissue ◽  
Soft Tissue Sarcomas ◽  
Chromosomal Translocations ◽  
Intrinsic Variability ◽  
Poor Survival ◽  
Genomic Alterations ◽  
Future Directions ◽  
Oncogenic Mutations ◽  
Targeted Treatments ◽  
Innovative Therapies

Soft-tissue sarcomas are rare tumors characterized by pathogenetic, morphological, and clinical intrinsic variability. Median survival of patients with advanced tumors are usually chemo- and radio-resistant, and standard treatments yield low response rates and poor survival results. The identification of defined genomic alterations in sarcoma could represent the premise for targeted treatments. Summarizing, soft-tissue sarcomas can be differentiated into histotypes with reciprocal chromosomal translocations, with defined oncogenic mutations and complex karyotypes. If the latter are improbably approached with targeted treatments, many suggest that innovative therapies interfering with the identified fusion oncoproteins and altered pathways could be potentially resolutive. In most cases, the characteristic genetic signature is discouragingly defined as “undruggable”, which poses a challenge for the development of novel pharmacological approaches. In this review, a summary of genomic alterations recognized in most common soft-tissue sarcoma is reported together with current and future therapeutic opportunities.

scholarly journals Molecular Mechanisms behind Inherited Neurodegeneration of the Optic Nerve

Biomolecules ◽  
2021 ◽  
Vol 11 (4) ◽  
pp. 496
Author(s):  
Alessandra Maresca ◽  
Valerio Carelli
Keyword(s):  
Optic Nerve ◽  
Mitochondrial Dysfunction ◽  
Molecular Mechanisms ◽  
Ganglion Cells ◽  
Mitochondrial Dynamics ◽  
Unmet Need ◽  
Deep Understanding ◽  
Genetic Landscape ◽  
Innovative Therapies ◽  
Energy Dependent

Inherited neurodegeneration of the optic nerve is a paradigm in neurology, as many forms of isolated or syndromic optic atrophy are encountered in clinical practice. The retinal ganglion cells originate the axons that form the optic nerve. They are particularly vulnerable to mitochondrial dysfunction, as they present a peculiar cellular architecture, with axons that are not myelinated for a long intra-retinal segment, thus, very energy dependent. The genetic landscape of causative mutations and genes greatly enlarged in the last decade, pointing to common pathways. These mostly imply mitochondrial dysfunction, which leads to a similar outcome in terms of neurodegeneration. We here critically review these pathways, which include (1) complex I-related oxidative phosphorylation (OXPHOS) dysfunction, (2) mitochondrial dynamics, and (3) endoplasmic reticulum-mitochondrial inter-organellar crosstalk. These major pathogenic mechanisms are in turn interconnected and represent the target for therapeutic strategies. Thus, their deep understanding is the basis to set and test new effective therapies, an urgent unmet need for these patients. New tools are now available to capture all interlinked mechanistic intricacies for the pathogenesis of optic nerve neurodegeneration, casting hope for innovative therapies to be rapidly transferred into the clinic and effectively cure inherited optic neuropathies.

Treatment of Genital Melanoma: Are We Ready for Innovative Therapies?

2017 ◽  
Vol 27 (6) ◽  
pp. 1063-1063 ◽  
Author(s):  
Antonino Ditto ◽  
Giorgio Bogani ◽  
Fabio Martinelli ◽  
Francesco Raspagliesi
Keyword(s):  
Innovative Therapies

The ethical assessment of innovative therapies: Liver transplantation using living donors

1990 ◽  
Vol 11 (2) ◽  
pp. 87-94 ◽  
Author(s):  
Peter A. Singer ◽  
Mark Siegler ◽  
John D. Lantos ◽  
Jean C. Emond ◽  
Peter F. Whitington ◽  
...  
Keyword(s):  
Liver Transplantation ◽  
Living Donors ◽  
Ethical Assessment ◽  
Innovative Therapies

New therapeutic challenges in metastatic breast cancer: the association of CDK4 / 6 inhibitors with radiotherapy. A short review

2020 ◽  
Vol 4 (4) ◽  
pp. 01-03
Author(s):  
Tudor Calistru
Keyword(s):  
Breast Cancer ◽  
Metastatic Breast Cancer ◽  
Volume Fraction ◽  
Multidisciplinary Treatment ◽  
Metastatic Breast ◽  
Palliative Radiotherapy ◽  
Short Review ◽  
Fda Approval ◽  
Dose Volume ◽  
Innovative Therapies

FDA approval of CDK4 / 6 inhibitors (Palbociclib, Ribociclib and Abemaciclib) for metastatic and advanced breast cancer, in combination or not with Fulvestrant or Letrozole, has improved the prognosis of this type of patient population. Palliative radiotherapy with antalgic purpose in most of the cases is often part of the multidisciplinary treatment of bone involvement metastatic breast cancer. In the context of the approval of these innovative therapies and of the development of radiotherapy techniques, including stereotactic radiosurgery, it is necessary to identify the best therapeutic sequence and parameters (dose, volume, fraction size) to obtain a synergistic effect. Considering the toxicity profiles of new therapies (especially lymphopenia and neutropenia) and the different mechanisms of the induction of these toxicities towards radiotherapy and chemotherapy, it is also necessary to demonstrate the safety profile of concomitant or sequential irradiation with the administration of CDK4 / 6 inhibitors in combination with radiation therapy.

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