Industry progress report on neuro-oncology: a biotech update.

With steadily rising revenue and large numbers of clinical trials utilizing novel treatment strategies, the field of neuro-oncology is at the core of the growing cancer therapy industry. In June 2012, the Weill Cornell Brain and Tumor Center hosted the first Brain Tumor Biotech Summit as a forum for fostering and encouraging collaboration between researches and investors to accelerate novel treatments for brain cancer. This event brought together neuro-oncologists, neurosurgeons, academicians, entrepreneurs, non-profits, CEOs and investors in an attempt to bring innovative treatments and concepts to the fore. Specific subjects presented at the meeting included new surgical devices and delivery techniques, targeted therapeutics, immunotherapy, and stem cell biology. The mission of the summit was to provide opportunities for researchers in neuro-oncology to directly interact with leaders from the investment community with insight into the commercial aspects of our work. Our shared goal is to shorten the time for basic science ideas to be translated into the clinical setting. The following serves as a progress report on the biotech industry in neuro-oncology, as presented at the Brain Tumor Biotech Summit.

Morbilliform Eruptions in the Hospitalized Child.

Morbilliform eruptions inspire a broad and varied differential spanning across inflammatory and infectious categories. The goal of this article is to help the clinician develop an approach toward the pediatric patient with a morbilliform eruption in the emergency room or hospital setting. The authors review several high-yield clinical scenarios with a focus on recently emerging and reemerging childhood diagnoses.

Safety and Efficacy of Lenabasum, a Cannabinoid Receptor Type 2 Agonist, in Patients with Dermatomyositis with Refractory Skin Disease: A Randomized Clinical Trial.

BACKGROUND: Treatment options are limited for skin disease in dermatomyositis. Lenabasum is a cannabinoid receptor type 2 agonist that triggers the resolution of inflammation. OBJECTIVE: The objective of this study was to evaluate the safety and efficacy of lenabasum in patients with refractory cutaneous dermatomyositis. DESIGN: This study was a single-center, double-blind, randomized, placebo-controlled phase 2 study conducted from July 2015 to August 2017. POPULATION: The population included subjects aged ≥18 years with at least moderately active dermatomyositis skin activity by Cutaneous Dermatomyositis Disease Area and Severity Index activity ≥ 14 and failure or intolerance to hydroxychloroquine. INTERVENTION: Participants received 20 mg lenabasum daily for 28 days and then 20 mg twice per day for 56 days or placebo. MAIN OUTCOMES AND MEASURES: The primary outcome was a change in Cutaneous Dermatomyositis Disease Area and Severity Index activity. Safety and other secondary efficacy assessments were performed till day 113. RESULTS: A total of 22 subjects were randomized to lenabasum (n = 11) or placebo (n = 11). No serious or severe adverse events were related to lenabasum, and no participants discontinued the study. The adjusted least-squares mean for Cutaneous Dermatomyositis Disease Area and Severity Index activity decreased more for lenabasum, and the difference was significant on day 113 (least-squares mean [standard error] difference = ‒6.5 [3.1], P = 0.038). Numerically greater improvements were seen in multiple secondary efficacy outcomes and biomarkers with lenabasum. CONCLUSION: Lenabasum treatment was well tolerated and was associated with greater improvement in Cutaneous Dermatomyositis Disease Area and Severity Index activity and multiple efficacy outcomes. TRIAL REGISTRATION: This study was registered at ClinicalTrials.gov, NCT02466243.

Classifying discoid lupus erythematosus: background, gaps, and difficulties.

To inform our ongoing efforts to develop defining features to be incorporated into a novel set of classification criteria for discoid lupus erythematosus (DLE), we conducted a literature review using the Ovid MEDLINE database. A search was performed to identify studies reporting criteria used to distinguish DLE from other cutaneous lupus erythematosus subtypes. We examined which clinical, histopathologic, and serologic features have data to support their use as effective features in distinguishing DLE from other potential disease mimickers and cutaneous lupus subsets. Through our search, we were also able to identify gaps that exist in the literature which can inform future directions for research endeavors. We found that localization of lesions, characteristic features of damage, and the absence of high titer Ro/SSA antibody seem most effective in differentiating DLE from other cutaneous lupus erythematosus subtypes. Histopathologic features and class of immunoreactant deposition appear to be less helpful.

Trachyonychia: Review and Update on Clinical Aspects, Histology, and Therapy.

Trachyonychia is a disorder of the nail unit that most commonly presents with rough, longitudinally ridged nails (opaque trachyonychia) or less frequently, uniform, opalescent nails with pits (shiny trachyonychia). The term trachyonychia refers to 'rough nails.' This article comprehensively reviews the clinical, histologic, and therapeutic aspects of trachyonychia. The authors' preferred evaluation and management strategies of trachyonychia are included.

Chronic Pruritus and Connective Tissue Disorders: Review, Gaps, and Future Directions.

Chronic itch is a common skin manifestation in many connective tissue diseases. However, the cause of chronic itch in these diseases is still not fully understood. The complex, and in some cases incomplete understanding of, pathogenesis in each condition makes it difficult to target specific mediators of chronic itch. It is important to better understand the pathophysiology of chronic itch in these conditions, as this information might provide a fuller understanding of the pathogenesis of the diseases in general and lead to the development of more specific treatments for connective tissue diseases in patients with chronic itch. We present a review of the literature on what is known about the prevalence, possible pathophysiology, and effect on quality of life of chronic itch in patients with scleroderma, Sjögren's syndrome, dermatomyositis, systemic lupus erythematosus, cutaneous lupus erythematosus, and mixed connective tissue disease.

Classifying discoid lupus erythematosus: background, gaps, and difficulties.

To inform our ongoing efforts to develop defining features to be incorporated into a novel set of classification criteria for discoid lupus erythematosus (DLE), we conducted a literature review using the Ovid MEDLINE database. A search was performed to identify studies reporting criteria used to distinguish DLE from other cutaneous lupus erythematosus subtypes. We examined which clinical, histopathologic, and serologic features have data to support their use as effective features in distinguishing DLE from other potential disease mimickers and cutaneous lupus subsets. Through our search, we were also able to identify gaps that exist in the literature which can inform future directions for research endeavors. We found that localization of lesions, characteristic features of damage, and the absence of high titer Ro/SSA antibody seem most effective in differentiating DLE from other cutaneous lupus erythematosus subtypes. Histopathologic features and class of immunoreactant deposition appear to be less helpful.

Conservative management of cavernous sinus cavernous hemangioma in pregnancy.

Cavernous sinus cavernous hemangiomas in pregnancy are extremely rare lesions. The precise management of these lesions remains unknown. The authors present a case of a cavernous hemangioma in pregnancy, centered within the cavernous sinus that underwent postpartum involution without surgical intervention. A 34-year-old pregnant patient (gravida 1, para 0) presented to an otolaryngologist with persistent headache and left-sided facial pain and numbness in the V1 distribution. While being treated for sinusitis, her symptoms progressed to include a left-sided oculomotor palsy and abducens palsy. Magnetic resonance imaging without contrast revealed an expansile mass within the left cavernous sinus consistent with a cavernous hemangioma. The patient was evaluated by a neurosurgeon who recommended close follow-up and postpartum imaging without surgical intervention. Although the lesion enlarged during pregnancy, the patient was able to undergo an uncomplicated cesarean section at 37 weeks. All facial and ocular symptoms resolved by 9 months postpartum, and MRI showed a decrease in lesion size and reduced mass effect. The authors conclude that nonsurgical management may be a viable approach in patients who have an onset or exacerbation of symptoms associated with cavernous sinus cavernous hemangiomas during pregnancy because postpartum involution may negate the need for surgical intervention.