Exploring the current state of technology transfer in the United States: perspectives and improvement strategies from the experts.

Technology transfer (TT) is a necessary, yet complex process to convey and disseminate scientific knowledge to the commercial sector. However, multiple barriers in TT can impede commercialization and innovative progress. To cultivate a deeper understanding, we conducted five interviews with strategic, elite leaders in different areas of TT in the United States. Experts shared their perspectives on the current state of TT, what needs improvement, and potential solutions to enhance the TT landscape, with a focus on biotechnology and medical devices. The formation of strong management teams, a comprehension of the regulatory, reimbursement, and funding pathways and policies, and thorough market assessments were noted as key aspects for venture success. Collaboration with Technology Transfer Offices (TTOs), industry experts, and strategic partners are also essential to support academic innovators and guide them throughout the complex commercialization process. There is agreement that a venture should have a defined vision and clear goals with a robust business case for the innovation; early involvement of TTOs is essential. Comprehension of the complexities and key facets of TT, while also streamlining the process, will better position biomedical innovators for success.

Dancing with uncertainties in the era of artificial intelligence.

In this commentary, a medical student reflects on the promise of artificial intelligence (AI) in mitigation of physician burnout and moral injury. The rapid introduction of AI technologies may present a challenge to medical professionals, especially those engaged in the transdisciplinary care of children with disabilities.

The Biomedical Entrepreneurship Skills Development Program for the Advancement of Research Translation: Foundations of Biomedical Startups course, metrics, and impact.

Background/Objective: A growing number of biomedical doctoral graduates are entering the biotechnology and industry workforce, though most lack training in business practice. Entrepreneurs can benefit from venture creation and commercialization training that is largely absent from standard biomedical educational curricula. The NYU Biomedical Entrepreneurship Educational Program (BEEP) seeks to fill this training gap to prepare and motivate biomedical entrepreneurs to develop an entrepreneurial skill set, thus accelerating the pace of innovation in technology and business ventures. Methods: The NYU BEEP Model was developed and implemented with funding from NIDDK and NCATS. The program consists of a core introductory course, topic-based interdisciplinary workshops, venture challenges, on-line modules, and mentorship from experts. Here, we evaluate the efficacy of the core, introductory course, "Foundations of Biomedical Startups," through the use of pre/post-course surveys and free-response answers. Results: After 2 years, 153 participants (26% doctoral students, 23% post-doctoral PhDs, 20% faculty, 16% research staff, 15% other) have completed the course. Evaluation data show self-assessed knowledge gain in all domains. The percentage of students rating themselves as either "competent" or "on the way to being an expert" in all areas was significantly higher post-course (P < 0.05). In each content area, the percentages of participants rating themselves as "very interested" increased post-course. 95% of those surveyed reported the course met its objectives, and 95% reported a higher likelihood of pursuing commercialization of discoveries post-course. Conclusion: NYU BEEP can serve as a model to develop similar curricula/programs to enhance entrepreneurial activity of early-stage researchers.

The development of a clinical research educational training for community health workers using the joint task force for clinical trial competency framework.

Introduction: The NYU Clinical & Translational Science Institute, in collaboration with a number of community-engaged initiatives, developed a training for community health workers (CHWs) to enhance health literacy about clinical research. This innovative research training provides CHWs with a basic level of competency in clinical research to convey the importance of research to communities and better advocate for their health needs. CHWs are an underutilized resource to engage diverse populations in clinical research. The training also addresses the need to expand and diversify the clinical research workforce-integrating CHWs into research teams and connecting underserved populations with research opportunities to enhance quality of care. Methods: Structured individual interviews and focus group sessions were held with CHWs as well as clinical research faculty and staff to identify knowledge gaps in clinical research and identify best practices for educating community members on research. Using the Joint Task Force (JTF) for Clinical Trial Competency framework, an online course was developed consisting of 28 modules offered asynchronously for internal and external audiences. Topics include the fundamentals of clinical research, scientific concepts and research design, research ethics, study management, clinical study operations, communications, and teamwork, as well as the importance of diversity and equity in research and the barriers to participation. Results: Learning was evaluated using multiple choice questions after each module to ensure the fundamental level of knowledge was obtained. A separate survey, completed at the conclusion of the course, evaluated the quality of training. Discussion: The course aims to enhance the knowledge and skills of CHWs to help promote greater understanding of clinical research within the communities they serve, including the risks and benefits of clinical research and opportunities for participation. As members of the research team, community stakeholders can help design interventions tailored to the unique needs, culture, and context of their communities. In addition, this research training equips trainees with skills to engage the community actively, involving them in the research process and ensuring community priorities are represented in research through more community engaged processes.

A rating scale for the functional assessment of patients with familial dysautonomia (Riley Day syndrome).

OBJECTIVE: To develop a reliable rating scale to assess functional capacity in children with familial dysautonomia, evaluate changes over time, and determine whether severity within a particular functional category at a young age affected survival. STUDY DESIGN: Ten functional categories were retrospectively assessed in 123 patients with familial dysautonomia at age 7 years ± 6 months. Each of the 10 Functional Severity Scale categories (motor development, cognitive ability, psychological status, expressive speech, balance, oral coordination, frequency of dysautonomic crisis, respiratory, cardiovascular, and nutritional status) were scored from 1 (worst or severely affected) to 5 (best or no impairment). Changes over time were analyzed further in 22 of the 123 patients who were also available at ages 17 and 27 years. RESULTS: Severely impaired cardiovascular function and high frequency of dysautonomic crisis negatively affected survival (P < .005 and P < .001, respectively). In the 22 individuals followed up to age 27 years, psychological status significantly worsened (P = .01), and expressive speech improved (P = .045). From age 17 to 27 years, balance worsened markedly (P = .048). CONCLUSION: The Functional Severity Scale is a reliable tool to measure functional capacity in patients with familial dysautonomia. The scale may prove useful in providing prognosis and as a complementary endpoint in clinical trials.

Chronic Non-infectious Osteomyelitis Mimicking Scurvy as the Presenting Sign of Crohn's Disease: Case Report.

Chronic non-infectious osteomyelitis (CNO) is a rare, inflammatory process associated with pediatric inflammatory bowel disease (IBD). Signs and symptoms of CNO parallel scurvy, a nutritional deficiency that can affect children with autism spectrum disorder (ASD). This is the first report of a child initially thought to have scurvy, then subsequently diagnosed with CNO as the presenting manifestation of Crohn's disease. This case enhances the literature elucidating extra-intestinal manifestations of IBD and pediatric nutritional deficiencies.

Kinetin improves IKBKAP mRNA splicing in patients with familial dysautonomia.

Familial dysautonomia (FD) is caused by an intronic splice mutation in the IKBKAP gene that leads to partial skipping of exon 20 and tissue-specific reduction in I-κ-B kinase complex-associated protein/elongation protein 1 (IKAP/ELP-1) expression. Kinetin (6-furfurylaminopurine) has been shown to improve splicing and increase WT IKBKAP mRNA and IKAP protein expression in FD cell lines and carriers. To determine whether oral kinetin treatment could alter mRNA splicing in FD subjects and was tolerable, we administered kinetin to eight FD individuals homozygous for the splice mutation. Subjects received 23.5 mg/Kg/d for 28 d. An increase in WT IKBKAP mRNA expression in leukocytes was noted after 8 d in six of eight individuals; after 28 d, the mean increase compared with baseline was significant (p = 0.002). We have demonstrated that kinetin is tolerable in this medically fragile population. Not only did kinetin produce the desired effect on splicing in FD patients but also that effect seems to improve with time despite lack of dose change. This is the first report of a drug that produces in vivo mRNA splicing changes in individuals with FD and supports future long-term trials to determine whether kinetin will prove therapeutic in FD patients.

Cultivating a New Generation of Biomedical Entrepreneurs.

In recent years, scientific and technological advances have brought great innovation within the life sciences industry, introducing the need for entrepreneurship training for medical and engineering graduates. With this in mind, Michal Gilon-Yanai, Dr Robert Schneider and their collaborators developed an academic program designed to provide students and faculty members with the skills they need to become successful entrepreneurs. The team of collaborators includes Dr Gabrielle Gold-von Simson, an expert in implementing academic programs, and Dr Colleen Gillespie, who specialises in education, evaluation and dissemination science. Their pioneering program trains students on how to bring new biomedical technologies to the market.

Factors Associated With Parental Participation in Family-Centered Rounds.

OBJECTIVES: Although families positively perceive family-centered rounds (FCR), factors associated with engagement have been examined in few studies. Our objective for this study was to test the hypothesis that inviting the parent to speak and nurse presence are associated with parent engagement during FCR. METHODS: We conducted a cross-sectional study with English-speaking parents (N = 199) of inpatients on the pediatric hospital medicine service at an academic medical center. We used a standardized checklist to record outcomes of engagement (number of questions asked and participation occurrences), predictor variables (team invited parent to speak, nurse presence), and other encounter-related variables. Parents were surveyed to assess parent and child characteristics and experiences during FCR. We examined parent, child, and encounter characteristic associations with the above outcomes using bivariate analyses and (for those associated in bivariate analyses) Poisson regressions. RESULTS: Inviting the parent to speak was independently associated with the number of questions asked (incident rate ratio [IRR] 1.4; 95% confidence interval [CI] 1.1-1.7). Trusting the medical team was inversely associated with questions asked (IRR 0.8; 95% CI 0.6-0.97). Factors associated with total participation included invitation for the parent to speak (IRR 1.5; 95% CI 1.3-1.6), nurse presence (IRR 1.3; 95% CI 1.1-1.5), white race (IRR 1.2; 95% CI 1.1-1.4), clerkship student presentation (IRR 1.2; 95% CI 1.03-1.3), and parent inclusion in rounding arrangement (IRR 1.5; 95% CI 1.05-2). CONCLUSIONS: Parents present during FCR are more engaged when invited to speak. Nurse presence was associated with total parent participation. Future studies to inform interventions to optimize engagement are warranted.

Characteristics of Hospitalized Children With SARS-CoV-2 in the New York City Metropolitan Area.

OBJECTIVES: To describe the characteristics of hospitalized children with severe acute respiratory syndrome coronavirus 2 in New York City metropolitan area. PATIENTS AND METHODS: This was a multicenter, retrospective cohort study at 4 hospitals comprising 82 hospitalized children (0-21 years) who tested positive for severe acute respiratory syndrome coronavirus 2 after symptoms and risk screening between March 1 and May 10, 2020. We subdivided patients on the basis of their admission to acute or critical care units and by age groups. Further subanalyses were performed between patients requiring respiratory support or no respiratory support. RESULTS: Twenty-three (28%) patients required critical care. Twenty-nine (35%) patients requiring respiratory support, with 9% needing mechanical ventilation, and 1 required extracorporeal support. All patients survived to discharge. Children with any comorbidity were more likely to require critical care (70% vs 37%, P = .008), with obesity as the most common risk factor for critical care (63% vs 28%, P = .02). Children with asthma were more likely to receive respiratory support (28% vs 8%, P = .02), with no difference in need for critical care (P = .26). Children admitted to critical care had higher rates of renal dysfunction at presentation (43% vs 10%, P = .002). CONCLUSIONS: Children with comorbidities (obesity and asthma in particular) were at increased risk for critical care admission and/or need for respiratory support. Children with renal dysfunction at presentation were more likely to require critical care.

Parent Perspectives on Participation in Family-Centered Rounds and Informational Resource Use.

Objectives: Family-centered rounds (FCR) can improve communication and patient/family engagement. While use of informational resources (e.g., tablets, computers on wheels, paper notes) can guide FCR, there are limited data concerning parental perspectives on how use of these resources during FCR, or other factors, affect their engagement. Our objectives were to examine parental perspectives on factors that affect their participation during FCR and preferences for informational resources used. Methods: We performed a cross-sectional study with English-speaking parents (n = 200), of pediatric inpatients at an academic medical center, present during FCR. We surveyed parents to ascertain factors they believed affect their engagement during FCR. We asked about their preferences regarding informational resources used by the medical team. Responses were analyzed using descriptive statistics. Parents described their reasoning behind resource preferences, and we categorized these responses. Results: Parents reported that participation was affected by: clarity of the medical team's explanations (78.5%), understanding the information (75.5%), the child's health (74.5%), and being asked for their input (71%). Few (25%) parents believed the informational resource affects participation. Tablets were the preferred resource (24%) due to portability and ease of use, although 56% of parents had no preference. Conclusions: Parents of hospitalized children placed importance on delivery of clear information and an "invitation" to participate during FCR. The resource used by the team was less important, although tablets were most preferred. Next steps are to examine factors associated with objective measures of participation and further study FCR in families with limited English proficiency.

Neoplasia in familial dysautonomia: a 20-year review in a young patient population.

We reviewed the charts of all patients with familial dysautonomia (n = 631) and found that 2% had been diagnosed with tumors. We hypothesize that the IkappaB Kinase-associated protein gene mutation, which causes aberrant RNA splicing in patients with familial dysautonomia, may contribute to tumorigenesis in this genetically homogenous patient population.

Kinetin in familial dysautonomia carriers: implications for a new therapeutic strategy targeting mRNA splicing.

Familial dysautonomia (FD) is caused by an intronic splice mutation in the IkappaB kinase-associated protein gene (IKBKAP) that leads to partial skipping of exon 20 and tissue-specific reduction of IkappaB kinase-associated protein/elongator protein 1 (IKAP/ELP-1 protein). Kinetin increases IKBKAP mRNA and protein expression in FD cell lines. To determine whether oral kinetin alters IKBKAP splicing in vivo, we administered kinetin to 29 healthy carriers of the major FD mutation for 8 d. Adverse effects, kinetin, and IKBKAP mRNA levels were monitored. In the highest dosing cohorts (23.5 mg/kg/d), the target plasma kinetin level was achieved in 91% of subjects at 2 h. After 8 d, IKBKAP mRNA expression in leukocytes increased as kinetin levels increased. There is a linear association between log plasma kinetin level and corresponding log change from baseline in IKBKAP mRNA expression that allows estimation of IKBKAP mRNA levels because of kinetin ingestion. Adverse effects were transient and mild. This is the first report of in vivo IKBKAP splicing modification and strongly suggests kinetin's therapeutic potential in FD and perhaps in other splicing disorders. Furthermore, our findings support our hypothesis that treatments, which target a particular splicing mutation, can be successfully developed.

A Longitudinally Extensive Spinal Cord Lesion Restricted to Gray Matter in an Adolescent Male.

Longitudinally extensive spinal cord lesions (LECL) restricted to gray matter are poorly understood as are their neurodevelopmental repercussions in children. We herein report the critical case of a 13-year-old male presenting with progressive quadriparesis found to have cervical LECL restricted to the anterior horns. Challenged with a rare diagnostic dilemma, the clinical team systematically worked through potential vascular, genetic, infectious, rheumatologic, and paraneoplastic diagnoses before assigning a working diagnosis of acute inflammatory myelopathy. Nuanced consideration of and workup for both potential ischemic causes (arterial dissection, fibrocartilaginous embolism, vascular malformation) and specific inflammatory conditions including Transverse Myelitis, Neuromyelitis Optica Spectrum Disorders (NMOSD), Multiple Sclerosis (MS), Acute Disseminated Encephalomyelitis (ADEM), and Acute Flaccid Myelitis (AFM) is explained in the context of a comprehensive systematic review of the literature on previous reports of gray matter-restricted longitudinally extensive cord lesions in children. Treatment strategy was ultimately based on additional literature review of treatment-refractory acute inflammatory neurological syndromes in children. A combination of high-dose steroids and plasmapheresis was employed with significant improvement in functional outcome, suggesting a potential benefit of combination immune-modulatory treatment in these patients. This case furthermore highlights quality clinical reasoning with respect to the elusive nature of diagnosis, nuances in neuroimaging, and multifocal treatment strategies in pediatric LECL.

Training scientists as future industry leaders: teaching translational science from an industry executive's perspective.

PhDs and post-doctoral biomedical graduates, in greater numbers, are choosing industry based careers. However, most scientists do not have formal training in business strategies and venture creation and may find senior management positions untenable. To fill this training gap, "Biotechnology Industry: Structure and Strategy" was offered at New York University School of Medicine (NYUSOM). The course focuses on the business aspects of translational medicine and research translation and incorporates the practice of business case discussions, mock negotiation, and direct interactions into the didactic. The goal is to teach scientists at an early career stage how to create solutions, whether at the molecular level or via the creation of devices or software, to benefit those with disease. In doing so, young, talented scientists can develop a congruent mindset with biotechnology/industry executives. Our data demonstrates that the course enhances students' knowledge of the biotechnology industry. In turn, these learned skills may further encourage scientists to seek leadership positions in the field. Implementation of similar courses and educational programs will enhance scientists' training and inspire them to become innovative leaders in the discovery and development of therapeutics.

Targeted drug discovery and development, from molecular signaling to the global market: an educational program at New York University, 5-year metrics.

Drug discovery and development (DDD) is a collaborative, dynamic process of great interest to researchers, but an area where there is a lack of formal training. The Drug Development Educational Program (DDEP) at New York University was created in 2012 to stimulate an improved, multidisciplinary DDD workforce by educating early stage scientists as well as a variety of other like-minded students. The first course of the program emphasizes post-compounding aspects of DDD; the second course focuses on molecular signaling pathways. In five years, 196 students (candidates for PhD, MD, Master's degree, and post-doctoral MD/PhD) from different schools (Medicine, Biomedical Sciences, Dentistry, Engineering, Business, and Education) completed the course(s). Pre/post surveys demonstrate knowledge gain across all course topics. 26 students were granted career development awards (73% women, 23% underrepresented minorities). Some graduates of their respective degree-granting/post-doctoral programs embarked on DDD related careers. This program serves as a framework for other academic institutions to develop compatible programs designed to train a more informed DDD workforce.