New York University

CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder

7 days ago

• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder. • The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein. • This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.

PRIM-ER Trial Finds Palliative Care Training in Emergency Departments Does Not Reduce Hospital Admissions

4 months ago

The PRIM-ER trial, a significant study involving 29 emergency departments across the United States, found that a comprehensive palliative care training program for emergency staff did not decrease hospital admission rates for seriously ill older adults. Despite the lack of impact on admissions, subsequent healthcare use, or short-term mortality, the trial underscores the importance of developing primary palliative care interventions due to workforce shortages in hospice and palliative medicine.

Innovative Approach to Cancer Treatment: Targeted Protein Degradation

4 months ago

A groundbreaking study by Fred Hutch Cancer Center introduces a novel cancer treatment method, targeted protein degradation, which tricks cells into destroying cancer-causing proteins. This approach, currently in clinical trials, has shown promising results in reducing tumor growth and improving survival rates in mice with lung and pancreas tumors caused by the KRAS G12V mutation.

Psilocybin-Assisted Therapy Shows Promise for Mental Health in Cancer Patients and Alcohol Use Disorder

5 months ago

• Psilocybin-assisted psychotherapy significantly reduced anxiety, depression, and other psychological distress in cancer patients, with benefits lasting up to six months. • In patients with alcohol use disorder, psilocybin therapy led to reduced impulsivity, depression, and increased openness to emotions, observed seven months post-treatment. • Studies highlight the potential of psilocybin, when administered under medical supervision and combined with psychotherapy, to induce lasting, positive personality changes. • Researchers plan to explore the efficacy of psilocybin-assisted psychotherapy for opioid-use disorder, building on observed benefits in alcohol use disorder and cancer patients.

ARPA-H Funds Multi-Institutional Consortium to Cure Blindness Through Eye Transplantation

6 months ago

• The University of Colorado Anschutz Medical Campus will receive up to $46 million from ARPA-H to advance vision-restoring human whole eye transplantation. • The project aims to achieve the first successful vision recovery in six human whole eye transplants by 2030, addressing optic nerve regeneration challenges. • The multi-disciplinary team includes experts from Johns Hopkins, University of Wisconsin, and the National Eye Institute, focusing on innovative technologies. • The Foundation Fighting Blindness will strategically align the consortium's progress with program requirements, driving advancements in treating blinding diseases.

Long COVID Patients Frustrated by Lack of FDA-Approved Treatments Despite Federal Research Funding

6 months ago

• Long COVID patients express frustration over the lack of FDA-approved treatments despite $1.15 billion in federal research funding. • The NIH's RECOVER initiative is criticized for prioritizing observational studies over clinical trials for potential therapies. • An additional $662 million in funding, including $300 million for clinical trials, aims to address the urgent need for effective treatments. • Researchers and patient advocates emphasize the importance of understanding biological mechanisms and prioritizing patient-centered research.

Eisbach Bio Relocates to Houston with $4.75M CPRIT Grant to Advance EIS-12656 Trial in HRD Tumors

6 months ago

• Eisbach Bio is relocating to Houston, supported by a $4.75 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT). • The funding will support Phase 2 clinical trials of EIS-12656, a therapy targeting homologous recombination deficiency (HRD) tumors. • EIS-12656 functions by suppressing cancer-related genome reorganization caused by DNA damage, offering a novel approach to cancer treatment. • CPRIT's investment aims to establish Texas as a hub for innovative cancer treatments and expand clinical options for HRD patients.

AJNA BioSciences Advances Psilocybin Antidepressant Toward FDA Approval

8 months ago

• AJNA BioSciences is developing a standardized, full-spectrum psilocybin antidepressant aimed at meeting strict FDA guidelines for botanical drugs. • Research suggests that psilocybin-assisted therapy could benefit over five million Americans suffering from depression, highlighting a significant market opportunity. • AJNA anticipates initiating a Phase 1 trial for its psilocybin-based antidepressant in early 2025, following a Phase 2 trial for its CBD-based Autism Spectrum Disorder drug in late 2024. • FDA approval is crucial for psilocybin drugs to gain acceptance in routine treatment and for insurance reimbursement, which AJNA is prioritizing.

Coverage Disputes Arise for Sarepta's Duchenne Gene Therapy Elevidys Despite FDA Approval

8 months ago

• Insurers are denying coverage for Sarepta's Elevidys, a gene therapy for Duchenne muscular dystrophy, despite its FDA approval for a broad patient population. • Coverage denials often stem from insurers aligning policies with clinical trial enrollment criteria, which focused on ambulatory patients, creating access barriers for non-ambulatory individuals. • The high cost of Elevidys, priced at $3.2 million, is a significant factor in coverage disputes, with insurers questioning the cost-effectiveness for patients with limited mobility. • Patient advocacy groups and medical professionals are pushing back against restrictive insurance policies, emphasizing the potential for Elevidys to prolong life and preserve function, even in wheelchair-bound patients.