Introduction/Aims: Lipid peroxidation is thought to play a biologically important role in motor neuron death in amyotrophic lateral sclerosis (ALS). 11,11 Di-deuterated linoleic ethyl ester (RT001) prevents lipid peroxidation in cellular and mitochondrial membranes. Herein we report on the use of RT001 under expanded access (EA).Methods: We provided RT001 to patients with ALS via EA at a single site. The starting dose was 2.88 g/day, which was increased to to 8.64 g/day as tolerated. Participants were not eligible for alternative clinical trials. Participants were followed for adverse events and pharmacokinetic (PK) parameters were measured approximately 3 months after RT001 initiation.Results: Sixteen participants received RT001 (5.6 ± 1.6 g/day; dose range, 1.92 to 8.64 g/day) for a mean period of 10.8 ± 7.1 months. After 3 months of treatment, PK studies showed that RT001 was absorbed, metabolized, and incorporated into red blood cell membranes at concentrations expected to be therapeutic based on in vitro models. The most common adverse events were gastrointestinal, including diarrhea, which occurred in 25% of the participants, and were considered possibly related to RT001. One participant (6%) discontinued due to an adverse event. Ten serious adverse events occurred: these events were recognized complications of ALS and none were attributed to treatment with RT001.

The coronavirus disease 2019 (COVID-19) pandemic has challenged the ability of harm reduction programs to provide vital services to adolescents, young adults, and people who use drugs, thereby increasing the risk of overdose, infection, withdrawal, and other complications of drug use. To evaluate the effect of the COVID-19 pandemic on harm reduction services for adolescents and young adults in Boston, we conducted a quantitative assessment of the Community Care in Reach (CCIR) youth pilot program to determine gaps in services created by its closure during the peak of the pandemic (March 19–June 21, 2020). We also conducted semistructured interviews with staff members at 6 harm reduction programs in Boston from April 27 through May 4, 2020, to identify gaps in harm reduction services, changes in substance use practices and patterns of engagement with people who use drugs, and how harm reduction programs adapted to pandemic conditions. During the pandemic, harm reduction programs struggled to maintain staffing, supplies, infection control measures, and regular connection with their participants. During the 3-month suspension of CCIR mobile van services, CCIR missed an estimated 363 contacts, 169 units of naloxone distributed, and 402 syringes distributed. Based on our findings, we propose the following recommendations for sustaining harm reduction services during times of crisis: pursuing high-level policy changes to eliminate political barriers to care and fund harm reduction efforts; enabling and empowering harm reduction programs to innovatively and safely distribute vital resources and build community during a crisis; and providing comprehensive support to people to minimize drug-related harms.

Introduction/Aims: Expanded access protocols (EAPs) are a Food and Drug Administration (FDA)-regulated pathway for granting access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. There is limited information about the use of EAPs in amyotrophic lateral sclerosis (ALS); the aim of this report is to share the design, operational features, and costs of an EAP program for ALS. Methods:The program was launched in 2018 at a single center. In alignment with FDA guidance, protocols were designed as individual (single participant) or intermediate size. Inclusion criteria were broad (e.g., no restrictions due to long disease duration or low vital capacity). Safety information was collected in all EAPs. Selected biomarkers were collected in nine of the EAPs.