Clinical Trial

• The Trump administration announced a multi-pronged initiative on Monday aimed at strengthening the early clinical trial ecosystem within the United States. • As part of this effort, the FDA will reconsider Regenxbio’s gene therapy designed to treat Hunter syndrome, a rare pediatric disease. • This move represents a reversal of previous regulatory rejections, signaling a shift in how the agency evaluates certain high-stakes therapies.

A clinical trial testing two drugs against the Bundibugyo ebolavirus, which is driving a fast-moving outbreak in Central Africa, is set to begin next week, WHO officials said.

• The U.S. Department of Health and Human Services (HHS) has launched a coordinated, department-wide initiative to restore American leadership in clinical research. • The ONC is working to integrate clinical trial notifications into electronic health records to connect eligible patients with research opportunities during routine care. • ARPA-H is implementing modernization programs, including THRIVE and CATALYST, which utilize AI and machine learning to optimize dosing and predict safety before patient enrollment.

• A federal grand jury in the Southern District of Florida charged a medical doctor and two staff members for falsifying data during clinical drug trials. • Starting around 2019, the defendants allegedly fabricated testing records to falsely indicate that human subjects had taken medications and undergone required protocol testing. • The scheme targeted trials sponsored by a pharmaceutical development company intended to evaluate the safety and efficacy of new drugs for FDA approval.

• Former President Trump signed an executive order in April titled “Accelerating Medical Treatments for Serious Mental Illness” to fast-track research and drug approvals for psychedelic-assisted therapy. • The order aims to increase clinical trial participation and explore innovative research models to treat serious mental health conditions. • This federal push provides a new path for psychedelic research in Missouri, where state lawmakers have failed for several years to pass similar legislation.

• Missouri State Rep. Dave Griffith is advocating for psychedelic-assisted therapy to treat veterans suffering from depression and post-traumatic stress disorder. • The initiative follows an April executive order signed by Donald Trump titled “Accelerating Medical Treatments for Serious Mental Illness,” which aims to speed up drug approvals and clinical trial participation. • The order directs the Secretary of Health and Human Services to allocate at least $50 million to support state governments developing programs for psychedelic drugs.

• Merck (known as MSD outside the U.S. and Canada) announced the publication of new clinical trial work on May 7, 2026. • The announcement was distributed via Business Wire from Rahway, N.J., targeting both healthcare professionals and consumers. • These updates are critical for tracking new drug approvals, safety alerts, and updated indications for medical treatments.

• A new clinical trial launched April 24, 2026 (NCT07549529), examines postural motor activation deficits following mild traumatic brain injury (mTBI) in US patients. • The study translates lab findings into physical therapy protocols for concussion rehabilitation, focusing on vestibular and balance impairments. • Affecting millions annually, mTBI recovery gaps could improve with targeted interventions, potentially reducing long-term disability rates by 30%.

• HHS through the National Cancer Institute announced a $200 million public-private partnership to fund clinical trials for cancer vaccines targeting high-risk recurrence patients. • The initiative supports mRNA and other vaccine types, aiming to accelerate development with private sector involvement including venture capital. • 'Nothing of this scale has been attempted in cancer vaccines before,' said Stacey Adam, VP of science partnerships for the Foundation for the National Institutes of Health.

• A Phase 2 clinical trial led by Memorial Sloan Kettering Cancer Center demonstrated that engineered CAR-T cells targeting mesothelin showed a 52% overall response rate in 89 patients with advanced pancreatic and ovarian cancers. • The trial, results published March 19 in Cancer Cell, showed durable responses lasting over one year in 68% of responding patients, compared to historical response rates below 20% with conventional chemotherapy. • CAR-T therapy has revolutionized blood cancer treatment but remained ineffective against solid tumors until recent engineering advances, potentially opening a new therapeutic avenue for 400,000 Americans diagnosed with pancreatic and ovarian cancers annually.