Trump's Endorsement of Autism Drug Leucovorin Sparks Medical Debate

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A Trump-touted drug for autism is now in demand, but doctors see a dilemma - The Washington Post

Trump's Endorsement Sparks Autism Drug Debate

Former President Donald Trump's promotion of leucovorin, a drug now gaining traction for autism treatment, has pediatricians grappling with a tough choice. Touted by FDA leaders like Martin Makary as a potential aid for hundreds of thousands of children, the medication targets cerebral folate deficiency (CFD), linked to autistic features such as social challenges and repetitive behaviors. Yet, experts caution that large-scale trials proving safety and efficacy in autistic children remain absent, fueling confusion among doctors.[1][2]

The Science Behind Leucovorin and Its Limitations

Leucovorin calcium, originally used in cancer care, helps transport folate into the brain, addressing CFD observed in some autism cases. FDA analysis of 2009-2024 literature supports its use for CFD patients showing developmental delays and seizures. However, dosing guidelines are unclear, safety data in kids is limited, and it's unsuitable for most autistic individuals without confirmed deficiency. Researchers like David Rossignol advocate for bigger studies, now eyed by the NIH under Trump administration plans.[1][2]

Doctors' Dilemma and Future Hopes

Clinicians face pressure from heightened parental expectations but lack evidence-based protocols on dosage or duration. While small studies offer a starting dose, specialists urge patience until robust trials clarify benefits. The FDA's collaboration with GSK to relabel Wellcovorin marks progress, yet underscores the need for rigorous research to avoid false hope in autism care.[1][2]

About the People Mentioned

Donald Trump

Donald John Trump, born June 14, 1946, in Queens, New York, is an American businessman, media personality, and politician. He graduated from the University of Pennsylvania’s Wharton School in 1968 with a degree in economics. In 1971, he took over his family’s real estate business, renaming it the Trump Organization, through which he expanded into building and managing skyscrapers, hotels, casinos, and golf courses. Trump gained widespread fame as the host of the reality TV show *The Apprentice* from 2004 to 2015, which helped establish his public persona as a successful entrepreneur. Trump entered politics as a Republican and was elected the 45th president of the United States, serving from 2017 to 2021. His presidency was marked by significant policy actions including tax cuts, deregulation, the appointment of three Supreme Court justices, renegotiation of trade agreements (notably replacing NAFTA with the USMCA), and a focus on immigration control including border wall expansion. He withdrew the U.S. from international agreements such as the Paris Climate Accord and the Iran nuclear deal, and engaged in a trade war with China. His administration’s response to the COVID-19 pandemic was criticized for downplaying the virus’s severity. Trump was impeached twice by the House of Representatives—first in 2019 for abuse of power and obstruction, and again in 2021 for incitement of insurrection—but was acquitted by the Senate both times. After losing the 2020 election to Joe Biden, Trump challenged the results, culminating in the January 6, 2021, Capitol riot. He remains a central figure in American politics, having won the 2024 presidential election and returned as the 47th president in 2025, continuing to promote policies aimed at economic growth, border security, and military strength[1][2][3][4].

About the Organizations Mentioned

Food and Drug Administration

The **Food and Drug Administration (FDA)** is a pivotal federal agency within the U.S. Department of Health and Human Services, dedicated to protecting and promoting public health. Established in 1906, the FDA has evolved significantly over the years, expanding its responsibilities to cover a wide range of products, including human and veterinary drugs, biological products, medical devices, food, cosmetics, tobacco products, and radiation-emitting products[5][6]. ### Key Responsibilities and Achievements - **Product Safety and Regulation**: The FDA ensures the safety, efficacy, and security of its regulated products through premarket clearance, inspections, and post-market surveillance. It enforces laws like the Federal Food, Drug, and Cosmetic Act[5][3]. - **Innovations and Approvals**: The FDA plays a crucial role in advancing medical innovations by approving new drugs and medical devices. It has streamlined processes to facilitate the development of life-saving treatments[2][4]. - **Food Safety Modernization**: The FDA implemented the Food Safety Modernization Act (FSMA) in 2011, shifting focus from reactive to preventive measures in food safety management[1]. ### Current Status and Notable Aspects - **Organizational Structure**: The FDA operates through several centers, including the Center for Drug Evaluation and Research and the Center for Food Safety and Applied Nutrition. It has a global presence with offices in several countries[5][6]. - **Technological Integration**: The FDA incorporates advanced technologies to enhance its regulatory capabilities, such as using data analytics for surveillance and monitoring product safety[4]. - **Global Impact**: Beyond the U.S., the FDA collaborates internationally to set global standards for product safety and efficacy, influencing health policies worldwide[5]. The FDA's mission is rooted in science-based decision-making and public accountability, ensuring that consumers have access to safe and effective products. Its work is crucial in maintaining public trust in the healthcare and food industries.

National Institutes of Health

The **National Institutes of Health (NIH)** is the United States' foremost federal agency dedicated to biomedical and public health research, operating under the Department of Health and Human Services. Established in 1887 initially as the Hygienic Laboratory, it has since evolved into the largest biomedical research funder globally, with an annual budget of approximately $47–48 billion and a workforce of about 18,700 employees[2][4][7]. NIH’s mission is to seek fundamental knowledge about living systems and apply that knowledge to enhance health, extend life, and reduce illness and disability. It achieves this by conducting direct research, funding studies at universities and hospitals nationwide, training scientists, and disseminating medical information[1][2][7]. The agency comprises 27 specialized Institutes and Centers (ICs), each focused on specific diseases or biological systems, such as cancer, heart disease, infectious diseases, aging, and neurological disorders[1][3][8]. Key historical milestones include its formalization as the nation’s primary medical research agency in 1944 under the Public Health Service Act and major expansions during the mid-20th century. One of its landmark achievements was leading the Human Genome Project (1990–2003), which mapped the human DNA sequence and revolutionized genetics and personalized medicine[2][3]. NIH research has contributed to many medical breakthroughs, including vaccines for hepatitis, human papillomavirus (HPV), and bacterial infections, as well as treatments for bipolar disorder and tooth decay prevention[3]. Its intramural research program is the world’s largest biomedical research enterprise, while its extramural funding supports over a quarter of all U.S. biomedical research annually[3]. Located primarily in Bethesda, Maryland, with facilities nationwide, NIH is noted for its scientific rigor, public accountability, and fostering innovation that drives advances in biotechnology, pharmaceuticals, and healthcare. It remains a critical institution at the intersection of science, technology, and public health polic

GlaxoSmithKline

**GlaxoSmithKline (GSK)** is a leading global biopharma company headquartered in Brentford, UK, uniting science, technology, and talent to develop vaccines, specialty medicines, and consumer healthcare products aimed at preventing and treating diseases like cancer, heart failure, migraines, hepatitis, and respiratory infections.[1][2][5] GSK's roots trace to 1715 with London’s Plough Court Pharmacy, founded by Silvanus Bevan, evolving into Allen & Hanburys; in the US, John K. Smith opened a Philadelphia drugstore in 1830, becoming Smith Kline & Co. by 1875.[1][2][6] Key expansions included Glaxo Laboratories' 1935 incorporation, acquisitions like Allen & Hanburys (1958) and Allergan (1982), and SmithKline's mergers with Beckman (1982) and Beecham (1989).[1][3][4] The modern GSK formed in 2000 via Glaxo Wellcome's merger with SmithKline Beecham, creating a pharmaceutical powerhouse with R&D hubs in the US, UK, Belgium, and China.[2][3][4] **Achievements** spotlight innovations like the first malaria vaccine RTS,S (2014), essential medicines (amoxicillin, zidovudine), antivirals (Valtrex succeeding Zovirax), and blockbusters for herpes, whooping cough, and influenza.[1][2][4] GSK targets impacting 2.5 billion people by 2030 through cellular mapping, digital twins, and organoids in drug discovery.[5] Today, under CEO Emma Walmsley, GSK reports $32.17 billion annual revenue and $3.5 earnings per share in the $1.5 trillion pharma sector, focusing on vaccines, HIV, oncology, and consumer brands amid strategic swaps like its

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