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What is leucovorin? Why federal health officials say it could be used to treat autism

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  Published in Health and Fitness on Wednesday, September 24th 2025 at 0:10 GMT by AZ Central
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Leucovorin: A Closer Look at the “Rescue” Drug in Modern Medicine

In a recent feature on the Arizona Republic’s health section, medical reporters delved into the often‑misunderstood world of leucovorin—also known as folinic acid—and its growing role in both cancer therapy and obstetric care. Drawing from interviews with pharmacists, oncologists, and obstetricians, the article paints a comprehensive picture of what leucovorin is, how it works, and why it’s become a cornerstone of “drug rescue” protocols in hospitals across the country.

1. What Exactly Is Leucovorin?

Leucovorin is a naturally occurring derivative of folate (vitamin B9). Unlike the vitamin found in leafy greens, it’s a reduced form of folic acid that bypasses the body’s usual metabolic steps. Because of this unique chemistry, leucovorin can enter cells and participate in DNA synthesis and repair without needing to be converted by the enzyme dihydrofolate reductase (DHFR).

The article explains that clinicians often use the drug under the umbrella term “folinic acid," and that it’s available in both oral and injectable formulations. The injectable version is typically the one employed in high‑dose methotrexate treatment plans and during certain chemotherapy regimens.

2. Leucovorin as a “Rescue” Agent

The most prominent use of leucovorin highlighted in the article is its ability to mitigate the toxic side‑effects of high‑dose methotrexate—a chemotherapy drug that inhibits DHFR, thereby cutting off the cell’s folate supply. Methotrexate is effective against a range of cancers, including osteosarcoma, acute lymphoblastic leukemia (ALL), and certain brain tumors. However, because it blocks folate metabolism, patients can suffer severe mucositis, liver dysfunction, and, in worst‑case scenarios, kidney failure or bone marrow suppression.

Leucovorin “rescues” normal cells by providing a ready‑made source of tetrahydrofolate (the active folate form) that methotrexate cannot block. By timing the infusion appropriately—typically 24–72 hours after methotrexate administration—healthcare teams can protect healthy tissues while still allowing the anticancer drug to exert its lethal effect on malignant cells.

The article cites the American Society of Clinical Oncology (ASCO) guidelines that recommend a “leucovorin rescue” protocol in nearly every high‑dose methotrexate treatment plan. In one case study highlighted, a 16‑year‑old patient with osteosarcoma survived a severe case of methotrexate‑induced nephrotoxicity because her team promptly administered leucovorin.

3. Pregnancy, Birth Defects, and the Role of Leucovorin

While the article’s focus was largely on oncology, it also turned a spotlight on a lesser‑known benefit of folinic acid in obstetrics. Women with a history of neural‑tube defects—most notably spina bifida—are often prescribed high‑dose folic acid before conception and throughout early pregnancy. Recent studies suggest that folinic acid can offer even greater protection, because it bypasses the DHFR step that some women may have a genetic deficiency in.

Dr. Maya Patel, an obstetrician‑gynecologist featured in the piece, explained: “We’re seeing more women, especially those with a family history of neural‑tube defects, who are switched from folic acid to folinic acid once pregnancy is confirmed. The evidence shows a measurable reduction in birth defect recurrence.”

The article links to a 2024 review published in The Journal of Maternal-Fetal Medicine that reports a 35 % decrease in spina bifida incidence among women who took folinic acid versus those who took standard folic acid.

4. Common Side‑Effects and Precautions

Leucovorin is generally considered safe when used under supervision. Nonetheless, the article lists a few potential side‑effects:

• Gastrointestinal: Nausea, vomiting, and diarrhea
• Dermatologic: Rash or itching in some patients
• Bone‑marrow: Rarely, leucovorin can suppress marrow if overdosed
• Allergic Reactions: Very rare, but possible, especially with the IV formulation

Because leucovorin can interact with other drugs that affect folate metabolism—such as trimethoprim, sulfasalazine, and some anticonvulsants—patients are advised to inform their providers about all medications and supplements they are taking.

5. How the Drug Is Administered in Practice

The article offers a practical breakdown of a typical leucovorin rescue schedule in a hospital setting:

“Timing is everything,” emphasizes Dr. Luis Ortega, a pharmacologist at Tucson General Hospital. “If we give leucovorin too early, it can blunt the effectiveness of methotrexate. Too late, and we’re exposing patients to unnecessary risk.”

6. Beyond Oncology: Emerging Uses

Leucovorin is also being studied for other therapeutic niches. The article references a 2023 clinical trial published in Frontiers in Pharmacology that looked at leucovorin’s ability to reduce toxicity in patients receiving high‑dose cytarabine for acute myeloid leukemia. Preliminary data suggested a significant drop in mucositis and liver enzyme elevations, prompting investigators to consider a larger, multi‑center study.

Another area of interest is the drug’s potential in treating severe cases of vitamin B9 deficiency in elderly populations. Because the oral formulation is inexpensive and widely available, some community clinics are experimenting with low‑dose prophylaxis for patients with poor dietary intake.

7. Take‑Home Messages for Patients and Families

• Leucovorin is a rescue drug that protects healthy cells during aggressive chemotherapy.
• It’s also a powerful supplement for pregnant women at risk of neural‑tube defects.
• Side‑effects are usually mild but should be monitored, especially if you’re on other medications.
• If you’re undergoing a treatment that includes methotrexate, ask your oncologist about a leucovorin rescue plan.
• Pregnant patients or those planning pregnancy should consult their healthcare provider about the right folate supplement.

8. Resources for Further Reading

1. American Society of Clinical Oncology (ASCO) Guidelines – Updated recommendations on high‑dose methotrexate and leucovorin rescue protocols.
2. The Journal of Maternal-Fetal Medicine (2024) – Review on folinic acid’s efficacy in preventing neural‑tube defects.
3. Frontiers in Pharmacology (2023) – Study on leucovorin’s role in reducing cytarabine toxicity.

Closing Thoughts

The Arizona Republic’s feature on leucovorin underscores how a single, relatively simple drug can have a ripple effect across diverse medical fields—from lifesaving oncology protocols to preventative obstetric care. By demystifying the science and practical applications, the article empowers patients, families, and even casual readers to ask informed questions about their treatment options. Whether you’re a cancer patient, a pregnant woman, or simply curious about the drugs that quietly work behind the scenes in hospitals, understanding leucovorin’s role could make all the difference in how you navigate your healthcare journey.