Understanding Normal Pressure Hydrocephalus: What is the drug of choice for normal pressure hydrocephalus?

October 13, 2025 •

3 min read

While some conditions are managed with medication, there is currently no approved drug of choice for normal pressure hydrocephalus (NPH). For most patients, the gold standard treatment involves surgical intervention to drain excess cerebrospinal fluid (CSF).

Quick Summary

The standard and most effective treatment for normal pressure hydrocephalus is surgical shunting, not medication. While some drugs, like acetazolamide, have been explored for temporary use, they do not offer a long-term solution. Research continues into pharmacological alternatives, but shunting remains the primary therapy.

Key Points

No Drug of Choice: There is currently no approved medication considered the drug of choice for normal pressure hydrocephalus.
Surgical Shunting is Standard Care: The most effective and standard treatment is the surgical placement of a ventriculoperitoneal (VP) shunt to drain excess CSF.
Limited Role for Acetazolamide: The carbonic anhydrase inhibitor acetazolamide is used off-label or temporarily, but it is not a long-term solution for NPH.
Positive Shunt Predictors: In some patients, a positive response to a diagnostic spinal tap or acetazolamide test can predict a better outcome with shunt surgery.
Pharmacology is Still Investigated: Research is ongoing for new pharmacological treatments, including studies on SGLT2 inhibitors, but these are still experimental.
Early Treatment Improves Outcomes: The earlier NPH is diagnosed and treated with a shunt, the better the chances are for significant symptom improvement.
Surgical Risks Exist: Shunt surgery carries risks such as infection and malfunction, which must be carefully weighed against the potential benefits.

The Role of Surgical Intervention in NPH

For individuals diagnosed with normal pressure hydrocephalus (NPH), the most effective and widely accepted treatment is the surgical implantation of a shunt. A shunt is a medical device that drains excess cerebrospinal fluid (CSF) from the brain's ventricles to another part of the body, most often the abdomen, where the fluid can be absorbed safely. This redirection of fluid helps relieve the pressure on the brain that causes NPH symptoms, which can include gait disturbances, cognitive decline, and urinary incontinence. A successful shunt procedure can lead to significant improvement in these symptoms, particularly when diagnosis and treatment occur early in the disease course.

There are several types of shunts used, including:

Ventriculoperitoneal (VP) Shunt: The most common type, which drains CSF from the brain to the abdominal (peritoneal) cavity.
Ventriculoatrial (VA) Shunt: An alternative option, where CSF is diverted into the heart's right atrium.
Programmable Valves: Many modern shunts feature adjustable valves that allow a physician to non-invasively change the drainage rate to optimize symptom relief and minimize complications like over- or under-drainage.

Limitations of Pharmacological Approaches

Unlike many other neurological conditions, NPH does not have a specific, approved pharmacological treatment. The core issue in NPH is a mechanical problem with CSF absorption, which most medications cannot effectively address long-term. While some drugs have been investigated, their use is limited, off-label, or experimental.

Acetazolamide: Limited and Off-Label Use

Acetazolamide, a carbonic anhydrase inhibitor, has been used to temporarily decrease CSF production in some instances. However, it is not a cure for NPH and its efficacy for long-term symptom management is inconsistent. In fact, one of its more established uses in the context of NPH is as a diagnostic aid. A transient reduction in intracranial pressure following an acetazolamide bolus has been observed to be predictive of a positive response to shunting in some patients.

Emerging Research on SGLT2 Inhibitors

In a promising but still early development, researchers are investigating the potential of sodium/glucose cotransporter 2 (SGLT2) inhibitors for treating NPH. These diabetes medications inhibit a receptor found not only in the kidneys but also in the brain's choroid plexus, which produces CSF. Early observations in patients taking SGLT2 inhibitors for type 2 diabetes have shown a reduction in brain ventricle size. Clinical trials are underway to formally evaluate their effectiveness and safety in NPH patients.

Drugs with No Proven Benefit

Other drugs have been anecdotally tested for NPH but lack definitive evidence of effectiveness. Levodopa/carbidopa, commonly used for Parkinson's disease, showed no reliable benefit in NPH patients and may simply have improved misdiagnosed cases of parkinsonism. General diuretics are also not effective for managing NPH symptoms.

Comparison: Surgical vs. Pharmacological Treatment for NPH


Feature	Surgical Shunting	Pharmacological Treatment (e.g., Acetazolamide)
Efficacy	The most effective option; often provides significant and lasting relief of symptoms, especially gait problems.	Very limited efficacy; may offer temporary, modest relief in a small number of patients.
Mechanism	Mechanically drains excess CSF from the ventricles to another part of the body, correcting the fluid imbalance.	Reduces the rate of CSF production by inhibiting carbonic anhydrase, but does not address the underlying absorption issue.
Risk Profile	Involves surgical risks, including infection, hemorrhage, and shunt malfunction, which may require additional procedures.	Associated with risks and side effects, such as nephrocalcinosis and metabolic acidosis, and is not suitable for long-term use.
Best Application	The standard of care for most NPH patients who are candidates for surgery and demonstrate a positive response to diagnostic CSF removal.	Sometimes used as a temporary measure when shunting is contraindicated or as a test to predict shunt responsiveness.

Conclusion

While a definitive drug of choice for normal pressure hydrocephalus does not exist, surgical shunting offers a treatable and often reversible solution for many patients. Pharmacological options have a very limited role, acting primarily as temporary measures or diagnostic tools, and are not a substitute for surgery. Emerging research, such as the potential use of SGLT2 inhibitors, offers hope for future pharmacological alternatives, but shunting remains the most reliable treatment. An accurate diagnosis and a comprehensive treatment plan developed in consultation with a specialized medical team are essential for achieving the best possible outcome for patients with NPH. You can find more information about NPH from the Hydrocephalus Association, a reliable resource for patients and families facing this condition. Hydrocephalus Association

Frequently Asked Questions

No, medication is not an effective long-term treatment for NPH. The underlying issue is a mechanical problem with cerebrospinal fluid (CSF) absorption that drugs cannot correct permanently. Surgical shunting is the standard for long-term management.

Acetazolamide is a medication that temporarily reduces CSF production. While it has been explored for off-label use in NPH, it is not a long-term solution. It can sometimes be used as a temporary measure or as part of a test to predict if a patient will respond well to shunt surgery.

Yes, NPH symptoms like gait difficulty and cognitive decline can be mistaken for other conditions such as Parkinson's or Alzheimer's disease. This misdiagnosis is why some drugs for those conditions, like levodopa/carbidopa, were anecdotally reported to help, but are now understood to be ineffective for NPH itself.

A VP shunt is a surgical device used to drain excess cerebrospinal fluid from the brain into the abdomen, where it is naturally absorbed by the body. This reduces the pressure on the brain's ventricles and often alleviates NPH symptoms.

Yes, drugs like acetazolamide can have side effects, including metabolic consequences such as electrolyte imbalances. This is one reason they are not suitable for long-term NPH management.

Doctors often perform diagnostic tests, such as a high-volume lumbar puncture or a lumbar drain trial, to see if removing a small amount of CSF improves the patient's symptoms. A positive response to this test suggests the patient is a good candidate for a permanent shunt.

NPH is a progressive condition, and if left untreated, the symptoms—including gait disturbances, cognitive decline, and urinary incontinence—will worsen over time. Early diagnosis and treatment are critical for the best outcomes.

Yes, researchers are exploring new pharmacological options. A current area of interest is the potential use of SGLT2 inhibitors, a class of drugs used for diabetes, which may help reduce CSF production. Clinical trials are underway to study their effectiveness for NPH.