Little-Known Cuban Alzheimer’s Drug Goes to Human Trials in Canada

By | May 17th, 2023

Cuba's trial results from its intranasal Alzheimer's drug, NeuroEPO, appear, at a glance, to be extraordinary. Canadian researchers are putting it to the test in a confirmatory trial.

Since the United States imposed an embargo on Cuba in 1961, the island nation has built a robust healthcare system on their own, with little U.S. collaboration. This includes developing their own drugs to treat their population’s most challenging diseases, from efficacious coronavirus vaccines to an experimental vaccine for lung cancer. Now, Cuban researchers are testing experimental neuro-protective drugs for Alzheimer’s.

Their NeuroEPO (brand name NeuralCIM), is currently in the final stages of clinical trials in Cuba. Recently, Cuba began collaborating with Canadian scientists at the University of Saskatchewan to run a confirmatory clinical trial, which will use imaging tools and techniques that have not been available to the Cuban research team. 

According to the Cuban trial data released, NeuroEPO has shown promising results in patients with early-stage Alzheimer’s. The trial was conducted at the Center of Molecular Immunology (Centro de Inmunología Molecular or CIM), a cancer research center in Havana.

The data has not as of now been publicly published or peer-reviewed by outside researchers. What it shows is that the drug appeared to stop cognitive decline from progressing in the majority of trial participants—a reported 80 percent. Being Patient does not have access to information about specific methodology or sample sizes in the study.

Trial administrators also reported that the drug improved cognition in 54 percent of trial participants who received the drug, while 80 percent of participants who received the placebo saw their condition worsen.

This is an extraordinary claim. No known drug so far has ever halted or reversed cognitive decline in humans.

However, there are many studies in mouse and cell models of Alzheimer’s that suggest EPO might treat cognitive impairment. (Mouse and cell results rarely translate to humans.)

In humans, there is less published data on the effects. One 2019 study looked at patients who regularly receive EPO as treatment for end-stage renal disease. People receiving this treatment had a 39-percent lower risk of developing dementia. But it is unclear whether there are other factors influencing this relationship or if the EPO exerts its effect in the brain. 

How does NeuroEPO work?

The drug is a laboratory engineered version of the erythropoietin (EPO) protein. EPO helps make red blood cells but also has neuro-protective properties.  The researchers are using a medical device to deliver the drug intranasally.

Delivering the drug via the nasal passages helps ensure it reaches the brain, while minimizing side effects.

In older populations, high levels of blood increase the risk of mortality and cardiovascular disease.

“Ultimately, the drug gets into the brain through the cerebrospinal fluid, and it may avoid the brain blood brain barrier by getting into the brain directly through the nasal passages,” University of Saskatchewan trial project director Konstantine Sarafis told Being Patient.

Other drugs developed from proteins tend not to be shelf-stable over extended periods of time, and stable storage of these medications can be costly.

What’s next for NeuroEPO?

Medical professors Ron Geyer and Dr. Andrew Kirk at the University of Saskatchewan in Canada are running a Phase 2 trial to confirm these findings.

“The trial provides validation that the trial is done in a westernized country outside of Cuba,” Sarafis said. “We are also using some updated cognitive assessments in addition to specific biomarkers, including beta amyloid, phospho-tau, APOE, and then MRI.”

Meanwhile, CIM also collaborated with researchers in China, at the University of Electronic Science and Technology, between 2015 and 2017, to run small, randomized Phase 1 and 2 human trials, testing NeuroEPO in 26 participants with Parkinson’s — the results suggested that the treatment was promising. In Cuba, the drug has also been tested for stroke and a balancing disorder called ataxia.

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