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Penetrating Brain’s Natural Barrier to Deliver Life-Saving Drugs

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By: Ruthie Edelstein

More than 400 million people in the world suffer from brain diseases that cannot be treated with modern drugs (biological therapies such as antibodies) because of one major obstacle: a barrier to the brain.

For the average healthy person, the blood-brain barrier (BBB), an extra layer found on blood vessels in the brain, is what protects and defends the sensitive organ from harmful substances, germs and many other sources of potential damage.

But when it comes to treating brain cancer and neurodegenerative diseases, the blood-brain barrier is less of a protective layer and more of a hindrance to effective treatments, presenting one of the biggest medical challenges in modern medicine today.

Nanocarry Therapeutics is hoping to bring an end to this devastating situation. The Ness Ziona-based biopharmaceutical company is developing a novel class of drugs, based on their innovative nano-platform technology, which are able to increase penetration to the BBB and deliver potentially life-saving therapies to the brain.

“This platform has limitless possibilities because we can attach any antibody to it and deliver it to the brain,” Michal Roytman Haham, Chief Business Officer of Nanocarry, tells NoCamels.

With a successful non-invasive technology for traversing the blood-brain barrier, Nanocarry could ultimately extend the reach of already existing therapies to the brain – a location that was previously out of reach.

To get the process started, the company selected HER2 positive breast cancer brain metastases as the area of focus for their first drug development.

The Mayo Clinic explains HER2 positive metastatic breast cancer as a condition that tests positive for a protein called human epidermal growth factor receptor 2 (HER2), which promotes the growth of cancer cells.

Cancer cells growing on healthy tissue. Nanocarry says it can help innovations in cancer treatments include tumors that have spread to the brain (Image: Depositphotos)

Nanocarry says it chose to tackle this medical condition because there is already a highly effective treatment for HER2 metastatic breast cancer, but it is in desperate need of what the company is offering – a successful platform to deliver the drug through the BBB to provide direct treatment to the brain.

Roytman Haham says that while HER2 positive metastatic breast cancer has undergone a “really dramatic revolution” over the past two decades, primarily due to the introduction of biological therapies, this unfortunately does not include the cases that have spread to the brain, which happens in 50 percent of patients, because of the inability of these biological drugs to cross the BBB in sufficient quantities.

“The drugs that normally work very well outside the brain cannot enter the brain and therefore, the prognosis actually plummets to less than one year,” Roytman Haham tells NoCamels.

“This is exactly what we were looking for when searching for a disease where our technology can make a real difference in patients’ lives. A disease that has a systemic and brain manifestation and while the systemic disease, meaning outside the brain, is treated effectively, the brain remains unreachable,” she says.

“That leads to an extremely tragic situation where women who achieved disease control and potentially still have a life to live, lose their lives because of brain metastases.”

Roytman Haham explains that for metastatic HER2 positive breast cancer that has not metastasized to the brain, there has been transformative advancement in treatment over the past 20 years, with the introduction of biological therapy.

Known commercially as Herceptin (trastuzumab) and Perjeta (pertuzumab), these two antibodies are considered the gold standard in treating HER2 positive metastatic breast cancer patients.

With the help of Nanocarry’s BBB technology platform, Herceptin and Perjeta could penetrate the blood-brain barrier, together, to reach the brain and deliver treatment directly to the site of the tumor, potentially transforming the survival of patients with brain metastases as well.

Indeed, the US government’s National Cancer Institute says HER2 positive breast cancer that has not spread to any other organs in the body or the axillary lymph nodes has a 5-year relative survival rate of between 97.3 to 98.8 percent.

Roytman Haham stresses that Nanocarry is not the only company that targets blood-brain barrier penetration, with both invasive and noninvasive technologies already under development.

While the technologies that are invasive do disrupt the blood-brain barrier, the noninvasive technologies are based on a scientific concept called the “trojan horse,” which leverages the brain’s natural processes by targeting receptors on the BBB, enabling large molecules to enter the brain.

Nanocarry says it has innovatively combined this approach with nanoparticle technology to enhance its effectiveness. The company is using insulin as its trojan horse, attached to nano-particles that also carry multiple copies of antibodies.

The insulin binds to insulin receptors that are abundant on the BBB cells, resulting in quick internalization across the barrier and providing efficient drug delivery.

What sets Nanocarry apart from the other “trojan horse” technologies is that it is the only company able to utilize insulin as the shuttle molecule, made possible by the distinctive design of their gold nanoparticles platform. Insulin is considered to be 10 times more effective than other shuttle molecules in penetrating the blood-brain barrier and delivering treatment.

This capability holds great promise in delivering sufficient doses required for diseases such as breast cancer brain metastases.

“It remains stable, and it reaches the brain, safely delivering the drug in high amounts. So basically, we’re the only company that has been able to devise this very, very effective shuttle across the blood-brain barrier,” Roytman Haham says.

The first therapy being developed with Nanocarry’s BBB technology carries multiple copies of Herceptin and Projeta, delivering them directly to the tumor site inside of the brain.

The potentially lifesaving technology was developed over the course of a decade at the Bar Ilan University Faculty of Engineering, in the lab of Prof. Rachela Popovtzer, a world expert in nanomedicine and today Nanocarry’s chief scientific officer. And the solution was formulated by Nanocarry COO Dr. Oshra Betzer as part of her PhD and postdoctoral work.

Popovtzer and Betzer founded Nanocarry in 2021, along with its CEO and experienced biotech executive Dr. Revital Mandil Levin. Today, the three women founders run the company with their small team, also made up primarily of women.

Having successfully completed proof of concept studies in animal models, the biopharmaceutical company is now starting their studies towards receiving Investigational New Drug (IND) status by the US Food and Drug Administration (FDA), which is granted to experimental therapies for serious or immediately life-threatening conditions that show promise in clinical testing.

With the urgent unmet needs in treatment of brain metastases in breast cancer patients, Roytman Haham explains, the FDA usually expedites the approval process for pharmaceutical companies in order to bring such life-saving treatments to market as soon as possible.

Based on the timeline for drugs that have been approved for similar life-threatening diseases, Nanocarry are confident that they will have that fast track to market. Nanocarry expects to run two clinical trials, which are estimated to begin next year, followed by the expedited FDA approval.

“Our results are truly unprecedented,” says Roytman Haham.

“We’ve shown massive brain penetration and accumulation within the tumor site and very significant tumor growth inhibition – exactly the effect these drugs achieve outside the brain.”

In its early days, Nanocarry’s main backer was the US-Israeli venture capital firm NFX. Additional firms now also provide funding, including Sapir Venture Partners and UnBox Ventures, the on-site venture studio at Bar-Ilan University. It has also received support from the Israel Innovation Authority, the branch of the government dedicated to promoting the national high-tech sector, and several private investors.

Nanocarry is focusing first on FDA approval, which it intends to follow with EU authorization, and hopes to be on the market in 2028.

If they achieve their date target, Nanocarry will have made it to market just seven years after the company’s founding. The development time of new drugs takes on average 10 to 15 years, and even longer for newer branches of medicine.

Ultimately, Nanocarry hopes to continue to develop their BBB drugs and provide more effective forms of treatment for many of the other devastating brain diseases for which treatment has proven elusive.

“Potentially, we can attach any drug and deliver it to the brain,” Roytman Haham concludes, “unlocking limitless possibilities in a wide range of central nervous system diseases, including Alzheimer’s disease, Parkinson’s disease, neuro-inflammatory diseases and beyond.”

          (NoCamels.com)

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