Brain tumors are one of the most common cancers in children. And although long-term survival is over 50%, it often comes at a cost. Because their brains are just developing, the toxicity of the treatment can affect learning and leave children with lifelong challenges.

One of the barriers to treating tumors in the brain is the literal barrier between the blood and the brain. Chemotherapy drug molecules cannot cross this membrane, which exists in part to keep toxins out of the brain.

A researcher at Rice University seeks to change that, by synthesizing a nanoparticle that can carry a gene-therapy product across the blood-brain barrier. His unique interdisciplinary approach encompasses both materials science and biology.

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Brain tumors are one of the most common cancers in children. And although long-term survival is over 50%, it often comes at a cost. Because their brains are just developing, the toxicity of the treatment can affect learning and leave children with lifelong challenges.

One of the barriers to treating tumors in the brain is the literal barrier between the blood and the brain. Chemotherapy drug molecules cannot cross this membrane, which exists in part to keep toxins out of the brain.

A researcher at Rice University seeks to change that, by synthesizing a nanoparticle that can carry a gene-therapy product across the blood-brain barrier. His unique interdisciplinary approach encompasses both materials science and biology.

Omid Veiseh was recruited to Rice from the Massachusetts Institute of Technology with the help of a CPRIT First-Time Tenure Track Investigator Award in 2017.

His research exploits the fact that the blood-brain barrier is not completely impermeable. The membrane has several different mechanisms to transport needed molecules, such as glucose, across it.

Veiseh hopes to synthesize a nanoparticle that can take advantage of these natural transport mechanisms to cross the barrier. He’ll be using mice to test the particles that he makes.

Inside these nanoparticles, Veiseh plans to insert a small piece of genetic material, which he hopes will gum up a genetic pathway unique to a tumor cell — its Achilles heel. The hope is to deliver a molecule that stops the tumor in its tracks, and prevent its cells from reproducing, without affecting normal cells.

“We start with polymers that are very diverse in composition,” he says. He plans to synthesize thousands of nanoparticles that vary in properties, like their electric charge, and whether or not they dissolve in water, for example. “Certain biologic molecules already cross the barrier, and maybe we’ll find a way to hitch a ride,” he says.

To figure out which of the thousands of nanoparticles he synthesized make it into the brain, and from there, into a tumor cell, Veiseh will essentially bar code each nanoparticle. “Each one will have a unique piece of genetic material inside, in a sequence that doesn’t exist naturally,” he said. “We can inject hundreds or thousands of these nanoparticles at a time into one mouse. So if one appears in the tumor, it would carry a unique payload.”

By removing part of the mouse’s brain tumor and sequencing the DNA, he can see if any nanoparticles hit the target.

Once he finds a candidate nanoparticle, he can experiment with the payload that exploits the tumor’s Achilles heel. Patients would still have to undergo surgery to remove the tumor, but if Veiseh is successful, his nanoparticle gene therapy would prevent any remaining cells from growing or metastasizing — without the collateral damage of traditional chemotherapy or radiation. While it’s still a long way from being commercialized, Veiseh can make and screen thousands of nanoparticles relatively quickly.

An inventor and entrepreneur, Veiseh cofounded a company, Sigilon Therapeutics (Sigilon.com), that uses biomaterials implants to treat diseases like hemophilia or diabetes without triggering a body’s natural immune response.

He received his undergraduate degree from Western Washington University. He received a Ph.D. dual degree in Materials Science & Engineering and Nanotechnology from the University of Washington. At MIT, he was a postdoctoral fellow in Chemical Engineering and Integrative Cancer Research, as well as an affiliate at Boston Children’s Hospital, Harvard Medical School, and the David H. Koch Institute for Integrative Cancer Research.

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