Micro Brain Robot Granted Orphan Status

Brain tumor graphic — (National Science Foundation)

23 June 2021. A tiny remote-controlled device that carries chemotherapy drugs to brain tumors received orphan drug status from the Food and Drug Administration. Bionaut Labs in Los Angeles says FDA granted the designation to the company’s device-drug combination code-named BNL-101 as a treatment for malignant gliomas in children and adults.

Bionaut Labs calls its therapy delivery a form of precision medicine, in that the robotic bionauts can deliver biologics, gene therapies, or small molecule drugs directly to precise targets in the brain. The company’s first applications are treatments for gliomas, a form of tumor affecting glial cells that support neurons or nerve cells in the brain. Bionaut Labs is also designing bionaut deliveries for Parkinson’s disease.

Bionaut deliveries are made with tiny robotic devices, custom designed and less than a millimeter in size. The devices move by remote control from external magnetic directors, and are shaped and configured with moving parts to interact with different types of tissue where the payloads are delivered. BNL-101 is designed to carry and deliver the chemotherapy drug doxorubicin, usually given as a systemic infusion to treat a number of solid tumor and blood-related cancers.

The company says Bionauts are designed to travel through cerebrospinal fluid, brain ventricles, and brain tissue itself thus bypassing the blood-brain barrier that impedes delivery of many drugs to the brain. And because its therapies are designed to work on precise targets in the brain or spinal cord, says Bionaut Labs, the treatments avoid adverse off-target effects as some systemic drugs.

Incentives to develop drugs affecting small numbers of patients

Gliomas are a family of tumors affecting glial cells, ranging from low-grade and slow-growing tumors to very aggressive and malignant glioblastoma tumors. Glioblastoma occurs in 3.19 of 100,000 individuals in the U.S., more often in males than females, and more White than Black or people of other races. “Gliomas,” says Bionat Labs’ chief scientist Alex Kiselyov in a company statement released through BusinessWire, “remain some of the most devastating tumors for which there are few, if any, effective treatment options and for which there remains significant unmet medical need.”

The Orphan Drug Act, first passed in 1983, gives FDA authority to designate an orphan drug for treatments being developed for diseases affecting fewer than 200,000 Americans. Therapies, both drugs and biologics, designated as orphan drugs qualify for incentives such as tax credits for clinical trials, exemptions from marketing application fees, and extended market exclusivity time.

“The granting of orphan drug designation for BNL-101,” notes Bionaut Labs co-founder and CEO Michael Shpigelmacher, “represents a significant milestone for Bionaut Labs as it recognizes the potential of our approach to transform the standard of care for devastating CNS diseases like malignant gliomas.”

Bionaut Labs started-up in 2016. In March, as reported by Science & Enterprise, the company emerged from stealth mode and raised $20 million in its first venture funding round.