People who suffer from a rare disease that produces tumors inside the brain and spinal cord are one step closer to getting a potentially less invasive treatment that rivals radiation therapy on the market.
Bionaut Labs, the Los Angeles startup behind tiny micro-robots that can swim through the body and deliver therapeutics, gained a special drug designation from the Food and Drug Administration that could speed up their clinical trials.
The company's goal is to begin clinical trials in 2023.
Bionaut's robotic capsule, which is smaller than a flea, holds doxorubicin (a chemotherapy drug) that can travel to delicate areas to treat malignant gliomas. The tumors often sit in parts of the brain that controls breathing.
Most doctors treat the disease with radiation therapy, a process that can be devastating especially for young patients.
"Because we work in orphan diseases that essentially a lot of very unfortunate patients with unaddressed medical needs have, it's a small but very significant step towards helping this very population," Alex Kiselyov, chief scientific officer for Bionaut Labs, said.
The FDA considers the disease rare since it only occurs in five of every 100,000 people, and gave the treatment the so-called orphan drug designation.
The special designation created in 1983 gives Bionaut access to clinical trial subsidies, patent protections and inflated marketing rights for companies that create new therapeutics. Perhaps as importantly, the FDA is providing transportation and other resources to malignant glioma patients to make it easier for them to access clinical trial sites and this new form of treatment.
Treatments for rare disease are uniquely difficult to push through clinical trials because it's difficult to find patients who can be tracked long-term. This is on top of a slew of problems that can occur during the clinical trial process, where multiple promising drugs have died. Furthermore, a company only has a certain number of years to speed through clinical trials and marketing, and make money off its patent before it expires and generics enter the market, which has failed big companies like Amgen. Most investors don't recoup expensive investments until decades later.
The designations have opened up the market for rare disease drugs, said Eunjoo Pacifici, a professor at the USC School of Pharmacy.
"Now almost half of all brand new drugs that are approved every year...are for rare diseases," she said.
The company still has a ways to go. While the drug itself is approved, the Bionaut platform isn't. The company is pursuing Humanitarian Use Device designation — another created from the agency's need to treat rare diseases — from the FDA.
Correction: This story has been updated to clarify the FDA's designation.
A small group of scientists and engineers are developing a device smaller than a flea that will wind its way inside the heads of people with brain tumors and deliver life-saving treatments.
Bionaut Labs, a Los Angeles startup, unveiled on Wednesday the tiny rigid remote-controlled device with metallic parts, a silica polymer exterior and a cavity to place treatments inside. It's a sort of drone for the body.
"This is the revolution and this is where it starts," said physicist and co-founder Michael Shpigelmacher. "I have no doubt that 10 years forward, there will be not one, but there will be multiple companies in this universe of ... remote-controlled micro robots."
Bionaut Labs began preclinical trials in 2018 to treat brainstem glioma, a tumor nestled in one of the most complex and delicate areas of the body — the brainstem, which controls everything from breathing to eating to heart rate. Because of its location, surgery is often not an option and some patients undergo radiation therapy.
"You really want to hit up that tumor and you don't want to just flood the whole body with a payload that would have significant toxicity or side effects," Shpigelmacher said.
Their "micro robot" or "Bionaut," as the company calls it, is still untested in humans. But, the device is meant to be injected by doctors into the brain and controlled with a magnetic device that moves it through the brain and to the affected area. Once treatment is complete, it's removed magnetically. It's biggest advantage, the company hopes, is to deliver targeted treatment, eliminating the need for physicians to turn to more invasive medicines such as radiation.
Physicist Michael Shpigelmacher is the co-founder of Bionaut Labs
"This is mostly useful for anatomical targets that are hard to reach where you need that level of anatomical precision," Shpigelmacher said.
He and his co-founders Aviad Maizels and Alex Shpunt previously worked together at a startup called PrimeScience that was later acquired by Apple in 2013. He began working with pharma companies in 2007, watching drugmakers formulate and reformulate drugs when they failed clinical trials.
Part of the problem is that medicine, most often pills, taken by patients flow through the bloodstream and often have side effects from depression and weight gain to even more serious ones. If it works, the Bionaut would only release chemicals to specific areas and not diffuse through a patient's bloodstream. That method, known as localized treatment, means drugs could be stronger and more targeted.
"That would maximize efficacy at the site of action, and minimize toxicity in all the places where it shouldn't have any activity," said Dr. Eunjoo Pacifici, a professor at the USC School of Pharmacy, about localized treatments.
But the process isn't easy. Drugs are highly regulated by the Food and Drug Administration. It can take years before a drug comes to market. Localized treatments often require a device to inject that treatment into the body. That, too, can take years for the FDA to approve.
"It's a combination product. Sometimes you need to have different parts of the FDA review that product as well. It's like developing two products at the same time," Pacifici said.
Bionauts are focused on brainstem glioma for now, but Shpigelmacher's background at McKinsey has him thinking big — the implications of Bionauts, he said, could change the way drugs are developed.
Pacifici said the drug-making process is a "high-stakes game" because companies often have to pump thousands of dollars into researching, developing and testing drugs without the promise of a return on investment. But if the FDA does not accept the benefit/ risk profile of a certain drug, it cannot get to the next step and be marketed or sold.
"You hope the product will do what you want it to do and there's some promising results in the lab, there's some promising results in the animals and the early stage clinical trials seem very promising," Pacifici said. "But then when you actually throw down the big money to conduct a big trial, more often than not it fails. A product can fail at any stage, but when it fails at a pivotal stage, it's a huge loss for the company."
If treatments can be localized, Shpigelmacher said, the amount of time companies spend in the research and development phase could shorten, and drugs would be conceptualized and tested to be used with a Bionaut.
The company raised $20 million in backing from Khosla Ventures, Upfront Ventures and Bold Capital.