Hard work and hope

By Sean Moore

In an old kitchen fridge in a third-floor lab, stashed away in what would normally be a produce drawer, is a vial of powder that resembles salty flour. UM graduate student Nitesh Sanghai confidently, yet gingerly, holds it up and twirls it around. The drug, which he co-named Borsantrazole, is not a cure, but it is overdue optimism for a longer, better life for people living with amyotrophic lateral sclerosis, ALS.

“Maybe this molecule can slow down progression by two, three, four, five years. Maybe it can halt the progression,” he says. “At least we can create hope so these patients can celebrate more birthdays.” 

Roughly 4,000 Canadians are living with this neurological disease that’s been likened to being buried alive. About 80 per cent will die within two to five years after diagnosis.

Sanghai’s ALS drug discovery, which earned him a prestigious 2025 Mitacs Innovation Award, wasn’t by fluke, but it very easily might not have happened. It took years of sacrifice, a fortuitous partnership with UM professor Geoffrey Tranmer, and a bold idea no one had thought to try. 

Sanghai grew up in a coal-mining town in northeastern India, in a one-room house with 10 family members. His father sold clothes from the back of a motorcycle before opening Sanghai’s Cloth Emporium, which made enough to send one child to school.

Sanghai missed every holiday to study and earned a place in India’s prestigious National Institute of Pharmaceutical Education and Research. And upon graduating, he and his wife got jobs with international drug companies testing the safety of new compounds. But in 2014, after his father died of cardiac arrest, the advice his dad gave him replayed in his head: “Nitesh, do something new. Bring purposeful change to human lives.”

So Sanghai quit his job and applied to graduate school in Canada. 

At the same time, Tranmer, in UM’s College of Pharmacy, was looking for a grad student with his specific skill set. Tranmer picked Sanghai up at the airport on a winter night in 2018 and after their hellos, Tranmer asked what he knew about ALS. Nothing, Sanghai said.

A few days later Tranmer handed Sanghai three scientific papers and challenged him to create a new type of molecule. Could he add boron to an ALS drug called Edaravone to make it better? 

Boron is the fifth element on the periodic table. Small, black, and brittle, it is a metal essential to good health in trace amounts.

There are only two drugs approved in Canada to treat ALS, Edaravone being the most recent, approved by Health Canada in 2018. Only two drugs for a disease that was first described in medical books in 1869 and known about widely since 1939, when baseball player Lou Gehrig shared his diagnosis with the world. It was this lack of options that had sparked Tranmer’s interest: When a family friend was diagnosed with ALS, he grew curious about what sort of therapies would be used and was stunned there were only two. 

“And the drugs had less than ideal properties. They’re mediocre,” he says. “And one day it just sort of occurred to me that you could add boron to Edaravone and I was surprised no one else thought of this.”

In their lab, Sanghai excitedly explains his work. He’s surrounded by glass vials connected by tubes, flowing from one to another, and flasks filled with substances of rich reds and yellows.

In people with ALS, the free radicals that are a natural byproduct of our bodily processes reach dangerous levels and degrade motor neurons to the point where muscles no longer receive any nerve signal, eventually leading to paralysis and death. 

Boron is exceptionally good at capturing free radicals. Edaravone also captures free radicals, but it has limitations, and this is what Sanghai focused on for his PhD thesis.

Edaravone’s drug properties limit how much of it crosses the blood-brain barrier, which is critical for a nerve drug, but that’s not its only issue. Edaravone is also unstable, breaking down into potentially harmful toxins and so needs a special formulation to be administered, which can cause serious allergic reactions in some patients. 

It took Sanghai about seven months to create a better, novel molecule using new methods he patented. The result is a drug that is 90 per cent pure and can fully pass the blood-brain barrier. 

“When you make a drug, it’s up to the quantum universe to decide if it’s going to work,” Tranmer says. “It’s up to the atoms to decide how they will interact. You don’t really know if the key you designed will work with the lock you want to open. And you don’t expect to get this right on the first try. But Nitesh did. That was really surprising.”

Borsantrazole (“bor” for boron, “san” for Sanghai, “tra” for Tranmer, “zole” for its class of compound) works in three phases: first, the boron captures a bounty of free radicals and then the drug is metabolized into Edaravone (without its usual baggage of unwanted toxins) to act as a secondary antioxidant. And then finally, as a parting gift, the drug becomes boric acid, a nerve-protecting substance, which makes this research development so groundbreaking.

“Every 90 minutes someone is diagnosed with ALS and someone dies from ALS,” Sanghai laments. “We need to do something. All major clinical drug trials have failed. They all fail.”

Borsantrazole looks promising though, and to fast-track the leap into medicine cabinets, Sanghai took the extraordinary step (for a pharmacist) of training to run animal studies and for years, once again missing holidays and vacations, ran proof-of-principle studies on mice, showing the drug to be effective and safe. More animal studies are needed before human trials can begin, but the results offer incredible hope. 

Tranmer says he calls Sanghai “a unicorn” to his colleagues, and then quickly adds “what’s really important to understand is that there is a real community of science on our campus. We’re so grateful because this was a real community effort.”

Armed with a patent and supported by UM’s Technology Mobilization Office, Sanghai and Tranmer launched their startup Borotherapeutics Ltd. to help bring their drug to ALS patients. Attracting investors may come easier since the CAS, a branch of the American Chemical Society, awarded Sanghai its elite Future Leader award on March 19. Only 31 graduate students from around the world were given this year’s honour, four of which were from Canada. Sanghai is the first honoree from UM. The research grabs attention, including here at home, where it provides pride for the ALS community, says Diana Rasussen, Executive Director and Client Services Coordinator of the ALS Society of Manitoba. 

“We’re very excited this is happening in Manitoba,” she says. “The work Nitesh, his supervisor and their team are doing is absolutely fabulous and we want to see more research funding coming to Manitoba so that we’re on the map. Often you see it in big cities where funding is available and exciting ideas are coming. But you know what? It happens all across the country. We just need to be supportive.”

Sanghai says his journey to UM was meant to be.

“I’m staying in Manitoba,” he says. “I’ve earned recognition here. Anyone can earn dollars some place, but earning recognition is harder. My supervisor empowered me here—we are co-inventors of a molecule. I’m an inventor because of him. And I want to keep working on this molecule with him to help ALS patients. This is a big, big deal.”