At Torrey Pines, striving to find medical game-changer

The next big thing in brain medicine could be hiding within the walls of the Torrey Pines Institute for Molecular Studies located in Tradition. Researchers there believe they’re onto something that could – eventually – help those with ALS (Amyotrophic Lateral Sclerosis) or Alzheimer’s disease.

Using compounds developed at Torrey Pines, spin-off company Autophagy Neurotherapeutics (ANT) has struck a licensing agreement to continue using those compounds commercially, in an effort to create what are called small-molecule drugs. Such drugs could eventually be taken in a pill form. But it could be decades before that happens.

Torrey Pines Institute for Molecular Studies has been waiting more than 20 years for one of its compounds to make it through a series of medical trials. It’s still undergoing testing and is in Phase 3 human trials.

The compound? Something that could be used to treat itching due to kidney disease as well as better treat pain.

Torrey Pines President Greg Welmaker said they are very excited about the partnership with ANT and what its work could mean for those with brain-related diseases.

“We fully support their efforts,” Welmaker said. If ANT can bring a drug to market, Torrey Pines will get royalty payments. The state of Florida, too, will get a royalty.

It’s part of how the nonprofit Torrey Pines brings in funds to keep the research going. Other ways include license agreements, equipment usage, and selling its “compound libraries” – boxes stacked on top of boxes filled with small vials containing trillions of peptides and millions of small molecules all for research purposes. Peptides are small chains of proteins that make up DNA.

Welmaker said that not only does the testing of potential new medicines take time, but requires a whole heap of money, too – $2 billion, on average.

Welmaker hails from the pharmaceutical world and explained that for every successful drug, there are potentially 50 that don’t make it. Successful drugs, then, have to not only bring in revenue enough to cover their own costs but also cover those that had failed.

For Alzheimer’s, Welmaker said billions of dollars have been spent industry-wide and little has been developed by way of therapies. “It’s the black hole” of research funding, he said, which is why the ANT agreement is exciting. ANT researchers have focused on two “very promising” compounds that could manipulate the way brain cells clear out used proteins and other cellular waste material.

It is believed that a buildup of cellular waste could be a contributing factor to Alzheimer’s disease. The compounds ANT’s researchers are working with have shown promise in enhancing the cells’ ability to clear those toxins. ANT will use the licensed technology to develop additional compounds active against neurodegenerative diseases.

For all the work happening under Torrey Pines Institute’s expansive roof at the Tradition Center for Innovation, Welmaker said the most frequent question he gets is, “You’re still here?”

“We’re still here!” Welmaker said, explaining that the institute is committed to Port St. Lucie and its mission.

Yes, Torrey Pines did not grow as it expected. Yes, it did not hire the number of researchers it had thought it would. Yes, it lost state funding.

But, for all that, Torrey Pines Institute for Molecular Studies is still up and running and working with partners around the country coming up with the next big thing.

Welmaker said they were sad to see VGTI – the Vaccine and Gene Therapy Institute – next door shut down.

“We want an active, vibrant scientific community here,” he said, adding that there was no competition between the two, only the potential for collaboration.

Both facilities came to the city prior to the market crash and Great Recession. Both received state and local incentives. Both had agreements with the City of Port St. Lucie.

One was able to weather the storm. The other did not.

For his part, Welmaker said Torrey Pines Institute has done what it can to meet its requirements. It has created jobs and has leased the third floor to two lab-testing companies. Now, Torrey Pines pays property tax on the third floor because the lease generates revenue. “We have to do whatever we can to survive,” he said.

As a nonprofit, Torrey Pines relies on revenues from its licensing agreements, selling its compound libraries, future royalties and grant funding. Donations from philanthropic residents, organizations and agencies, too, would greatly appreciated, Welmaker said. “We’re scraping all the time,” he said of making the bill payments. The carrying cost of the building alone is nearly $2 million annually.

What the future holds for Torrey Pines Institute for Molecular Studies remains murky. For now, and into the foreseeable future, the facility will remain in Tradition and continue plugging away on molecular research.

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