A Patient’s Desire to Help

Mar 01, 2016

A Patient's Desire to Help

By Meaghan Casey

Laabs’ foundation and Aura Biosciences advance efforts to develop new and better therapies to treat rare cancer patients

Four years ago, Memphis native Mark Laabs’ future was bright. He was 28, working in China as chief operating officer of a company dedicated to fighting climate change, and living out his calling to follow his “personal North Star.”

A single moment completely shifted where that North Star was leading him. While in a meeting in Beijing, Laabs lost vision in his right eye. Nearly 48 hours and five consultations later, he was sitting with the dean of ophthalmology at a major medical center, being told it was ocular melanoma.

A rare cancer, ocular melanoma is diagnosed in about 2,500 adults every year in the U.S. Although produced from the same cells, called melanocytes, ocular melanoma is different from skin cancer. It develops in the uvea, or uveal tract, of the eye. In approximately half of those diagnosed, the tumor can aggressively grow and spread to other parts of the body, becoming fatal.

As Laabs quickly discovered, there have been no targeted therapies available and the cancer is usually treated with an invasive radioactive plaque placed against the exterior of the eye near the tumor. Known as brachytherapy or plaque therapy, this treatment can require multiple surgeries and can lead to cataracts, retinopathy and loss of vision. The alternative to radiation is surgery to remove the eye.

“I was essentially a biohazard for eight days while the radioactive plaque was on my eye and at the end of that process, the tumor was dead,” said Laabs, who had flown back to the U.S. for treatment in Philadelphia. The treatment left him in remission, but he was unable to regain most of the vision in his right eye.

Aura Biosciences, a Cambridge-based biotech company developing highly tumor targeted breakthrough therapies for rare cancers, is hoping to improve the options for future ocular melanoma patients. The new class of therapies would potentially target and destroy cancer cells selectively, while leaving surrounding tissue unharmed—an approach Aura calls molecular surgery. It would effectively transform the treatment of ocular melanoma into a routine outpatient procedure.

“By enabling physicians to treat cancer more selectively, effectively and safely than they can do today, we aim to eliminate the need for risky procedures that carry significant morbidity and often do little to improve a patient’s overall survival,” said Aura’s Founder and CEO Elisabet de los Pinos. “When you’re focusing on a rare disease and thinking about the limited and highly invasive alternatives out there, it creates an incredible sense of purpose. Everything is aligning at the moment and, if successful, the end product will be extremely meaningful for patients.”

In May, Aura was granted Orphan Disease Designation by the FDA for its lead product candidate, AU-011. The company’s pre-clinical research, ‘Evaluating the in vivo efficacy of a first-in- class drug for the treatment of primary uveal melanoma,’ was delivered that same month by McGill University Health Centre researchers at the 2015 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.

“Aura’s novel approach has the potential to dramatically improve the outcomes, and hope, for patients with ocular melanoma,” said Grant Allen, Co- Founder and Chairman of the Ocular Melanoma Foundation. “The Orphan Drug Designation for AU-011 is a huge step forward, enabling Aura to potentially bring this drug to patients in an expedited manner.”

AU-011 consists of a viral-like particle drug conjugate that binds selectively to cancer cells in the eye and upon activation with an ophthalmic laser, the small molecule selectively destroys the membrane of cancer cells, killing them without damaging the adjacent retina.

Aura is in the process of submitting an Investigational New Drug (IND) application to the FDA and is advancing toward clinical testing. De los Pinos hopes to enroll 24 patients in the trial and has 10 hospitals throughout the U.S. lined up as sites. Dr. Evangelos Gragoudas, Director of Retina Service at the Massachusetts Eye and Ear Infirmary of Harvard Medical School, and Dr. Carol Shields at the Wills Eye Hospital in Philadelphia, are serving on the Clinical Advisory Board and helping to guide the clinical development.

Dr. Ivana Kim, Co-Director of the Ocular Melanoma Center at Mass. Eye and Ear, will also participate as one of the investigators in the trial.

“It has the potential to be transformative,” said Kim. “The main benefit would be eliminating some of the side effects of radiation we’re seeing now, such as damage to the retina or optic nerve, that lead to vision loss. I would also love to see it developed into a diagnostic tool, to test borderline lesions that could be treated early. That would be a big step forward.”

As a survivor, Laabs is encouraged by the progress being made and is hopeful he, too, can play a role. Following his experience with ocular melanoma, he founded the Rare Cancer Research Foundation (RCRF) to advance efforts to develop new and better therapies to treat rare cancer patients.

Laabs, who built a successful career in global renewable energy development and solar product
distribution, sold his clean energy finance company, Climate Bridge— which had become one of the largest players in the global carbon markets—to embark on this, a different kind of socially innovative project.

“In remission, I was left with questions of what to do next—really where I could make the greatest positive impact as possible over the course of my lifetime,” said Laabs. “I spent a lot of time thinking, ‘should I work directly in ocular melanoma?’ But all these cancers have common needs, and I realized that I don’t want anyone dying of cancer, no matter what type. So I came to the conclusion that the best use of my time and talent would be to connect with groups doing really important work and help them to do that work more efficiently.”

Working with more than 200 foundations and medical research organizations, RCRF administers the research infrastructure and enables researchers to focus on novel, incremental innovation. The foundation offers solutions for patient registries, tissue samples, cell lines, animal models, and genome sequencing, available to researchers through a single, virtual, integrated platform. The hope is that it will radically accelerate the time-to-market for new, life-saving therapies. At the same time, researchers can reallocate time, money and attention to novel drug development efforts and bringing new therapies to market.

“We’re a supporting player, supplying the tools and the building blocks,” said Laabs. “The most important thing is improving patient quality of life and longevity. To see better patient outcomes, it’s necessary to bring together different groups to solve problems that not one individual group could solve on its own. It’s that collaboration that I would want to see in my own treatment.”

See all MassBio News