Cell and gene therapy advances coming fast and furious

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A treatment to restore eyesight and hearing. New hope for a devastating form of brain cancer. Cell therapies for gut diseases that affect digestion. These are just a few of the cell and gene therapy technologies that are poised to significantly improve healthcare for patients over the next decade — some of them close at hand.

Each of these innovations was recently highlighted by Mass General Brigham, a Massachusetts-based academic healthcare system and biomedical research organization, in its annual “Disruptive Dozen” list, chosen by a group of Harvard Medical School faculty members. The list showcases a small number of ongoing research projects and illustrates just how far the cell and gene therapy field has evolved from formative concepts developed as far back as the 1960s.

The first person successfully treated with gene therapy was a 4-year-old patient suffering from a rare immune system disorder in 1990. But the FDA didn’t officially approve the first gene therapy treatment, for a type of acute lymphoblastic leukemia (ALL) , until 2017.

Since then, the cell and gene therapy market has seen a huge infusion of cash aimed at advancing the field. In 2019, nearly $20 billion was poured into the cell and gene therapy space, breaking the previous record of $13.5 billion set the prior year. Today, cell and gene therapy initiatives are making progress toward treating conditions such as Parkinson’s disease, sickle cell disease and even Type 1 diabetes.

But while there has been substantial investment and nearly two dozen cellular or gene therapy treatments have made it to market, the field has also had its share of setbacks ranging from disappointing trial results to safety issues, including serious complications such as inflammation, infections and even the development of certain cancers following treatment.

Despite these hurdles, cell and gene therapy research is advancing so rapidly that many of the treatments in the works today seemed unfathomable a decade ago. Ten years from now, the story could be much the same.

Chris Coburn, chief innovation officer at Mass General Brigham

Permission granted by Mass General Brigham/Chris Coburn.

“When you talk to the clinicians they say, ‘We’re going to look back on this period and recognize it for how primitive it was regarding these technologies,’” says Chris Coburn, chief innovation officer at Mass General Brigham (previously known as Partners HealthCare). In the very near future, he says, the whole model is going to be different.

Here, we’re looking at some of the potentially game-changing treatments being developed in the Mass General Brigham network, which includes a number of high-profile hospitals in the Boston area.

Seeing a cure for blindness

Many of the treatments being explored target once incurable conditions, such as blindness caused by genetic disorders. A method known as CRISPR-Cas9 gene editing, which made Mass General Brigham’s list this year, repairs faulty genes in the eye by inserting healthy replacements to treat conditions like Leber congenital amaurosis type 10, a currently untreatable and severe form of childhood blindness.

Patrick Fortune, VP of strategic innovation leaders at Mass General Brigham

Permission granted by Mass General Brigham/Patrick Fortune.

Researchers are also developing cell-based therapies and designed to replace lost or injured cells to restore vision in people with damage to the retina or cornea.

A suite of complementary treatments may allow doctors to treat multiple problems in different parts of the eye at once, Patrick Fortune, vice president of strategic innovation leaders at Mass General Brigham, says.

A needed therapy booster for glioblastoma

Gene therapy advances are bringing doctors closer to helping patients with glioblastoma, the devastating brain cancer with a five-year survival rate of just 6.8%. To date, precision medicine approaches have targeted proteins, but new research is focusing on a previously overlooked actor — RNA.

This treatment approach at Mass General Brigham uses molecules called microRNAs, which join to form a single unit that can be inserted into the brain using a virus. This therapy may help enhance the effectiveness of chemotherapy and other existing treatments.

Another separate approach blocks the activity of a type of microRNA called miR-10b, which prompts tumor cells to die. There has been little change in how glioblastoma has been managed over the past 50 years, Coburn says.

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