Why Gene Therapy Can be a Boom or Bust for ADA Patients

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Have you ever heard of “ex vivo” gene therapy?

If you haven’t, it means removing some cells, modifying them, and putting them back in. The goal? Curing the diseases that are caused by genetic mutations in those cells. Seems simple enough, right?

Well, not so fast.

The concept of gene therapy as a whole was only recently developed. While its innovation has provided a great deal of hope to the rare disease community, there are also downfalls. Primarily, it takes a long time to get approved, and most companies don’t want to sell it.

The first person to EVER commercially use gene therapy tried the treatment in 2015 with the drug Glybera. It’s been called “the world’s most expensive medicine” costing 1 million dollars.


The drug was later taken off the market because of a lack of demand. Talk about frustrating. I mean, let’s break this down.

  • A treatment was developed to cure a RARE disease
  • A company decided to sell the treatment for an exorbitant amount of money
  • The company didn’t get many buyers because their consumers were people with RARE diseases
  • The company stopped selling the drug

But in any journey, there are potholes. Soon another gene therapy drug was approved for sale. This one, for treating adenosine deaminase deficiency (ADA-SCID). The drug’s name is Strimvelis and it was approved in 2016. This year, it will receive its first patient.

However, while not as expensive as Glybera, at around $648,000 it’s still very costly. And, it can only be obtained in Milan, Italy due to the nature of the treatment. While convenience isn’t the first thing patients look for when trying to access medicine that could change their lives, it’s undoubtedly a factor.

It’s yet to be seen exactly how many people will purchase the drug. Given the fact that it’s been on the market for a year, things aren’t looking very promising. Many patients are turning to clinical trials instead to receive treatment. And who can blame them?

Why pay for gene therapy when you can get it for free by volunteering?

In the U.S., for example, studies are currently being held at the University of California, Los Angeles. The funny part is the treatments in that study are being licensed by FORMER executives of the company trying to sell Strimvelis. In addition to being cheaper, clinical trials can also be more convenient for patients (depending on where they’re held) and companies are starting to see that.

There’s still a long way to go, but no one can deny that it’s at least hopeful these new treatments are being studied. Fingers crossed that gene therapy gets cheaper, and more accessible SOON.

Read more about Strimvelis here, and the potential it, and treatments like it, could have for ADA patients.




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