Please ensure Javascript is enabled for purposes of website accessibility

bluebird bio Says Its Drug "Unlikely" to Have Caused Blood Cancer

By Todd Campbell - Updated Mar 10, 2021 at 12:03PM

You’re reading a free article with opinions that may differ from The Motley Fool’s Premium Investing Services. Become a Motley Fool member today to get instant access to our top analyst recommendations, in-depth research, investing resources, and more. Learn More

A clinical hold on the biotech company could be lifted following Bluebird's results.

In February, bluebird bio (BLUE -8.01%) announced a temporary suspension of two clinical trials evaluating LentiGlobin, an experimental gene therapy, after a trial patient was diagnosed with acute myeloid leukemia (AML), a blood and bone marrow cancer. The possibility that a lentiviral vector used in Bluebird's drugs caused the cancer called into question the viability of its genetic program, causing a sharp sell-off in its shares.

Today, Bluebird unveiled updated results from its internal investigation, suggesting it's unlikely the AML case reported in its phase 1/2 (HGB-206) study of LentiGlobin for sickle cell disease (SCD) was caused by its lentiviral vector. 

A woman scientist looking through a microscope.

Image source: Getty Images.

Specifically, Philip Gregory, its chief scientific officer, said:

Analyses identified the integration site for the vector within a gene called VAMP4. VAMP4 has no known association with the development of AML nor with processes such as cellular proliferation or genome stability. Moreover, we see no significant gene misregulation attributable to the insertion event.

Gregory went on to say it's "very unlikely" Bluebird's lentiviral vector played a role in this case.

Bluebird is providing this research to the Food and Drug Administration, and the clinical holds that are currently hamstringing its beta-thalassemia and sickle cell disease programs could be lifted if the FDA agrees with Bluebird's assessment.

A positive opinion could also allow Bluebird to restart commercialization efforts in the European Union for Zynteglo, a gene therapy for transfusion-dependent beta-thalassemia patients. Previously, it had voluntarily stopped marketing Zynteglo because it relies on the same viral vector used in its sickle cell disease study.

Separately, the company continues to investigate a second Suspected Unexpected Serious Adverse Reaction (SUSAR), involving a patient diagnosed with myelodysplastic syndrome in February. 


Invest Smarter with The Motley Fool

Join Over 1 Million Premium Members Receiving…

  • New Stock Picks Each Month
  • Detailed Analysis of Companies
  • Model Portfolios
  • Live Streaming During Market Hours
  • And Much More
Get Started Now

Stocks Mentioned

bluebird bio, Inc. Stock Quote
bluebird bio, Inc.
$3.33 (-8.01%) $0.29

*Average returns of all recommendations since inception. Cost basis and return based on previous market day close.

Related Articles

Motley Fool Returns

Motley Fool Stock Advisor

Market-beating stocks from our award-winning service.

Stock Advisor Returns
S&P 500 Returns

Calculated by average return of all stock recommendations since inception of the Stock Advisor service in February of 2002. Returns as of 05/18/2022.

Discounted offers are only available to new members. Stock Advisor list price is $199 per year.

Premium Investing Services

Invest better with The Motley Fool. Get stock recommendations, portfolio guidance, and more from The Motley Fool's premium services.