It’s that time of the month where the BSB readers get their chance to put us on the hot spot!
Here, we take a look at reader questions that have been submitted and argue the toss – is there evidence preclinically or clinically that is useful or instructive?
We can’t promise to answer every question, sometimes there simply isn’t any data to help either way.
This week, the topic is CAR T cell therapies, a subject that seems to be very high on many people’s minds and many of you had similar questions, so here goes…
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The 2015 annual meeting of the American Society of Hematology (ASH) (Twitter #ASH15) in Orlando has a bumper crop of interesting data.
ASH is one of the my favourite meetings on our conference calendar. I’ve been attending for many years, starting with when I was a commercial account manager for Hematology, Immunology, Transplantation and Oncology in the UK, then at Novartis in the US, when I was part of the team that brought Gleevec to market.
Hematologists make for an interesting group of people to talk to! They are very focused on the science behind a disease and how translational research can move the needle forward and generate better outcomes for their patients.
As part of our continuing preview of #ASH15, I’ve taken a quick look at the late-breaking abstracts that were released today. We will have more in-depth coverage after we’ve heard the data presented in the 7.30-9.30 am session on Tuesday December 8.
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If you’re not already a subscriber, but what to know “What’s hot at ASH15?” then you should purchase access. Additional ASH previews are already planned. By the time you’ve read them, you should “hit the ground running” in Orlando.
As Warren Buffett famously said, “Price is what you pay. Value is what you get.” I couldnt agree more. We have subscribers who just purchase our ASH coverage every year, so do “check it out“ if you haven’t done so already.
Bluebird bio ($BLUE) is an emerging biotech company with a novel lentiglobin gene therapy in development that could revolutionize the treatment of beta-thalassemia and sickle cell disease.
There’s a tremendous global unmet medical need for new effective treatments that could potentially result in transfusion independence.
Earlier this week we published an interview with Dr Alexis Thompson, the PI for the Northstar clinical trial of lentiglobin in beta-thalassemia that was presented at ASH last month.
The story continues with an interview with bluebird bio CEO Nick Leschly in which he discusses in more detail how their “one time” treatment could potentially be transformative.
He says, “we have to figure out a way to get this in the hands of global regions, because thalassemia and sickle cell together are the most common genetic disorders in the world.”
Whether countries that aren’t as wealthy as the US can afford novel gene therapy treatments is a big question, it will be interesting to see how that pans out, but it’s exciting to have potentially transformative new treatments in development.
Leschly says the mission of Bluebird bio is “making hope a reality.” It’s a compelling vision that he’s well on the way to pulling off with lentiglobin.
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Beta thalassemia isn’t something you read much about in the medical lay press, at least until recently. Part of the problem is the lack of approved therapies, as well as the dearth of new products being evaluated in this condition. It’s also more common in the Mediterranean, Middle East and Asia compared to the US, where it’s medical cousin, sickle cell anemia, dominates.
Things are changing on the horizon though with the advent of new approaches in gene therapy and gene editing, which have enabled new compounds to be developed that strike right at the source of the problem – mutated genes – rather than tackle the symptoms associated with the complications that can arise.
As such, this new approach is potentially transformative and therefore of great medical interest.
In the first of our two part series, we take a look at what the clinical impact of treating thalassemia patients really means and what’s happening next in sickle cell disease in a interview with Dr Alexis Thompson, the PI for the Bluebird Bio Northstar trial with lentiglobin that was presented at ASH last month.
Her perspectives offer a fascinating insight into this novel therapy now that the first 4 patients have been successfully treated.
In the second part of the series later this week, we will also take a look at Bluebird Bio the company, and their approach to gene therapy with lentiglobin and CAR T cell therapy with an interview with their CEO, Nick Leschly.
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