RICHMOND, Va. -- Another glimpse of hope for the more than 100,000 Americans with sickle cell anemia.
The FDA recently approved two breakthrough gene therapies that can now be used to treat and possibly cure the genetic blood disorder, which mostly impacts the African-American community.
According to the Virginia Department of Health, between 2,500 to 4,500 Virginians live with the disease.
One in 325 African-Americans have the disease and one in 12 have the sickle cell trait in the state.
CBS6 Anchor GeNienne Samuels sat down with Dr. Wally Smith, who leads the adult sickle cell research and treatment clinic at VCU Health to better understand the gene therapy process, where the patient would essentially donate their cells to be altered and then used during treatment.
Below is a recap of Dr. Smith’s answers.
What should CBS 6 viewers know about this FDA-approved treatment?
"Maybe the first thing to reiterate to those who've been following this treatment, this is not the first so-called cure for sickle cell disease ever offered. The first so-called cure was actually a bone marrow transplant offered many, many years ago. What this is it’s the first gene therapy to be offered.
And gene therapy is different from bone marrow transplants. Bone marrow transplant, you get your cells from somebody else. Those cells have already been genetically fixed to not have sickle cell disease.
In this instance, you actually donate your cells to a company and the company goes off, and depending on which company goes off and adds something to them or cuts and changes the DNA in them, and then gives you your cells back the same way it would somebody else's cells if you were getting a bone marrow transplant."
Is gene therapy the same as a bone marrow transplant?
"Yes and no, except it's your cells and you're getting them back after they've been manipulated. One company adds something to the cells, and that's the Bluebird bioproduct, and the other company snips and rearranges the genes, and that's the Vertex product, and that's the one that got the most attention because that's the first disease for which there's ever been a genetic rearrangement, so called CRISPR rearrangement. First disease ever in the country that's had this done. So it's a real day in science, not just a day in sickle cell."
You mentioned the word “cure”. Is this treatment a cure?
"Yes, and we use the word advisedly. Let me explain what I mean. It's supposed to stop the sickling process in its tracks. If you've already had disease damage, you already have ear, eye, lung, heart, kidney, or other trouble, bone trouble, it does not reduce that. It does not reverse that. It does not take that away, but it does stop the cycling process in its track.
So you have to be advised, when you say the word cure, people want to be able to return back to their usual lifestyles. And they may not be able to, if they were so damaged to begin with that they could not have a normal lifestyle. But we are certainly excited that we can stop this disease in its tracks."
Is there an upper age limit to qualify for this treatment?
"Practically, no. I think that we will take it on a case-by-case basis, but we did not put a ceiling age range on this. Certainly, a person who has had a lot of organ damage already and is approaching 65 or 70 years of age is unlikely to be recommended for a therapy like this. On the other hand, if that same person says, I'm 50, I'm 55 and I'd like to live to 75, there might be situations where we could offer the therapy even to them. And certainly, the younger you are and the more able, we're able to predict that you're going to have a bad course of your disease, the more likely you are to be able to qualify for this therapy."
Where can people go for the gene therapy treatment?
"The community needs to recognize that not just any medical center will be able to give this therapy. There's contractual and structural requirements for you to be able to process the cells, harvest the cells and send them off to the company. VCU will be one of the first in the nation to have these facilities up and running, both for the Vertex and for the bluebird bio product.
Secondly, you have to have a person who's going to do the transplant, and not every medical center has people who can do these transplants.
Third, you need to have a sickle cell specialist who's going to follow the patient very closely, such as we do, even during the transplant process. And those are unfortunately rare in the state of Virginia, especially for the adult population. Not so much for the childhood population, but for the adult population. So you have to have a certified center.
You'll be able to go on the company's website to see if there's a certified center near you, and we recommend if there is, that you would get a consultation from that certified center in order to access either the Bluebird or the vertex product."
When will VCU be able to offer these therapies?
"It can be done now at VCU Health via the Bluebird process, but not Vertex yet. And that's because the contracting itself takes months, and we've been working on this for months. So I'd imagine the first of the year we'll be up and running for the Bluebird product and by March of 2024, up and running for the Vertex product.
We are taking referrals now. People want to come see us. It's not too soon to sign up to come talk to us about this. So if somebody needs an appointment just to discuss it, we anticipate many people will want to just discuss what's going on and we're ready for that."
According to Dr. Smith, for those that can afford to take advantage of this treatment, which is expected to cost more than $1Million, it’s a yearlong process with 15 years of monitoring for possible side effects, the biggest of which could be cancer.
As a reminder, other treatments are available to sickle cell patients including four drugs and bone marrow transplants. These options will be discussed with you if you schedule an appointment at VCU Health.
For more information, you can visit the VCU Health website, here.
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