Growing up, I really didnt have as many crises with sickle cell, Hymes said in a recent virtual interview from Arkansas. When I was getting older, I started having more and more sickle cell crises. But Ive always had this dream of becoming a physician, and as I got older, the more crises I had, the harder it made that journey.

A report from the Centers for Disease Control and Prevention (CDC) stated the origins of the pain: Sickled cells traveling through small blood vessels can get stuck and block blood flow throughout the body, causing pain. A pain crisis (vaso-occlusive episode or VOE) can start suddenly, be mild to severe, and can last for any length of time.

If you got hit by a bus and survived, all that excruciating pain that you would feel, thats how Id describe it. Id spend a week, two weeks, in the hospital, and then you have to recover, thats another week, and Id miss two weeks of class, Hymes said when explaining what living through a pain crisis has been like for him.

So, he looked for a solution by doing some online research that led him to McKinneys work.

We shot a few emails back and forth and it just felt like the right place to be I knew that the life I wanted and how it was going couldnt really co-exist, he said. Leading up to around the time I reached out to Dr. McKinney, I started to have crises almost every other month or sometimes even every month.

After he and McKinney developed an email relationship, Hymes came to Colorado for some blood work and testing and discovered he was a match for the trial.

Once it was determined that I could, I became a little more hopeful in the journey, said Hymes, who didnt dwell on the challenges of that journey, and instead honored the people who helped him through it: his mother, his girlfriend, and his grandparents all of whom came to visit him in Colorado, while he spent months coping with the complicated procedure.

We evaluate the patient to ensure their organs are healthy enough to tolerate the gene therapy process; we then start putting them on chronic transfusions where they get a monthly transfusion to decrease how much sickle hemoglobin they have," explained McKinney. "We give them medications to help them release their stem cells from their bone marrow into their blood and we hook them up to machines to collect those stem cells.

Its those cells that then get the gene therapy, they are edited to stop forming the sickle shape.

And that may take several months to get enough stem cells ... once weve collected those stem cells, we have to send those stem cells off to manufacture and to change the genes so that they make non-sickling hemoglobin. And that process can take about 12 weeks, McKinney said.

Patients then return for high doses of chemotherapy, which gets rid of the old bone marrow and makes room for the new.

Once they get that chemotherapy, we then infuse the stem cells, and it can take about four weeks for those cells to grow, McKinney said. The patient remains in the hospital while the new stem cells grow. Upon discharge, they have years of follow-up monitoring. Ultimately, patients who receive gene therapy need to be followed for about 15 years after the infusion to make sure theyre doing well."

For Hymes, things went according to plan. Because he was from out of town, he had to stay in Colorado for about six months in total. He said part of it was to have my edited genes put back in my body, and thats when I would have the chemo and stay in the hospital for an extended period of time.

Recovering from the chemotherapy, Hymes said, was one of the hardest parts.

I was still having to deal with the chemo and all the problems that it can cause. Its just this really bad taste you could have from the chemo and it could knock out your taste and you have a sore throat ... I guess Im a big eater, so food was the important part to me. But I drank a lot of Ensures just recovering from the chemo, said Hymes.

Now, two years since his journey began and one year after the transplant, Hymes said he feels like a new man.

It was a total change in my energy level and how I was able to just go about life! I didnt have to worry about having pain crises every other day or every month, said Hymes.

Regarding his pain crisis, Hymes said, Its almost non-existent at this point. I dont say almost non-existent. Its been non-existent at this point.

Like about 90 percent of McKinneys other trial participants, its all over now.

I havent had any crises since I had the stem cell transplant, and sickle cell-related problems since the transplant. So I would say it has been extremely beneficial in my life, said Hymes.He said it's also been good for his mother.

I only seen my mom cry one or two times, Hymes explained. One time was seeing him struggle with the disease, the other time, Hymes said, was seeing him leave it behind.

The two FDA-approved treatments are slightly different from what Hymes received but they work the same way, editing cells, and then returning them to the patient.

Now that the drug therapy has been approved, another component of McKinneys job is to let the rest of his patients the ones who werent part of a trial know about it. Some of those who werent in any of his trials experience pain from the disease serious enough to make the therapy worth considering.

A few weeks after the FDA announced its approval of the drugs in early December, McKinney met with Mia Hilton, a 20-year-old esthetician from Green Valley Ranch in Denver. Hilton has been McKinneys patient for the past 10 years. Vivacious and upbeat, she spent most of the meeting making funny comments. After a quick overall exam, he asked if shed heard about the newly approved gene therapies.

They are meant to be curative, he explained. Youd no longer have sickle cell after administration of them.

For her, that would be a big change. Like Hymes, sometimes, her pain got bad enough to go to the ER, where shed sometimes receive intravenous drugs.

Usually a pain crisis feels kind of like burning or stabbing a little bit. It can feel pretty hot internally in your muscles. I'll take a Benadryl and then they'll give me morphine through my IV, and that usually works, said Hilton. If not, she gets a nasal dose of fentanyl. In the ER, medical staff monitor her progress.

They usually do a chest X-ray to make sure that I'm not going to have a chest crisis, explained Hilton. And after a few hours, if the pain was under control, shed get discharged. I'll have somebody come and pick me up from the ER just because of the heavy medicines, they don't want me to drive.

Sometimes, when she didnt bounce back, she would be admitted from the ER into the hospital.

Usually my stay can range from three to seven days, so it does get pretty strenuous, she said.

She told McKinney that shed heard some pieces of information about the new gene therapies, but wanted to know more. In a cautious, gentle voice, he told her, The side effects of the chemo could cause infertility and hair loss.

Hilton had a quick answer to that.

Ive never wanted babies, she said, So its a free form of birth control.

I'm very into make-up and hair, so I already have 40 wigs in my closet, the bubbly woman added while already wearing eyelashes nearly an inch long. When McKinney mentioned that painful sores could form in her mouth, she didnt care about that either.

I bite my jaws a lot, so Im already there. I bit my tongue and my jaw four times in the elevator on the way up here, said Hilton.

Her reservations focused on the time shed lose working, and concerns about whether her case was severe enough to warrant the sacrifice.

I love science, so to hear that you're going to take something out, fix it, and put it back, it's like, Oh my gosh, that's kind of cool. Personally, I would definitely look into it, but ... I don't know if I would have the energy to do that. You know what I mean? I would be pretty much down to do it because I feel like in the long run, it could really help because having sickle cell, if you're not on top of it and you're not maintaining it, it can really slow you down ... So to get rid of crises altogether, that would be pretty ideal, said Hilton.

Although the side effects didnt bother Hilton, the cost could if her insurance wont cover it. One therapy costs $2 million, and the other is $3 million. But, according to Dr. David Rind, chief medical officer at the Institute for Clinical and Economic Review, a nonprofit that evaluates the prices and effectiveness of medications, that price wouldnt come out of the patients pocket. He said he expects insurance companies to cover the gene therapy.

I dont think any manufacturer would charge a price like that thinking only the patients who can afford it will get it, Rind said. I think the assumption is that insurance will pay for this ... I think this will be paid for, for almost anyone who wants it.

The rationale for why insurers should cover the gene therapies relates to slavery, he said.

The reason there is sickle cell in the U.S. is because we brought Africans over as slaves, Rind said. He explained that the disease is a gene mutation that fights malaria, a disease common in Africa. Thats why the people who are here have sickle cell . This is a population obligation in the United States to get this right.

Hymes has a sense of obligation, too, to other people born, like him, with the disease. Now that he has gotten rid of his crises as a clinical trial participant, what he thinks would be ideal is to become a doctor caring for people with sickle cell.

My entire life, Ive been extremely interested in hematology-oncology, of course, because that is what [sickle cell disease] is grouped as. But lately, Ive been interested in emergency medicine as well, because I know a lot of times we get seen on the front end in the emergency room, and thats where a lot of chronic pain patients get perceived as opioid-seeking, said Hymes.

He is now participating in an academic program for aspiring medical students while waiting to hear if hes been accepted to medical school. Once he has completed his medical degree, he said, he knows hed have the sensitivity to understand a sickle cell disease patients experience as real, not one of faking pain in the ER.

So either helping on that side or helping in the hematology and oncology area is what Im interested in. If I could do both or figure out a way to help all across the board, then Id probably choose that, too, said Hymes.

McKinney looks forward to offering his patients the choice of a different ending.

Now, when those families come in for their first visit with us to talk about what its going to look like for their child to live with sickle cell disease, we can at least provide them some hope that this is something that is very, very treatable, McKinney said. Something that if theyre having a lot of problems related to this, that we can potentially cure and get rid of.

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New FDA-approved sickle cell gene editing therapies offer hope for a pain-free life to patients some living in Colorado - Colorado Public Radio