Advocating for your child’s health can mean the difference between managing symptoms and finally seeking to cure his or her disease. Deborah Cromer is all too familiar, and her child was first in line for a new treatment seeking to cure sickle cell disease.
This past May, her 12-year-old son, Kendric received his first treatment of LYFGENIA, a gene therapy that would help his body rebuild more robust blood cells. Kendric went into his treatment with trepidation, uncertain of what life would look like on the other side.
He recalls, “I didn’t know what life would be like without sickle cell. I didn’t know if I wanted the treatment, but I couldn’t let sickle cell define me as a person.”
Sickle cell disease is a hereditary condition that impacts the hemoglobin, the part of the red blood cell that carries oxygen. Instead of being plump and robust, red blood cells end up in a sickle shape. These cells don’t adequately carry oxygen throughout the body, which affects every major system.
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Sickle cell disease mostly impacts people of African descent worldwide. In the US, 1 in 13 Black people are born with the sickle cell trait. Of those with the trait, about one out of every 365 Black babies are born with sickle cell disease. The disease can cause debilitating pain, and without treatment, individuals may face serious organ damage and other life-threatening complications.
According to his mother Deborah, the decision was not entered into lightly at all. “We’ve really been steadfast in making sure that Kendric’s never missed a doctor’s appointment and that we have to get him in front of the right people,” she explains.
The right people included three different sets of clinical trials, and a hematologist early on, something that she was surprised to know other sufferers didn’t have access to. At one point, one of her neighbors expressed just that. “She told Kendric she had sickle cell, but she was just seeing a general practitioner,” Deborah recalls“Why hasn’t the general practitioner moved her over to [a hematology practice]?”
Even with Kendric’s care, Deborah has had to go beyond to ensure her son gets the best treatment available. “Like many Black people, our cries have been ignored. So I know that it’s important that people have a clear understanding about how to move forward even when those things happen.”
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Deborah believes Kendric’s access to LYFGENIA has been due to her and her husband’s constant advocacy for his health. She hopes that other parents will push for the same. “I would encourage all parents to find a way to make sure that you or your child are getting the best possible care.”
Sickle cell disease has long been considered understudied, despite shortening the lifespan of thousands of sufferers. That was until a breakthrough gene therapy, LYFGENIA, showed promise in providing a cure. While Deborah welcomed a new treatment, she had no idea that her son Kendric would be the first to receive it.
The treatment began at Children’s National Hospital. According to David Jacobsohn, MD, MBA, chief of the Blood and Marrow Transplantation division, “The recent approval of gene therapies to treat patients with sickle cell is an enormous breakthrough in patient care and a silver lining to families witnessing their children’s struggles with this condition. In the face of immense burden, gene therapy is a groundbreaking treatment alternative and a beacon of hope for a better future.”
The gene therapy LYFGENIA is approved for patients 12 and older. The treatment takes months and involves removing stem cells and then modifying them. After, the patient receives chemotherapy, and then the cells will be reintroduced to the patient. The result will hopefully be red blood cells that no longer form a c shape, known as a “sickle.” The treatment requires millions of stem cells, and harvesting them takes time. Each of Kendric’s treatments will take six to eight hours.
Kendric has had his second stem cell harvesting and he gets a three-month break. He was recently inducted into the National Junior Honor Society at his school, despite having little energy to complete his studies. “Even with everything he’s got going on, he still is [my] Wonderboy,” Deborah praises. “My kid is super resilient.” Kendric is anxious to see what life will be like for him and what doors open when he has more energy and is not constantly in pain, she adds. That has always weighed on Deborah, and she says the decision to start LYFGENIA was not taken lightly.
Until recently, sickle cell disease was treated with long-term medication and/or a bone marrow transplant. Despite how promising gene therapy seems, it is limited to the number of people who can be treated every year. The treatment process is time-consuming, and Bluebird Bio, the company behind the LYFGENIA treatment, believes it can only treat 85 to 105 patients per year. Medical centers, including Children’s National where Kendric began his treatment, can accept roughly 10 gene therapy patients per year.
Deborah says that people ask her how they can get on a list or who they can contact, and she feels that research trials have been helpful. “A lot of times Black people don’t look to research as an option, but for us, it was a means to an end. We did not see this coming though for years. Honestly, I had no idea that it was right under our noses. We’re honored, of course, and we can’t see what the future holds, but Kendric has big dreams and we just want to help him fulfill those dreams.”
