Editor's note: A version of this press release was issued December 19, 2023, by the office of New York State Senator Tim Kennedy.

BUFFALO, N.Y. — Senator Tim Kennedy (D-Buffalo) joined pediatric hematologist/oncologist Steven Ambrusko MD, MS, at Roswell Park Comprehensive Cancer Center to urge the New York State Department of Health to expedite the coverage approval process for a new groundbreaking gene therapy that is being hailed as a cure for sickle cell disease. On December 8, the Food and Drug Administration (FDA) approved the transformative treatment, which works by editing the DNA in a patient’s stem cells. Doctors say this one-time, multi-month treatment will replace the many years of painful treatments and finally provide relief and hope for patients who have long suffered from the disease, which disproportionately impacts Black communities. In New York State, one of every 365 Black infants are born with sickle cell disease, while one in 13 Black infants are born with the sickle cell trait. 

In Western New York, there are about 160 patients under the age of 21 being treated for the disease. Through the collaborative Roswell Park Oishei Children’s Pediatric Cancer and Blood Disorders Program, these patients are treated at the Pediatric Center at Roswell Park Comprehensive Cancer Center in Buffalo. Not all patients will qualify for the gene therapy, but most who do will have Medicaid or Managed Medicaid insurance. This makes it all the more pressing that Medicaid offer a plan to get this life-saving therapy to patients and end a disparity that impacts New York’s Black and underserved populations. 

“Health disparities have persisted within communities of color in Buffalo for decades, and in this moment, we have an opportunity to bridge one of those gaps in coverage and care in real time,” says Senator Kennedy. “By expediting the Medicaid approval process for these life-saving new treatments, New York can provide compassion, relief, and hope to patients and populations who have been consistently overlooked and denied comprehensive access to care for decades. Roswell Park doctors and patients have been waiting years for this day. Now, we need to make sure New York is doing everything possible to get this cure into the clinics treating people, so that we can immediately improve these health outcomes.”

“Even though sickle cell disease was one of the first genetic disorders to be identified, many decades ago, until now we’ve had limited treatment options to offer our patients,” says Dr. Ambrusko, who also serves as Director of the Sickle Cell & Hemoglobinopathy Center of Western New York. “Gene therapy has been the Holy Grail for sickle cell disease, and we finally have a more accessible cure to offer many of our patients, especially those with severe disease. These patients often go through debilitating pain and other difficult complications that dramatically reduce quality of life, so being able to offer a cure for even more patients — the improvements in quality of life and opportunities to live a full, rich, rewarding life are immeasurable.”

“As a Sickle Cell Warrior, I believe these new therapies should be available to everyone who can benefit from them,” says Juanita McClain, founder of the nonprofit organization Sickle Cell Warriors of Buffalo. “Sickle Cell Warriors often battle this disease every day of their lives. They’re looking for treatments to live every day with more comfort and with the hope for a longer, richer life. We’re all here to celebrate this great progress and the promise that lies ahead for more patients to have a healthier sickle cell experience through new and groundbreaking therapies.” 

Patients with sickle cell disease experience a shortage of red blood cells, due to abnormally shaped hemoglobin. According to the CDC, symptoms can include sudden mild to severe pain episodes, blockage of blood flow to the lungs, long-term eye damage, and increased susceptibility to infections, among other complications. According to the CDC, children with SCD may start to have signs of the disease during their first year, usually around 5 months of age.