Historic Milestone: First Patient Undergoes New Gene Therapy for Sickle Cell Disease
Landmark in Medical Innovation: Revolutionary Lyfgenia Treatment Begins
In a groundbreaking medical feat, a new gene therapy aimed to treat and potentially eradicate sickle cell disease had its initial trial with its first-ever patient, Kendric Cromer, on May 1st. The 12-year-old African-American boy from a Washington D.C. suburb embarked on his inaugural treatment journey in Bluebird Bio’s care at the Children’s National Hospital within the D.C. metro vicinity. This treatment is significant due to the prevalence of sickle cell disease within the African-American community, a debilitating genetic disorder which affects about 100,000 individuals in the U.S.
Lyfgenia: A Rigorous yet Promising Treatment
The gene therapy, known as Lyfgenia, imposes an arduous processing on the patient. Kendric became eligible for this therapy after satisfying key criteria such as the severity of his illness and insurance clearance. His mother, Deborah Cromer communicated her mixed feelings of fear and hope, considering the strict therapeutic protocols her son has to undergo. Kendric, who has been impacted by the disease since he was three, is hopeful for a cure.
The therapy involves extracting Kendric’s bone marrow stem cells after administering a medication, plerixafor, to assist in the extraction process. The cells harvested are subsequently sent to a lab in Allendale, New Jersey, where a healthy hemoglobin gene is introduced to correct the mutation responsible for the disease.
A Costly Hope: An Investment Towards a Revolutionary Cure
Valued at a staggering $3.1 million, Lyfgenia is celebrated as a revolutionary treatment in the healthcare industry despite its cost. Bluebird Bio reports that given the duration required for the entire treatment, it can only accommodate around 85 to 100 patients annually.
DPH Hot Take
The onset of Lyfgenia treatment brings a beacon of hope to the forefront for those affected by sickle cell disease, particularly for those in the African-American community where the disease is prevalent. This ground-breaking treatment, albeit at a significant cost, signals the potential of medical science to tackle hereditary diseases in a freeing and innovative way. The challenges and opportunities that lie ahead will invariably shape not only the lives of patients but also the shifting landscape of genetic therapy. The story of Kendric Cromer acts as an illustration of the far-reaching effects of such medical evolution – an impetus for continued development in gene therapy.