The only known cure for Sickle Cell Disease is an allogeneic stem cell transplant. Most patients, more then 75%, don’t have a suitably matched, unaffected family donor. Sadly, African American and other minority donors are also poorly represented in the unrelated donor pools. This lack of suitable donors makes it difficult for patients to receive curative stem cell transplant. This disparity led to the formation of the Haploidentical Sickle Cell Disease Consortium (Haplo SCD). Seven centers around the nation, led by Dr. Cairo and his team at Maria Fareri Children’s Hospital/New York Medical College, have collaborated and joined efforts on a Clinical Trial The Safety and Efficacy of Prophylactic Defibrotide Administration Children, Adolescents, and Young Adults with SCD or Beta Thalassemia Following Myeloablative Conditioning and Haploidentical Stem Cell Transplantation Utilizing CD34 Enrichment and T-Cell Add Back (IND 127812). The trial is currently accruing patients and showing promise for patients with Sickle Cell Disease who may otherwise have not had an option for cure. The Children’s Cancer Fund is a proud supporter of this trial and the patients treated on it at MFCH.
Dr. Cairo and his team are leading numerous clinical trials that explore novel targeted and immune therapies that aim to improve or maintain excellent outcomes while minimizing short-and long-term complications.
The Children’s Cancer Fund supports a nationally recognized research laboratory that is investigating strategies designed to determine the safety, feasibility, dose, schedule and efficacy of new treatments during experimental clinical investigation in patients with both malignant and non-malignant disorders.