Dr. Donald Kohn helps Young Girl with Rare Disease Live Life to the Fullest

Dr. Donald Kohn and researchers at UCLA opened the door to a brighter future for a young girl previously isolated due to a rare and life-threatening disease. Twelve-year-old Eliana Nochum spent her early years in a sterile environment, diagnosed with severe combined immunodeficiency disorder (SCID), a condition that left her without the necessary immune system to fight off infections. Her form, ADA-SCID, is extremely rare, with fewer than ten cases reported each year.

Eliana's family, from Fredericksburg, Virginia, faced tremendous challenges to keep their home safe for her, including removing pets and limiting exposure to plants and fresh foods. “We had to create a bubble,” her mother recalled, emphasizing the drastic measures they took to protect her.

Hope emerged when they learned about a clinical trial that could potentially provide a cure through gene therapy or a bone marrow transplant. Under the guidance of Dr. Donald Kohn and his team at UCLA, Eliana received treatment that has transformed her life. After the trial, her family received the life-changing news that she could finally step outside and enjoy normal activities, including sharing kisses with her mother, a gesture forbidden during her early years.

Today, Eliana is navigating a world full of possibilities. Reflecting on her journey, she admits it’s emotional to revisit her past but is excited about the future that lies ahead, now fully within her grasp.