Where is ashanti desilva today in history

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In 1976, pop culture icon John Travolta starred in the made-for-television movie The Boy in the Plastic Bubble about a young man with a compromised immune system. The premise of the film is that Travolta’s character has to decide between living in isolation behind a protective bubble or dying engaged with the physical world. Hollywood revisited the theme in 2001 with Bubble Boy starring Jake Gyllenhaal.

Although the movie industry makes these plots seem larger than life, they are based in reality. The characters’ compromised health is loosely based on severe combined immune deficiency (SCID). In recent decades, clinical gene therapy trials have underpinned the research that has significantly contributed to advancements in the development of therapies for this and other diseases – and these trials can be traced back to a young girl named Ashanthi DeSilva.

The four-year-old that changed history

In 1989, the parents of four-year-old Ashanthi lived with the horror that their beloved daughter was suffering from an incurable gene-based immune deficiency. She had been diagnosed at two years old after suffering a string of debilitating infections.

Her particular type of SCID was deemed adenosine deaminase (ADA) deficiency. Treatment involved regular injections of PEG-ADA, an a

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When she was just two days old, Ashanti DeSilva developed an infection. It turned out to be the first of many infections that would baffle her parents and doctors until she turned 26 months old, when she was finally diagnosed with adenosine deaminase (ADA) deficiency, a form of severe combined immunodeficiency disease (SCID).

Like other children with the disorder, DeSilva was started on pegylated ADA (PEG-ADA), an enzyme therapy that emerged in the 1980s. The treatment worked well enough for her to gain weight, develop a normal peripheral T-cell count, and experience fewer infections. Yet her immune deficiency persisted.

At the age of four years, DeSilva made history at 12:52 p.m. on September 14, 1990, as the first patient to undergo an approved application of gene therapy. She was part of a trial authorized by the FDA and the NIH’s Recombinant DNA Advisory Committee (RAC). The trial included just one other patient, Cynthia Cutshall (now Kisik). Cynthia began treatment January 31, 1991, when she was nine years old.

Researchers drew blood from both girls, inducing the T cells from their blood to replicate in culture. Retroviral vectors infused with functional