BIO 2018: Anything is possible, says first-ever human gene therapy patient
“I have a message for all of you searching for a treatment and a cure in your lifetime; I hope you can look at my story and know that anything is possible,” said Ashanti DeSilva to attendees at the 2018 BIO International Convention.
In September 1990, the first approved human gene therapy attempt took place on four-year-old DeSilva, who was cured of severe combined immunodeficiency, a rare genetic disorder that disturbs the development of functional T cells and B cells.
DeSilva had two broken copies of the gene that contains the instructions for manufacturing a protein called adenoside deaminase.
“By age four, she was slipping away. But her physician knew a team of scientists at the National Institutes of Health,” said Jim Greenwood, BIO president and CEO, who told DeSilva’s story before she came on stage.
Within months, she had shown real improvement.
Greenwood added: “They knew it worked when the whole family got the flu and Ashanti was the first one to get better. My friends, this is the power of biotechnology.”
DeSilva was awarded the first-ever BIO History-Maker award yesterday, June 5.
She said: “Patients and their families are now at the forefront of biomedical research. We’re connecting with scientists, we’re sharing information and, most importantly, we’re telling our own stories, so please have hope, be your own advocate and never give up.”
Three years after DeSilva’s treatment, BIO was formed, in 1993. According to Greenwood, its core mandate is “to create a policy environment that helps convert medical miracles like [DeSilva’s] into accessible therapies that can reach all patients in need”.
As part of this mission, BIO has worked to modernise the Food and Drug Administration (FDA).
Greenwood added that under Scott Gottlieb, who is commissioner of the FDA, the agency has shown an “unprecedented willingness to work with the research community to get it right”.
In August last year, the FDA made history by making the first gene therapy available in the US. The FDA approved Novartis’s Kymriah (tisagenlecleucel) for certain paediatric and young adult patients with a form of acute lymphoblastic leukaemia.
Seven weeks later, the FDA issued a second landmark approval for a CAR T therapy, a treatment by Gilead-owned Kite.
This has the potential to send cancer into remission for thousands of patients, claimed Greenwood.
This was followed in December by approval of a novel gene therapy to treat patients with a rare form of inherited vision loss. The treatment, Luxturna (voretigene neparvovec-rzyl), is owned by Spark Therapeutics.
And this is just the beginning, there are 81 gene therapies and 46 CAR T medicines in the pipeline, and hundreds more in pre-clinical development.
The 2018 BIO International Convention is taking place in Boston between Monday, June 4 and Thursday, June 7.
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