Issue 220
The FDA has granted accelerated approval for Bristol Myers Squibb’s Breyanzi cell therapy to target a new indication: adult patients with relapsed or refractory follicular lymphoma (FL) who have undergone at least two previous systemic therapies.
This comes after Breyanzi’s positive results in a global phase II trial – TRANSCEND FL – which evaluated efficacy and safety. Notably, the study had an overall response rate of 95.7 percent and a complete response rate of 73.4 percent.
Responses were also reported to be quick, with a median time of one month. However, the median duration of response has not yet been reached, with 77.1 percent of responders still in remission at 18 months. By comparison with Gilead’s Yescarta, the first CAR-T therapy to enter FL, this showed overall response rates of 91 and complete response rates of 60 percent in third-line trials.
“The approval of Breyanzi for relapsed or refractory FL provides an option with potential for lasting remission in a one-time infusion and a safety profile that allows for administration and monitoring in both the inpatient and outpatient setting in an increasing number of certified treatment centres in the US,” said Bryan Campbell, Cell Therapy Commercial Head and Senior Vice-President at Bristol Myers Squibb.
Given the recent advancements in CAR-T cell therapy, what are your thoughts on the future potential and challenges of this treatment in oncology? Let me know: [email protected]
Until next week,
Jamie Irvine | Associate Editor
Essential Reading
The therapeutic future
Researchers at the Children’s Hospital of Philadelphia have developed a gene therapy model for X-linked sideroblastic anemia (XLSA), a rare congenital anemia caused by ALAS2 gene mutations. In the study, they created a mouse model lacking the ALAS2 gene and treated it with a lentiviral vector that directs ALAS2 expression in erythroid cells. Notably, they observed a significant increase in hemoglobin and red blood cell levels, as well as normalizing hormone levels that control red blood cell production in mice that received the vector. The researchers now aim to adapt this model to explore pharmacological treatments and in vivo gene editing for milder forms of the disease.
My back!
Scientists from Ohio State University have set their sights on ending disc-related back pain using a gene therapy delivered by naturally derived nanocarriers that, as the study shows, repairs damaged discs in the spine and lowers pain symptoms in mice. The team used mouse fibroblasts and loaded them with genetic material for a protein key in tissue development. Then, they injected a solution containing the carriers into damaged discs in mice at the same time the back injury occurred. After 12 weeks, the researchers found – through imaging, tissue analysis, and mechanical and behavioral tests – that the gene therapy restored structural integrity and function to degenerated discs and reduced signs of back pain in the animals.
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