Summary of A Promising New Treatment for Pediatric Brain Cancer:
Professor Adrian Krainer of Cold Spring Harbor Laboratory has developed an antisense oligonucleotide (ASO) drug for treating diffuse intrinsic pontine glioma (DIPG), a deadly pediatric brain cancer. DIPG is a lethal disease that often kills within a year of diagnosis, and surgery is near impossible due to the tumors’ location, while chemotherapy has debilitating side effects. The ASO drug slows tumor growth, reverses some cancer cell changes, and increases survival rates in mice. The treatment would likely need to be combined with radiation or immunotherapy before clinical trials can begin, and the team is exploring ways to enhance the therapy’s effectiveness.
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Research Team Develops Potential Therapeutic for Pediatric Brain Cancer
Diffuse intrinsic pontine glioma (DIPG) is a type of pediatric brain cancer that often leads to death within a year of diagnosis. With limited treatment options available, Cold Spring Harbor Laboratory researchers have developed a potential therapeutic for DIPG using antisense oligonucleotides (ASOs).
Groundbreaking Research on ASOs
Professor Adrian Krainer, known for his pioneering research on ASOs and the development of Spinraza® for spinal muscular atrophy (SMA), has created a drug that slows down tumor growth, reverses some cancer cell changes, and increases survival rates in mice.
The dominant mutation in DIPG blocks closely related proteins from turning genes on and off, leading to uncontrolled cell growth and cancer. The ASO drug, developed using technology similar to that in Spinraza, targets the mutated protein H3.3K27M. The ASO drug works by shutting down H3.3K27M, leading to a decrease in tumor growth and the return of affected genes to normal.
Exploring Ways to Enhance Effectiveness
While the new ASO drug has shown promising results, Krainer believes there is still a long way to go before it can begin clinical trials. Additionally, it may need to be combined with radiation or immunotherapy to be maximally effective. Despite these obstacles, Krainer and his team are committed to exploring ways to enhance the drug’s effectiveness and bring it to clinical trials as soon as possible.
Hope on the Horizon for DIPG Patients
With limited treatment options, developing a potential therapeutic for DIPG is a significant breakthrough. While there is still a long way to go before this ASO drug can be marketed, the findings provide hope for DIPG patients and their families.
References:
“Antisense oligonucleotide therapy for H3.3K27M diffuse midline glioma,” Science Translational Medicine, April 12, 2023, doi: 10.1126/scitranslmed.add8280.
Funding: Cure Starts Now Foundation, Simons Foundation, The V Foundation, St. Giles Foundation, NIH/National Cancer Institute
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