Glioma therapy THIO granted FDA rare pediatric disease designation
Anti-cancer agent aims to treat children with aggressive glioma type
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to the anti-cancer agent THIO as a potential treatment for an uncommon form of glioma, called pediatric-type diffuse high-grade gliomas, or PDHGG, that affects children.
This designation is awarded by the FDA to provide extra incentives to companies that are investing in the development of treatments for rare diseases affecting children.
As a boon of the designation, if THIO is ultimately approved for PDHGG, then the treatment’s developer Maia Biotechnology would receive a voucher that could be used to get FDA priority review on a future drug application. That priority status would shorten the agency’s normal regulatory review time from about 10 months to six months. Maia could use the voucher itself, or sell it to another company.
“We are proud to receive the FDA’s rare pediatric disease designation for THIO, which significantly bolsters our plans for continuing research in the PDHGG indication,” Vlad Vitoc, MD, chairman and CEO of Maia, said in a company press release.
K. Robinson Lewis, vice president and head of regulatory and quality at Maia, added that this designation “offers a highly valuable incentive” for the company.
THIO designed for difficult to treat cancer types, such as PDHGG
Gliomas are the most common type of brain cancer in children. PDHGG refers to certain types of glioma that mainly affect children and young adults. These tumors grow aggressively — and thus are known as high-grade — and infiltrate through brain tissue so that there’s usually not a clear border between the normal brain tissue and the tumor. In such cases, the glioma is known as diffuse. PDHGG has historically been extremely difficult to treat, and survival outcomes are very poor.
THIO, more formally known as 6-thio-dG or 6-thio-2’-deoxyguanosine, is an experimental anti-cancer agent that’s designed to target telomeres, which are structures that act like caps on the end of a cell’s DNA. Telomeres are normally important for protecting cells’ DNA, but in cancer cells, they play key roles in helping the cancer to grow. They also help the cancer resist currently available treatments, many of which aim to target cancer cells’ DNA.
Maia’s therapy candidate is essentially a faulty version of a building block that’s usually used to make telomeres. When THIO is incorporated into a cancer cell’s growing telomeres, it causes the telomere to fragment, and this in turn triggers the body’s immune system to activate — which can ultimately spur the immune system to attack the cancer cells.
THIO is a versatile anti-cancer agent that has demonstrated positive results in multiple difficult to treat cancer types, including pediatric high-grade glioma, which is among the most treatment-resistant cancers in children.
The experimental therapy has already shown promise in lab models for treating a type of PDHGG called diffuse intrinsic pontine glioma or DIPG.
“THIO is a versatile anti-cancer agent that has demonstrated positive results in multiple difficult to treat cancer types, including pediatric high-grade glioma, which is among the most treatment-resistant cancers in children,” Vitoc said. “THIO is shown to activate the immune system while evading tumor immunosuppression, a novel therapeutic approach for this devastating childhood disease.”
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