A new treatment for gastrointestinal stromal tumors, certain leukemias and systemic mastocytosis (SM) called HT-KIT has shown promising results in early laboratory studies, according to a press release from the biopharmaceutical company Hoth Therapeutics.
HT-KIT targets the KIT gene, which can drive these conditions. By turning off this faulty gene, researchers hope to stop cancer growth. Because HT-KIT is very precise in targeting only the faulty KIT gene, they think it may cause fewer side effects in the rest of the body compared with existing drugs.
What is a KIT gene mutation?
SM is usually caused by a sporadic mutation in the KIT gene, which codes for a protein called CD117 transmembrane tyrosine kinase. The protein is involved in the growth, survival and migration of mast cells. The most common KIT mutation associated with SM is the D816V mutation, which results in the amino acid aspartic acid being replaced by the amino acid valine in the protein chain.
In preclinical studies (studies done before human testing), HT-KIT caused tumor cells to die within 24 hours and led to noticeable tumor shrinkage within just over a week. It also reduced the activity of the cancer-causing KIT gene by more than 80%. These results suggest that HT-KIT could be effective against cancers driven by the KIT gene.
Animal studies indicated that HT-KIT did not harm major organs, including the liver, kidneys, spleen, bone marrow and thymus gland. This suggests that the treatment may be well-tolerated in humans.
Read more about SM treatment and care
Researchers also tested how stable HT-KIT is under strict laboratory standards. The treatment remained stable in blood serum for 37 days at minus 112 degrees Fahrenheit (minus 80 degrees Celsius), which is longer than the required 28 days. This shows that the drug can be stored without losing its effectiveness. The Hoth Therapeutics lab also passed all necessary quality checks.
“We believe HT-KIT has the potential to transform outcomes in KIT-driven cancers,” said Robb Knie, CEO of Hoth Therapeutics.
The next step is an Investigational New Drug (IND) application, which is submitted to the Food and Drug Administration (FDA) to gain permission to start testing a new drug in people. Because the data look strong, Hoth Therapeutics hopes to move quickly with this step. If the IND is approved, the company can begin testing the drug in human volunteers.
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