Waiting for a breakthrough in oncology
The drug, which induces the self–destruction of tumor cells, was approved for testing on people suffering from anaplastic astrocytoma and glioblastoma multiforme - the most common and malignant brain tumors. Phase 1 of clinical trials will determine whether the use of the experimental drug PAC-1 in combination with temozolomide, used as standard chemotherapy, is safe.
Anaplastic astrocytoma is currently being treated surgically, followed by the appointment of a course of chemotherapy with temozolomide. With glioblastoma multiforme, treatment is also surgical, then the patient receives courses of chemotherapy and radiation therapy. Glioblastoma multiforme is dangerous because it can quickly and asymptomatically spread to different parts of the brain through blood vessels. Its complete excision during surgery is almost impossible. This is the main reason for the ineffectiveness of treatment and frequent recurrence of the tumor. The survival rate for glioblastoma is on average only 15 months.
The study will involve patients whose tumor continues to progress after routine treatment.
In a study on dogs with glioblastoma, combined treatment with PAC-1 and temozolomide was well tolerated by animals and had a persistent positive effect: the tumor decreased by an average of 30%. According to MRI data, one of the dogs was confirmed to have a full recovery on the 84th day of therapy.
The peculiarity of the drug PAC-1 is that it is able to penetrate the blood-brain barrier, unlike many other antitumor drugs. The point of application is the enzyme procapspase-3, which is synthesized in large quantities by tumor cells and damages healthy neurons. PAC-1 normalizes the level of procapspase-3, affecting only tumor cells and not affecting healthy ones.
In 2016, PAC-1 was tested on domestic dogs with osteosarcoma and lymphoma. In combination with the chemotherapy drug doxorubicin, PAC-1 stopped tumor progression in four out of four dogs with lymphoma and in three out of six dogs with osteosarcoma. Among the side effects, only a disorder from the gastrointestinal tract was noted. In general, PAC-1 proved to be a fairly safe drug.
In an ongoing study on people with solid tumors and lymphoma, it was found that the drug PAC-1 is well tolerated at a dosage of 400 mg / day. In the planned study for the treatment of brain tumors, PAC-1 will be prescribed at a dosage of 375 mg / day. Then the dose will be gradually increased to assess the safety of the drug when administered together with temozolomide.
So far, there have been no data on serious side effects of PAC-1 in people who would require the drug to be discontinued or the patient to be excluded from the study. It is impossible to judge the effectiveness of the drug at this stage, since the main purpose of this phase of testing is to assess its safety.
It will take several years to determine the safety and effectiveness of PAC-1 in humans.
Article by Avadhut D. Joshi et al. Synergistic and targeted therapy with a procaspase-3 activator and temozolomide extends survival in glioma rodent models and is feasible for the treatment of canine malignant glioma patients published in the journal Oncotarget.
Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru Based on the Illinois News Bureau: Cancer drug starts clinical trials in human brain-cancer patients.