Transkrip medicine fights 3 types of cancer

• April 25, 2024

The preleukemia or myelodysplastic syndrome , the cervical cancer and the cutaneous lymphoma can be treated through a combination of drugs that for years have been used to treat hypertension and epilepsy .

This development was carried out by the group of Alfonso Dueñas González , from the Institute for Biomedical Research (IIBm) of the UNAM, which after several years showed that the combination of hydralazine and magnesium valproate It is effective against these conditions.

The drug is called Transkrip , and through it, today patients can be given a few more months of survival with respect to traditional cervical cancer therapies, and important therapeutic effects have been observed to counteract myelodysplastic syndrome and cutaneous lymphoma. specialist in medical oncology.

Myelodysplastic syndrome

Myelodysplastic syndrome is a condition that, in common terms, is also known as preleukemia . "As we know, in leukemia there is a defect in the cells that produce the blood and, therefore, patients suffer from alterations derived; that is to say, the cells of the bone marrow stop producing the normal constituents of the blood and, consequently, they often have problems with coagulation, defenses, and anemia occurs ".

It is part of the group of diseases called leukemia, and is considered rare, because its prevalence is less than 200 thousand cases. Without treatment, most progress to a myeloblastic leukemia acute in a period of approximately 12 to 24 months, or die as a result of complications, he added.

Dueñas González said that this condition is malignant and the genetic or molecular alterations that originate it are not yet known. However, it is known that so-called epigenetic alterations that, without damaging the DNA chain, change the expression of genes , participate in the development and progression of it.

These modifications occur through two phenomena: the DNA methylation and the deacetylation of histones, which combine to quench suppressor genes and alter the functioning of cells, he said.

Until recently, treatments for this condition had been ineffective; however, a few years ago a couple of medications called azacitidine Y decitabine They proved to be effective, by improving blood production and delaying the onset of acute leukemia. These drugs are called demethylating agents because they inhibit DNA methylation, explained the also member of the National Cancer Institute.

The Transkrip It is a pharmaceutical preparation that consists of two products: hydralazine , which inhibits DNA methylation, and magnesium valproate , an antiepileptic that inhibits the deacetylation of histones, combines the inhibitors of these phenomena and when used in the myelodysplastic syndrome, we have seen favorable responses in practically half of the patients, he said.

The academic commented that in March 2010, the Federal Commission for the Protection against Sanitary Risks (Cofepris) approved the registration of this product for your commercialization and use in advanced cervical carcinoma. "We showed that patients who received chemotherapy, plus Transkrip, lived longer without progression of the disease."

"Based on our studies we observed that in the case of the referred syndrome, the Transkrip, used as a single therapy, is also effective," he said.

The advantage in the development of drugs under the concept of therapeutic repositioning is that since they are part of an already known drug, the preclinical and clinical process is more accelerated, which allows faster registration and subsequent commercialization.

Transkrip, orphan drug

Dueñas González indicated that in the United States there is a program that depends on the Food and Drug Administration (FDA), equivalent to Cofepris, to investigate and develop drugs for diseases that they call "orphans", in the sense that the prevalence in that country should be less than 200 thousand.

Then, the drugs that have potential against these conditions are designated "orphan drug", and one of the incentives of that program is that the FDA, through a support system, facilitates pharmaceutical companies and / or to public institutions, nonprofit, the development of clinical studies in the American Union, for its eventual approval and commercialization in that and other nations.

In the case of Mexico , he added, the Transkrip has just been "designated" by the FDA as an "orphan drug" for myelodysplastic syndrome and cutaneous T-cell lymphoma, also considered a rare condition.

This means that clinical studies will be conducted in the neighboring country with the drug to demonstrate its usefulness and, based on this, the FDA will approve your registration for future marketing he explained.

"We will develop the protocol and the pharmaceutical company that produces Transkrip will seek to carry out the Clinical studies in an institution that does cancer research in that country, "he added.

This step is a practically necessary condition for the medicine to be approved and marketed internationally, he concluded.