The raw material for the drug used in the treatment of Familial Mediterranean Fever is now produced in Türkiye.

Prof. Dr. Ahmet Gül, Head of the Rheumatology Department at Istanbul Faculty of Medicine, spoke to an Anadolu Agency (AA) reporter about the treatment of Familial Mediterranean Fever and drug development studies in Türkiye.

Prof. Dr. Gül stated that Familial Mediterranean Fever, classified among rare diseases in the world, is a genetic disease and causes recurrent attacks of inflammation.

Gül, noting that this disease, which is seen in communities located east of the Mediterranean, is most prevalent in Türkiye worldwide, said, "The disease is characterized by inflammatory attacks that often last 1-3 days in the peritoneum, pleural effusion, joints, and sometimes skin. These attacks, which begin in childhood, can recur with varying frequency. If left untreated, or if treatment is delayed, recurrent inflammatory attacks can lead to the development of a significant complication called amyloidosis. Amyloidosis, a complication that can lead to kidney failure, intestinal malabsorption, heart failure, and death, can develop when inflammatory symptoms are not adequately controlled."

Gül emphasized that treatment with the active ingredient colchicine should be started after diagnosis, and stated that this drug is obtained from the Colchicum plant, which is an endemic species in Türkiye but is imported from abroad in its purified form as a medicine.

Pointing out that the raw material in the drug colchicine is entirely of plant origin, Gül noted:

Although at least half of the more than 100 species of Colchicum are endemic in Türkiye, we source purified colchicine from abroad. When used in adequate doses, this medication can prevent recurrence of FMF attacks. Therefore, patients should take the appropriate dose of colchicine regularly from the moment of diagnosis. Even with adequate colchicine use, it may not be possible to suppress attacks or stop inflammation in approximately 5-10 percent of patients. In some patients, it may not be possible to reach the desired dose due to side effects such as diarrhea. In such cases, biologic drugs that reduce the effect of the inflammatory cytokine Interleukin-1 (IL-1) are used.

Gül explained that biological drugs that reduce the effect of Interleukin-1, directly targeting the disease-causing agent, are used in patients who do not respond adequately to colchicine, and that three different biological drugs have been shown to be beneficial in reducing the frequency of recurrence of attacks and suppressing inflammatory findings in FMF patients by blocking the effect of Interleukin-1.

Gül emphasized that two of the three drugs that block the effects of interleukin-1 are among the drugs licensed and reimbursed for the treatment of Familial Mediterranean Fever in Türkiye, and continued:

In addition to these drugs, we always need new ones. In this context, we have conducted the first human study in Türkiye and recently completed the phase 2 study for another drug. Early results will be announced at a meeting in October. Since its beneficial effect has been demonstrated, the phase 3 study will begin. After this phase is completed, the regulatory process will begin. The drug will be deemed effective and safe, and once the regulatory process is complete, it will be available to patients. Turkey, a country with a high prevalence of the disease and a relatively high number of patients with inadequate response to colchicine, is among the countries particularly preferred for drug research. We are making significant contributions to newly developed drugs, including currently licensed drugs. The introduction of new drugs will increase patient options and perhaps make it easier to access the drug.

Prof. Dr. Gül also reported that the INFLAM-IST platform, established in Türkiye under the leadership of Istanbul University with the collaboration of three academic institutions and two national industry partners, has begun producing the Colchicum plant, the raw material for the drug colchicine.

Gül said, "As part of the TÜBİTAK 1004 program, the INFLAM-IST platform, spearheaded by Istanbul University, is conducting important studies on the development of new treatments that block the effects of interleukin-1. As part of these studies, studies on the production of the Colchicum plant, the raw material for colchicine, in Türkiye and the extraction of colchicine from plants have been successfully carried out to date. Once these studies are completed, it will be possible to obtain colchicine from plants in Türkiye. This will enable the supply of raw materials to meet the increasing demand for colchicine due to its increased use resulting from the emergence of new applications."

Prof. Dr. Gül stated that colchicine has been widely used in the treatment of gout, Familial Mediterranean Fever, and Behçet's disease since ancient times, and in recent years, it has also been used as a preventative measure against cardiovascular diseases.

Gül stated that the drug is expected to become widely used in other disease groups where similar inflammatory pathways play a role, emphasizing that this will increase the need for the drug's raw material and create a resource for both Turkey and other countries in need of this drug.

Gül also stated that biomarker studies are being conducted within the INFLAM-IST platform to answer the questions, "Which patients are resistant to colchicine?" and "Can there be objective findings to identify patients who do not respond adequately?" and provided the following information:

We are continuing the INFLAM-IST platform's colchicine-related studies at the Atatürk Horticultural Central Research Institute in Yalova, in collaboration with our Faculty of Pharmacy. Here, the cultivation of the highest-yielding Colchicum species in our country is a process that begins in pots and is then transferred to the fields. Studies to increase this production and obtain colchicine have reached their final stages. Both production studies and studies to extract colchicine from these plants are ongoing at our Faculty of Pharmacy.

Gül, who also announced the establishment of a production facility, said, "In this production facility, we will produce the raw material for colchicine in our country without needing to import it. By producing the raw material for the drug in our country, we will no longer be dependent on foreign sources. Producing colchicine in Türkiye will not only enable the drug to be obtained from a largely endemic plant, generating economic gains but also eliminate our dependence on foreign sources, and provide an advantageous opportunity for exporting the drug abroad. We expect significant economic gains for our country."

Professor Dr. Gül also stated, "Studies on developing new, highly bioavailable forms of colchicine are nearing completion. This will allow our patients to use colchicine more easily in situations where some have difficulty taking it or cannot continue treatment due to side effects such as diarrhea. This will also allow children to take the medication more easily in different forms."