The drug Omomyc, created at the Vall d'Hebron Institute of Oncology (VHIO), has begun to be tested in patients with pancreatic cancer with metastasis after the good results obtained in its first clinical trial in humans.
Omomyc works in a different way than any previous therapy, as it is the first drug capable of inactivating the MYC protein in cancer cells, so it is expected to be effective in patients where other therapies they are not
The results of the first trial, presented yesterday in the journal Nature Medicine, indicate this. But it is still a few years before the drug can be approved, the researchers warn.
The first trial involved 22 volunteers who had various types of cancer with metastasis and in which the disease progressed after all treatment options had been exhausted.
In one person with pancreatic cancer, the volume of tumors was halved, there was an 83% loss in the amount of tumor DNA detectable in the blood, and the disease stabilized for seven months.
In another patient with a type of sarcoma that had not responded to the last two treatments, the cancer stopped progressing for eight months.
A third patient with salivary gland cancer remained stable 26 months after starting treatment and continued to receive Omomyc after the clinical trial ended.
Other types of cancer that have stopped progressing for a while with Omomyc include lung and colorectal.
These data extend the initial results of the Omomyc presented at a congress 15 months ago. "We now have proof that this treatment has a long-term clinical benefit", declares Laura Soucek, Icrea researcher at VHIO and project leader. "The challenge is to get it to the patients as quickly as possible and to as many patients as possible."
A priori, Omomyc can be effective against multiple types of tumors, as it inactivates the MYC protein, which is altered in most cancers. This protein, which accelerates the proliferation of tumor cells, has traditionally been considered undruggable, that is, impossible to attack with drugs. "Everyone said it was impossible," Soucek declared to La Vanguardia in 2002. But "where others saw an impossible problem, I saw technical difficulties that I hoped could be solved."
He teamed up with the pharmacologist Marie-Eve Beaulieu to develop a drug capable of accessing the inside of the cell nucleus, where MYC acts, and they founded the Peptomyc company.
To get the drug approved and reach patients, the Peptomyc team has now decided to focus on pancreatic cancer, because "it is a type of tumor in which there is a great need to develop new therapies and that we had two patients who responded in the first clinical trial", declares Soucek. And also "because we have to focus on a specific indication to move forward; in the future we hope to study the potential of Omomyc in other types of tumors".
The first three participants in the pancreatic cancer clinical trial have already received the first dose of Omomyc, reports Soucek. In the coming months, up to thirty patients will be enrolled from Vall d'Hebron, ICO-Bellvitge, Miguel Servet in Zaragoza and Gregorio Marañón hospitals in Madrid.
Patients will receive Omomyc in combination with chemotherapy, rather than receiving it as a single therapy like participants in the first clinical trial. For this reason, it is expected that the effectiveness of the treatment will be higher, since Omomyc can have a synergistic effect with chemotherapy and prevent the cancer from becoming resistant to treatment.
In addition, in the first clinical trial, a biomarker has been identified that predicts which patients will respond to Omomyc. This is making it possible to select patients who can in principle benefit from the drug for the second trial, which will conclude in two years.
If the results are positive, as expected, "our goal is to partner with a large pharmaceutical company to continue the development of the treatment, because a small biotech like Peptomyc does not have enough resources to get it to a large number of patients", declares Soucek.
