Roswell Park Cancer Institute later this month expects to start the first patient in a much-anticipated trial of a lung cancer drug developed in Cuba.
Cancer center officials express confidence the trial will move forward despite uncertainty about future relations with the island nation following the election of Donald J. Trump.
President-elect Trump pledged during the campaign to roll back the Obama administration's moves to ease trade restrictions with Cuba to get a better deal. And, following the Nov. 25 death of Cuban leader Fidel Castro, Trump in a tweet again threatened to reverse the efforts to normalize relations between the countries.
“If Cuba is unwilling to make a better deal for the Cuban people, the Cuban/American people and the U.S. as a whole, I will terminate deal,” he said in a Twitter message.
Roswell Park officials say any change in policy would probably not alter the first U.S. study of CIMAvax, the promising lung cancer drug developed by Cuba's Center for Molecular Immunology. The steps to gain approval to bring the drug into this country and conduct the study were taken before the U.S. eased trade restrictions with Cuba, they say.
“We’re moving ahead regardless,"said Candace Johnson, president and chief executive officer of the cancer center. "We started our relationship in Cuba before the sanctions were lifted, and we navigated quite well, and we have no indication yet that anything will change.”
If Cuba-U.S. relations dramatically reverse, she said Roswell Park will accommodate to them. Travel between the countries by researchers could become more cumbersome if Trump terminates the agreement. But she said Roswell Park has all the appropriate federal permissions to receive shipments of the drug from Cuba.
Still, uncertainty remains about what happens beyond this initial two-phase study, expected to take about three years to complete.
Will Trump follow through with his threats? If so, what will that mean for further studies of CIMAvax, as well as the potential marketing of CIMAvax and other Cuban drugs in the U.S. if they prove to be safe and effective?
Johnson said it's all speculative at this point.
"Right now we're trying to bring a potentially life-saving drug to patients in the United States, and we're trying to keep politics out of it," she said.
CIMAvax will be offered to as many as 90 patients with advanced non-small-cell lung cancer to determine the optimal dosage and effectiveness in combination with the immunotherapy nivolumab – brand name Opdivo.
CIMAvax was approved by Cuba in 2008, but U.S. doctors can't prescribe the drug without FDA approval after studies in the U.S.
CIMAvax is a vaccine, but it was developed to stop cancer from growing, not to prevent it. Rather than attempting to kill cancer cells like a chemotherapy, CIMAvax targets epidermal growth factor, a protein found naturally in the body that signals cells to grow. The vaccine stimulates the immune system, leading the body to produce antibodies that stop epidermal growth factor from attaching to cancer cells and signaling them to grow out of control.
About 5,000 patients in other countries, including 1,000 Cubans, have received CIMAvax. The results have been modest but interesting enough to raise hopes for progress in treatment, especially among patients with high levels of epidermal growth factor.
Advanced lung cancer patients on CIMAvax lived three months longer than those in a control group in a Cuban study of 405 participants published this year in the journal Clinical Cancer Research. But the survival benefit was even greater for patients with high concentrations of the protein in their blood, and researchers are hopeful for even better results with the addition of nivolumab.
Slightly more than 20 percent of the patients in the study with high levels of epidermal growth factor who received CIMAvax were still living five years later, while none of the participants in the control group were alive after five years. Moreover, CIMAvax appears to be well-tolerated compared to standard chemotherapy.
Roswell Park has had informal exchanges with students and scientists in Cuba since 2011. But this is the first formal scientific relationship in the country.
The Treasury Department's Office of Foreign Assets Control in 2014 authorized the cancer center to import the drug, and that license was renewed this year for three more years. Roswell Park in 2015 struck a deal with the Center for Molecular Immunology to study the drug following a foreign trade mission to Havana that included Johnson and Gov. Andrew M. Cuomo.
In October, the Treasury Department approved the joint venture with the Center for Molecular Immunology, which developed CIMAvax and produces it. The Food and Drug Administration at that time also authorized the cancer center to conduct the trial. Roswell Park anticipates it will start enrolling patients before Christmas.
The Roswell Park Alliance Foundation committed $2.4 million in donor funds to cover costs of the study. The cancer center posted information about eligibility for the clinical trial at roswellpark.org/cancer-vaccine.