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January 29, 2013
In 1999, Project HOPE began to implement the Gaucher Disease Initiative in Egypt in partnership with Genzyme Corporation, to relieve the suffering and prevent the untimely death of people affected by the rare genetic disease. Since the program’s beginning, Genzyme has donated more than 30 million units of Cerezyme (imiglucerase for injection) the drug used to treat the incapacitating and often deadly disease, and helped to improve the lives of more than 300 children. As Genzyme and Project HOPE commit to another year of the successful partnership, it is a good time to reflect upon the success of the program.
“In 1998, when we began developing the program, no one with Gaucher Disease had ever been treated in Egypt,” says Dr. Reda Mansour, Chief of Party for Project HOPE in Egypt. “Now there are seven centers for treatment across the country and seasoned clinical specialists who are experts in the treatment of the debilitating disease. This program has not only saved children’s lives, it has built capacity and strengthened the local health care system in Egypt and contributed to the treatment of Gaucher Disease around the world.”
Dr. Pramod K. Mistry, Professor of Pediatrics and Medicine Chief, Pediatric Gastroenterology and Hepatology at Yale University School of Medicine has served on the Project HOPE’s Gaucher Initiative’s expert medical committee since 2001 and now serves as chairman. He describes the program in Egypt as “transformative.”
“The overarching goal of this humanitarian program among Project HOPE, Genzyme and key opinion leaders in the Gaucher Initiative is to treat children and adults with Gaucher Disease in countries where there is no access to treatment and to develop a cadre of medical professionals with expertise in this rare disease who can not only treat patients, but contribute to the research in the treatment of the disease,” he says. “From its small beginnings, this program has helped treat hundreds of patients and develop sustainable health care centers for the disease. Now anyone in Egypt who is diagnosed with Gaucher Disease has somewhere to go.”
While Project HOPE coordinates the program and works with Egyptian physicians to identify patients and the expert medical committee helps mentor the doctors and offer support, it is Genzyme that provides the expensive drug free of charge to the patients.
“Providing Cerezyme to the patients is the right thing to do,” says Simone Azevedo, Genzyme’s Vice President Global Disease Leader, Global Gaucher. “By working with the right partners, training doctors to become leading experts in the field and involving the community, we are able to make this program successful and sustainable.”
Genzyme and Project HOPE staffs along with dedicated physicians and ethicists on the Expert Medical Committe have been able to travel to Egypt to see the program success first-hand. Azevedo values the detailed reports. “I am lucky enough to understand what Cerezyme treatment brings to patients. The program not only changes the patients’ health, but transforms entire families and communities. Patients before treatment have difficulties even going to school, or growing properly. With treatment, the children live normal lives. We have patients now who are university students, engineers, married with families, all contributing to society.”
Over the past decade, the program has survived volatile times, including a shortage of the drug Cerezyme as well as the unrest in Egypt. “That is the beauty of the partnership with Project HOPE,” Azevedo says. “We enjoy openness and trust in knowing Project HOPE is taking care of the patients, delivering the drug and providing feedback on what works and what doesn’t. During the challenging times, Project HOPE staff worked hard to move clinical appointments around. No one stopped treatment, and we never lost a patient. ”
As the successful program continues to give care to children in Egypt, Azevedo says she hopes that the lessons learned from this successful program can be applied to other countries as well.
“Genzyme does not look at this program from a cost perspective. We look at it from a patient perspective. Paying and non-paying patients are treated the same.” With the opportunity to serve true global Gaucher community, comes responsibility.