Genzyme and Project HOPE Treat Children with Gaucher Initiative in Mansoura

Egypt is one of the most populous countries in Africa and the most populated in the Middle East. Slightly over 74 million people call the north African country home, the majority of these live near the banks of the Nile River. Large areas of the country are covered by the Sahara Desert. Half of the population lives in urban areas.

 

Egypt is a semi-presidential republic. It is a low to middle income country with a sluggish economy, low economic growth and difficulty creating jobs. This has lead to high poverty and unemployment rates. Tourism is an important source of income for the country and terrorist attacks have had a negative impact on the economy.

 

Ad Daqahliyah is one of Egypt’s 29 governorates. Its capital El Mansoura is home to 420,000 and sits in the Nile Delta 120 km northeast of Cairo. Located in El Mansoura is the Mansoura University Children’s Hospital, a site of treatment for the Gaucher Initiative.

 

The Gaucher Initiative is an innovative humanitarian program that was established in 1999 to assist people in developing countries who are afflicted with Gaucher Disease. The program is a collaborative effort with Genzyme Corporation, a leading biotechnology company that innovates on behalf of people with serious diseases and provides medicines that meets major unmet medical needs. The Gaucher Initiative is the first alliance of its kind between a biotechnology company and a non-profit organization.

 

Genzyme provides free Cerezyme^® (imiglucerase for injection) for the Gaucher Initiative and Project HOPE distributes the drug through its international infrastructure. Cerezyme is a targeted enzyme replacement therapy that helps alleviate and even reverse many effects of Type 1 Gaucher disease. Cerezyme has been the standard of care for treating Gaucher disease since it was introduced in 1994.

 

Gaucher disease is an inherited, genetic disorder that afflicts approximately 1 in 50,000 people, or about 10,000 people worldwide. People with Gaucher disease lack sufficient levels of a particular enzyme that breaks down a fatty material, or lipid, into waste that can be removed from the body. Without enough of the enzyme to break it down, the fatty material accumulates in the cells of organs and bones. This accumulation can cause mild to severe symptoms that can appear at any time throughout life, but usually are first noticed in childhood or adolescence.

 

The symptoms of Gaucher Disease are easy bleeding and bruising, excessive fatigue, anemia, weak bones

fracturing too easily, bone and joint pain, and enlargement of the belly through increase in the volume of spleen and liver. Gaucher disease is progressive and, if left untreated, will usually become worse over time. With treatment, the disease process itself can be addressed, alleviating and usually reversing the many effects of Gaucher disease for most patients.

 

The Gaucher Initiative is guided by an expert medical committee of six internationally respected physicians. The group makes patient treatment decisions, monitors and reviews patient progress and advises the local physicians on appropriate patient care and correct administration of the treatment. Patients accepted into the program are provided with the enzyme replacement therapy More than 300 patients in 19 countries across Asia, Africa, Latin American and the Middle East have benefited from the program.

 

Donors and partnership: Genzyme, Project HOPE, Mansoura University Children’s Hospital

Tomye Tierney, Vice President and General Manager of Emerging Markets at Genzyme Corporation, has a blog www.facetofacegenzyme.com where she chronicles her travels for the Gaucher Initiative. Below is a post from her visit to Mansoura, Egypt.

We got off to an earlier start this morning even though Mansoura, our destination, was about half the distance as Alexandria. We took what is known as the "agriculture road," and as the name implies it was green with crops and vegetation. I learned that yesterday's route is known as the "desert road," and aptly so!

 

Mansoura is a pleasant city, built along the Nile as most of the cities are. We arrived at Mansoura University Children’s Hospital, or MUCH. (I like that, very MUCH!). We were welcomed by Dr. Ahmed Abdallah, Prof. of Pediatrics and Director of the pediatric gastrointestinal unit. And yes, he even has MUCH printed on his card.

The patients and their families were waiting. Many had come quite a distance just to meet us, so Dr. Abeer, their treating physician, took us upstairs to two large, windowed rooms where the patients come every two weeks for their Cerezyme infusions.

 

Since this was my first trip to Mansoura, it was also the first time for me to meet these patients. The smallest little guy - about a year old - was sound asleep while getting his 2-hour IV infusion.

 

Dr. Abeer said it was touching how tightly everyone has bonded. She amusingly told us that all she has to do is explain something to one patient and before she can begin contacting the others the word has spread like wildfire. Amazing since they are all children, the oldest being 11.

 

She is impressed by the extent to which the parents have become thoroughly informed about the disease and the infusion process. This helps make things run more smoothly and quickly at the hospital.

 

Everyone comes to the hospital on the same day and so have become like family. Recently a new patient arrived for the first infusion and the group immediately welcomed them and explained about what to expect. And most importantly to give them their moral support.