Treating a rare disease

People suffering from a rare affliction called Gaucher disease are receiving free medicines through a partnership between US-based Genzyme Corp and the China Charity Federation, or CCF.

Since 1999, the biotechnology company has provided its Cerezyme medicine to more than 130 patients in China through the international Project HOPE healthcare charity.

Project HOPE has been operating in China for more than two decades.

About 5,000 people around the world have been treated with Cerezyme through Project HOPE.

In December, the company and CCF reported plans to reach more victims of the rare genetic disease in China with the "CCF Cerezyme Charity Drug Donation Project."

Donations to fund the project were delivered by Project HOPE to CCF in July.

Already, more than 100 new patients with Gaucher disease have been approved for the medication.

Gaucher disease is a progressive, often life-threatening disease that is caused by an enzyme deficiency.

Symptoms can include bone abnormalities and severe neurological disorders.

Genzyme in the 1990s developed Cerezyme as a life-long treatment that now is in use in 90 countries.

The high cost of the drug -- more than 1.5 million yuan annually per patient -- led to the Genzyme/CCF partnership.

Participating patients can receive free medicines and treatment at six hospitals.

The hospitals are: Beijing Children Hospital, Peking Union Hospital, Shanghai Xinhua Hospital, Shanghai Children Medical Center, Zhejiang University Affiliated Children Hospital and First Affiliated Hospital of Sun Yat-Sen University.

CCF also has established a Rare Disease Office to promote public awareness about other rare diseases and available treatments.

While Genzyme is donating free medicines to treat Gaucher disease in 15 countries, the China project is unique in partnering with a country's charity federation, according to Tomye Tierney, vice-president and general manager of the company's emerging markets division.

"This partnership enables us to continue discussions with CCF and the government about which national policies should be adopted to treat rare diseases," said Erik Tambuyzer, senior vice- president of public policy for Genzyme.

Tierney said the goal is to reach as many people suffering from Gaucher disease as possible.

"We really want to be able to treat everyone who needs it," Tierney said.

Tierney said the company has built three treatment and education centers in China during the past decade and plans to double that number in coming years.

"I envision sharing responsibility for this project with local healthcare systems as a major challenge that we are working on collaboratively," she said.

Genzyme's Tambuyzer said that government support will be crucial to reaching a broader range of patients with Gaucher and other treatable rare diseases.

"This is something that we have to do in a public-private partnership, because this is too big for one company alone," Tambuyzer said.

"We need to work together with patients, with clinics, with the government and possibly with other companies to develop policies for treatment of patients with rare diseases," he said.

Tambuyzer said developing a biotechnology platform for public education and treatment of rare diseases also can fuel economic development in China.

"We strongly support China's healthcare reforms and its efforts to make biotechnology one of the pillars of future economic policies, since China is deciding on those types of priorities right now," he said.

(China Daily 08/03/2009 page10)