New Hope for MPS II, ETR Launches in Taiwan

New Hope for MPS II, Enzyme Replacement Therapy Launches in Taiwan

Mucopolysaccharidosis II (MPS II) is a mucopolysaccharide storage disorder also  relatively known as Hunter Syndrome, a rare and life-threatening genetic  disorder, affecting at least 1 in 162,000 people in the world. All individuals with  Hunter Syndrome have deficiency of the lysosomal enzyme iduronate sulfatase,  which results in accumulation of mucopolysacchairdes. With progressive storage  of mucopolysacchairdes, the affected individuals may have clinical problems, such  as slowing development of intelligence, chronic ear infection, joint stiffness,  respiratory system and central nervous system damage. 

It was considered to be no cure for individuals affected by Hunter Syndrome. However,  an enzyme replacement therapy could be new hope for affected individuals. In 2006,  U.S. Food and Drug Administration granted marketing approval for Elaprase  (idursulfase), which is a recombinant enzyme replacement therapy. After 53-weeks,  randomized, double-blind, placebo-controlled trial, Elaprase (idursulfase) demonstrates  medical benefits on people suffering Hunter Syndrome. Since Elaprase (idursulfase) is  designed to replace the insufficient enzyme, it is unable to reconstruct pre-existing  illness and damaged functions in Hunter Syndrome patients' bodies. 

Elaprase(idursulfase) is not applicable to all Hunter Syndrome patients.  Some patients may have learning disabilities and mild physical problems, while others  don't. Patients who have intelligence damages and under 5 years old are excluded  from the treatment. It is because the molecules of enzyme are too large to get  through the blood-brain barrier which may prevent enzyme therapy from directly  helping the brain. 

In Taiwan, it is estimated there are 300 patients suffering from Mucopolysaccharidosis,  including 49 patients with MPS II. Treatment for MPS I and MPS VI have launched in  Taiwan last year, 12 patients have received the therapy. From now on, the privileges are not only for MPS I and MPS VI but also for Hunter Syndrome patients. Expected for  many years, enzyme replacement treatment for Hunter Syndrome finally launches in  March, 2007. The new enzyme replacement treatment is under the reimbursement of  National Health Insurance, which is prior to Asian countries. The individuals without  intelligence damages and above 5 years old get chance to be included in the treatment.  There are 5 lucky patients to receive the therapy, intravenous infusion weekly. To each  patient, the National Health Insurance predicts to input 330 thousand U.S. dollars to the  new therapy every year. The amount of reimbursement to this treatment totally granted  to 1.65 million U.S. dollars in 2007. 

A 13-year-old junior high school student, Yang Luo, who is affected by Hunter Syndrome  starts to receive infusion of Elaprase (idursulfase) every week. Except for Japan, this  lucky patient is the first one to receive the new MPS II treatment in Asia. Because the  new treatment is effective to reduce intelligence, organ and bone damage, Yang Luo  is expecting the future life and desirous to go to university. 

To individuals suffering from Hunter Syndrome, it's inspiring to hear the enzyme  replacement launched and under government reimbursement in Taiwan. In addition to  new treatment, the society with open mind and friendly circumstances is also expected  for MPS II patients. 

The successful ERT treatment demonstration also encourages patients in Hong Kong,  who still haven't got the chance to try the new treatment. Patients in Hong Kong have  no national insurance reimbursement for the ERT treatment and it is almost impossible  for them to afford the medicine. August 2006, TFRD and MPS patients from Taiwan met  MPS members of Hong Kong Mucopolysaccharidoses (MPS) and Rare Genetic Diseases  Mutual Aid Group. It was a great opportunity for MPS patients from Taiwan and Hong  Kong to meet each other. Yung-Shiang, Yang, deputy executive director of TFRD,  attend the meeting and to share our experience in advocating and social legislation. 

So far, Hong Kong MPS patients' situation remains the same. However, there will always  be hope that someday patients in H.K. can receive treatment. 

Besides, as the director of Taiwan MPS Association, Ms. Tsai, Chung-Wei indicates, as the  ERT treatment brings hope to MPS II patients, we should also consider how to make MPS  patients' life better, instead of simply prolonging their lives. Issue such as how to make  MPS patients’ quality of life better as their live longer and how to settle their lives when  their parents pass away have to be taken into serious consideration.