The road to treatment is slow for people who suffer from rare diseases in Hong Kong

The road to treatment is slow for people who suffer from rare diseases in Hong Kong

Access to medicine for people who suffer from Mucopolysaccharidoses has come a long way – but there is still a way to go


Xiao Hiu-chun (left) suffers from a type of MPS not covered by the Hospital Authority.
Photo: Dickson Lee/SCMP

For a long while, people in Hong Kong who suffered from Mucopolysaccharidoses Type 6 (MPS) weren’t able to afford the drugs used to combat the rare disease. Then, thanks to a government subsidy that was announced seven years ago which helps to pay for the treatment, they were. Not all MPS sufferers were as lucky.

Stephen Ma On-tat and his wife were two Hongkongers who benefited from the subsidy. When their son was two, they realised that something was wrong.

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There was no cure, they told him

“My son was clumsy, and had found it hard to move around,” Ma recalls. Doctors told Ma the boy had MPS, and said he would probably only live for around 20 years.

MPS causes the body’s tissues and organs to enlarge and become inflamed, or scarred. The last few years of his life would probably be spent in bed. The disease might also affect his son’s IQ. There was no cure, they told Ma.

The news devastated the couple, but when Ma saw an article that talked about another MPS patient, he started the Hong Kong Mucopolysaccharidoses & Rare Genetic Diseases Mutual Aid Group (HKMPS). He got in touch with the reporter who wrote the article so he could contact the patient in the story, and the group had their first meeting in 1998.

Now, HKMPS has 50 members and 16 consultants – health professionals, lawyers and consultants. They provide patients and their families help, support, and advice.

“MPS is a very rare disease,” Ma says, which is why he wanted to create a group to help those who suffer from it, and the families who are affected by it.

The group became a way for families to share their experiences with each other, and a place where they could ask for advice on how to take care of people with MPS.

“Later on, when treatments were developed in the 2000s, our group also started pushing for patients to have their treatment subsidised by the government, because the drugs are very expensive.”

While the treatment does not cure the disease, it does prevent the condition from getting any worse. Being able to take the drug means an MPS sufferer can live longer.

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The treatments are for life

Ma says that he was crushed when he realised that the treatment would cost US$500,000 a year, and that someone who had MPS would need the treatments for life.

At that time, the Hong Kong government was not providing MPS patients with any support, apart from the ability to claim up to HK$400 from a fund for the disabled. Not enough people suffer from it to make it worth a pharmaceutical company’s time and effort.

This changed in 2010, when the government announced that it would cover the costs of treatment for MPS patients who suffered from Types 1, 2, or 6. Ma’s son would get weekly treatments at Queen Elizabeth Hospital in Kowloon. Today, he is in his early 20s; a university graduate, who holds a full-time job.

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Which leads us to the latest news

It wasn’t all good news, as the government subsidy didn’t cover those who suffer from Types 4, 7, and 9. This is because some of the drugs that are needed are not on the Hospital Authority’s subsidy approval list.

Xiao Hiu-chun, who suffers from MPS Type 4, made headlines late last year after being told his treatment would be subsidised.

Hiu-chun, seven, has a bell-shaped chest and deformed wrists and legs. His family talked to doctors in Hong Kong, the mainland, and Taiwan before he was officially diagnosed at the age of three. His family made a public appeal for help after waiting for years to hear that his treatments would not be covered.

Then, in October, it was announced that the Hong Kong government had made a deal with a drug manufacturer to split the cost of his medication. Hopefully this victory for Hiu-chun is a sign that, soon, more people will be able to access the treatments they badly need.

What is Mucopolysaccharidoses (MPS)?

Humans need to have 11 special chemicals to break down long-chain sugars in their cells. Some people don’t produce one of the chemicals at all, while others might produce chemicals that don’t work. In either case, they are diagnosed with Mucopolysaccharidoses.

The long-chain sugars build up in the person’s cells, causing damage that can be very severe. In some forms of this disease, a bone-marrow transplant can help. In other cases, it needs to be combined with drug therapy.

Edited by Ginny Wong

This article appeared in the Young Post print edition as
The slow road to treatment


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