Using only two fingers, Hong Kong girl types 22-page proposal calling for life-saving muscle drug
Josy Chow Pui-shan, 23, wants government to look into new but costly medication for spinal muscular atrophy patients
Using just two fingers, a 23-year-old Hong Kong woman who is almost completely paralysed by a rare disease has written a proposal calling for patients to be treated with a new drug that could be life-saving.
Josy Chow Pui-shan, who was born with spinal muscular atrophy (SMA), a genetic disorder that gradually destroys physical abilities such as walking and breathing, urged the government to introduce nusinersen, marketed as Spinraza, the first approved drug for SMA by the US Food and Drug Administration.
She will submit the proposal in a petition on Sunday staged by SMA patients and families outside the office of the chief executive in Admiralty.
The therapy, approved in December last year, was deemed as a lifesaver for the incurable disease. But it has not been registered in Hong Kong yet.
Typing with her right thumb and right index finger on a special mouse pad, Chow, an English and linguistics student at the University of Hong Kong, could only form four to five words a minute on her laptop, and needed to rest for five to 10 minutes between lines.
“It is really tiring to write with two fingers only ... but I don’t want other children [in a similar condition] to experience the hardship I have had growing up,” Chow said.
To cope with her breathing difficulties, she wears a face mask for ventilation and has a plastic suction tube in her mouth to clear away mucus in her throat.
“I could write and press phone keys in the past, but now I can’t do so,” Chow said. Two years ago, her daily use of the ventilator extended from eight hours to about 16 hours.
Since June, she has compiled a 22-page long report with over 100 pages of appendix, aiming to explain the rare disease and analyse the cost effectiveness of nusinersen. The cost of the drug per patient is about HK$6 million in the first year of treatment and HK$3 million annually in subsequent years.
There are an estimated 85 SMA patients in the city, with 20 of them suffering from the most severe form of the disease and requiring frequent intensive care in hospital.
Chow said that spending HK$6 million a year on medication for a patient would be more cost effective than forking out HK$24,400 a day in medical bills for intensive care in a public hospital – which totalled about HK$8.9 million annually.
She called for the government to work on a programme with the drug maker to allow patients with the biggest need “compassionate use” of nusinersen.
Compassionate use refers to access to free treatment before a drug is approved in a patient’s country, usually involving an arrangement between the local government and the pharmaceutical firm.
If Chow’s hopes are realised, patients like Wong Yuet-ka, a four-year-old girl who suffers from the most severe form of SMA, may benefit from such medication.
Wong, affectionately known as Ka Ka to her family, was diagnosed with the illness when she was about a year old.
Ka Ka has to use a ventilator 24 hours a day, and spends all her time lying on a sofa at home.
“I want to go to Ocean Park,” she said in a soft voice. But the travel from her home near Choi Hung to the park in the south of Hong Kong Island would be too much for her fragile body to bear.
Biogen, the firm that produces nusinersen, said it was “continuously assessing the possibility of expanding access” of the drug across the globe, including to Hong Kong.
The Hospital Authority said it had been in contact with the drug maker to gather available data for clinical trials.
“From what we understand, the company’s registration and supply plan to Hong Kong have not yet been confirmed,” a spokesman said.
A Food and Health Bureau spokesman said the government had been contacting the drug company through different channels as part of its plan to introduce the drug in Hong Kong.