Desperate parents launch S$2.4 million crowdfunding campaign to save baby Lucas from rare genetic disorder

SINGAPORE — Lucas, a 2-month-old baby diagnosed with a rare genetic disorder, has parents who have been left with no choice but to appeal for crowdfunding from the public. Lucas was diagnosed with Spinal Muscular Atrophy (SMA) Type 1 which requires Zolgensma treatment priced at S$2.4 million, which is not subsidised by the government. SMA Type 1, also known as Werdnig-Hoffmann disease, is a genetic neuromuscular disorder that progressively weakens and damages muscles over time, affecting the brain and spinal cord. Unfortunately, the damage caused by this disorder is irreversible. According to the parents, Andy and Lavin, who shared information on the crowdfunding platform Ray of Hope, they noticed abnormal signs when Lucas turned two months old. He did not seem to enjoy using his legs to stretch or kick. By his 9th week, the parents decided to consult with doctors. However, their hearts sank when they observed the doctors' unexpected reactions, followed by a flurry of tests. On June 26, 2023, Lucas was diagnosed with SMA Type 1, which typically has a life expectancy of only two years. "SMA will relentlessly ravage his tiny muscles, denying him the simple pleasures we often take for granted—sitting, crawling, standing, walking, swallowing, and even breathing." Currently, Lucas relies on two temporary medical supports to survive: a nose mask connected to a ventilator and a feeding tube painfully inserted into his nose. Unlike other children, Lucas drinks his milk from his nose through the tube. There is a treatment called Zolgensma that can save Lucas' life. However, the cost of S$ 2.4 million is an overwhelming sum beyond Andy and Lavin's means, in which they described it as 'the world's most expensive drug'. "Thus, we are humbly turning to the compassionate community for help. We know that this is a big ask and an ambitious goal. But as parents, we must do everything we can to give our son a fighting chance at life." The parents also ask for assistance in sharing their crowdfunding campaign and reaching out to organisations, communities, or sponsors who may be willing to support them. Individuals who wish to help save baby Lucas' life can donate via the following link: https://rayofhope.sg/campaign/baby-lucas/, or reach out to the parents on [email protected] for further details. The public can also stay updated on the crowdfunding and Lucas' treatment progress through Instagram and Facebook. As of 12 July, the parents have managed to raise S$ 824,950, which is only 34% of the total cost. Nearly S$ 1.5 million is still needed to secure the treatment for baby Lucas.

Other parents who also had to sought crowdfunding for their kids dignosed with the rare genetic disorder

In April 2023, TOC reported that another 19-month-old baby, Pilapitiya Shamel Adrian, also diagnosed with SMA when he was 14 months old, raised a fund of S$3 million to secure the one-time gene therapy drug Zolgensma. A spokesperson from National University Hospital (NUH) revealed that Onasemnogene abeparvovec (Zolgensma) is a novel drug that was approved by the US Food and Drug Administration (FDA) in May 2019 as a one-time gene therapy for children. In November 2022, The Straits Times reported that the parents of a five-month-old boy with SMA type 1 had just managed to raise $3 million through crowdfunding for his treatment. Available in Singapore only via the Special Access Route – which allows for unregistered life-saving drugs to be imported into the country – it is often touted as the most expensive drug in the world, costing almost $3 million. A written reply from Singapore Health Minister Ong Ye Kung in March 2023 to a Parliamentary question filed by Louis Chua, Member of Parliament for Sengkang GRC, revealed that about 40 to 50 persons in Singapore are currently diagnosed with SMA. Risdiplam is the only treatment option registered with and approved by Health Science Authority (HSA), and It costs around S$375,000 a year in Singapore's public healthcare institutions.

Health Minister: Singapore's healthcare financing system is not designed to support such high-cost treatments

On 8 November 2022, the Health Minister, in a written reply to parliamentary questions filed by MP for Aljunied GRC, Leon Perera, stated that Singapore’s healthcare financing system is not designed to support such high-cost treatments for rare illnesses. He noted how the financial needs of Singapore citizens with rare diseases are supported collectively through the Rare Disease Fund (RDF) with the Government providing three dollars of matching grants for every dollar of donation. He pointed to examples of how some patients are able to obtain support from the community through crowd-funding or other charity funds and shared that the Ministry of Health (MOH) will continue to monitor the situation and work towards expanding the scope of RDF treatments that can be supported. “We also encourage members of the public to support the RDF with donations so that more medicines and conditions can be listed, and more patients can be supported.” According to RDF’s website, a rare disease is defined as one which affects fewer than one in 2,000 patients. Currently, the RDF has listed 7 medicines for 5 conditions.

$19.2million raised by RDF in 2022

In another parliamentary reply, Mr Ong revealed that the RDF raised $4.9m in public donations in the calendar year of 2022.

$2.3m was contributed by foundations, $1.3m was from companies, and the remaining was from individuals and others.

Together with the Government’s $3 to $1 matching grant, this brings the total funds raised by RDF in 2022 to $19.2m.

SDP Chairman: healthcare is a basic human right, not appropriate to fund treatment of rare diseases through crowd-funding or charity funds

However, in November last year, Dr Paul Ananth Tambyah, the Chairman of the Singapore Democratic Party (SDP), expressed his viewpoint that relying on crowdfunding or charity-based funds to support treatments for patients with rare diseases is not appropriate for Singapore. Dr Tambyah highlighted several problematic issues associated with crowdfunding, despite its widespread practice worldwide for drugs like Zolgensma. These issues include loss of privacy, overstating the benefits of drugs, and various other concerns. “The drug company which makes zolgensma has tried to respond to global criticism of its pricing by introducing a lottery in which 100 children get the drug free – that is even more problematic as it creates a kind of “hunger games” situation.” “The SDP believes that healthcare is a basic human right and there should not be a price to that. There are alternative approaches which can and should be used.” said Dr Tambyah. Dr Tambyah, who established the National University Hospital’s Division of Infectious Diseases in 2003, noted that SDP’s healthcare policy proposed negotiating intensely with the manufacturers of these drugs and devices to force lower prices. He said that this has already worked even for a drug like zolgensma which is cheaper in Japan than in most other countries due to Japanese government negotiations. “If this does not work, the SDP plan advocates compulsory licensing, which is to enable parallel drug manufacturers to produce the drugs – this has been done for other drugs in Thailand, India and Brazil and there is a good chance that this will happen with these expensive gene therapies with time.” “This is actually allowed under the WTO’s TRIPs but only in low and middle-income countries because the assumption is that high-income countries will pay for these drugs for their citizens.” “Unfortunately for those in Singapore and the United States, that is not always true and children born with these conditions may have to depend on charity.” “This is really unfair as it is no fault of the child and that is why we in the SDP believe in a universal insurance policy which has a reinsurance component. The whole concept of insurance is risk pooling against rare and unusual situations.” said Dr Tambyah.