This blog is totally independent, unpaid and has only three major objectives.
The first is to inform readers of news and happenings in the e-Health domain, both here in Australia and world-wide.
The second is to provide commentary on e-Health in Australia and to foster improvement where I can.
The third is to encourage discussion of the matters raised in the blog so hopefully readers can get a balanced view of what is really happening and what successes are being achieved.
Sunday, February 25, 2024
It Seems There Are Some Encouraging Signs Coming From The Gene Therapy Arena.
This appeared a few days ago
‘Years my kids didn’t have’: Boost to Australian gene
therapy development
February 24, 2024 — 10.00pmWhen Megan Maack’s two children were diagnosed with a rare genetic condition causing childhood dementia in 2013, research teams overseas had a drug that could dramatically slow the disease’s progress.
But it took years for the drug to be developed to a clinical grade, and it is extremely difficult to enrol in trials of such drugs when they are held abroad.
“They were years my kids didn’t have,” said Maack, who is now CEO of the Childhood Dementia Initiative, a group advocating for the 2800 children born with conditions causing childhood dementia in Australia each year, including her children Isla, 14, and Jude, 12, who have Sanflippo syndrome.
“We need to get these drugs to kids when they are young. Once they hit a point of decline, the drug is just not effective,” she said.
“Unfortunately, at the moment, there’s just not the capability in Australia to develop clinical trials locally. It is more about developing small batches to test on mice.”
But Maack is optimistic about the future of treatment in Australia, with major investment in gene therapy in NSW that could see life-changing drugs for rare childhood diseases being produced and trialled in Sydney as early as 2025.
A $134.5 million world-leading “viral vector” manufacturing facility in western Sydney is expected to be fully operational by the end of this year.
Viral vectors – microscopic tools which deliver genetic material into cells – are providing hope in the treatment of rare genetic diseases and cancers through gene therapy, an area of medicine which has progressed rapidly in the past five years.
“It’s just unbelievable what’s evolving: the way we can fix genes is now more precise,” said Professor Ian Alexander, head of the gene therapy research unit at Sydney Children’s Hospital.
“Instead of putting an extra copy of a healthy gene in, we can go in and fix ‘spelling errors’ in existing genes.”
A recent success story has been the treatment of spinal muscular atrophy (SMA), a rare condition causing muscle wastage which is the leading cause of infant death.
Maack said this would be an ideal outcome for therapies developed for conditions causing childhood dementia. Like SMA, she said, parents are unlikely to know if their child is at risk, and early genetic treatment, before a child’s decline, is critical.
“Both parents need to have a mutation in the same gene, so you can’t look at your family history,” she said.
“The majority of families will be completely blindsided by the diagnosis.”
The state government announced today it had created a new company, registered as Viral Vector Manufacturing Facility Pty Ltd, to manage operation of the Westmead plant as it manufactures the therapies on a commercial scale, managing relationships between research teams, universities, hospitals, pharmaceutical companies and other stakeholders.
“My priority as health minister has been to embrace innovative initiatives in improving health outcomes for our community,” Ryan Park said.
“These therapies are expanding rapidly, targeting more and more genetic diseases, cancer and infections, significantly increasing the number of patients who can and will benefit from viral vectors.
It really must be one of the worst outcomes people can have to find their nipper(s) have a genetic flaw that will lead to disability, suffering or worse.
What is proposed here is clearly complex and expensive work with no guarantee of success and all most of us can do is watch and wait as the experts do their work and progressively more young lives can be saved / improved!
It’s good to see funding and effort are going to these endeavours. It is hard to see the rewards won’t be pretty large over time! I sure hope so….
"When Jobs and Apple’s other cofounders, including Steve Wozniak, first realized how big their company would be, they decided to go out and hire what they called “professional management,” or folks who just knew how to manage people. But it quickly backfired.
“It didn't work at all,” Jobs said in a 1985 interview. “Most of them were bozos. They knew how to manage, but they didn't know how to do anything.”
Jobs’ comment gets at the crux of a central debate in management: whether managers should actually want to be managers or not. Jobs argued the people who were least expecting to be leaders ended up being the best managers in the long run. That’s because other employees were more likely to actually learn something from them because they’ve mastered that skill—rather than only focusing on management techniques."
What an excellent insight. As a proven and effective leader and as a proven and competent manager I absolutely concur with your perspective. Steve Jobs understood. Thank you.
This may provide a clue as to why My Health Record and Digital Health have stagnated over the past twelve years.
ReplyDeleteThe only people ADHA can hire are those who don't want to or can't work in jobs that actually deliver useful stuff.
It starts at the top. This is from the CEO's bio
Ms McGrath has a Bachelor of Arts from Macquarie University; and a Master of Business Administration from Macquarie Business School;
Australia's National Digital Health strategy is being run by someone with an arts degree and who is a professional manager.
Steve Jobs adopted a no ‘bozos’ policy and said the best managers are those who never wanted the job—here are his 3 best management tips
https://www.aol.com/finance/steve-jobs-adopted-no-bozos-130000644.html
"When Jobs and Apple’s other cofounders, including Steve Wozniak, first realized how big their company would be, they decided to go out and hire what they called “professional management,” or folks who just knew how to manage people. But it quickly backfired.
“It didn't work at all,” Jobs said in a 1985 interview. “Most of them were bozos. They knew how to manage, but they didn't know how to do anything.”
Jobs’ comment gets at the crux of a central debate in management: whether managers should actually want to be managers or not. Jobs argued the people who were least expecting to be leaders ended up being the best managers in the long run. That’s because other employees were more likely to actually learn something from them because they’ve mastered that skill—rather than only focusing on management techniques."
What an excellent insight. As a proven and effective leader and as a proven and competent manager I absolutely concur with your perspective. Steve Jobs understood. Thank you.
ReplyDelete