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June 2018 - ISSCR special edition

Gene and stem cell therapies restoring eyesight, saving skin, and more

  • Two people who had vision loss can now read again, thanks to a stem cell ‘eye patch’ treatment trial.
  • A Syrian refugee boy’s life was saved when Italian researchers grew new GM skin from stem cells to replace his fragile ‘butterfly skin’.
  • A gene therapy that cures an inherited form of blindness has been approved by the FDA.
  • A therapy for haemophilia A and B is showing long-term protection and a dramatic drop in bleeds in clinical trials.
  • A commercial factory-made stem cell treatment for a life-threatening complication of bone marrow transplants has dramatically improved children’s survival rates in a US-based trial.

After years of promise, stem cell treatments are now starting to move out of the lab and changing lives.

This was one of the take-home messages from last week’s gathering of leading gene therapy and stem cell scientists and physicians from around the world. They were in Melbourne for the International Society for Stem Cell Research 2018 Annual Meeting.

We’ve included highlights from Australian and visiting speakers below.

Delegates also heard about CRISPR/Cas9 gene editing from its co-inventor Jennifer Doudna, the huge progress in developing mini-organs from stem cells, and the potential to use stem cells for drug screening to help with personalised medicine.

And they discussed the challenges of unproven treatments and the moves by Australian and US regulators to tighten the rules.

We sponsored a public forum in the lead up to the meeting so that patients could bring their questions directly to the experts. We will share highlights and a link to a video recording of the event in a future bulletin.

Melissa Little, chair of the meetings scientific program committee and a stem cell researcher at the Murdoch Children’s Research Institute, shares her insights from the event in this newsletter.

Next week the 2018 Metcalf Prizes for Stem Cell Science will open for applications, offering two $50,000 prizes for Australia’s emerging leaders in stem cell research. Think about the rising stem cell scientists you know, and encourage them to enter.

Kind regards,
Dr Graeme L Blackman AO Chairman, National Stem Cell Foundation of Australia

In this bulletin:

  • What we learnt when 2,800+ stem cell scientists invaded Melbourne
  • Saving skin, restoring eyesight, treating diabetes, and taking treatments to the clinic
  • Stem cell news from around the world

What we learnt when 2,800+ stem cell scientists invaded Melbourne

An overview of ISSCR 2018 from Melissa Little 

2,832 delegates from 53 countries heard from 150+ speakers at last week’s International Society for Stem Cell Research Annual Meeting, held at the Melbourne Convention and Exhibition Centre.

Professor Melissa Little

Professor Melissa Little

Melissa Little, who led the development of the scientific program, says there were moving stories of successful treatments, unusually strong involvement from the business and industry sector, and scientific presentations showing awe-inspiring progress.

Roger Barker, chair of the ISSCR’s Clinical Translation Committee gave a great overview of the progress of treatments to the clinic, telling delegates what we’ve tried, where we’ve failed and why; what we’ve learnt and where we’re going. Steven Goldman showed you can repopulate the brain from stem cells,” says Melissa, a mini-kidney ‘organoid’ pioneer based at the Murdoch Children’s Research Institute.

“And the patient address from Daniel Fuller, the father of seven-year-old Harry who has a genetic deaf-blindness condition, was very moving.

“The science presented showed we’ve moved so far towards translation in the last decade. The promise is getting closer. Three years ago, at the Stockholm meeting, there was one presentation on using iPS cells in clinical trials. This year there were several. It’s no longer if, but when we will try treatments with this relatively new source of stem cell.”

Melissa, who is also President of Meeting co-host, the Australasian Society for Stem cell Research, was particularly proud of the contribution from Australian plenary and keynote speakers.

“Jane Visvader’s [Walter and Eliza Hall Institute] presentation was a tour-de-force, not only detailing where all breast cells come from but how they can develop into cancers,” says Melissa.

“Patrick Tam’s [Children's Medical Research Institute] lecture elegantly described how his lab created the raw data for a whole genome ‘satellite navigation’ map of early mouse development, showing Australia is still leading in fundamental research. And Clare Parish’s work developing cell therapies for Parkinson’s disease is truly world-leading.”

See social media highlights from the meeting via the hashtag #ISSCR2018, and look out for Australian content via #GDayStemCells.

Saving skin, restoring eyesight, treating diabetes, and taking treatments to the clinic

Our round-up of the life-changing scientific highlights from ISSCR 2018.

How new skin from genetically-modified stem cells saved a refugee boy’s life

Michele De Luca and Graziella Pellegrini (Italy) led the team that developed genetically-engineered stem cells to produce sheets of disease-free skin cells to save the life of a seven-year-old Syrian refugee. The patient suffered from ‘butterfly skin’, the genetic disease Epidermolysis Bullosa that made his skin as fragile as a butterfly’s wings. Now living with his family in Germany, Hassan is able to attend school, play soccer and live a full life.

Fighting type one diabetes with stem cells

Doug Melton (USA) reported on a way to use stem cells to produce the massive quantities of insulin-producing beta cells needed to treat people with diabetes, including life-threatening type 1 diabetes, which his two children have. Doug shared his lab’s work towards providing human islet cells for diabetics which have found ways to thwart immune rejection so that patients no longer require blood checks and insulin injections.

Michele De Luca and Doug Melton joined Foundation director and Hudson Institute stem cell scientist Caroline Gargett in a special episode of The Conversation Hour, talking about stem cell science with Jon Faine and science communicator Tanya Ha on ABC Radio Melbourne. Listen online.

(Left to right) Michele De Luca, Doug Melton, Caroline Gargett, Tanya Ha and Jon Faine at ABC Studios Southbank.

Stem cell eye patch cures blindness in two trial patients

Two patients with a common type of blindness are recovering their vision, thanks to a new stem cell ‘eye patch’ treatment.

The two patients, both with age-related macular degeneration (AMD), were the first to have diseased eye cells replaced with stem cell-derived retinal eye patches, which restored vision to both patients. They’re both now able to read again.

Age-related macular degeneration (AMD) is one of the leading causes of blindness in the world. Peter Coffey, who led the trial, hopes it will lead to an affordable ‘off-the-shelf’ therapy that could be made available to patients within the next five years.

This is in stark contrast with the cases of three US women with AMD whose eyesight worsened after paying for experimental and unproven stem cell treatment.

New cell therapy a life-saving peacemaker between warring patient and donor cells

A new factory-made stem cell product has been shown to dramatically increase the 100-day survival rates of children with an often-fatal complication of bone marrow transplants—acute graft versus host disease (GVHD).

Australian stem cell scientist and Mesoblast CEO Silviu Itescu announced the results of a Phase 3 clinical trial treating 55 children suffering from the most severe form of GVHD, a condition in which the patient’s white blood cells attack the transplanted donor cells.

The treatment involves mesenchymal stem cells, which have an anti-inflammatory and immune system-calming effect. 75 per cent of patients had survived 100 days after treatment, compared with a survival rate as low as 30 per cent in patients who fail to respond to the standard initial steroid therapy.

Mesoblast, an Australian biotech company, expects this will lead to the treatment becoming the first FDA approved ‘off the shelf’ commercially-manufactured stem cell product.

Treating haemophilia and eye disease with gene therapy

Katherine High (USA) reported on an FDA approved gene therapy for a form of blindness, and on a clinical trial in people with haemophilia.

She’s the head of research and development for Spark Therapeutics, a spin-out from the Children’s Hospital of Philadelphia.

Last year, the US FDA announced the approval of the first directly administered gene therapy targeting a rare inherited form of blindness caused by mutations in a specific gene. The therapy uses a modified virus to deliver a normal copy of a defective gene directly into retinal cells in the eye. The corrected code makes the eyes’ cells function normally, producing the proteins they need to restore vision.

In May, Spark Therapeutics also announced progress in clinical trials of gene therapies for bleeding disorders haemophilia B and A.

Stem cell news from around the world

Between newsletters, we share stem cell news on social media:

Here are a few media stories we’ve shared recently from the meeting:

Herald Sun: (Subscriber only) Two patients cured of blindness with new stem cell treatment

ABC Radio National: The hope (and hype) of stem cells

ABC TV 7.30: 'Buying hope': Unproven stem cell treatments facing tighter regulation

ABC Radio Melbourne: The Conversation Hour: Stem cell pioneers Doug Melton, Michele De Luca, and Caroline Gargett

The Age: Watch this fish regrow its spinal cord—in real-time