November 2017

Finally! Better stem cell regulation in Australia and USA; private vs public cord blood banking


Welcome to the National Stem Cell Foundation of Australia’s bulletin on stem cell medicine and research in Australia.
 
The Australian and US governments are finally acting so that regulations to protect patients can catch up with the rapid proliferation of unethical clinics offering unproven stem cell treatments.
 
Australian patients are spending vast sums on unproven and ineffective therapies and patients have died as a result. In the US, one company was mixing stem cells with smallpox vaccine. Read on for details.
 
In contrast, established stem cell treatments, such as cord and bone marrow transplants, have been saving lives for years.
 
This month we also ask, ‘Should you store cord blood?’ More below.
 
Australia’s Chief Scientist Dr Alan Finkel has also published an explainer on the topic of gene editing. Read on for more information.
 
Who can you trust on Twitter? US stem cell scientist and author Paul Knoepfler has written a handy list of the top 50 stem cell influencers on Twitter to follow, which we’re delighted to share (particularly since we’ve been included).
 
Finally, we look forward to sharing the news of the winners of the 2017 Metcalf Prize for Stem Cell Research in the coming days.  

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

In this bulletin:

  • Regulation to reign in rogue stem cell practices
  • US FDA crack down on dubious clinics
  • Cord blood: to bank or donate?
  • What is CRISPR and what are its implications?—Chief Scientist explains
  • Stem cell news from around the world

Regulation to reign in rogue stem cell practices 

The Therapeutic Goods Administration has announced that 2018 will see long-awaited reforms to the regulation of stem cell therapies in the hope of curtailing unsafe or unproven treatments, and the unethical marketing of them to vulnerable people.
 
 The announcement follows two rounds of public consultations (in 2015 and 2016) in response to serious concerns raised by doctors, researchers and patient advocates. It also follows a coronial inquest finding a dubious stem cell treatment for dementia led to the death of a NSW woman, and a rapid proliferation of stem cell clinics offering unproven treatments for conditions including arthritis, pain, spinal cord injury, multiple sclerosis, diabetes and infertility. They’re even offered for ‘facial rejuvenation’.
 
Previously, a loop-hole allowed registered medical practitioners to offer treatments using ‘autologous’ stem cells—stem cells derived from a patient’s own tissues, usually obtained from fat tissue by liposuction.
 
Read full TGA announcement: Regulation of autologous cell and tissue products.
 
While the details of the new regulations are still to be determined, it is hoped this loop hole will be closed, subjecting autologous treatments to the safety standards required of drugs and other new treatments.
 
The Foundation’s Science and Ethics Committee has issued a statement in response. 
 
The Foundation is pleased to see the announcement by the Therapeutic Goods Administration (TGA) regarding regulation of autologous stem cell products used for treatment in Australia. 
 
It addresses the current anomaly where autologous cell products—prepared from patients' own tissues for re-administration as treatments for a range of illnesses—do not have to comply with the strict standards that apply to drugs and protein products used in patients.
 
The proposed regulatory changes briefly outlined in the announcement will be drafted into specific and detailed regulations by the Office of Parliamentary Counsel, and submitted to Government for approval.
 
The important points are: 
  • a ban on direct advertising of stem cell ‘treatments’ (specifically ‘products’) to consumers,
  • a restriction of the regulatory exemption to cell and tissue products manufactured and used in a hospital by a medical or dental practitioner for a patient in his or her care, and 
  • the introduction of regulations to bring products currently exempt under the TGA’s regulatory umbrella. Products previously outside this class will be subject to the more stringent requirements of the Biologicals Regulatory Framework.
Salient aspects of the review recommendations include the addition of dental practitioners to the approved user category—there is high quality research into dental applications of stem cells in Australia—and defining accredited hospitals as sites approved for stem cell treatments.
 
Much will depend on the details of the regulations that emerge. How will the term “accredited hospitals” be interpreted for instance? In its responses to the TGA’s consultations, the Foundation expressed particular concern over the lack of ethical review or effective capture of information about treatment outcomes, which is currently a feature of the stem cell ‘industry’.
 
Will we see new hospitals opened specifically to comply with the regulations, but still lacking ethical review or useful data collection? We also pointed out the lack of Good Manufacturing Practice standards that are compulsory for the pharmaceutical industry but have not been followed by current stem cell practitioners. 
 
We are pleased that a regulatory framework is under development.  We hope our deep concerns for the wellbeing of patients and their relatives will be fully addressed.
 
Related story: Finally, unproven stem cell clinic practices might be curtailed via The Conversation.
 

US FDA crack down on dubious clinics

Smallpox vaccine used in dangerous stem cell treatments seized
 
The questionable and failed treatments performed by US stem cell clinics—from macular degeneration therapies that left three patients legally blind, to smallpox vaccine used in risky cancer treatment—have led to action from the Food and Drug Administration.
 
On Friday 25 August, US Marshalls seized five vials (500 doses) of smallpox vaccine from StemImmune Inc. in San Diego to prevent its use to treat cancer in the California Stem Cell Treatment Centers clinics. Four vials were intact; the fifth was partially used.
 
The treatment reportedly involved mixing the vaccine with stem cells derived from a patient’s fat and injecting the mixture intravenously and directly into malignant tumours—an unproven and potentially dangerous approach for both patients and any unvaccinated people they have close contact with who are pregnant or have certain heart, immune system or skin problems.
 
In a statement, the FDA expressed concerns about how the company sourced the vaccine, and a determination to investigate these kinds of unscrupulous clinics.
 
“Our actions today should also be a warning to others who may be doing similar harm, we will take action to ensure Americans are not put at unnecessary risk,” said FDA Commissioner Scott Gottlieb, M.D., who is also a cancer survivor.
 
Earlier in the year, The New England Journal of Medicine reported the cases of three patients of the US Stem Cell Clinic in Florida who had their macular degeneration treated with injections of stem cells derived from their fat, only to experience further vision loss.
 
There are nearly 600 stem cell clinics operating in the USA, offering treatments for everything from cancer to Parkinson’s disease to rheumatoid arthritis, typically costing $5,000 to $10,000 per round. The FDA has updated its consumer information page about stem cell treatment claims.
 

Cord blood: to bank or donate?

Should you collect your cord blood and donate it to the community? Or should you bank it privately on the off-chance your child will need it in the future? 
 
 Expectant parents are confused about their options, with some paying thousands to privately store cord blood, but few ever needing to use it. We asked the director of Melbourne’s public cord blood bank to outline the pros and cons of both.
 
Cord blood—collected from the umbilical cord at birth—is rich in blood-forming stem cells, and can be used as a replacement for bone marrow in bone marrow transplants.
Prospective parents can either pay for a private bank to collect and store the cord blood in case the child (or a family member) needs it in the future, or donate their cord blood to a public cord blood bank, making it available to people from around the world looking for a bone marrow transplant match.
 
A rapidly growing number of parents are paying thousands of dollars for the services of private cord blood banks, following the trend set by celebrity mums, such as Rebecca Judd and Tori Spelling. But is it a necessity, or a very expensive insurance policy?
 
Public cord blood banking started in Australia more than 20 years ago. Dr Ngaire Elwood is Director of the BMDI Cord Blood Bank, one of Australia’s three public cord blood banks, and where more than 13,000 cord blood units are stored.
 
“We answer phone calls every day from people wanting to know whether they should bank privately or publicly, and the differences between the two,” Ngaire says. “There is a lot of confusion out there, and a lot of misinformation.”
 
Ngaire says that only a very small portion of the cord blood units stored privately are ever used.
 
“It’s very expensive to collect and store cord blood,” says Ngaire. “Private banks charge a collection fee and an annual storage fee up to 18 years. It varies between banks, but that blood can only ever be used for that child or a family member.”
 
Generally, it costs $3,000 to $4,000 to have cord blood collected and stored for a period of 18 years, or $5,000 to $6,000 to have both cord blood and tissue collected and stored.
 
“In Australia, we’re fortunate. The handful of private cord blood banks we have are pretty good because they’re regulated. I have inspected cord blood banks around the world where they are happy to take people’s money to bank 10 millilitres of cord blood, which is nothing!”
 
Ngaire says there are some instances where banking a child’s cord blood for potential use in a transplant may be worthwhile. For example, a family with a child with leukaemia may wish to save the cord blood from a subsequent pregnancy.
 
“However, you have to keep in mind there’s a chance people won’t be a tissue match with their sibling. Also, with hereditary conditions like thalassaemia and aplastic anaemia, you can’t assume the baby doesn’t have the condition the older sibling has. Or if your child developed leukaemia, for example, you wouldn’t want to be using their own cord blood because it’s likely to have the leukaemic clone.”
 
Public cord blood donation is free, and is done by specially trained collectors in a handful of participating hospitals. Collected blood is taken to a processing lab at the children’s hospital. The red blood cells and plasma are removed, leaving the ‘buffy coat’ which is rich in blood stem cells, then carefully frozen and stored until they’re requested for transplant.
 
The number of cells in the unit are counted and the human leukocyte antigen (HLA) tissue type is checked, which is used for blood matching in transplants. This information is put into an international registry.
 
“One of the fantastic things about cord blood is that, unlike bone marrow, you don’t need a perfect HLA match. The cells in cord blood are still quite naïve; there’s a little bit of leeway,” says Ngaire, who is also head of the Cord Blood Stem Cell Research Program at Murdoch Childrens Research Institute.
 
“Nearly everyone who needs a bone marrow transplant will be able to find a suitable cord blood donor in one of the public cord blood banks around the world.”
 
Melbourne’s cord blood bank has released more than 540 units to children and adults, both in Australia and overseas. It’s part of an international network of public cord blood banks.
 
On rare occasions and with a treating doctor’s approval, a hospital may collect and store a baby’s cord blood without charge for a family member who has an illness that can be treated with cord blood transplantation.
 
If you decide to privately bank cord blood, check the following:
  • Ask what the minimum volume they’ll bank is.
  • Many private banks also offer to bank cord tissue. Consider whether it’s worth paying extra to bank cord tissue. It’s currently unknown whether you can get healthy, viable cells from cryogenically frozen cord tissue.
  • Ask what arrangements are in place should the cord blood banking company go broke and fold.
  • All private cord blood banks in Australia are licensed by the Therapeutic Good Administration. Don’t assume cord blood banking operations overseas have the same level of regulation. Check what they’re certified for and regulated for.
 
For more information about public cord blood donation, visit:
www.abmdr.org.au/public-cord-blood-donation
 
15 November 2017 is World Cord Blood Day. For information, visit www.worldcordbloodday.org
 

What is CRISPR and what are its implications? Australia’s Chief Scientist explains

Gene editing in the spotlight
 
 US scientists have successfully repaired a heart defect-causing gene in human embryos, reporting their results in a Nature paper published in early August to wide media attention.
 
The researchers used a gene-editing tool ‘CRISPR-Cas9’ that acts like a pair of molecular scissors to find and edit the gene mutation that causes the heart-weakening condition hypertrophic cardiomyopathy.
 
The findings have put the spotlight on CRISPR and raised the hope of one day preventing a range of inherited diseases. But we’re still a long way off.
 
The Office of the Chief Scientist has published an occasional paper Gene Editing and CRISPR. The paper provides a general introduction to gene editing, and explores the implications of CRISPR.
 
Read more at the Office of the Chief Scientist website.
 

Stem cell news from around the world

 
Between newsletters, we share stem cell news on social media: Here are a few of the stories we’ve shared recently:
 

Phys.org: Life-long blood production depends on hundreds of cells that form prior to birth

BioWorld: Zika virus kills glioblastoma stem cells in early research

The Australian: Newborn babes at heart of life-saving research

Financial Times: Stem cells: Japan’s scientific ‘moonshot’

The Guardian: Dr Con Man: the rise and fall of a celebrity scientist who fooled almost everyone

Space.com: Deep-space challenge: broken bones may heal best in Earth-like gravity

Reuters: 'Reprogrammed' stem cells fight Parkinson's disease in monkeys

Los Angeles Times: FDA cracks down on clinics selling unproven stem cell 'therapies'

New York Times: F.D.A. cracks down on ‘unscrupulous’ stem cell clinics

Tass Russian News Agency: International research team discovers new system of liver regeneration

Cosmos Magazine: Immune cells may prevent stem cell growth in spinal cord repair

Japan Times: Six arrested in connection with unauthorized cord blood treatments

Science Daily: Adult brains produce new cells in previously undiscovered area

New Scientist: Stem cell technique could reverse a major type of infertility

ABC: First embryo gene repair holds promise of wiping inherited diseases out of family's bloodline. Nature paper.

ABC: What you need to know about scientists fixing mutant genes
 

About the Foundation

The National Stem Cell Foundation of Australia is an ATO-registered tax-deductible Health Promotion Charity dedicated to promoting the study and responsible use of stem cells to reduce the burden of disease.

The Foundation’s activities include:

  • supporting research that pursues cures for as-yet-untreatable diseases
  • building a community of people with a shared interest in stem cell science
  • providing the Australian public with objective, reliable information on both the potential and risks of stem cell medicine.

We are working to build a community of people with a stake in stem cell science and to promote collaboration between scientists locally and internationally.

Please feel free to contact the Foundation’s Executive Officer Julia Mason via jmason@stemcellfoundation.net.au

Resources

We aim to:

  • Promote the study and use of stem cells

  • Prevent or control diseases or illness

  • Enhance public education about stem cells