Interesting news story on stem cell transplants for Type 1 diabetes

Note:
I am not medically trained and it is 13 years since I worked in this area. 
Single words below in bold italics are explained in the Jargon Buster at the end.

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The news story
London [as in Ontario, Canada] woman off insulin for Type 1 diabetes after a single dose of experimental manufactured stem cells (21 June 2025) CTV News
- read to the end for a cracking typo / correction :D

It's really exciting news and hopefully can be expanded to more people with Type 1 diabetes but, as is almost always the case, it's not a simple fix.

When I worked at Diabetes UK a question that came up frequently at any research talk I gave, or through our question and answering service, was along the lines of "Can't people with Type 1 diabetes just have their beta cells replaced with stem cells?"

Yes.... but...

Type 1 diabetes is an autoimmune condition
A person with Type 1 diabetes has an autoimmune condition in which their immune system selectively goes after and destroys the insulin-producing beta cells in the islets of their pancreas. Destruction is a gradual process but at some point there won't be enough beta cells to produce enough insulin (an essential hormone) and the person will start to show unavoidable symptoms of Type 1 diabetes, requiring daily insulin injections or use of an insulin pump. 

Their immune system has attacked their own insulin-producing cells. 

Using your own stem cells, or other cells, as a source of potential replacement cells
If a person with Type 1 had stem cells taken from their own body, the cells tweaked in some way to convert them to insulin-producing beta cells and then returned to the person's body I think there's a high chance that the immune system would attack these genetically-identical-to-the-last-lot-of-beta-cells in the exact same way. Possibly the cells would survive for a period of time before the autoimmune system got round to munching through all of them, but I'm fairly certain it would happen. 

Using cells from elsewhere
If you were to take or make beta cells, derived from stem cells or another source, from any other person you'd also have the problem of tissue rejection or organ rejection that happens whenever your immune system meets something 'foreign' (after all that is what it has evolved to do). 

For the autoimmune side of things I don't know if it's because the immune system has some objection to (or at least is triggered by) -

(a) beta cells in particular - perhaps there is some marker on the surface of the cell, e.g. a glycoprotein in the cell membrane to which the immune system 'takes offence' and so will attack any beta cell. An analogy to this might be a computer virus that attacks any PDF file.
or
(b) any cell that secretes insulin (so perhaps tweaking other types of cells to be able to produce insulin wouldn't solve the problem). An analogy to this might be a computer virus that attacks any file that has the word 'insulin' in it, whether it's in a doc, spreadsheet, PDF or reminder note to collect a prescription.

The need to suppress the immune system
The 'solution', such as it is, when giving anyone a donor organ (pancreas) or cell transplant (islets) is to accompany that with immune suppressing medication that stops the immune system from attacking the newly transplanted tissue. That's good but having your immune system suppressed is not an easy way to go through life (for example think back to the groups of people needing the most protection from Covid).

The news story about a stem cell trial
The news article above reports on an encouraging development in using an external ('allogeneic') source of stem cell-derived insulin-producing cells* to help people with Type 1 diabetes in Canada (Canadian scientists have been doing amazing work in Type 1 diabetes and transplantation research), where their diabetes was difficult to manage. This was a small trial with 14 patients.

The woman interviewed has been able to come off insulin for now (I don't know if that will be permanent) but she does have to take immune suppressing medicine for life (or for the length of time that the transplanted cells survive). The article also notes the death of two of the trial participants, one was possibly linked to immune suppression but the person died from infection following an unrelated surgery. The other death seems entirely unrelated to the trial as they died from dementia.

*the stem cells were used to make more than just the insulin-producing beta cells, so the end result was slightly more similar to the grouping of cells (islets) found naturally in the pancreas.

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Further reading
The news article (also linked above): London [as in Ontario, Canada] woman off insulin for Type 1 diabetes after a single dose of experimental manufactured stem cells (21 June 2025) CTV News

which is about the study reported here: Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes (20 June 2025) New England Journal of Medicine, DOI: 10.1056/NEJMoa2506549

and this paper has also been reviewed here: Can Stem Cells Cure Type 1 Diabetes? (10 July 2025) NEJM Journal Watch
"Phase 2 trial results look promising... Although the treatment potentially obviates some issues with pancreatic transplants (e.g., organ availability and surgical complexity), it still requires immunosuppression."

See also the section headed "Promising results from beta cell therapy trials in type 1 diabetes" in Diabetes UK's research news roundup for 3 July 2025.

Jargon buster
allogeneic / allotransplantation: 'allo' here means 'other' and refers to cells or organs being transplanted from someone else. Allogeneic just means 'contains material from someone that isn't you'.

glycoprotein - a molecule that contains a sugar element and a protein element. All sorts of molecules are found inside, within and outside the cell membrane. Some are used as a sort of 'flag' to say 'this is the type of cell I am'.

islets - 'islets of Langerhans' - groups of hormone-secreting cells scattered within the pancreas. Islets include beta cells which release insulin, and alpha cells that release another hormone glucagon. Both help to control the level of glucose in the blood.

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Anyway let's all enjoy the news site's correction - "The Edmonton Protocol involves cells from deceased organ donors, not diseased organ donors."

#Burzynski - is this normal clinical trial practice?

The year before last I wrote a blog post expressing surprise at how many trials the Burzynski Clinic had running and after recent events (rumours that the Burzynski clinic is under unprecedented scrutiny from the FDA, but note this caveat tweeted by @medtek) I wondered what had changed.

Back in November 2011 I found 61 trials, this year by searching for the name Burzynski it seems there are now 62 (however the last one, this trial, actually relates to a completely different Burzynski so no change in numbers at all, h/t @dianthusmed).

It also appears that there have been more trials but not all of them registered, according to a paragraph on a page from 1998 on Quackwatch's website, highlighted to me by @jonmendel

"Working outside peer review, Burzynski is conducting 71 concurrent, preliminary phase II trials that cover most cancer indications-an unheard of number for a single investigator, and for a drug which is yet to be proven effective for any indication."

What struck me was that the ones I looked at on the first page (of 'unknown' status) all follow the same format of "Antineoplaston therapy in treating X (patients) with Y (type of cancer)" and all on the first page (shown below) are followed by this line (indicating the type of treatment given) appears below all of them: Interventions:     Drug: antineoplaston A10;   Drug: antineoplaston AS2-1

From the first page of results (brown = status unknown for more than two years, red = withdrawn, purple = terminated, green = completed - though is it published and what happened?):

• Antineoplaston Therapy in Treating Patients With Stage IV Adrenal Gland Cancer
• Antineoplaston Therapy in Treating Patients With Residual or Recurrent Anaplastic Astrocytoma
• Antineoplaston Therapy in Treating Patients With Brain Tumors
• Antineoplaston Therapy in Treating Children With Brain Tumors
• Antineoplaston Therapy in Treating Children With Low-Grade Astrocytoma
• Antineoplaston Therapy in Treating Patients With Anaplastic Astrocytoma 
• Antineoplaston Therapy in Treating Patients With Recurrent or Refractory Mixed Gliomas
• Antineoplaston Therapy in Treating Patients With Primary Malignant Brain Tumors
• Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors
• Antineoplaston Therapy in Treating Children With Visual Pathway Glioma
• Antineoplaston Therapy in Treating Patients With Refractory or Recurrent Intermediate-Grade Stage II, Stage III, or Stage IV Non-Hodgkin's Lymphoma
• Antineoplaston Therapy in Treating Patients With Recurrent or Refractory High-Grade Stage II, Stage III, or Stage IV Non-Hodgkin's Lymphoma
• Antineoplaston Therapy in Treating Patients With Mantle Cell Lymphoma
• Antineoplaston Therapy in Treating Patients With Primary Central Nervous System Lymphoma
• Antineoplaston Therapy in Treating Patients With Advanced Mesothelioma
• Antineoplaston Therapy in Treating Patients With Stage IV Melanoma
• Antineoplaston Therapy in Treating Patients With Multiple Myeloma
• Antineoplaston Therapy in Treating Patients With Recurrent or Refractory Waldenstrom's Macroglobulinemia
• Antineoplaston Therapy in Treating Patients With Metastatic, Recurrent, or Refractory Neuroblastoma
• Antineoplaston Therapy in Treating Patients With Neuroendocrine Tumor That Is Metastatic or Unlikely to Respond to Surgery or Radiation Therapy

Are all these trials running at the same time? The way the titles are written it seems as if the antineoplastons A10 and AS2-1 are being trialled in children and adults with a fairly broad range of cancer types (or stages of a particular cancer type).

Searching for antineoplaston A10 brings up 61 hits and searching for antineoplaston AS2-1 brings up 60, so possibly there's at least one Burznyski trial listed in the 62 that has nothing to do with antineoplastons (could be someone with the same name of course).

Is that usual where the type or stage of cancer has a very poor prognosis and no-one has the luxury of time to wait and see how these ANPs perform in one situation before trying them in the next?

For comparison I had a look at GLP-1 and spotted a few that had similar titles, but there seemed to be much more variance in title, and trial status. One is even listed as having results...

Feb 2012 - Searching just for the antineoplaston A10 trials, we get 61 of which:

• 0 are recruiting
• 50 are of unknown status
• 7 are withdrawn
• 2 are terminated
• 1 is completed
• 0 are active, not recruiting
• 1 is not yet recruiting

Note that Unknown status incorporates some of the other categories

UNKNOWN (RECRUITMENT STATUS)

A clinical study in ClinicalTrials.gov with a status of Recruiting; Not yet recruiting; or Active, not recruiting and whose status has not been confirmed within the past 2 years. Studies with an Unknown recruitment status are considered open studies or closed studies, depending on their last recorded recruitment status.

Nov 2011 - Here's what I found in November 2011 (but I think I searched on Burzynski rather than antineoplaston A10 so there's a caveat there)

• 10 are recruiting
• 35 are of unknown status
• 7 are withdrawn
• 2 are terminated
• 1 is completed - it began in March 1996 and was completed in 2005. Its clinical identifier is NCT00003509 and this does not appear anywhere in PubMed.
• 5 are active, not recruiting
• 1 is not yet recruiting

While writing this post @majikthyse also published a post on the Burzynski Clinic which makes very interesting reading.

Comments relating to conspiracy theories or personal attacks on anyone will be ignored.

I hope lots of medical research charities will sign the #alltrials petition

UPDATE 9 February 2013: The AMRC (Association of Medical Research Charities) signed the petition yesterday morning - but of course I'd still like to see other health / patient charities (AMRC members and non-members) signing up.

Update 12 February 2013: A whole bunch of medical research charities have signed as part of a big gang of patient groups, including my beloved former employer Diabetes UK - hooray :D
http://www.alltrials.net/supporters/pin-statement/

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"Trials with positive results are twice as likely to get reported as negative results," Goldacre said. "You can't make informed decisions about which treatment is best for the patient on the basis of just half the results, especially as it's the unflattering ones that are withheld."
Source: GlaxoSmithKline to publish clinical trial data, The Guardian, 5 February 2013

There seems to have been widespread under-reporting of clinical trial data along with an incomplete record of registered trials in the first place. This makes it harder to track what happened next. It also means that healthcare professionals only have part of the picture available to them when looking at which medications to consider for their patients.

The #alltrials petition calls for all clinical trial reports to be published, not just the positive stuff.

I've heard of the petition mostly through following Ben Goldacre on Twitter where he's been enthusiastically tweeting about organisations who have signed, including a surprise entry from GSK (GlaxoSmithKline) today who are pretty much 'big pharma' by any definition, or haven't signed in the case of the Association of the British Pharmaceutical Industry, aka ABPI.

The Wellcome Trust has signed the petition and they are a big funder of medical research, they're also members of the Association of Medical Research Charities (AMRC) as are lots of other lovely charities and I hope those charities might also sign this petition.

Signing is A Good Thing and as Ben says in his recent blog post "The eccentric position is now not supporting alltrials.net. There is no serious defense for withholding information about clinical trials from doctors and patients. It is simply unethical, and it harms patients."

• Organisations who have already signed up to support the #alltrials petition

Click to visit the AllTrials site

It's important to have all the information to know which drug to prescribe (or which drug to take) but it's also important to know about other problems with drugs that might show up only after the drug is on the market when many more people are taking it. This is 'postmarketing surveillance' (the drug is now on the market and regulatory people keep their beady eyes on it to spot any problems).

Conveniently I heard a discussion about precisely this topic on Radio 4 this evening - Dr Margaret McCartney and someone from the MHRA (Medicines and Healthcare products Regulatory Authority) were discussing, with host Dr Mark Porter, how anyone - you don't have to be a doctor to do this - anyone can use the MHRA's Yellow Card scheme to report any side effect that they experience when taking any medicine.

The scheme was opened up to the public a few years ago - previously it had been just healthcare professionals that would fill these cards in. Now anyone can fill in the 'card' online. The MHRA has noticed that the number of these filled-in reports is dropping and is keen to raise awareness of the yellow card scheme.