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RESEARCH

Milan gene research hope for childhood disease

A new type of gene therapy has shown promise in wiping out two rare childhood diseases, apparently without the risks of causing cancer, international researchers said on Thursday.

Published: 12 July 2013 08:37 CEST
Milan gene research hope for childhood disease
Researchers in Milan say a new type of gene therapy is showing hope in eradicating two rare childhood diseases. Photo: Wikicommons

The method used an HIV virus vector and the patients' own blood stem cells to deliver a corrected version of a faulty gene, said the report in the US
journal Science.

As a result, six children are doing well, 18 to 32 months after their operations, said lead scientist Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy in Milan.

"Three years after the start of the clinical trial, the results obtained from the first six patients are very encouraging. The therapy is not only safe, but also effective and able to change the clinical history of these severe diseases."

Three of the children suffer from metachromatic leukodystrophy, a disease of the nervous system which is caused by mutations in the ARSA gene.

Babies with the disease appear healthy when they are born, but as they grow, they start to lose cognitive and motor skills. There is no cure.

The new gene therapy approach has halted progression of the disease in these three children, researchers said.

The other three children in the study have Wiskott-Aldrich syndrome, which is caused by mutations in the WAS gene and results in recurring infections, autoimmune diseases, frequent bleeding, and a high risk of cancer.

The treatment has caused the children's symptoms to lessen or vanish altogether, the researchers said.

Previous attempts at gene therapy for a range of diseases, including Wiskott-Aldrich Syndrome, have shown some success, but over the long term it was discovered that immune-compromised patients sometimes developed blood cancer.

Researchers now believe that the viral vectors used in the past may have somehow activated a part of the DNA that caused cancer.

Scientists have been greatly cautious with human trials on gene therapy since the 1999 death of an American teenager, Jesse Gelsinger, whose participation in a trial for a rare metabolic disorder overwhelmed his immune system and led to death by multiple organ failure.

The new method is based on taking hematopoietic stem cells from the patients' bone marrow and introducing a corrected copy of the defective gene
using viral vectors derived from HIV.

When the treated cells are re-injected into the patients, they begin to restore the missing protein to key organs.

"Until now we have never seen a way to engineer stem cells using gene therapy that is as effective and safe as this one," said researcher Eugenio
Monti.

"These results pave the way for new therapies for other more common diseases."

The two trials began in 2010 and include six patients with Wiskott-Aldrich syndrome and 10 with metachromatic leukodystrophy.

The research published in Science relates to the first six patients, for which enough time has passed for scientists to reach their first conclusions on safety and efficacy.

However, scientists involved in the project say longer follow-up time and more patient trials are needed to confirm the safety and effectiveness of the therapy.

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RESEARCH

ANALYSIS: Why are Denmark’s politicians criticising university researchers?

The Danish parliament has recently adopted a controversial text asking universities to ensure that "politics is not disguised as science". The Local's contributor Sophie Standen examines why Denmark's politicians are criticising university researchers.

Published: 9 June 2021 17:13 CEST
ANALYSIS: Why are Denmark's politicians criticising university researchers?
Populist politicians have singled out courses at Copenhagen Business School (CBS) for following a so-called 'woke' agenda. Photo: Bjarke MacCarthy/CBS

What has happened? 

On the 1st of June, a majority in the Danish parliament adopted a written declaration that aimed to combat ‘excessive activism in certain humanities and social science research environments’.

The initial debate was led by Morten Messerschmidt from the Danish People’s Party (DF) and Henrik Dahl from Liberal Alliance (LA). The declaration was then voted through, with all of the major parties in favour, including the governing Social Democratic party.

What does the controversial declaration say? 

The declaration stated that the Danish parliament expects that university managements will ensure the self-regulation of scientific research, so that ‘politics is not disguised as science’.

However, it also asserted that Danish parliament has no right to determine the method or topic of research in Danish universities, and stressed the importance of free and critical debate in the research community.

Who is upset by it? 

The adoption of this position by Danish parliament has proven extremely controversial for many academics and researchers, with over 3,200 Danish and international researchers signing an open letter denouncing the stance adopted by the Danish government.

The authors of the letter stated that ‘academic freedom is under increasing attack’, and described the developments as ‘highly troubling’.

Furthermore, in another open letter to the Minister for Higher Education and Science, Ane Halsboe-Jørgensen, published in the Politiken newspaper, 262 Danish university researchers complained that they were facing increasing occurrences of personal intimidation and harassment due their research.

What is concerning university researchers and professors? 

Professor Lisa Ann Richey, a professor at Copenhagen Business School, told The Local that the parliament’s move was “illiberal” as “it doesn’t support freedom”. 

Richey, who has been a professor in Denmark for more than 20 years, was one of co-organisers of the open letter, and a co-signatory of the letter published in Politiken.

“I am one of the international recruits who finds the Danish research environment a great place to work,” she said. “We have a strong university system and good research environments. One of the things we are risking here is that reputation, and also the possibility of recruiting internationally.”

She said that in her opinion, academia in Denmark was self-policing due to the exhaustive peer-review process and oversight by university authorities. 

“There are lots of checks and balances within academia, and sometimes it doesn’t seem like that because they [the politicians] have no idea how many evaluations we go through,” she said. “We have peer reviews, student reviews, and university assessments to ensure quality in research.” 

Is there a populist campaign behind the statement? 

Richey complained that long before the parliamentary statement, prominent populist politicians “came out on social media calling out particular courses”. 

“They did this to a course I taught in, saying now even CBS has become part of this ‘woke agenda’,” she complained. “This statement about politics dressed up as science, it’s meant to intimidate. We need university leadership to support us and we need everyone to recognise that this is a threat towards academic freedom and also to make sure that we don’t expose individuals”

Anders Bjarklev, the rector of the Danish Technical University (DTU), and president of the rector’s college for Danish universities, echoed this sentiment. Writing on social media, he has called the position adopted by parliament, ‘an attack on research freedom’. 

“When subjects are singled out by politicians, such as gender studies or post-colonial studies, then academics get worried because much of our funding is from the government,” he told The Local. 

“I am also worried that academics will be scared to take part or publish research in these subjects”.  As rector of DTU, he says he is “not sure what we could do differently”, as academics at the university “always want to ensure the highest quality standard of research”.

What has the government said to defend itself? 

In an interview with the Politiken newspaper, Bjørn Brandenborg, the Social Democrat’s spokesperson for higher education and science, insisted that despite the statement, there was “no general distrust of universities” on the part of the government. 

“The Danish parliament has a right, like all other citizens, to have an opinion on research results”, he continued, while stressing that “the Danish parliament will not become involved in decisions over what is researched in Danish universities”.

In his view, he said, the text voted on by the parliament was “completely unproblematic”, as  “all it says is that universities should take responsibility for the quality of their research”.

This adopted stance by the Danish government has shaken the arms-length principle of trust between Danish research institutions and the Danish government. Many have denounced the politicians who have singled out specific researchers on social media as examples of political activism within research in Denmark.

In a statement to Politiken, the minister responsible for Higher Education and Science in Denmark, Ane Halsboe-Jørgenson, remarked that the 3,241 researchers that had signed the open letter had “reached the wrong conclusion” about the adopted declaration.

She insisted that the Danish government is “fighting for research freedom”, while also remarking that she thinks “we politicians must stay far away from judging individuals and individual research areas”.

What will happen next? 

For Professor Lisa Ann Richey, “now, when major political parties are part of this, making a ‘non-problem’ a problem, then it’s really time that we [academics] have to respond.”

“Our work is important and it is not acceptable behaviour to try and bully individual researchers and to police research environments,” she continued. “This is something that will be moving forward now that universities have spoken out officially”. 

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