Tag Archives: Research2Reality (R2R)

Gene therapy in Canada; a November 2020 report and two events in December 2020

There’s a lot of action, albeit quiet and understated, in the Canadian gene therapy ‘discussion’. One major boost to the discussion was the Nov. 3, 2020 release of a report by the Canadian Council of Academies (CCA), “From Research to Reality; The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada.”

Dec. 2 – 3, 2020 Breaking Through

Another boost is the the free and virtual, upcoming 2020 Gairdner Ontario International Symposium “Breaking Through: Delivering on the Promise of Gene Therapy“; an international symposium on gene therapy research and practice, which will feature a presentation on the CCA’s report,

Breaking Through brings together Canadian and international leaders to explore the past, present, and future of somatic gene therapy research and practice. This two-day virtual event will examine the successes, challenges and opportunities from the bench to the bedside. It will also feature:

  • Speaker sessions from Canadian and international researchers at the forefront of gene therapy research.
  • A panel discussion exploring the opportunities and challenges facing Canadian scientists, regulators, clinicians, decision-makers, and patients (Presented by NRC).
  • A presentation and Expert Panel discussion on the Council of Canadian Academies’ latest report, From Research to Reality, and a closing panel discussion about the future of gene therapies and gene editing (Presented by Genome Canada).

The title for the CCA report bears an uncanny resemblance to the name for a Canadian initiative highlighting science research, Research2Reality (R2R). (If you’re curious, you can check out my past postings on R2R by using ‘Research2Reality’ as the term for the blog’s search engine.

Glybera

This name stood out: Michael Hayden (scroll down to his name and click), one of the featured speakers for this Dec. 2 – 3, 2020 event, reminded me of the disturbing Glybera story,

Dr. Hayden identified the first mutations underlying lipoprotein lipase (LPL) deficiency and developed gene therapy approaches to treat this condition, the first approved gene therapy (Glybera) in the western world.

Kelly Crowe’s Nov. 17, 2018 story for the Canadian Broadcasting Corporation (CBC) lays it out,

It is one of this country’s great scientific achievements.

The first drug ever approved that can fix a faulty gene.

It’s called Glybera, and it can treat a painful and potentially deadly genetic disorder with a single dose — a genuine made-in-Canada medical breakthrough.

But most Canadians have never heard of it.

A team of researchers at the University of British Columbia spent decades developing the treatment for people born with a genetic mutation that causes lipoprotein lipase defficiency (LPLD).

If you have the time, do read Crowe’s Nov. 17, 2018 story but as I warned in another post, it’s heartbreaking.

Fora brief summary, the company which eventually emerged with the licensing rights to Glybera, charged $1m per dose and a single dose is good for 10 years. It seems governments are reluctant to approve the cost and for many individuals, it’s an impossible price to meet, every 10 years. So, the drug is dead. Or perhaps not? Take a look at the symposium’s agenda (scroll down) for description,

GLYBERA REINVENTED: A WINDING STORY OF COMMITMENT, CREATIVITY, AND INNOVATION

Michael Hayden, MB, ChB, PhD, FRCP(C), FRSC, C.M., O.B.C University Killam Professor, Senior Scientist, Centre for Molecular Medicine and Therapeutics, Department of Medical Genetics,

University of British Columbia (Vancouver, BC)

Money issues

One theme from the agenda jumped out at me: money. The focus seems to be largely on accessibility and costs. The Nov. 3, 2020 CCA news release (also on EurekAlert) about the report also prominently featured costs,

Gene therapies are being approved for use in Canada, but could strain healthcare budgets and exacerbate existing treatment inequities [emphasis mine] across the country. However, there are opportunities to control spending, streamline approvals and support fair access through innovation, coordination and collaboration, according to a new expert panel report from the Council of Canadian Academies (CCA).

“Rapid scientific advances mean potentially life-changing treatments are approaching the clinic at an accelerated pace,” said Janet Rossant, PhD, C.C., FRSC, and Chair of the Expert Panel. “These new therapies, however, pose a number of challenges in terms of their introduction into the Canadian healthcare system and ensuring access to those who would most benefit.”

Gene therapies and gene editing

Before moving on, you might find it useful to know (if you don’t already) that gene therapy can be roughly divided into somatic cell gene therapy and germline gene therapy as per the Gene Therapy entry in Wikipedia.

Two other items on the symposium’s agenda (scroll down) drew my attention,

Genome editing and the promise for future therapies

Ronald Cohn, MD, FACMG, FCAHS President and CEO,
The Hospital for Sick Children (SickKids) (Toronto, ON)

COMING SOON: THE FUTURE OF GENE EDITING AND GENE THERAPIES

Presented by: Genome Canada

Rob Annan, PhD President and CEO,
Genome Canada (Ottawa, ON)

R. Alta Charo, J.D. Warren P. Knowles Professor of Law & Bioethics,
University of Wisconsin Law School (Madison, USA)

Jay Ingram, C.M. Science broadcaster and writer, Former Co-Host, Discovery Channel’s “Daily Planet” (Calgary, AB)

Vardit Ravitsky, PhD, FCAHS Full Professor, Bioethics Program, Department of Social and Preventative Medicine, School of Public Health, Université de Montréal; President, International Association of Bioethics (Montréal, QC)

Janet Rossant, PhD, C.C., FRSC President,
Gairdner Foundation (Toronto, ON) [also a member of the CCA expert panel for report on somatic cell therapies ‘From research to reality …’)

Genome editing, by the way and if you don’t know, is also known as gene editing. The presence of the word ‘future’ in both the presentations has my antennae quivering. Could they be hinting at germline editing possibilities? At this time, the research is illegal in Canada.

If you don’t happen to know, somatic gene editing, covered in the CCA report, does not affect future generations as opposed to germline gene editing, which does. Should you be curious about the germline gene editing discussion in Canada, I covered as much information as I could uncover in an April 26, 2019 posting on topic.

Jay Ingram’s presence on the panel sponsored by Genome Canada is a bit of a surprise.

I saw him years ago as the moderator for a panel presentation sponsored by Genome British Columbia. The discussion was about genetics and ethics, which was illustrated by clips from the television programme, ReGenesis (from its IMDB entry),

[Fictional] Geneticist David Sandstrom is the chief scientist at the prestigious virology/micro-biology NORBAC laboratory, a joint enterprise between the USA, Canada and Mexico for countering bio-terrorism.

Ingram (BA in microbiology and an MA that’s not identified in his Wikipedia entry) was a television science presenter for a number of years and has continued to work in the field of science communication. He didn’t seem all that knowledgeable about genetics when he moderated the ReGenesis panel but perhaps his focus will be about the communication element?

For anyone interested in attending the free and virtual “Breaking Through” event, you can register here.

CAR-T cell therapies (a type of somatic cell therapy)

One final note, the first week of December seems to be gene therapy week in Canada. There is another free and virtual event, the second session of the Summit for Cancer Immunotherapy: 2020 Speaker Series (Hosted by BioCanRx, Canada’s Immunotherapy Network), Note: I made a few changes to make this excerpt a bit easier to read,

Session Two: Developing better CAR T-Cell Therapies by engaging patients, performing systematic reviews and assessing real-world and economic evidence
Wednesday, December 9, 1:30 pm – 3:15pm EST [emphasis mine]

Chimeric Antigen Receptor T-cell (CAR-T) therapy is a personalized immunotherapy, currently being assessed in a Canadian Phase I/II clinical trial to test safety and feasibility for relapsed/refractory blood cancer (CD19+ Acute Lymphoblastic Leukemia and non-Hodgkin’s Lymphoma).

This virtual seminar will provide an overview of a multidisciplinary team’s collaborative efforts to synthesize evidence for the development of this clinical trial protocol, using a novel approach (the ‘Excelerator’ model). This approach involved the completion of a systematic review (objective review of existing trial data), engagement of patients and clinicians, and drawing from real world and economic evidence.

Dr. Fergusson will provide a brief introduction. Dr. Kednapa Thavorn will discuss the team’s use of economic modelling to select trial factors to maximize economic feasibility of the therapy, and Mackenzie Wilson (HQP) will discuss the current efforts and future directions to engage diverse stakeholders to inform this work. Gisell Castillo (HQP) will speak about the interviews that were conducted with patients and hematologists to identify potential barriers and enablers to participation and recruitment to the trial.

The team will also discuss two ongoing projects which build on this work. Dr. Lalu will provide an overview on the team’s patient engagement program throughout development of the trial protocol and plans to expand this program to other immunotherapy trials. Joshua Montroy (HQP) will also discuss ongoing work building on the initial systematic review, to use individual participant data meta-analysis to identify factors that may impact the efficacy of CAR-T cell therapy.

Dr. Justin Presseau will moderate the question and answer period.

And there’s this,

Who should attend?

Scientific and health care community including researchers, clinicians and HQP along with patients and caregivers. Note: There will be a plain language overview before the session begins and an opportunity to ask questions after the discussion.

If you want to know more about CAR T-cell therapy, sometimes called gene or cell therapy or immune effect cell therapy, prior to the Dec., 9, 2020 event, this page on the cancer.org website should prove helpful.

Congratulations to Molly Shoichet (her hydrogels are used in regenerative medicine and more) for winning the $1 million Gerhard Herzberg Canada Gold Medal

I imagine that most anyone who’s been in contact with Ms. Shoichet is experiencing a thrill on hearing this morning’s (November 10, 2020) news about winning Canada’s highest honour for science and engineering research. (Confession: she, very kindly, once gave me a brief interview for a posting on this blog, more about that later).

Why Molly Shoichet won the Gerhard Herzberg Canada Gold Medal

Emily Chung’s Nov. 10, 2020 news item on the Canadian Broadcasting Corporation (CBC) website announces the exciting news (Note: Links have been removed),

A Toronto chemical engineering professor has won the $1 million Gerhard Herzberg Canada Gold Medal, the country’s top science prize, for her work designing gels that mimic human tissues.

The Natural Sciences and Engineering Research Council of Canada (NSERC) announced Tuesday [Nov. 10, 2020] that Molly Shoichet, professor of chemical engineering and applied chemistry and Canada Research Chair in Tissue Engineering at the University of Toronto is this year’s recipient of the award, which recognizes “sustained excellence” and “overall influence” of research conducted in Canada in the natural sciences or engineering.

Shoichet’s hydrogels are used for drug development and  delivery and regenerative medicine to heal injuries and treat diseases.

NSERC said Shoichet’s work has led to the development of several “game-changing” applications of such materials. They “delivered a crucial breakthrough” by allowing cells to be grown in three dimensions as they do in the body, rather than the two dimensions they typically do in a petri dish.

Hydrogels are polymer materials — materials such as plastics, made of repeating units — that become swollen with water.

“If you’ve ever eaten Jell-o, that’s a hydrogel,” Shoichet said. Slime and the absorbent material inside disposable diapers are also hydrogels.

Shoichet was born in Toronto, and studied science and engineering at the Massachusetts Institute of Technology and the University of Massachusetts Amherst. After graduating, she worked in the biotech industry alongside “brilliant biologists,” she said. She noticed that the biologists’ research was limited by what types of materials were available.

As an engineer, she realized she could help by custom designing materials for biologists. She could make materials specifically suit their needs, to answer their specific questions by designing hydrogels to mimic particular tissues.

Her collaborations with biologists have also generated three spinoff companies, including AmacaThera, which was recently approved to run human trials of a long-acting anesthetic delivered with an injectable hydrogel to deal with post-surgical pain.

Shoichet noted that drugs given to deal with that kind of pain lead to a quarter of opioid addictions, which have been a deadly problem in Canada and around the world.

“What we’re really excited about is not only meeting that critical need of providing people with greater pain relief for a sustained period of time, but also possibly putting a dent in the operation,” she said. 

Liz Do’s Nov. 10, 2020 University of Toronto news release provides more details (Note: Links have been removed),

The  Herzberg Gold Medal is awarded by the Natural Sciences and Engineering Research Council (NSERC) in recognition of research contributions characterized by both excellence and influence.

“I was completely overwhelmed when I was told the good news,” says Shoichet. “There are so many exceptional people who’ve won this award and I admire them. To think of my peers putting me in that same category is really incredible.”

A pioneer in regenerative medicine, tissue engineering and drug delivery, Shoichet and her team are internationally known for their discovery and innovative use of 3D hydrogels.

“One of the challenges facing drug screening is that many of the drugs discovered work well in the lab, but not in people, and a possible explanation for this discrepancy is that these drugs are discovered in environments that do not reflect that of the body,” explains Shoichet.

Shoichet’s team has invented a series of biomaterials that provide a soft, three-dimensional environment in which to grow cells. These hydrogels — water-swollen materials — better mimic human tissue than hard two-dimensional plastic dishes that are typically used. “Now we can do more predictive drug screening,” says Shoichet.

Her lab is using these biomaterials to discover drugs for breast and brain cancer and a rare lung disease. Shoichet’s lab has been equally innovative in regenerative medicine strategies to promote repair of the brain after stroke and overcome blindness.

“Everything that we do is motivated by answering a question in biology, using our engineering and chemistry tools to answer those questions,” says Shoichet.

“The hope is that our contributions will ultimately make a positive impact in the cancer community and in treating diseases for which we can only slow the progression rather than stop and reverse, such as with blindness.”

Shoichet is also an advocate for and advisor on the fields of science and engineering. She has advised both federal and provincial governments through her service on Canada’s Science, Technology and Innovation Council and the Ontario Research Innovation Council. From 2014 to 2018, she was the Senior Advisor to the President on Science & Engineering Engagement at the University of Toronto. She is the co-founder of Research2Reality [emphasis mine], which uses social media to promote innovative research across the country. She also served as Ontario’s first Chief Scientist [emphasis mine], with a mandate to advance science and innovation in the province.

Shoichet is the only person to be elected a fellow of all three of Canada’s National Academies and is a foreign member of the U.S. National Academy of Engineering, and fellow of the Royal Society (UK) — the oldest and most prestigious academic society.

Doug Ford (premier of Ontario) and Molly Shoichet

She did serve as Ontario’s first Chief Scientist—for about six months (Nov. 2017 – July 2018). Molly Shoichet was fired when a new provincial government was elected in the summer of 2018. Here’s more about the incident from a July 4, 2018 article by Ryan Maloney for huffingtonpost.ca (Note: Links have been removed),

New Ontario Premier Doug Ford has fired the province’s first chief scientist.

Dr. Molly Shoichet, a renowned biomedical engineer who teaches at the University of Toronto, was appointed in November [2017] to advise the government and ensure science and research were at the forefront of decision-making.

Shoichet told HuffPost Canada in an email that the she does not believe the decision was about her, and “I don’t even know whether it was about this role.” She said she is disappointed but honoured to have served Ontarians, even for a short time.

Ford’s spokesman, Simon Jefferies told The Canadian Press Wednesday that the government is starting the process of “finding a suitable and qualified replacement.” [emphasis mine]

The move comes just days after Ford’s Progressive Conservatives officially took power in Canada’s largest province with a majority government.

Almost a year later, there was no replacement in sight according to a June 24, 2019 opinion piece by Kimberly Girling (then the Research and Policy Director of the Evidence for Democracy not-for-profit) for the star.com,

Premier Doug Ford, I’m concerned for your government.

I know you feel it too. Last week, one year into your mandate and faced with sharply declining polls after your first provincial budget, you conducted a major cabinet shuffle. This shuffle is clearly an attempt to “put the right people in the right place at the right time” and improve the outcomes of your cabinet. But I’m still concerned.

Since your election, your caucus has made many bold decisions. Unfortunately, it seems many are Ontarians unhappy with most of these decisions, and I’m not sure the current shuffle is enough to fix this.

[] I think you’re missing someone.

What about a Chief Scientist?

It isn’t a radical idea. Actually, you used to have one. Ontario’s first Chief Scientist, Dr. Molly Shoichet, was appointed to advise the government on science policy and champion science and innovation for Ontario. However, when your government was elected, you fired Dr. Shoichet within the first week.

It’s been a year, and so far we haven’t seen any attempts to fill this vacant position. [emphasis mine]

I wonder if Doug Ford and his crew regret the decision to fire Shoichet especially now that the province is suffering from a new peak in rising COVID-19 case numbers. These days government could do with a little bit of good news.

The only way we might ever know is if Doug Ford writes a memoir (in about 20 or 30 years from now).

Molly Shoichet, Research2Reality, and FrogHeart

A May 11, 2015 posting announced the launch of Research2Reality and it’s in this posting that I have a few comments from Molly Shoichet about co-founding a national science communication project. Given how busy she was at the time, I was amazed she took a few minutes to speak to me and took more time to make it possible for me to interview Raymond Laflamme (then director of the Institute for Quantum Computing at the University of Waterloo [Ontario]) and a prominent physicist.

Here are the comments Molly Shoichet offered (from the May 11, 2015 posting),

“I’m very excited about this and really hope that other people will be too,” says Shoichet. The audience for the Research2Reality endeavour is for people who like to know more and have questions when they see news items about science discoveries that can’t be answered by investigating mainstream media programmes or trying to read complex research papers.

This is a big undertaking. ” Mike [Mike MacMillan, co-founder] and I thought about this for about two years.” Building on the support they received from the University of Toronto, “We reached out to the vice-presidents of research at the top fifteen universities in the country.” In the end, six universities accepted the invitation to invest in this project,

Five years later, it’s still going.

Finally: Congratulations Molly Shoichet!

The decade that was (2010-19) and the decade to come (2020-29): Science culture in Canada (an addendum)

I missed a few science journalists (part 1 of this series, under the Science Communication subhead; Mainstream Media, sub subhead) as the folks at the Science Media Centre of Canada (SMCC) noted on Twitter,

Science Media Centre @SMCCanada Apr 16 Replying to @frogheart

Thanks for the mention. But I think poor @katecallen at the Toronto Star would be dismayed to read that @IvanSemeniuk is the only science reporter on a Canadian newspaper. And @row1960 Bob Weber at Canadian Press is carried in every newspaper in the country.

Science Media Centre @SMCCanada Apr 16 Replying to @frogheart

In addition, @mle_chung at CBC News Online (#1 news source in Canada) is read more than any other science writer in the country, as is her colleague @NebulousNikki

Thank you.

***ETA April 29, 2020 at 0910 PT: Yesterday, April 28, 2020, Postmedia announced that it was closing 15 community newspapers and a number of jobs elsewhere in the organization. Earlier in the month on April 7, 2020 Postmedia announced that 85 positions were being eliminated, including 11 in the editorial department of TorStar (Toronto Star). I hope they keep a position for a science writer at the Toronto Star.***

Alice Major, a poet mentioned in Part 3 under The word subhead; Poetry sub subhead, wrote with news of two other poets who focus on science in their work.

  • Christian Bök
  • Adam Dickinson

From Bök’s Wikipedia entry (Note: Links have been removed),

Christian Bök[needs IPA] (born August 10, 1966 in Toronto, Canada) is an experimental Canadian poet. He is the author of Eunoia, which won the Canadian Griffin Poetry Prize.

On April 4, 2011 Bök announced a significant break-through in his 9-year project to engineer “a life-form so that it becomes not only a durable archive for storing a poem, but also an operant machine for writing a poem”.[7][8] On the previous day (April 3) Bök said he received confirmation from the laboratory at the University of Calgary that my poetic cipher, gene X-P13, has in fact caused E. coli to fluoresce red in our test-runs—meaning that, when implanted in the genome of this bacterium, my poem (which begins “any style of life/ is prim…”) does in fact cause the bacterium to write, in response, its own poem (which begins “the faery is rosy/ of glow…”).”[9]

The project has continued for over fifteen years at a cost exceeding $110,000 and he hopes to finish the project in 2014.[10] He published “Book I” of the resulting Xenotext in 2015.

Xenotext: Book 1 published by Coach House Books is described this way,

Internationally best-sellling poet Christian Bök has spent more than ten years writing what promises to be the first example of ‘living poetry.’ After successfully demonstrating his concept in a colony of E. coli, Bök is on the verge of enciphering a beautiful, anomalous poem into the genome of an unkillable bacterium (Deinococcus radiodurans), which can, in turn, “read” his text, responding to it by manufacturing a viable, benign protein, whose sequence of amino acids enciphers yet another poem. The engineered organism might conceivably serve as a post-apocalyptic archive, capable of outlasting our civilization.

Book I of The Xenotext constitutes a kind of ‘demonic grimoire,’ providing a scientific framework for the project with a series of poems, texts, and illustrations. A Virgilian welcome to the Inferno, Book I is the “orphic” volume in a diptych, addressing the pastoral heritage of poets, who have sought to supplant nature in both beauty and terror. The book sets the conceptual groundwork for the second volume, which will document the experiment itself. The Xenotext is experimental poetry in the truest sense of the term.

Adam Dickinson is a poet and an associate professor at Brock University (Ontario). He describes himself and his work this way (from the Brock University bio page),

Adam Dickinson is a poet and a professor of poetry. His creative and academic writing has primarily focused on intersections between poetry and science as a way of exploring new ecocritical perspectives and alternative modes of poetic composition. His latest book, Anatomic (Coach House Books), involves the results of chemical and microbial testing on his body, and was shortlisted for The Raymond Souster Award. Sections of it were also shortlisted for the Canadian Broadcasting Corporation (CBC) Poetry Prize. His book, The Polymers (House of Anansi [2013]), which is an imaginary science project that combines the discourses, theories, and experimental methods of the science of plastic materials with the language and culture of plastic behaviour, was a finalist for both the Governor General’s Award for Poetry and the Trillium Book Award for Poetry. He has published two previous books, Kingdom, Phylum (also nominated for the Trillium Book Award for Poetry) and Cartography and Walking (nominated for an Alberta Book Award). His scholarly work (supported by SSHRC [Social Sciences and Humanities Research Council of Canada]) brings together research in innovative poetics, biosemiotics, pataphysics, and Anthropocene studies.

His current research-creation project, “Metabolic Poetics,” (also supported by SSHRC) is concerned with the potential of expanded modes of reading and writing to shift the frames and scales of conventional forms of signification in order to bring into focus the often inscrutable biological and cultural writing intrinsic to the Anthropocene, especially as this is reflected in the inextricable link between the metabolic processes of human and nonhuman bodies and the global metabolism of energy and capital.

He has been featured at prominent international literary festivals, such as Poetry International in Rotterdam, The Harbourfront International Festival of Authors in Toronto, and the Oslo International Poetry Festival in Norway. Adam has also been a finalist for the K.M. Hunter Artist Award in Literature, Administered by the Ontario Arts Council. Adam welcomes potential student supervisions on topics in poetry and poetics, environmental writing, science and literature, and creative writing.

Thank you.

This last addition may seen a little offbeat but ARPICO (Society of Italian Researchers & Professionals in Western Canada) has hosted a surprisingly large number of science events in Vancouver. Two recent examples include: The Eyes are the Windows to The Mind; Implications for Artificial Intelligence (AI) -driven Personalized Interaction on March 4, 2020 and, the relatively recent, Whispers in the Dark: Underground Science on June 12, 2019.

Hopefully, I’ll be able to resist the impulse to make any more additions.

***ETA April 30, 2020: Research2Reality (R2R) was launched in 2015 as a social media initiative featuring a series of short video interviews with Canadian scientists (see more in my May 11, 2015 posting). Almost five years later, the website continues to feature interviews and it also hosts news about Canadian science and research. R2R was founded by Molly Shoichet (pronounced shoyquette) and Mike MacMillan.***

For anyone who stumbled across this addendum first, it fits on to the end of a 5-part series:

Part 1 covers science communication, science media (mainstream and others such as blogging) and arts as exemplified by music and dance: The decade that was (2010-19) and the decade to come (2020-29): Science culture in Canada (1 of 5).

Part 2 covers art/science (or art/sci or sciart) efforts, science festivals both national and local, international art and technology conferences held in Canada, and various bar/pub/café events: The decade that was (2010-19) and the decade to come (2020-29): Science culture in Canada (2 of 5).

Part 3 covers comedy, do-it-yourself (DIY) biology, chief science advisor, science policy, mathematicians, and more: The decade that was (2010-19) and the decade to come (2020-29): Science culture in Canada (3 of 5).

Part 4 covers citizen science, birds, climate change, indigenous knowledge (science), and the IISD Experimental Lakes Area: The decade that was (2010-19) and the decade to come (2020-29): Science culture in Canada (4 of 5).

Part 5: includes science podcasting, eco art, a Saskatchewan lab with an artist-in-residence, the Order of Canada and children’s science literature, animation and mathematics, publishing science and more: The decade that was (2010-19) and the decade to come (2020-29): Science culture in Canada (5 of 5).