Open Science for Children’s Health

M4K Pharma is using open science to revolutionize how affordable new treatments are discovered and developed. Through our Medicines4Kids program, we are aggregating and aligning the work of global academics, foundations and pharma/biotech researchers to advance new cures for childhood diseases not well served by current business models.

M4K Pharma’s first program is the discovery, development and commercialization of an ALK2 enzyme inhibitor which will provide the first therapeutic developed specifically for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG).

In line with M4K Pharma’s open science principles, we are committed to sharing our research data freely in the public domain. Our data is currently available for public viewing in the video recordings of our monthly open scientific updates, which are posted to our blog and also available through our YouTube Channel.

Latest Project Meetings

Online Meetings
M4K Pharma Open Scientific Update Meeting October 2024- Recording
Scientific Meetings
M4K Pharma Open Scientific Update Meeting October 2024- Recording
Scientific Meetings

Recording of M4K Pharma's Scientific Update Meeting - October 23, 2024. Listen to our scientists and collaborators discuss the latest progress in M4K Pharma's quest for a medicine for DIPG- a deadly children's brain cancer. Everyone is invited to dial-in and join the live meetings.

Scientific Meetings
September 2024 M4K Open Scientific Meeting
Scientific Meetings
September 2024 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from September 25th, 2024 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Everyone is invited to dial-in and join the live monthly meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
September 2020 M4K Open Scientific Meeting
Scientific Meetings
September 2020 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from September 23rd, 2020 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Everyone is invited to dial-in and join the live monthly meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
July 2020 M4K Open Scientific Meeting
Scientific Meetings
July 2020 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from July 15th, 2020 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Everyone is invited to dial-in and join the live monthly meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
March 2020 M4K Open Scientific Meeting
Scientific Meetings
March 2020 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from March 11th, 2020 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Everyone is invited to dial-in and join the live monthly meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
February 2020 M4K Open Scientific Meeting
Scientific Meetings
February 2020 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from February 12th, 2020 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Everyone is invited to dial-in and join the live monthly meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
December 2019 M4K Open Scientific Meeting
Scientific Meetings
December 2019 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from December 4th, 2019 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Everyone is invited to dial-in and join the live monthly meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
November 2019 M4K Open Scientific Meeting
Scientific Meetings
November 2019 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from November 6th, 2019 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Join us to hear first-hand how we are developing a medicine using an open science collaborative approach!

In today’s virtual meeting, Dr. Methvin Isaac from OICR opened with an overview of progress against CTIP grant milestones. The team is collectively working towards optimizing compounds from three chemical series, including five lead compounds that have met our target product profile, as inhibitors to the mutated ALK2 protein.

Our Structural Genomics Consortium (SGC) collaborators from Prof. Alex Bullocks’ lab at Oxford University shared an update on in vitro assays testing for potency and off target activity of backup compounds in DIPG cell lines.  They also presented a 3D structure model to explain why two of the compounds bound poorly to the G356D- mutated ALK2 protein.

Dr. Ahmed Aman (OICR) shared results from formulation studies confirming that aqueous methylcellulose can be used as an alternative to the DMSO-containing vehicle currently used for in vivo studies. There was a typical slight reduction in exposure of the compound in the plasma relative to the DMSO formulation. The urgent need to change the vehicle is in response to results from recent DIPG efficacy studies in mice which suggested that the vehicle may be causing toxicity in the form of weight loss.

Dr. Angel M Carcaboso from Hospital Sant Joan de Déu Barcelona shared an update on the efficacy studies with M4K2009, one of our lead compounds, using a DIPG xenograft mouse model. The treated group lost less weight than the control group and survived 7-9 days longer. As the vehicle contains DMSO and seems to affect the health of the mice, interpreting the data is difficult. The experiment will need repeating with the new formulation using aqueous methylcellulose.

Dr. Sue Cramp from Charles River Laboratories (CRL) presented their progress to improve the physical and chemical properties of backup compounds from the Blueprint series. CRL chemists have been volunteering their time with M4K as part of their Corporate Social Responsibility program. Working with the AI company Atomwise, CRL is also identifying compounds related to M4K2009 that will have improved selectivity for ALK2 over ALK1 and ALK6, sister proteins to our target. The CRL team is also collaborating with SGC to develop brain penetrant and cell permeable PROTACs with newer analogues, to be used both as tools for knockdown and as potential drugs.

Everyone is invited to dial-in and join the live monthly meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
October 2019 M4K Open Scientific Meeting
Scientific Meetings
October 2019 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from October 2nd, 2019 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Scientific Meetings
September 2019 M4K Open Scientific Meeting
Scientific Meetings
September 2019 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from September 12th, 2019 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Scientific Meetings
August 2019 M4K Open Scientific Meeting
Scientific Meetings
August 2019 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from August 7th, 2019 where our scientists and collaborators discussed the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Scientific Meetings
July 2019 M4K Open Scientific Meeting
Scientific Meetings
July 2019 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from July 3rd, 2019 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Scientific Meetings
June 2019 M4K Open Scientific Meeting
Scientific Meetings
June 2019 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from June 12th, 2019 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Scientific Meetings
May 2019 M4K Open Scientific Meeting
Scientific Meetings
May 2019 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from May 8th, 2019 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Scientific Meetings
April 2019 M4K Open Scientific Meeting - 2
Scientific Meetings
April 2019 M4K Open Scientific Meeting - 2
Scientific Meetings

Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

Everyone is invited to dial-in and join our live meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
April 2019 M4K Open Scientific Meeting - 1
Scientific Meetings
April 2019 M4K Open Scientific Meeting - 1
Scientific Meetings

Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

Everyone is invited to dial-in and join our live meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
March 2019 M4K Open Scientific Meeting
Scientific Meetings
March 2019 M4K Open Scientific Meeting
Scientific Meetings

Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

Everyone is invited to dial-in and join our live meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
December 2018 M4K Open Scientific Meeting
Scientific Meetings
December 2018 M4K Open Scientific Meeting
Scientific Meetings

Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

In this meeting, the team provides a comprehensive summary of the project accomplishments at the one year mark. We also highlight the discovery and profile of M4K2009, a selective, orally bioavailable and brain penetrant ALK2 inhibitor. M4K plans to make this molecule available to the broader scientific community by the end of February, 2019.

Everyone is invited to dial-in and join our live meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
November 2018 M4K Open Scientific Meeting
Scientific Meetings
November 2018 M4K Open Scientific Meeting
Scientific Meetings

Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

Everyone is invited to dial-in and join the live meetings.

To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
September 2018 M4K Open Scientific Meeting
Scientific Meetings
September 2018 M4K Open Scientific Meeting
Scientific Meetings

Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

Everyone is invited to dial-in and join the live meetings.

To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
August 2018 M4K Open Scientific Meeting
Scientific Meetings
August 2018 M4K Open Scientific Meeting
Scientific Meetings

Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

Everyone is invited to dial-in and join the live meetings.

To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
July 2018 M4K Open Scientific Meeting
Scientific Meetings
July 2018 M4K Open Scientific Meeting
Scientific Meetings

Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

Everyone is invited to dial-in and join the live meetings.

To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
June 2018 M4K Open Scientific Meeting
Scientific Meetings
June 2018 M4K Open Scientific Meeting
Scientific Meetings

Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

Everyone is invited to dial-in and join the live meetings.

To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
May 2018 M4K Open Scientific Meeting
Scientific Meetings
May 2018 M4K Open Scientific Meeting
Scientific Meetings

Full recording from M4K Pharma’s Scientific Update Meeting – May 9th, 2018. Listen to our scientists and collaborators discuss the latest progress in M4K Pharma’s quest for a medicine for DIPG- a deadly children’s brain cancer.

Everyone is invited to dial-in and join the live meetings. To find out when our next meeting will be, follow us on twitter: @M4KPharma

Scientific Meetings
April 2018 M4K Open Scientific Meeting
Scientific Meetings
April 2018 M4K Open Scientific Meeting
Scientific Meetings

Catch M4K Pharma’s virtual Scientific Update Meeting from April 6th, 2018 where our scientists and collaborators discussed the latest progress in our Medicines4Kids project for diffuse intrinsic pontine gliomas (DIPG). 

Scientific Meetings
March 2018 M4K Open Scientific Meeting
Scientific Meetings
March 2018 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s virtual Scientific Update Meeting from March 7th, 2018 where our scientists and collaborators discussed the latest progress in M4K’s Medicines4Kids project for diffuse intrinsic pontine gliomas (DIPG). Everyone is invited to see the chemistry first-hand!

Scientific Meetings
February 2018 M4K Open Scientific Meeting
Scientific Meetings
February 2018 M4K Open Scientific Meeting
Scientific Meetings

Listen to M4K Pharma’s Scientific Update Meeting from February 6th, 2018 where our scientists and collaborators discuss the latest progress in our Medicines4Kids project for a deadly children’s brain cancer called DIPG. Everyone is invited to hear first-hand how we are developing a medicine in the open using an open science collaborative approach.

Scientific Meetings

Latest Blog Posts

Blog & News
Peter Sampson Joins Agora as VP, Drug Discovery and Development [Press Release]
Peter Sampson Joins Agora as VP, Drug Discovery and Development [Press Release]

M4K Pharma is pleased to announce the appointment of Dr. Peter Sampson as the new Vice President of Drug Discovery and Development for M4K’s parent Canadian charity, Agora Open Science Trust (Agora).

Childhood Cancer Awareness Month
Childhood Cancer Awareness Month

In a piece to mark Childhood Cancer Awareness Month, Dr. Diana Martins Carvalho reflects on the vital support of families who generously donate tissue samples and funds that enable her research into childhood brain tumours.

M4K Team Publishes in J. Med. Chem.
M4K Team Publishes in J. Med. Chem.

In the May and September 2020 issues of the Journal of Medicinal Chemistry, the M4K team and collaborators published two articles on M4K’s open science approach and the chemistry progress to develop the inhibitors for ALK2 as a treatment for Diffuse Intrinsic Pontine Glioma (DIPG).

Hope of New Medicine for Childhood Cancer
Hope of New Medicine for Childhood Cancer

Our collaborator Dr Chris Jones and his fantastic team at The Institute of Cancer Research were profiled in The Observer for their work on DIPG with support from Abbie’s Army.

Poster Award for CRL and M4K
Poster Award for CRL and M4K

Congratulations to Senior Scientist Stefano Levanto from Charles River Laboratories (CRL) on winning the early career poster prize for his poster describing CRL’s open science collaboration with M4K Pharma at the 20th SCI/RSC Medicinal Chemistry Symposium in Cambridge, UK last month.

Oxford Team Supports Brain Tumour Charity’s Twilight Walk
Oxford Team Supports Brain Tumour Charity’s Twilight Walk

On September 29th 2019, the Brain Tumour Charity organized The Twilight Walk in Warwick, UK, to raise funds for research and patient support. M4K Pharma’s DIPG project collaborators at the Structural Genomics Consortium (SGC)- University of Oxford took part in the event by hosting an activity booth and joining the walk.

Fostering Open Collaboration for Paediatric Cancer Drug Development
Fostering Open Collaboration for Paediatric Cancer Drug Development

Wong et al. describe in Biochemical Society Transactions how open collaboration and an open drug discovery model, like the one used by M4K Pharma, can help overcome the challenges faced while researching therapies for rare paediatric brain tumours such as DIPG.

Oxford Team Shares First ALK2 Protein Structure Bound to M4K Compound
Oxford Team Shares First ALK2 Protein Structure Bound to M4K Compound

M4K Pharma’s collaborators Dr Alex Bullock and his team from the Structural Genomics Consortium (SGC) at the University of Oxford shared the details of the ALK2 kinase crystal structure bound to M4K’s potent inhibitor M4K2117 in the RCSB’s Protein Data Bank (Structure 6SRH).

Study on New Drug Class Targeting ACVR1 Mutation for DIPG
Study on New Drug Class Targeting ACVR1 Mutation for DIPG

Scientists at The Institute of Cancer Research, London, along with colleagues at the Structural Genomics Consortium in Oxford, have published their findings on a new drug class that can kill brain cancer cells with mutations in the ACVR1 gene and shrink tumours in mice.

Open Science Drug Discovery Model Expands to Neurodegeneration
Open Science Drug Discovery Model Expands to Neurodegeneration

Following in the path of M4K Pharma, M4ND will tackle neurodegenerative diseases such as Parkinson’s disease, thanks to funding from the Krembil Foundation, and will commit to open science practises, open sharing of data, and affordable pricing.

For Innovation, Open Science Means Open for Business
For Innovation, Open Science Means Open for Business

Richard Gold (McGill University) and Max Morgan (SGC, M4k Pharma) penned an opinion piece in the Globe and Mail discussing how open science projects with no patents can result in local economic development, citing the recent $1Billion investment deal that Celgene signed with the Ontario Institute for Cancer Research (OICR) and their commercialization arm FACIT to advance a potential drug for leukemia.

M4K’s Open Science Model
M4K’s Open Science Model

How can a company take advantage of regulatory exclusivity to launch an open science drug discovery program? Max Morgan et al explain in their paper.

Can Open Science Find a Solution for Rare Brain Cancers in Children?
Can Open Science Find a Solution for Rare Brain Cancers in Children?

OICR-funded drug discovery project’s unique ‘open science’ business model is accelerating the search for a solution to lethal pediatric brain cancers.

Reaction Biology Joins the Fight Against DIPG with M4K Pharma [News Release]
Reaction Biology Joins the Fight Against DIPG with M4K Pharma [News Release]

Reaction Biology Corporation (“RBC”), a leading contract research organization providing early stage drug discovery services, announced that it has partnered with M4K Pharma (“M4K”), an open science drug discovery and development company, in the fight against diffuse intrinsic pontine glioma (or DIPG), a rare pediatric brain cancer with no curative therapies.

M4K’s Lemon Face Challenge for DIPG
M4K’s Lemon Face Challenge for DIPG

Watch the M4K team in Toronto complete the #LemonFaceChallenge to spread DIPG awareness, along with the Structural Genomics Consortium (SGC) and the Ontario Institute for Cancer Research (OICR).

Bio2040 interviews M4K Pharma’s Owen Roberts [Podcast]
Bio2040 interviews M4K Pharma’s Owen Roberts [Podcast]

In a podcast interview with Bio2040, CEO Owen Roberts discusses M4K Pharma’s business model and why open science and sharing can be the path to finding medicines for rare diseases like DIPG.

SGC Open Notebookers Share DIPG Drug Discovery Research in Real Time
SGC Open Notebookers Share DIPG Drug Discovery Research in Real Time

In January 2018, a group of post-doctoral fellows at the Structural Genomics Consortium (SGC) undertook the bold task of sharing their real-time and unpolished experimental results through open lab notebooks online in a mission to facilitate collaboration and transparency and to reduce duplication of effort.

Research Community Ready for Open Source Drug Discovery [Editorial]
Research Community Ready for Open Source Drug Discovery [Editorial]

In an Editorial to the Canadian Science Policy Centre, Drs. Aled Edwards and Aidan Hollis describe the need for Open Source Drug Discovery to address the inefficiencies in developing new drugs and the resulting exorbitant prices of new medicines.

OICR’s CTIP Initiative Funds M4K’s DIPG Program
OICR’s CTIP Initiative Funds M4K’s DIPG Program

The Ontario Institute for Cancer Research (OICR) announced its Cancer Therapeutics Innovation Pipeline (CTIP) initiative and the first 10 projects selected for the inaugural round of funding. CTIP will support the local translation of Ontario discoveries into therapies by creating a new drug development pipeline and by working with the research teams to attract further investments for clinical development.

Making Medicine, Not Money: How One UofT Researcher’s Startup Is Rethinking Big Pharma’s Business Model
Making Medicine, Not Money: How One UofT Researcher’s Startup Is Rethinking Big Pharma’s Business Model

The latest medical innovation to spring from Aled Edwards’s University of Toronto lab isn’t a new protein structure or potential drug target – it’s a business model.

Thanks to advances in genomics research, it’s rapidly becoming apparent many illnesses can be splintered into ever smaller categories of disease, each affecting a relatively small number of people. Hence all the buzz about developing “personalized” medicines.