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Leading Mitochondrial Disease Research Center in Canada

Liam Pharma: Turning Research Breakthroughs into Treatments for Mitochondrial Disease

Liam Pharma is 100% dedicated to the fight against mitochondrial disease. Born from The Liam Foundation's mission, it works with doctors, hospitals and research centres around the world to bring the first therapies for POLG and mitochondrial DNA depletion disorders from the clinical trial to the patients who need them.

Why Liam Pharma Exists

Because a Breakthrough Only Matters When It Reaches Patients

The effects of mitochondrial diseases are profound and multifaceted:

For decades, families facing POLG and mitochondrial DNA depletion disorders were told the same thing: there is no treatment. Then, a clinical trial funded mainly by The Liam Foundation changed that. Led by Dr. Kenneth Myers at the Montreal Children’s Hospital and the Research Institute of the McGill University Health Centre, the trial showed that deoxycytidine/deoxythymidine (dC/dT) combination therapy is safe and effective for POLG-related disorders.

A research result, however, is not yet a treatment that patients can get from a pharmacy. Liam Pharma was created to bridge that gap. It carries the science forward through the clinical, regulatory and manufacturing steps needed to make therapies available to children and adults in Canada and around the world.

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Liam Pharma | Bringing Mitochondrial Disease Treatments to Patients
Our Research Program

Safety and Efficacy of an Oral Deoxynucleoside for Mitochondrial DNA Depletion Disorders

Recent years have seen promising developments in the understanding and treatment of mitochondrial diseases:

  • Gene Therapy: Innovative gene therapy approaches are being explored to correct genetic defects causing mitochondrial dysfunction. Clinical trials are underway to assess the safety and efficacy of these treatments

  • Pharmacological Interventions: New medications are being tested to enhance mitochondrial function and alleviate symptoms. For instance, a clinical trial almost entirely funded by the Liam Foundation and led by the Research Institute of the McGill University Health Centre investigated a treatment for POLG-related disorders, showing potential in improving patients’ quality of life.

  • Diagnostic Tools: Advances in genomic testing have improved the accuracy of diagnoses, enabling personalized treatment plans. A study by the Murdoch Children’s Research Institute highlighted the importance of genomic testing in diagnosing mitochondrial diseases in children, achieving a 71% success rate among young patients.

One Mission

To Find a Cure for Mitochondrial Disease

Develop Therapies

Advance deoxynucleoside therapy and other promising approaches through clinical research, so that patients with mitochondrial DNA depletion disorders have real treatment options.

Improve Access

Work to give patients and families access to essential medications and vitamins, known as the Mito Cocktail, that support people living with mitochondrial disease, under the guidance of their medical team.

Collaborate Worldwide

Partner with The Liam Foundation, physicians, hospitals, research centres and organizations around the world, united by one goal: a cure.

Our Research Program

Safety and Efficacy of an Oral Deoxynucleoside for Mitochondrial DNA Depletion Disorders

In November 2025, CQDM, Québec’s biopharmaceutical research consortium, selected Liam Pharma’s project for funding through its SynergiQc program, with a grant from the Government of Québec. Liam Pharma and Dr. Kenneth Myers of the Research Institute of the McGill University Health Centre are working together on this project.

The project evaluates how a combination of deoxycytidine and deoxythymidine, two natural building blocks of DNA, can help mitochondria produce energy more efficiently in children and adults living with rare mitochondrial DNA depletion disorders. Early data from the clinical study in Montreal show promising efficacy and good tolerability, paving the way for expanded patient recruitment.

Key facts :

  • Partner: Dr. Kenneth Myers, Research Institute of the McGill University Health Centre
  • Funding: CQDM SynergiQc program, Government of Québec grant
  • Therapy: oral deoxycytidine/deoxythymidine (dC/dT)
  • Patients: children and adults with mitochondrial DNA depletion disorders, including POLG-related disorders
  • Goal: bring one of the first therapies for these disorders to market

Expected impact :

  • Fewer repeated hospitalizations
  • Tangible health improvements for patients
  • Less pressure on the healthcare system
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How It Works

Helping Mitochondria Rebuild Their Energy Supply

Mitochondria are the power plants of our cells, and they carry their own DNA. In POLG-related and other mitochondrial DNA depletion disorders, cells cannot copy that DNA properly. The amount of mitochondrial DNA drops, and cells lose their ability to produce energy, especially in the brain, muscles and liver.

dC/dT therapy provides two natural DNA building blocks, deoxycytidine and deoxythymidine. These help cells rebuild their mitochondrial DNA and restore energy production. The treatment is taken by mouth or feeding tube.

Results to date (from the long-term trial funded by The Liam Foundation):

  • 25 patients treated, ages 1 to nearly 48
  • Clinical scores improved at every checkpoint from 1 to 24 months (Newcastle Mitochondrial Disease Scale: 30.9 → 18.8)
  • Quality of life improved significantly
  • Only 11% of early-onset children died in the first year, compared with more than half expected from natural history data
  • Published in Neurotherapeutics (2026)

Disclaimer :
dC/dT therapy is currently available through clinical research and is not yet an approved commercial treatment. Patients should always consult their physician before starting any medication or supplement.

Partners

Our Research and Funding Partners

Research Institute of the McGill University Health Centre (RI-MUHC) , partner of Liam Pharma for Mitochondrial Disease

Research Institute of the McGill University Health Centre (RI-MUHC)

The Montreal Children's Hospital Foundation partner with the Liam Foundation

Montreal Children's Hospital

CQDM, SynergiQc program , partner of Liam Pharma for Mitochondrial Disease

CQDM, SynergiQc program

Government of Québec , partner of Liam Pharma for Mitochondrial Disease

Government of Québec

Frequently Asked Questions About
Liam Pharma

What is Liam Pharma by the Liam Foundation ?

Liam Pharma for Mitochondrial Disease Inc. is a company 100% dedicated to the fight against mitochondrial disease. Working with The Liam Foundation and with doctors, hospitals and research centres around the world, it develops therapies and improves access to medications for people living with POLG and other mitochondrial diseases.

Liam Pharma was born from The Liam Foundation’s mission and works in close collaboration with it. The foundation funds research and supports families, while Liam Pharma carries research breakthroughs toward treatments that patients can access.

Liam Pharma is working with Dr. Kenneth Myers of the Research Institute of the McGill University Health Centre on oral deoxycytidine/deoxythymidine (dC/dT) therapy for mitochondrial DNA depletion disorders, including POLG-related disorders. The project receives funding from CQDM’s SynergiQc program, with a grant from the Government of Québec.

dC/dT therapy is currently available through clinical research in Montreal at the Liam Mito Centre. It is not yet an approved commercial treatment. Liam Pharma’s goal is to help bring it to market so that more patients can benefit from it.

The Mito Cocktail is a combination of vitamins, supplements and medications commonly prescribed to support people living with mitochondrial disease.

Liam Pharma works to make these essential products more accessible to patients and families. They should always be taken under the guidance of a physician.

Researchers, clinicians, hospitals and industry partners who share our mission can contact Liam Pharma through The Liam Foundation.

We welcome collaborations that accelerate treatments and, ultimately, a cure for mitochondrial disease. You can also donate to the Liam Pharma by clicking Here.

Resources

Resources for Families

The Liam Foundation is committed to supporting families affected by mitochondrial disease by providing resources, access to specialists, and community support. Whether you’re looking for a doctor, seeking information about treatments, or simply need emotional support, we’re here to help. 

Join a Fundraising Event

Be part of community events like walks, runs, and charity galas organized by The Liam Foundation. Your participation not only raises funds but also spreads awareness about mitochondrial disease.

Volunteer Your Time

If you’re looking to make a direct impact, consider volunteering with The Liam Foundation. From event planning to support group facilitation, your efforts can bring hope to families in need.

Spread Awareness

Help us amplify the conversation around mitochondrial disease by sharing our resources on social media, joining awareness campaigns, and educating others about this often-overlooked condition.

Reach Out for Help

If you or a loved one is affected by mitochondrial disease, don’t face it alone. Contact The Liam Foundation at contact@liamfoundation.com.

Together, We Can Make a Difference

Living with mitochondrial disease is a challenging journey, but with the right help and support, it’s possible to improve the quality of life for patients and their families. The Liam Foundation stands as a pillar of hope, offering resources, advocacy, and funding to drive progress in research and treatment.

By donating, volunteering, or simply spreading the word, you can help bring us closer to a future where mitochondrial diseases are better understood, managed, and ultimately cured. Join us in this fight today. Together, we are stronger. 💚