New Drug Has Promise for Treating Diffuse Intrinsic Pontine Glioma and Fibrodysplasia Ossificans Progressiva

Diffuse Intrinsic Pontine Glioma

Diffuse intrinsic pontine glioma (DIPG) is a rare form of childhood brain cancer that has a 100% mortality rate. Children diagnosed with DIPG only typically live 9 to 12 months after birth. Some patients may receive radiotherapy to slow the progression of the disease, however it is not a life-saving treatment. The tumors form in the brainstem, and therefore surgery is not an option. The use of chemotherapy in this condition has also been unsuccessful.

This patient population is desperate for more options, but in the last 20 years there have been no new approved therapies for any form of brain cancer, let alone this rare form.

However, an update from researchers at The Institute of Cancer Research (ICR) in London has just been released and there are exciting developments underway.

A New Development

The ICR, with collaborative help from an international team of scientists, has uncovered what they believe could be an effective treatment for DIPG. It could become the first available therapy for DIPG patients.

This development began in 2014 when the ICR discovered that mutations in the ACVR1 gene occur in 25% of all DIPG tumors. Since then, ACVR1 has been their research target.

Working with Oxford, ICR was able to create a new series of molecules that actually target ACVR1. They developed 11 prototype drugs in total that they believed had potential to stop the activity of mutated ACVR1. Specifically, these drugs targeted the protein that this mutated gene produces. They tested all 11 of these prototypes on DIPG brain cancer cells that they grew in the lab. Two of the prototypes were more effective than the others. They were able to kill the cancerous cells while leaving the healthy cells unscathed.

They then moved to a mice model. The drugs effectively stopped the activity of the mutated ACVR1 and shrunk the cancer tumors. Overall, the therapies extended the survival of the mice from 67 days to 82 days (25%).

While no other cancer involves ACVR1 mutations, they are the cause of another rare disease called fibrodysplasia ossificans progressiva (FOP), or stone man syndrome. The condition causes ligaments and muscles in the body to turn to bone. For this reason, the researchers believe that this treatment could also potentially serve those with FOP.

Additionally, a similar reaction involving Activin A occurs in both conditions.

Activin A is a molecule that is present while the brain is developing. But the researchers found that the mutated ACVR1 gene responds inappropriately to it. This triggers the growth of the tumors.

The results from this study were published in Communications Biology.

M4K Pharma

M4K Pharma is currently working on the development of this treatment and they hope to begin clinical trials in 2021. It will be M4K Pharma’s first big project. Specifically, this new company aims to serve children facing rare or under-researched diagnoses.

ICR is excited about their new partnership with M4K Pharma. Their goal is to create innovative therapies for small patient populations at a price range which will actually be affordable. Typically, it is difficult to get companies to support the development of therapies for small patient populations. But partnerships like this can change the game for rare disease patients.

Abbie’s Army

Abbie’s Army was founded by Amanda and Ray Mifsud in memory of their daughter who they lost to DIPG. They have helped support this research and are so excited to see what benefits this potential treatment could bring to patients. Their hope is that one day, families will not be told “there is no cure and no hope” like they were.

You can read more about this new development for DIPG and FOP here.


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