Advancing a drug from the laboratory to clinical trials typically takes 17 years and around £1 billion. In only 10 years, Breakthrough has developed a new treatment, a PARP inhibitor, in ‘near record speed’ and ‘at a fraction of cost’.
The journey of PARP-inhibitors
Following research to target hereditary breast cancer Breakthrough scientists found that cancers caused by faulty BRCA genes were uniquely sensitive to drugs called PARP-inhibitors (reported in 2005).
This laboratory work paved the way for early clinical trials in patients with inherited forms of advanced breast, ovarian and prostate cancers, caused by faulty BRCA1 and BRCA2 genes. Despite having previously received many standard therapies, more than half of the patients’ tumours shrank or stabilised.
The results of the Phase II clinical trial created much excitement when published in 2010 where more than a third of patients taking the maximum dose showed some improvement in their tumours. Due to the drug working in a targeted way, it killed cancer cells while leaving healthy cells relatively unaffected, which meant fewer side effects for patients.
Following this breakthrough, Alan Ashworth’s team went on to discover that PARP inhibitors also kill cancer cells with faulty PTEN genes. Faults in the PTEN gene account for between 30 and 80% of breast, prostate, melanoma, endometrial and colon cancers and therefore the future potential of this drug is enormous.
Professor Alan Ashworth says: "It is highly likely that PARP inhibitors will become the standard care for BRCA mutation carriers in the next 5 years. I believe that they will also be important in the treatment of the more common sporadic breast cancers, as well as having an impact on treating other cancers."
This intelligent approach to cancer has changed the direction of cancer research;
the field has moved very rapidly and there are now over 40 clinical trials ongoing worldwide using PARP inhibitors in many types of cancer. If these trials are successful, these drug could be used to treat a wide range of cancers this would mean new treatments for thousands of patients across the world.
“Whatever the future holds, I count myself lucky and privileged to have been a patient in this trial and I am grateful and full of admiration for the scientists who made it possible and the clinicians who have made it a reality.” (Patient and BRCA carrier in the phase I clinical trial)
Looking to the future
PARP-inhibitors are now moving into the next phase of clinical trials. If these phase III trials are successful we hope that, within a matter of years, these drugs will be available to treat hereditary breast cancer.
PARP-inhibitors are one of the first of Breakthrough’s treatments to be tested in the clinic, but others are close behind. Breakthrough research has led to the development of 10 clinical trials.
We have made great progress but the reality remains that around 12,000 women in the UK die from breast cancer each year. We still have a long way to go until breast cancer is no longer a life-threatening disease.
Everything we do is breaking through. Our scientists are working every day to ensure our research makes as big an impact as possible – as fast as possible. With the help of our supporters, we want to make sure that we can continue to change and save lives now, and for generations to come.
