Former Labour cabinet minister, Tessa Jowell, has called for patients with life-threatening conditions to have wider access to experimental treatments.
In her first speech to the House of Lords since last year, Baroness Jowell, who is receiving treatment for brain cancer, appealed for increased use of so-called “adaptive clinical trials” and more global collaboration to accelerate the development of new treatments.
The type of brain cancer that Baroness Jowell has – glioblastoma – is usually treated with surgery, followed by radiotherapy and chemotherapy. Unfortunately, the disease nearly always returns. The average survival time, from diagnosis, is 12-15 months.
Clinical trials are the standard way for assessing new drugs in a safe and controlled way. Traditionally, they investigated one new drug at a time. In order to become an accepted treatment, that drug has to pass several hurdles, or phases, that test its safety and effectiveness.
While this approach has undoubtedly been successful, it is not without its faults. Each new phase needs its own set-up and funding, which is costly and takes several years before the final results are known. Even then, fewer than 7% of the cancer drugs that start the process will become approved treatments.
Adaptive clinical trials are a relatively new type of clinical trial. They are designed to improve the speed and efficiency of drug development. They do this by taking a more flexible approach, allowing multiple drugs to be tested within the same trial.
Drugs that do not meet pre-specified targets can be discarded and new ones added, while those that show promise can move more seamlessly through the different phases. In this way, patients can have quicker access to new drugs as they can be added within the existing adaptive trial structure, rather than having to establish a whole new trial. For patients with rapidly progressing, life-threatening conditions, such as glioblastoma, this can make the difference between having access to a new drug or not.
A more flexible approach for a new era
We are entering an era of precision medicine. That means that if we notice that a patient’s cancer has mutated, we can tailor the treatment to specifically target that mutation – assuming a treatment is available. This is currently done for breast and lung cancers. Given that researchers – through global collaboration – have identified mutated targets in glioblastoma, this should be possible for future treatments for this deadly disease.
To speed up development of new drugs for mutated cancers, researchers have designed so-called umbrella trials and basket trials. Umbrella trials involve patients with the same cancer – say, lung cancer – being assigned different treatments based on their mutations. This way, several drugs – or drug combinations – can be tested at once. This gives researchers a quicker way to find the best candidate medicine for a specific mutation.
And basket trials involve patients with the same cancer cell mutation, regardless of where the tumour is – lung, kidney, spleen, say – testing a single drug. That way, researchers can quickly discover the drug’s effects on several cancer types at the same time.
Both basket and umbrella designs can be combined with an adaptive trial approach to provide a powerful technique for accelerating drug development.
Because glioblastoma is a relatively rare form of cancer, global collaboration is essential, from basic research, that might help identify new biomarkers, say, to the development of precision medicine clinical trials. The sharing of basic scientific knowledge among scientists, and the sharing of anonymised clinical data from patients (with their consent) will lead to an accelerated discovery of powerful new treatments.
Of course, there will be challenges, particularly with coordinating and unifying different experimental and treatment protocols, across the globe. There will also be debates about how to fund a patient if they travel to another country to participate in a trial.
Baroness Jowell ended her speech in the House of Lords by saying:
But I also have such great hope. So many cancer patients collaborate and support each other every day. They create that community of love and determination wherever they find each other.
We now need policymakers and health care professionals, the world over, to show the same love and determination by taking Baroness Jowell up on her challenge.