Pamela Goldberger was given months to live after a diagnosis of glioblastoma, a highly aggressive brain cancer which has an extremely poor prognosis even with surgery. Now, she is healthy and “living her best life” after taking part in a clinical trial for a novel cell therapy.[1]
What is glioblastoma?
Glioblastoma is a type of tumour that develops from glial cells, which are cells supporting neurons in the brain and spinal cord. It is fast growing and very aggressive, and is the most common type of brain tumour in adults.[2][3][4]
There is no cure for glioblastoma[4], but it is treatable. The standard treatment consists of surgery to remove as much of the tumour as possible, followed by both radiotherapy and chemotherapy.[2][5] Without treatment, glioblastoma can result in death in fewer than six months; even with treatment, most people live an average of 12 to 18 months after diagnosis, and the five-year survival rate is approximately 5%.[3] This is because the tumour is highly invasive and spreads microscopic cancer cells into the surrounding brain tissue, making it impossible to remove completely.[6] The cancer can also effectively silence the body’s immune response, protecting it from attack.[1] Thus, treatment can lessen symptoms and improve quality of life, but the cancer almost always reoccurs.[5][7]
Survival rates and treatments for glioblastoma haven’t changed in 20 years[1], and although there are some risk factors, most cases occur in people with no risk factors at all,[6] says Dr Joseph Georges, a US-based neurosurgeon who led the clinical trial Pamela Goldberger took part in.[1] Symptoms depend on the tumour’s location and can include headaches, seizures, speech difficulties and weakness on one side of the body, as well as confusion and unusual behaviour.[6][8]
What does the new cell therapy entail?
Because glioblastoma is a highly mutated tumour which has different cells in each patient,[1] the therapy, too, needs to be specific to each patient treated. It is based on dendritic cells, a type of white blood cell which is part of the immune system, and is delivered alongside the standard treatment.
First, the patient has surgery to remove as much of their glioblastoma as possible. Then, scientists collect dendritic cells from the patient through a process called apheresis. During this process, a centrifuge machine draws blood from the patient and spins it to separate it into its various elements. The machine then collects specific elements, in this case dendritic cells, and returns the rest of the blood to the patient.[9] Afterwards, researchers use samples of the removed tumour to train the dendritic cells to specifically recognise the cancer and, in turn, teach the immune system to attack it.
After the surgery, patients undergo the standard six-week regimen of chemotherapy and radiation. Then, they receive three doses of modified dendritic cells, one every two weeks, along with weekly doses of pegylated interferon, a type of medication which helps regulate the immune system. This process is then followed by another year of maintenance chemotherapy.[1][10][11]
What were the trial results?
In the phase 1 clinical trial, the therapy was tested on sixteen patients with newly diagnosed glioblastoma.[1][10] Most of the patients (94%) had tumours resistant to chemotherapy, and 25% could not have their tumour fully removed in surgery. Two patients whose glioblastoma had recurred were also treated.[10]
The therapy proved to be safe, with only mild adverse events and no issues that would limit treatment dosage. What’s more, the therapy appears to have generated a desirable immune response in patients, resulting in improved survival. The 12-month overall survival rate for the study cohort was 88%, which is significantly higher than the expected 60% resulting from standard treatment; four of the patients are still alive after 22-33 months. The patients with recurrent glioblastoma survived for 10-12 months.[10]
According to Dr Georges, these are highly encouraging results, given the poor prognosis generally associated with glioblastoma, particularly the chemotherapy-resistant type. In some cases, the tumour has disappeared in MRI scans, which is, Georges said, “something you don’t see in glioblastoma”.[1]
One of these lucky patients is Pamela Goldberger, who, two and a half years after her initial diagnosis, is alive and healthy.[1]
The future of cell therapies for cancer
Although promising, this particular therapy is still in the early stages of development. There is now an ongoing phase 2 trial, with the first patient having recently been dosed.[12] In addition to this, the therapy is also being trialled for pancreatic cancer.[13]
Nevertheless, it is clear that cell therapies could, in the future, provide effective treatment for conditions and diseases that right now are a death sentence. As these therapies are still in development, it is impossible to know which of the many sources of cells available will prove to be the most effective for each specific therapy. It could be cells from peripheral blood, as used in this therapy, or, just as easily, cells from the umbilical cord or even the placenta.
Your baby’s future health could depend on having access to as many cell sources as possible, to enable the widest variety of treatment options – yet some of these are only available to collect and store in the few minutes right after birth. To learn more about how you could preserve this precious resource for your baby, request your free guide to cord blood banking below.
References
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