This is a story about the amazing collaboration of 2 biotechnology firms, a pharmaceutical company, and a leading health care institution to help save the life of a little girl who has been battling a rare cancer since the age of 3.
An unusual beginning
Zaida Mattson is a 12-year-old from Lafayette, Colorado, who was diagnosed with cancer when she was just 3 years old. For the past 9 years, Zaida has been in and out of hospitals all over the United States, working with some of the best oncologists in the country. Her cancer diagnosis has ranged from mesothelioma to papillary serous carcinoma but has ultimately remained unclear; physicians only know with certainty that she is battling a very rare form of cancer. In her fight against this disease, she has endured multiple rounds of chemotherapy and radiation, a myriad of drug interventions, and multiple surgeries, including a complete hysterectomy. The cancer, however, has always come back, and this last time, it came back much worse.
This past fall, the news of Zaida’s deteriorating condition spread across social media and reached the ears of scientists at ArcherDX, a local start-up biotech company located in Boulder, Colorado. ArcherDX specializes in detecting gene fusions and other mutations in tumor samples using sophisticated next-generation sequencing (NGS) tools. The scientists at ArcherDX thought they might be able to use their technology to discover something that others had missed: a mutation in Zaida’s DNA that could lead to a drug intervention. They ran Zaida’s sample through a sequencing-based diagnostic platform and found nothing, but they didn’t give up. They then asked a partner diagnostic company to perform an NGS-based test, which identified a variant of unknown significance involving AKT1, a gene known to promote cancer. This caught the eye of ArcherDX scientists, who promptly designed a research test to re-examine AKT1 in Zaida’s tumor biopsy samples. Lo and behold, a breakthrough—they identified a novel fusion in AKT1 in Zaida’s RNA.
Collaborating to save a life
As it turned out, Array BioPharma (another local Boulder biotech company) had developed a drug that targets AKT1 called Ipatasertib, so ArcherDX promptly reached out to Array BioPharma for help. There are no approved cancer drugs targeting AKT1. Ipatasertib was being used in clinical trials sponsored by Genentech, however, for the treatment of a number of different types of cancer. The problem was that all of these clinical trials were exclusive to adults. Ipatasertib had never been used, and was not approved for use, in children. Not to be dissuaded by that, pro bono networking efforts initiated by scientists at ArcherDX and Array BioPharma led Genentech and a devoted group of physicians and scientists at Memorial Sloan Kettering Cancer Center (MSKCC) in New York City to collaborate and write a compassionate-use trial for a single patient: Zaida. The trial was approved and, although she was almost too weak to travel to New York, Zaida was enrolled as the first child ever to receive Ipatasertib. Her treatments started in October. Incredibly, after just 3 weeks, PET scans revealed that Zaida’s tumors had shrunken drastically.
Zaida’s parents recently reported that she has a better appetite, has more energy, and has been running up and down stairs and doing handstands late at night—the results have been amazing.
This dramatic and quick response far exceeded expectations and speaks to the power of using targeted therapies customized to act against specific gene mutations.
Ipatasertib wasn’t necessarily designed with a childhood mesothelioma or other rare cancer in mind. Likewise, the tools developed at ArcherDX weren’t designed with a single drug, or even Ipatasertib wasn’t necessarily designed with a childhood mesothelioma or other rare cancer in mind. Likewise, the tools developed at ArcherDX weren’t designed with a single drug, or even a single cancer, in mind. All of these came together, however, to help one child, Zaida, and in doing so, they will help many others—adults and children alike—who are battling AKT-driven cancers, providing them with hope and a new treatment option.
While the scale of this response is impressive, it goes without saying that Zaida’s journey is far from over. Zaida will continue to travel to and from MSKCC in New York City for the foreseeable future as doctors continue to monitor her progress as part of the clinical trial. In response, ArcherDX hosted a fundraising event that raised over $25,000 in direct donations and contributions to support her treatment.
While a lot has happened in a short time, Zaida’s journey is far from over. You can follow and directly support Zaida’s journey by visiting her GoFundMe page.
Read more about Zaida’s story and watch a short clip on Zaida’s story below