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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
Here’s what’s been going on with pharma and biotech regulatory intelligence:
FDA grants priority review to sNDA for full approval of Ibrance (palbociclib) in breast cancer. Based on the phase III PALOMA-2 trial and supporting conversion of accelerated to full approval of Ibrance for use in combination with letrozole as a frontline treatment for postmenopausal women with ER-positive, HER2-negative metastatic breast cancer. (OncLive)
FDA approves Spinraza (nusinersen), the first drug to treat children and adults with spinal muscular atrophy (SMA),a rare and often fatal genetic disease affecting muscle strength and movement. The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. (FDA)
FDA issues final guidance “Clinical Pharmacology Data to Support a Demonstration of Biosimilarity to a Reference Product.” The guidance will help sponsors develop a clinical pharmacology program to support a decision that a proposed biosimilar is not meaningfully different clinically from a branded or reference product. (FDA)
FDA issues complete response letter (CRL) to Cempra regarding submissions for Solithera (solithromycin)for the treatment of community-acquired bacterial pneumonia (CABP) in adults. Specifically, the FDA noted that, before the application can be approved, additional clinical safety information is needed and that manufacturing deficiencies must be resolved. (FirstWord Pharma)
FDA officials call for inclusion of older adolescents in adult oncology trials. In a recent article in Clinical Cancer Research, officials wrote that “In order to facilitate earlier access to investigational and approved drugs for adolescent patients with cancer, and because drug exposure is most often similar in adolescents and adults, we recommend the inclusion of adolescents (ages 12-17) on disease and target-appropriate adult oncology trials.” (RAPS)
“PwC’s pharma and healthcare predictions for 2017 are out. And though, yes, next year may be “a year of uncertainty” with U.S. President-elect Donald Trump set to step in, it’s also a year of “opportunity,” the consultancy says.”
While patient connection is not a new concept, in 2017 it will be more valuable than ever, according to the new PwC healthcare report. Patients are becoming more engaged and are open to connecting directly with pharma companies. The report offers some predictions for the new year, one being that patients could become pharma’s new strategic partner.
It’s a year of opportunity, and to connect with patients means to communicate effectively with them. In our work with clients, we find that there are 4 qualities that make patient communication effective and valuable:
Clear – The purpose is clear to the reader and easy to digest.
Concise – No beating around the bush. People are overwhelmed with information. Get to the point in as few words as possible.
Compelling – Learn what evokes the reader’s interest and use that to engage them with a story that catches attention and prompts action. Do more than just recite facts and figures.
Clinically correct – Even if you have the first 3 C’s nailed down, if you miss this one, it’s game over. The language has to be scientifically accurate, but—and this is the hard part—it must also be understandable to someone without a science background.
Like to know more? Give us a ring at Aegis Creative. We’d love to have a conversation with you.
Here’s a look at what’s going on this week with pharma and biotech regulatory intelligence!
CDER’s accomplishments for 2016 and priorities for 2017. CDER Director Janet Woodcock’s priorities for 2017 include: developing a plan to automate processes for new drug reviews, implementing provisions of the 21st Century Cures Act, and supporting reauthorization of the user fee acts. (FDA)
New postmarketing safety requirements for combination products. On December 19, FDA finalized new postmarket safety reporting requirements for combination drug/device, biologic/device, biologic/drug, or drug/biologic/device products. (FDA)
FDA grants accelerated approval to Clovis Oncology’s Rubraca (rucabarib) for BRCA+ ovarian cancer. Rubraca was approved for the treatment of women with advanced ovarian cancer who have received two or more prior chemotherapies and whose tumors have a BRCA gene mutation, as identified by the CDxBRCA companion diagnostic test. (FirstWord)
FDA approves removal of boxed warning for neuropsychiatric events for Pfizer’s Chantix (varenicline). Chantix is approved to help adults quit smoking. Removal of the boxed warning was based on the results of a randomized clinical trial designed to determine whether smokers with a history of psychiatric disorders are at a greater risk than other smokers for developing clinically significant adverse neuropsychiatric events. (Pfizer)
European Medicines Agency (EMA) to launch a tailored scientific advice pilot to support the development of biosimilars. The pilot will advise developers on the studies they should conduct, based on a review of their available quality, analytical, and functional data. (RAPS)