By Angela Woodall, Healthcare and Environmental Reporter.
Parkinson’s disease was first diagnosed two centuries ago. Today it ranks among the most common degenerative disorders. However, a resolution is stubbornly elusive, as is the case with many diseases of the central nervous system despite a multitude of clinical trials.
Patients and advocates are keenly aware of the figures as well as the pace of treatments coming to market. The last breakthrough treatment, Rytary, was a reformulation of an existing dopamine replacement therapy. The Parkinson’s Foundation praised drug makers for listening to patients managing Parkinson’s, but also noted that there remained “a lot of room for improvement and more formulations in the marketplace.” Their concerns surface during events meant to bring attention to the disease — World Parkinson’s Day on April 11 and Parkinson’s Awareness month, which in the United States starts April 1. This year marks the 201st anniversary of an essay that established Parkinson’s disease — then called Shaking Palsy — as a recognized medical condition.
Such unmet needs were the topic of a recent two-day workshop populated by more than 400 industry and government leaders gathered to identify how regenerative medicine can help fill in the gaps. They convened to set an agenda for spending $30 million earmarked for regenerative treatments in the 21st Century Cures Act. In the words of NIH Director Francis Collins it was for what’s really possible and not just hypothetical. The Cures Act, along with patients and their advocates, have pushed the research agenda forward and given an incentive to the NIH and FDA to “seize the ripe opportunities” afforded by the legislation. As former director of the Genome Project, Collins helped steer research toward the stem cell and gene-based therapies involved regenerative medicine, which has ignited the hopes of patients suffering from Parkinson’s.
It has long been known the disease, which affects the brain, is caused by a breakdown of cells that produce dopamine. Those cells are developed early, in utero, and the body does not generate replacements. The only way to replenish them is to re-create them in a dish and put them back, as NYSTEM researcher Lorenz Studer, stated. This would have seemed farfetched until recently but Studer is part of a team developing a therapy that uses stem cells to help the brain produce the dopamine neurons Parkinson’s patients need. The therapy does not cure Parkinson’s but it does “cure” the motor symptoms caused by the disease. Current options are, at least in Studer’s estimation, “sub-optimal”, a driving force in research.
Regenerative therapies have challenged the regulatory and research apparatus of agencies like the NIH and FDA. For one, cell therapies and some gene-based therapy products blur distinctions between clinical trial phases, according to Larissa Lapteva, an FDA associate director whose department oversees advanced therapies.
This can be a challenge for regulators but an advantage for sponsors. Lapteva explains an early study may not only assess safety and tolerability, but also provide some basic information about the preliminary efficacy of the product. Early studies can yield valuable data. “We do encourage sponsors to maximize the design of those very first studies because of the value of the information that can be obtained.” Her office is looking for sufficiently informative pre-marketing development programs, not just ” flawless or textbook-like cases.” The FDA, she added, wants to see good quality products — not only when a treatment is ready to market, but when it is being investigated in clinical trials.
“A small fraction of trials involving regenerative therapies are aimed at Parkinson’s” shared Robert W. Mays, Vice President, Regenerative Medicine and Neuroscience Programs at Athersys. In 2017, the studies numbered 58, which works out to about 6 percent of the nearly 1,000 trials. Advocates like the research foundation founded by the actor Michael J. Fox want to see more but echo the cautious optimism of the NIH. “We’re a step closer, but we still have ways to go to prove the safety and efficacy of stem cells in people with Parkinson’s.”
PRC Clinical is involved with multiple sponsors in the Parkinson’s Disease research space. Our extensive experience managing and supporting clinical studies for CNS and neurodegenerative diseases outside of Parkinson’s include amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease, Multiple sclerosis, pediatric rare diseases, and various forms of neuropathic pain. PRC Clinical is currently supporting multiple programs, including a study with University of California, San Francisco (UCSF) with trial management services for a Phase I, investigator sponsored IND Parkinson’s disease clinical study. The four-year clinical study is being conducted at UCSF and the Oregon Health and Science University (OHSU), and is evaluating safety for a vector based, gene-therapy treatment, one of the many regenerative medicine programs we support not only in CNS, but other indications.
Our involvement and commitment to Parkinson’s Disease research goes beyond clinical trial management. On Friday, April 27th, PRC Clinical will sponsor Foxy Nights 2018 in San Francisco, CA, a fundraising event benefiting the Michael J. Fox Foundation and the fight against Parkinson’s disease. Last year’s Foxy Nights raised $27,000 for the Foundation for Parksinon’s Research. This year, the event organizers, along with PRC Clinical and other sponsors, are aiming for $35,000. If you would like to be involved, we invite you to read more about the event, register to attend, and donate on the Foxy Night’s website.
PRC is celebrating its 15th year anniversary in 2018 with conferences and events, starting with the Outsourcing in Clinical Trials West Coast Conference in Burlingame, California. We’ll also be publishing special reports and offering giveaways. In the meantime, you can keep up with PRC blogs and ClinPulse events here.