We all want more research into Peripheral Neuropathy! Right??
We hope all of us take the opportunity to help in some small way. Often the studies are simple, like filling in a survey or being interviewed by a researcher on a specific topic. It doesn’t usually take long, and sometimes there is a bonus in it for participants. If provided the chance, please pitch in by taking an active part in research.
WINSANTOR – CLINICAL TRIAL Update
WinSanTor has completed two Phase 2 clinical studies. Phase 2 is a study phase meant to demonstrate safety and more importantly, PROOF OF CONCEPT, which basically means: Does the drug do what it is meant to do? After Phase 2 comes Phase 3, which precedes approval. We have come a long way, but still have a distance to go.
Our Phase 2 data demonstrated unprecedented small fiber nerve growth, or nerve regeneration, after six months of administration of WST-057. Based on this data, it appears that WinSanTor is the first and only company regenerating peripheral nerves in humans. This result supports WST-057 as a disease-modifying drug that works to change the underlying pathology that causes peripheral neuropathy and small fiber nerve degeneration.
The primary objective for Phase 2 studies is safety. The secondary objective is to show efficacy. While generally most of the focus of the Phase 2 program is on efficacy, it is a requirement to demonstrate safety in order to move forward in development.
Not surprisingly, few, if any, adverse events were seen with respect to WST-057. The few events that were seen were related to the formulation drying out the skin, which in most cases, were resolved through the use of a high-quality skin lotion.
The strongest efficacy results observed in the Phase 2 program was the increase in nerve fiber density after six months of drug administration. This data showed that WST-057 not only prevents nerve loss (the placebo lost nerves during the same period), but that nerves were regenerated in the active dose groups.
Interestingly, the sural nerve velocity, which measures large fiber nerve conduction, also showed improved nerve conduction after three and six months of administration of WST-057. Historically, nerve conduction velocity was the good standard for measuring nerve health. We are the first group to show clinically relevant changes for both small fiber nerve growth and sural nerve conduction.
Two of the most common assessments used in clinical settings are the modified Toronto Clinical Neuropathy Score (mTCNS), which combines patient-reported information and physician-directed neurological assessment, and the Utah Early Neuropathy Score (UENS) neurological assessment. After three months of administration of WST-057, both assessments showed strong improvement in the active group, particularly for functions associated with nerve regeneration/repair, such as decreased numbness and weakness. Again, we are one of the first groups to show positive results in symptoms such as numbness and weakness—symptoms usually ignored by the pharmaceutical industry.
Additional endpoints evaluated include quality of life (QoL), temperature sensitivity and activity. Although the sample sizes were too small to show statistically significance in these endpoints, the patients in the active group improved their QoL, could feel temperature changes better and when monitored with FitBit® watches, were walking more (distance and steps) and slept better.
With regard to pain, the WST-057 mechanism of action, does not impact pain like other pain drugs. These drugs might make the pain feel better temporarily, but they do nothing to impact the underlying cause or pathology of the neuropathy.
Our Phase 2 studies were designed to guide the design of Phase 3 protocol. The data from Phase 2 will be presented to the U.S. FDA in August 2023, with recruitment for Phase 3 to begin shortly after the FDA’s response to this data. We will discuss the endpoints and the number of patients necessary to reach statistical significance with the U.S. FDA. Phase 3 will be performed in North America and Europe. We are exploring clinical studies in other regions as well, including Asia, Middle East and Latin America. After Phase 3 is completed, we will review the data and submit for a New Drug Application for approval of the drug.
Being the first is not the easiest—and that is especially true for drug development. We are creating new tools and new processes, changing opinions and perceptions. Our progress is obviously contingent on the FDA and other regulatory agencies buying into our program, but based on the results from our Phase 2, we are clearly on the right track to be the first to develop a disease-modifying treatment.
Additionally, we have other ongoing studies:
The HIVN (HIV-induced neuropathy) study was completed and will be re-evaluated at a future date.
The CIPN study, which focuses on the neuropathy associated with chemotherapy, looks promising in preventing neurotoxicity from chemotherapy, though we are still relatively early on in this study. Chemotherapy is the first line treatment for most cancers, treating approximately two-thirds of all cancers in the U.S. and almost exclusively used to treat most cancers in the rest-of-the-world (ROW).
Living with Charcot-Marie-Tooth (CMT) can present unique challenges and experiences for individuals. CMT is a genetic disorder that affects the peripheral nerves, leading to muscle weakness, loss of sensation, and difficulty with coordination and balance.
There are thousands of research studies going on around the world. Below are some of the databases to help you find research studies of interest to you. You do not need to restrict yourself to neuropathy when searching. If you have an underlying condition, such as CMT or Diabetes, include that in your search criteria in the database.
Sponsored by Winsantor, The University of Alberta and other research institutes are conducting a randomized outpatient, double-blind, placebo-controlled, multiple-site study of the safety, tolerability, and exploratory efficacy of topically administered WST-057 (4% pirenzepine free base monohydrate) for 24 weeks in subjects with T2DM with peripheral neuropathy. Inclusion criteria include: diagnosis of T2DM; age rage 18-75 years, and presence of definite diabetic neuropathy of at least 12 months duration in the lower extremities. Recruitment for this trial is complete.
Our December speaker, Dr. Douglas Zochodne, Professor & Divisional Director of Neurology at the University of Alberta has recently published Our Wired Nerves: The Human Nerve Connectome. Dr. Zochodne is a world renowned expert in the field of peripheral neuropathy. This book is intended for patients or “non-specialists”. This book describes the make of peripheral nerves, discusses various neuropathies, the associated pain, and how nerves may be regenerated.
You can view details about the book, download the first chapter for free and purchase his insightful book on Amazon by clicking this link.
The CNA is striving towards raising funds for research, but we aren’t there yet. In fact we are a long way off. We need help.
Want to join us in building something special that will one day perform miracles? We believe it could happen with people like you!