Planning for Clinical Trials
Assessment of the factors relevant to the design of clinical trials has been the major focus of recent planning. Noscapine has now been identified as the preferred drug candidate for the clinical trials based on its effectiveness on HSP stem cells, safety data, toxicology data, patent status, availability, and future affordability to HSPers.
Opinion has been sought from consultants specialising in clinical trial design regarding how much of a ‘jump start’ in the process might be possible based on the long-established good safety and toxicology record with the candidate drug. This is an important and critical process as the decision that will be made will significantly influence how long clinical trials will take and how much they will cost. It will also have bearing on the likelihood of acceptability and approval from ethics and regulatory authorities.
Preliminary discussions have also been held with contract research organisations regarding implementation of early stage trials. Several levels of highly specialised, technical documentation are required for submission to one or more ethics committees, and to regulatory bodies, for approval. Funding of the overall process through government, institutional and philanthropic grants, private equity investors and commercial partnership with a pharmaceutical company has also been discussed and now requires concerted investigation and consideration to ensure the best decisions are made over time in the interests of establishing an effective treatment that is widely available and affordable to HSPers globally.
2016 was by far the best year yet for fundraising with a grand total of $139,196 being raised over the 12 months, close to a whopping 40% increase over 2015 and a very healthy 17% above target.
This reflects the high levels of both generosity and commitment from around 100 members of the HSP community. We know that many in the HSP community have very limited means and therefore capacity to give, and on behalf of all, our sincere thanks goes to those who have given so generously on a continuing basis.
No part of clinical trials has yet been costed as planning is not sufficiently advanced, nor decisions made, on which costing would be based. Those figures will be forthcoming in time. Let’s hope that accumulated funds are adequate to at least get the process started.
The next three months
Working to come up with an optimal trial design with an advantageous starting point, good collaborators, good partners and further clarity on the path forward into trials is the objective for the next quarter with the next report due at the start of June.