Stem Cells and Regenerative Therapies – How to Extract the Essence
the time of action of finding and developing new drugs is long and costly. It takes years and millions of dollars for one single new drug to be developed from the stage in which a candidate molecule was identified to the ultimate stage of regulatory approval for marketing. The new era of stem cells and regenerative medicine does not shorten this course of action. On the contrary, it presents new challenges in the road by translation into approved therapy. The challenges are due to the completely new and different structure of the final product. Patients are treated with living cells instead of active molecules. The road to develop new cell based therapy is not less long, complicated or costly than the road to develop traditional drugs.
The amount of information in the media, both specialized and popular, is huge. Nonetheless, most of it, although interesting and supportive for further development, is not applicable for present patient’s needs. If we want to estimate the current possible of a new therapy, the most important parameter is how close it is to clinical utilization. nearly, we want to know if the therapy is used already to treat diseases in human beings. Such treatment can be either experimental in its early stages, or ultimately, approved by regulatory authorities and provided by companies, clinics and hospitals.
Let us look at the various stages of development and consequently be able to clarify the most applicable ones according to this simple rule. Research is conducted on the molecular and cellular levels, in addition as in experimental animal models. Such research generates large amount of data, and set the basis for identifying new modules of therapies. Once a promising candidate is identified, the time of action of drug or cell based therapy starts. It needs complete characterization, understanding mechanism of activity, optimization of large extent production, thorough examination of safety profile, establishing quality control tests and more. The first stage is a general or theoretical research, while the second is called pre-clinical development. Once this stage is achieved, the product is ready for clinical studies, following the submission and approval by regulatory body. This third stage is termed clinical development. The clinical studies are conducted at three stages and if successful, approval for marketing is obtained. A product or course of action which is presently being tested in clinical studies, can be better evaluated for its current application. Being in the clinical stage of development is meaningful for functional purposes, although only few products will turn to be safe and effective, and approved for clinical use. The clinical phase of the development may take about 2 to 5 years, and sometimes longer.
In some countries, it is legal and possible to offer cell based treatments already before the complete extent clinical studies were completed. Clinics and hospitals in such countries are treating patients for life threatening and chronic diseases before thorough testing and approval by regulatory agencies in the US, Europe or other countries. Most of these clinics do not conduct or publish methodic clinical trials, but they usually provide information on the technologies and therapeutic approaches used. Can this be regarded as “tested in human patients”? The answer is no, but it is up to the patient to decide whether he would like to take the risk with all its implications, instead of wait for the therapy to be approved.
Most reliable information on current clinical studies is found in the FDA (US Food and Drug Administration) database – Clinicaltrials.gov. Although US based, most clinical studies from many other countries in addition are published in this comprehensive website which also includes effective internal search option. However, early pilot and phase I studies are usually not listed there. Results on clinical studies can be found in the well known scientific literature data base website, the US NIH (National Institute of Health) PubMed -Ncbi.nlm.nih.gov. Here again, the applicable clinical data will be difficult to extract from the plethora of scientific information, and it is mostly used by specialized scientists. Large medical centers will also provide updated information on scientific achievements, along with clinical trials and results. Many other blogs and websites, in addition as specialized organizations, provide information on stem and cell therapy news. For clinical applicable information on cell therapies – My New Therapy website is patient oriented and focuses on clinical data. As suggested, you can estimate the current clinical relevance of a piece of news according to its closeness to being tested in human patients, or preferably, if being already a part of reliable clinical study. This rule of thumb, being tested in human patients, can help to better estimate for functional purposes or needs, the clinical value of a product, service, technology or course of action.