Stand-alone drugs

To make matters more difficult for non-stand-alone drugs, an additional group of people has to be added to the trial. If you are trying to treat, say, rheumatoid arthritis (RA), a chronic progressive disease, the trial takes 24 to 48 months. All of the many patients have to be X-rayed, which in itself is not cheap, to see if the drug slows deterioration of their... 

Importantly, the currently available transporter models only cover a small fraction of all transporters involved in drug disposition. Other than incorporating current stand-alone transporter models into systemic models to directly predict drug pharmacokinetic properties, continued efforts are still needed to investigate other transporters such as MRP, BCRP, NTCP, and OAT, to get a more complete understanding of the drug pharmacokinetic profile. 

OWENS has prepared antibodies to PCP in goats. When administered to mice the PCP levels in blood rose tenfold as an antibody-bound form that was readily excreted in urine. BROWNE tested the selfadministration by rats of 1,000 compounds related (and not related) to PCP, some of which produced PCP-like effects. One compound that was self-administered prevented the entrance of PCP into brain. BALSTER gave a general review of the effects produced by PCP in laboratory animals and showed that some effects were similar to those produced by amphetamine, some to barbiturates, and some to antipsychotics. This response profile makes PCP a unique drug that stands alone in its complex effects and toxicity. 

As described in the following chapter, there are many biopharmaceutical applications of protein assays. Assigning the protein concentration for the drug substance, drug product, or in-process sample is often the first task for subsequent analytical procedures because assays for purity, potency, or identity require that the protein concentration be known. Hence it is typical for several different methods to be employed under the umbrella of protein concentration measurement, depending on the requirements of speed, selectivity, or throughput. The protein concentration is valuable as a stand-alone measurement for QC and stability of a protein. However, protein concentration methods provide no valuable... 

Apart from disease entities, one recognizes a variety of symptoms that makes up a disease, like pain, fever, convulsions, cough, rhinitis, nausea, meteorism, diarrhea, headache, dyspnea, anorexia, insomnia, constipation, etc. All these symptoms could make up a symptom complex or syndrome, or may stand alone by themselves without being part of a disease entity. The term of symptomatic drug treatment usually refers to its use for the alleviation of symptoms, whether as a part of a syndrome or standing alone. 

Intuitively such delivery systems or excipients would best achieve this objective if they were to be composed of natural products or their modifications an overwhelming presence of excipients under this category (discussed in Ref. 1) provides credence to this observation. The substitution of natural products comprising complex proteins, antibodies, chimera, or toxins in lieu of stand-alone simple inorganic molecules as excipients for parenteral drug delivery represents a paradigm shift in the introduction of emerging excipients in the therapeutic armamentarium. 

The current buzzwords, chemical biology, somewhat degrade peptide chemistry to a service of biology. With the current book, we demonstrate that peptide-based drug design is an independent science that is well and on the rise. Our ultimate satisfaction will arrive when the first molecules, helped to conception or manufacture from the ideas set forth in this volume, gain regulatory approval and market share as stand-alone or combination medicines. 

The statistical review may also take a more global view and, in addition to evaluating the aspects of each protocol in a stand-alone fashion, evaluate how it fits in with and adds to the overall drug development program. 

An important concept to appreciate regarding receptors is that they are not static, stand-alone components of a cell. On the contrary, they are an integral part of the overall homeostatic balance of the body. For example, receptors can become desensitized upon continuous exposure to an agonist. When desensitization involves a specific class of agonists it is referred to as homologous desensitization. If response is reduced by disparate classes of drugs, the term heterologous desensitization is applied. 

Most regulatory bodies, concerned with the approval of new drugs for clinical use, do not accept the results of uncontrolled studies as stand-alone evidence of efficacy. There are, however, circumstances under which data from uncontrolled studies can provide acceptable evidence of efficacy (a) the treatment results in consistent and clinically significant improvement in a disease with a well-established natural course and remission rate, (b) the treatment consistently results in significant improvement in all or almost all patients. 

The picture painted here doesn t appear any rosier if you consider that most of the drugs in use as stand alone or in combination were approved before 1960 and, today, many of those would not be approved. Aspirin, for example, would not be approved today because of the frequency of stomach bleeding in its users. This would be true even if you also consider that we have come up with new applications— indications — for aspirin, such as to lower thrombosis risk by utilizing its recognized major side effect. 

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