Targeting the delivery of a pharmacological substance to its intended physiological site of action ensures that adequate concentration of the therapeutic is made available to elicit the desired clinical benefit and limit off-target / systemic toxicity. Advances in cell biology and molecular probing techniques have revealed the presence of several potential therapeutic targets for a number of difficult-to-treat diseases, localized within the confines of the cell membrane. Therefore, efficient methods of facilitating intracellular drug delivery to various sub-cellular compartments are a prominent need within the modern biopharmaceutical industry.
Need for Targeted Drug Delivery
Considering the fact that many modern pharmacological interventions are being designed against specific biological targets, there is an evident need for robust drug delivery technologies. Some of the key advantages offered by targeted drug delivery systems / devices are summarized below:
- Reduction of Systemic Toxicity and Side Effects: Targeted drug delivery is particularly beneficial in the case of highly potent drugs, which include the chemotherapy drugs and biologics (such as cell therapies, gene therapies and immunotherapies), making it possible to altogether avoid off-target toxicity related concerns
- Blood Brain Barrier Penetration: Over time, several types of drug delivery devices capable of facilitating the transport of drugs / therapeutic substances across the elusive blood braon barrier. Such technologies have revolutionized the treatment of many neurodegenerative, neuro-oncological disorders and other debilitating neurological conditions.
- Simplification of Introducing Medication in the Subretinal Space: Traditionally, drug delivery to the retina was a complex process, requiring a surgeon to perform retinotomy – a complicated procedure, involving the removal of the vitreous humor of the eye to inject a drug / therapy into the through retina.
- Intracellular drug delivery technologies use vehicles that encapsulate (52%) the drug payloads and facilitate them into the cytosol of the cell. Examples of technologies which utilize encapsulation includes (in alphabetical order) DeliverEX platform (Evox Therapeutics), Exosome Platform (ReNeuron), LUNAR (Arcturus Therapeutics) and SNIM RNA platform (ethris).
- Provisions for Sustained Delivery: In most cancers, the dynamic nature of the tumor microenvironment renders it difficult for a drug’s therapeutic effect to persist for a therapeutically beneficial time frame. This is primarily due to the fact that tumor cells eventually become programmed to tolerate active pharmacological substances or excrete / eliminate them from the vicinity. In order to circumvent the development of therapeutic tolerance, medical researchers have developed the means to ensure that pharmacologically active substances are delivered in formulations / devices that enable sustained release of the API over a prolonged duration of time. In this direction, intratumoral implants are now available / under evaluation and are deemed to offer better and lasting therapeutic outcomes.
Advanced Approaches for Delivery of Drug Payloads
Liposomes are described as aqueous-filled structures, surrounded by one or more double layers of amphiphilic lipids or phospholipids. These molecules are generally spherical in shape and their size ranges from 20 nm to 10 μm. Liposomes exhibit certain polymeric muco-adhesive properties, which prolong their retention time in the intestine. Further, they have been shown to be an effective method of delivery for both lipophilic and hydrophilic drugs.
Nanoparticles / microparticles are typically fabricated using certain organic / inorganic components, such as lipids or other polymers, which have a size range of 1 nm – 1,000 nm. These are essentially used to entrap or attach drug molecules, in order to facilitate their entry into target cells. Over time, several types of nanoparticles have been shown to be viable drug delivery agents, with reports of long circulating times.
Linking peptides to PEG can help increase the bioavailability of proteins / peptides. When a molecule is fused with PEG, each PEG subunit firmly couples with water molecules. This enhances the solubility index of the drug and enlarges the molecular structure of the protein / peptide. The increase in the size of the molecule prevents its clearance from the kidney, increasing its in vivo half-life. A few examples of PEGylated proteins are PEGylation of IFN-α2a (used for the chronic hepatitis C disease as a preliminary therapy), Peginterferon α2b (PegIntron) and mono-PEGylated TNF-α (used for antitumor treatment).
- Cell Penetrating Peptides (CPPs)
Cell penetrating peptides (CPPs) are short (10–30 amino acids), water-soluble, cationic or amphipathic peptides that are used as vehicles for the delivery of a wide range of macromolecular payloads into cells. These vehicles are able to penetrate the selectively permeable cell membrane through endocytosis and thereby, transport their respective cargos to the cytosol, where they are further processed into their clinically active forms.
CPPs have been demonstrated to facilitate the delivery of biologics, (such as proteins, and oligonucleotides), and small molecule drugs. The carrier molecules themselves may be protein-derived, chimeric, and / or synthetic.
Although several unique variants of CPPs have been identified, till date, only a few CPP-linked drugs have entered clinical trials. However, many preclinical studies have reported success in the delivery of fluorophore-labeled CPPs or CPP-cargo fusions, into specific target cells. One of the prominent limitations of CPPs is that at time the conjugates have been observed to remain trapped within endosomes, unable to deliver their cargo into the cytoplasm.
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