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Posted On: 10/23/2020 7:35:04 AM
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Can Growth Factors Cure Parkinson’s Disease?
Https://www.cell.com/trends/pharmacological-sciences/fulltext/S0165-6147(20)30217-0
Concluding Remarks
Although attempts to use GFs in clinical settings have a long history, these proteins are yet to demonstrate their full potential in PD patients. GF-based therapeutics are different from conventional small-molecule drugs in many aspects and their use requires careful design of clinical trials (see Outstanding Questions). Patient stratification, dose, and the delivery method should be meticulously considered in the future studies of GFs in PD patients. Nonlinear dose–response and pulsatile nature of GF release under physiological conditions favors intermittent administration of moderate doses of the proteins or the use of regulated gene therapy vectors in clinical trials in order to limit upregulation of various negative feedback mechanisms. Patient recruitment strategy should favor the selection of early stage PD patients to ensure the participation of people with sufficient numbers of remaining DNs in SNpc. Clinically favorable alternatives such as mutated GFs with improved tissue distributions, small molecules targeting GF receptors, and BBB-penetrating peptides should be seriously considered for further development of disease-modifying treatments against PD. Using alternatives to GF with improved tissue-penetrating ability, for example, small molecules, can also provide a way to target not only motor, but also non-motor symptoms of PD. In summary, clinical trials with GF-based therapeutics in PD patients should be continued in the future to find a cure for millions of affected people, but their design should be considerably improved.
Https://www.cell.com/trends/pharmacological-sciences/fulltext/S0165-6147(20)30217-0
Concluding Remarks
Although attempts to use GFs in clinical settings have a long history, these proteins are yet to demonstrate their full potential in PD patients. GF-based therapeutics are different from conventional small-molecule drugs in many aspects and their use requires careful design of clinical trials (see Outstanding Questions). Patient stratification, dose, and the delivery method should be meticulously considered in the future studies of GFs in PD patients. Nonlinear dose–response and pulsatile nature of GF release under physiological conditions favors intermittent administration of moderate doses of the proteins or the use of regulated gene therapy vectors in clinical trials in order to limit upregulation of various negative feedback mechanisms. Patient recruitment strategy should favor the selection of early stage PD patients to ensure the participation of people with sufficient numbers of remaining DNs in SNpc. Clinically favorable alternatives such as mutated GFs with improved tissue distributions, small molecules targeting GF receptors, and BBB-penetrating peptides should be seriously considered for further development of disease-modifying treatments against PD. Using alternatives to GF with improved tissue-penetrating ability, for example, small molecules, can also provide a way to target not only motor, but also non-motor symptoms of PD. In summary, clinical trials with GF-based therapeutics in PD patients should be continued in the future to find a cure for millions of affected people, but their design should be considerably improved.
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