Academic pharma focus on the discovery and development of innovative and new medicinal products (or new targets) and repurposing already existing medicinal products. These innovative medicinal products will strongly contribute to the advancement of health care.
Vision and mission
Most medicinal products available are the result of:
- academia delivering new knowledge based on biomedical and chemical research.
- pharmaceutical companies that further develop these new insights in safe and effective medicinal products.
However, there are only a few examples of effective collaborations between academia and industry that resulted in registered medicinal products. That these collaborations are unsuccessful is, in part, caused by differences in culture and goals between these sectors. In addition to this problem, there is limited interest in developing medicinal products for extremely rare diseases or in patent-expired products.
To address these problems there is a need for a more active involvement of academia in the discovery and development of new medicinal products, and setting up collaborations with industry in which academia plays a more prominent role.
In Leiden there is excellent research performed at the Leiden University, the Centre for Human Drug Research and the Leiden University Medical Centre. These parties are all located in the Leiden Bio Science Park in the vicinity of pharma companies and biotech-start-ups. This is an excellent environment for an active role of academia in the discovery of new targets, and (clinical) development of medicinal products.
“In Leiden all expertise and facilities for design, development and testing of new innovative medicine are present.”
Jacques (Sjaak) Neefjes, Professor of Chemical Immunology
Jacques (Sjaak) Neefjes is Professor of Chemical Immunology and head of the Department of Cell and Chemical Biology. One of the topics where Sjaaks research focuses on is understanding the working mechanism of anti-cancer medicine. This knowledge can improve medicines or diminish the toxic side effects of the medicines. Recently, his team discovered that chemically changing the chemotherapy doxorubicin, made it less cardiotoxic. This new compound, N,N-dimethyldoxorubicin is now further developed under academic pharma.
Teun van Gelder, Professor in Clinical Pharmacology
Teun van Gelder was trained in internal medicine, nephrology and clinical pharmacology. Ever since he has been involved in the clinical development of several innovative drugs, and in developing personalised treatment strategies, based on novel therapeutic drug monitoring strategies and on clinical implementation of pharmacogenetics. In December 2019, he was appointed as professor in clinical pharmacology, in particular Drug Discovery & Development at the LUMC. One of his goals is to set up a clinical research unit, which makes it possible to conduct early phase medicinal product studies in patients.
Laurel Schunselaar, Postdoc researcher
Laurel Schunselaar works as post-doc in the LUMC. She leads multiple projects within academic pharma, including a multicentre phase III trial of the new chemotherapy aclarubicin in acute myeloid leukemia, a LUMC phase I trial of aclarubicin in uveal melanoma and the N,N-dimethyldoxorubicin project. Within these projects she is responsible for outsourced non-clinical research and setting up clinical studies. In addition, she interacts with regulatory authorities and ethics committees to support the desired indication and market approval.
Research collaborations and alliances
Paul Janssen Futurelab develops international online and on campus learning-by-doing courses for entrepreneurial biomedical professionals working in academia, start-ups, industry and at authorities. Collaboration between the LUMC and Paul Janssen Futurelab improves processes for discovery and development of new medicinal products.
Read more about Paul Janssen Futurelab.
With Academic Pharma we aim to bring new medicinal products to the market. The full clinical development will not be done by Academic Pharma in Leiden alone, but in collaboration with the industry. If you are interested in setting up a collaboration, please contact us.
For PhDs and Postdocs
We are designing a PhD/ postdoc program within Academic Pharma at the moment. The program focuses on interdisciplinary PhD-students/ postdoc or PhD-student/ postdoc teams that work on discovery, development, social, or legal aspects within Academic Pharma. If you are interested in a PhD or postdoc position let us know what you would like to accomplish within academic pharma.
For students we have multiple master internships available. We offer opportunities in the field of discovery, non-clinical development, clinical development and regulation. Let us know what kind of internship you are looking for, and in which field or fields.
The development of new medicinal products involves many aspects. It all starts with the discovery of a new medicinal target or candidate medicinal product. To further develop the candidate medicinal product it is important that it is properly protected.
Often the chemical or biological candidate can be tested in cells and animals, but it is not yet suitable for use in humans. A new production process according to good manufacturing practice (GMP) is necessary. With more patients receiving personalised treatment, there is a need for innovations in drug manufacturing. For further development of the candidate medicinal product, it is essential to define which non-clinical studies are important to conduct.
Clinical development is all about testing the drug in healthy volunteers / patients. Distribution in the body, safety and efficacy are amongst many topics that are tested in clinical trials. The ultimate goal for the medicinal product is market registration. To achieve this, the product must meet the requirements of the regulatory authorities.
Research and discovery
Fundamental biomedical and chemical research could lead to new candidate medicinal products.
This research involves screening of compound libraries in which hits are further characterised, researched and ultimately further developed. But a better understanding of diseases, pathways involved in these diseases, and working mechanisms of therapies, will also give new insights into treatment options for which new medicinal products could be developed.
Not every compound will be suitable to develop further in an academic setting. The Academic Pharma drug development board has the goal to review and evaluate scientific, clinical and technical issues related to the discovery and development of candidate medicinal products. The board provides recommendations to investigators who submitted requests or proposals for new candidates medicinal products.
Towards precision drug application by studying drug uptake pathways
Tissue-specific drug-uptake has not been well studied. Many drugs need active or defined transporters to pass the cell membrane. Tissue-specific uptake of anti-cancer drugs may directly impact the side effects of many drugs when they accumulate in healthy tissues. Therefore, linking anti-cancer drugs to their respective drug import transporters would directly help to predict drug responses whilst minimising side effects. We aim to use CRISPR screening technology to identify potential drug importers of commonly used anti-cancer drugs such as anthracycline drugs. By this method, a comprehensive anti-cancer drug importer catalogue will be assembled for major anti-cancer drugs.
Contact person: Baoxu Pang - email@example.com
Related research facilities
If you would like to submit a request or proposal for a candidate medicinal product to the Academic Pharma development board, contact Teun van Gelder.
Academic Pharma focuses on innovative manufacturing and collaborate with companies for upscale production.
In manufacturing, you can distinguish between raw material, also called the active pharmaceutical ingredient (API), and the candidate medicinal product.
During the research and discovery phase, only small amounts of the API are produced. However, for non-clinical studies, production of the API should be increased. Thereby for clinical studies, the API should be manufactured according to good manufacturing practice (GMP). The active pharmaceutical ingredient should be further formulated to a candidate medicinal product. This could, for instance, be a tablet or a powder that is dissolved for infusion.
To change the manufacturing process GMP, and for large scale manufacturing, Academic Pharma works together with other companies. Within Academic Pharma, there are some projects in which the candidate medicinal product is manufactured on a small scale or formulated on a larger scale. Thereby Academic Pharma focusses on innovations in drug manufacturing, especially for personalised treatment.
Innovations in drug manufacturing: how 3D printing can enable the production of personalised medicine
Treatment with drugs has moved from a blockbuster ‘one size fits all’ to a personalised medicine approach. Indeed, children, patients with rare diseases, and patients in need of an individualised adapted drug dosage based upon the DNA profile cannot always be treated with standard dosage strengths. 3D printing of tablets will allow small scale manufacturing of tablets perfectly adapted to individual patients’ personal needs.
In our research, we focus both on technical aspects as well as implementation. We believe that this will lead to increased knowledge and applicability of the innovative 3D tablet printing technology. Besides, it is an essential step in the further development and implementation of personalised medicine in healthcare.
Contact person: Kirsten Schimmel – firstname.lastname@example.org
Related research facilities
Contact us for more information.
After discovery of the candidate medicinal product, it is further developed in non-clinical studies. These include in vitro (cell, organoids, organ on a chip) and in vivo (animal) experiments.
A large part of these studies focus on safety. You have to show the candidate medicinal product is safe for pharmacokinetics and toxicokinetics, (safety) pharmacology, and toxicology including general toxicity, reproduction toxicity, genotoxicity, carcinogenicity, immunotoxicity and photo safety . But dose finding, drug delivery, drug-drug interaction and efficacy studies are also part of the non-clinical development studies. It depends on the candidate medicinal product which non-clinical studies should be performed.
LUMC’s research community
Most of the studies should be performed before you can start your clinical trials. However, non-clinical development continues during clinical development. It is therefore useful to design a development plan before starting any studies.
The LUMC has a large research community that can help with the non-clinical studies. When the study could not be performed in the LUMC, we have contact with reliable partners that could help you further.
The development of N,N-dimethyldoxorubicin
Doxorubicin is a chemotherapy that is used for the treatment of various tumors. In most cases it is very effective, but also gives a lot of side effects. Cardiotoxicity is, for instance, the dose limiting factor.
N,N-dimethyldoxorubicin is a semi-synthetic product, produced by dimethylation of the amine group of doxorubicin. It has antitumor activity against various cell lines. In addition, it has anti-tumor activity against experimental murine tumor models and a human AML patient-derived xenograft (PDX) model in mice. Mice experiments showed N,N-dimethyldoxorubicin give less side effects than doxorubicin, especially cardiotoxicity. This was in different models, not observed.
The non-clinical development of this project now involves general toxicity studies in different animal models and dose finding studies.
Contact person: Sjaak Neefjes email@example.com or Laurel Schunselaar firstname.lastname@example.org
Related research facilities
If you are interested in designing or conducting non-clinical studies, we can think along with your development plan and bring you in contact with other researchers in the LUMC.
The clinical development of the candidate medicinal product is all about testing the drug in healthy volunteers or patients. These tests are also known as phase I, phase II and phase III clinical trials.
The clinical trials are designed and conducted to answer several questions. The main questions to answer are:
- Does the candidate medicinal product reach the site of action? This includes analysis of absorption, distribution, metabolism, and excretion.
- What is the safe therapeutic window of the product?
- Which dose could be safely administrated and in what schedule?
- Is the functional and/ or clinical effect of the candidate medicinal product reached? If a product is functionally effective, this does not automatically mean it is also clinically effective. For instance, a chemotherapy can reduce the tumour, but not give a survival benefit. Other endpoint like side effects and quality of life are then important factors.
- Is the intended population, the correct population and how does variability in response changes the development?
For each candidate medicinal product there are more specific questions. The questions then need to be translated to clinical trials in which endpoints, comparative treatment, inclusion and exclusion factors etcetera need to be determined. The LUMC has a lot of experience in designing and conducting clinical trials for all types of diseases.
Postbiotics to boost mucosal immunity
Severe viral respiratory infections can lead to hospitalisation and even death. There is no effective medication for these viral infections. Strategies to protect risk groups are urgently needed. Until now antimicrobial therapies have focused on targeting invading pathogens directly.
A novel approach is to reduce acute respiratory infections in at risk groups by strengthening their own mucosal immunity. Gut commensals profoundly impact host immunity. They generate bioactive molecules – postbiotics – that promote gut integrity and antimicrobial activity in immune cells, but without tissue-damaging inflammation.
Researchers from Leiden University Center of Infectious Diseases (LU-CID) plan to administer postbiotics (in this study butyrate) to healthy volunteers for a proof-of-concept study to reduce viral load and clinical symptoms in a controlled human rhinovirus infection. The hypothesis is that nasal mucosal responses should prevent pathogens from spreading to the lower airways. Academic Pharma is supporting the investigators in developing a nasal spray containing the postbiotic butyrate, under GMP conditions.
Contact person: Hermelijn Smits - email@example.com
Bladder carcinoma patients undergo transurethral resection of the bladder tumor. In patients with a high risk of recurrence and progression this surgery is followed by adjuvant intravesical instillations with Bacillus Calmette-Guérin (BCG). However, patients do not always tolerate BCG and the risk of relapse in BCG-treated patients varies between 30-40 percent. Therefore, more effective therapies for high-risk bladder cancer patients are warranted.
Cationic Amphiphilic Drugs (CADs) are a class of commonly used antidepressants, antihistamines and antipsychotics. Preclinical research at the department of Urology at LUMC, in close collaboration with the Erasmus MC, has shown that treatment with CADs resulted in a significant decrease in viability and proliferation of multiple human UCB cell lines. Treatment of mice with CADs resulted in a significantly reduced tumour burden. Penfluridol treatment of ex-vivo cultured human UCB tissue slices caused significant anti-tumour responses
The goal of the department of Urology and Academic Pharma is to repurpose penfluridol for the treatment of bladder cancer. A phase I study in patients is currently being prepared.
Contact person: Gabri van der Pluijm - G.van_der_Pluijm@lumc.nl
Related research facilities
If you are interested in designing or conducting a clinical trial in the LUMC, we can think along with you and bring you in contact with physicians.
Regulation for market registration
The ultimate goal for a candidate medicinal product is market authorisation. An application for market authorisation must comply with all kinds of legal and regulatory requirements.
The key principle of a medicine assessment, performed by the regulatory authorities, is the balance between the benefits and risks of a medicine. The medicine should be safe, effective, and of good quality to be suitable for use in patients. A medicine will only be authorised if its benefits outweigh the risks. The application should therefore at least include information on:
- the pharmaceutical quality, including its chemical and physical properties
- the mechanism of action
- the distribution of the medicine
- the benefits for the patient group
- the safety of the medicine
- and how risks will be managed and monitored
All experiments and studies should be performed in compliance with international requirements such as good manufacturing, laboratory, and clinical practice. It depends on the candidate medicinal product and the intended patient group for whom the medicine is developed, which preclinical and clinical studies are necessary. It is, therefore, important to know the guidelines before designing a development plan.
If you would like more information on the topic of regulation for market authorisation, have any questions or need any guidance with the regulatory part of your development plan, please contact Laurel Schunselaar.