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Final Report by the Commission on Professional Self Regulation in Science of the Medical Faculty LMU Munich on Gene Therapy Study of the Wiskott–Aldrich Syndrome

26.07.2017 -

The final report by the Commission on Professional Self Regulation in Science of the Medical Faculty of Ludwig Maximilians University (LMU) Munich was presented to the University Hospital of LMU Munich on 10 July, 2017. The investigation mandated by the Executive Board of University Hospital of LMU Munich focused on the clinical phase I/II study entitled "Feasibility, Safety and Effectiveness of Transplanting of Retrovirally Transduced Hematopoietic Stem Cells for the Therapy of Wiskott-Aldrich Syndrome", which was led by Professor Dr. med. Dr. sci. Christoph Klein. The investigation showed no evidence of any scientific, medical, legal or ethical misconduct on the part of Professor Dr. med. Dr. sci. Christoph Klein.


By a letter dated 28 April, 2016, the executive board of University Hospital of LMU Munich requested the chairperson of the Medical Faculty's Commission on Professional Self Regulation in Science to investigate the serious allegations made against Dr. med. Dr. sci. Christoph Klein in an article published by SZ-Magazin on 22 April, 2016, entitled "Arzt ohne Grenzen" ("Doctor without Borders"). The allegations were raised in connection with a clinical phase I/II "WAS Gene Therapy Study" designed by Professor Klein at Hannover Medical School (MHH), proposed by him to MHH in July 2004, and implemented under his responsible leadership since October 2006. This experimental pilot study was the first gene transfer study addressing to patients/children suffering from Wiskott–Aldrich syndrome. For molecular transport vehicles ("gene shuttles" or "gene taxis"), the study used the first generation of the so-called retroviral vectors available at the time, i.e., non-replicable, modified, retroviral virus constructs. Gene shuttles of this type had previously been used by a few other international medical research scientists as part of their gene therapy studies for children suffering from other severe hereditary (monogenetic) diseases. Professor Klein and his team, however, were the first physicians and research scientists to conduct a gene therapy study specifically of children suffering from WAS, i.e., they were venturing into unchartered territory in that regard.

The investigation mandate given to the Commission on Professional Self Regulation in Science of the Medical Faculty of Ludwig Maximilians University (LMU) Munich covered the complex medical-scientific, legal, and ethical issues of the WAS gene therapy study. The study was commenced at Hannover Medical School (MHH) in 2006, and upon Professor Klein’s transfer to LMU in 2011, was transferred to LMU. Munich no longer saw any treatment of study patients, merely follow-up examinations and follow-on therapies.

Special attention must be paid in the case at hand to the ethical assessment of risks and benefits in the triangle of conflicting priorities between the physician's pursuit of "individual benefits" and the medical research scientist's generally farther-reaching pursuit of "global benefits". In other words: to either continue using previously existing treatment procedures, albeit fraught with known serious side effects, to attempt to cure or alleviate an individual child's suffering who is affected by a disease which in all likelihood will lead to premature death. Or, at the same time, to venture into unchartered territory, embarking from an adequately validated scientific starting point, with due respect of and compliance with the existing statutory and ethical rules and guidelines, in an effort to achieve significant improvement with new procedures, i.e., progress in the treatment outcomes, both for the individual and in general for all patients eligible.

Implementation of the investigation

The purpose of the investigation mandated by the executive board of University Hospital of LMU Munich was to contain the following battery of questions, quoted verbatim in the following, which to some extent go beyond purely scientific questions and also concern issues of medical conduct and the assessment of ethical risk for new, experimental therapy studies:


  1. Have all patients and their families (parents) enrolled in the study been informed about all risks (notably about tumor development) entailed by the trial therapy using gene vectors and alternative treatment options (stem cell/bone marrow transplants), and have declarations of consent been obtained from all parties involved?
  2. What was the state-of-the-art at the time of the study? What was the state-of-the-art in gene therapy at the time of study, specifically with regard to viral vectors?
  3. What was known about the risk of leukemia development and how high was the risk of alternatives (stem cell therapy)?
  4. Were the procedures for approval of the study by MHH and the government authorities properly followed?
  5. Was the study properly verified by the University Hospital on MHH's take-over of the sponsorship?
  6. What where the existing ethical standards at the time serving as (mandatory) decision-making basis for such an experimental therapy on children, and which boards were involved in the decision-making process?


The investigation of this multi-faceted topic by the extended Commission on Professional Self Regulation in Science of the Medical Faculty of LMU, which was explicitly declared from the outset as being highly confidential, without prepossession, and strictly neutral, was conducted by means of a retrospective analysis of extensive file materials. What needed to be taken into account retrospectively for that investigation, to the greatest extent possible, were the conditions relevant for the WAS gene transfer study and valid at the time the study was proposed and implemented. The investigation of the files primarily provided was supplemented by a detailed interview of Professor Klein over the course of several hours during one of the Commission's meetings, as well as additional written and telephone queries addressed to him. This was later followed by additional written documents which had been additionally requested on the basis of the experts' instructions and which were incorporated in the Commission's work.

External expert's reports

As mentioned earlier, it seemed advisable, both from the outset and following the intense involvement of the Commission on Professional Self Regulation in Science in the issues and questions associated with the above-described and commented clinical phase I/II–WAS gene therapy study led by Professor Klein, to have a complementary scientific expertise conducted by external scientists/experts. The task was to identify experts possessing outstanding expertise who are known to be impartial in every respect and who consider themselves impartial in every respect. After they had responded favorably to a request, two highly renowned scientists were commissioned with the investigation in August 2016, by a letter dated 28 August 2016. For that purpose, the experts were provided with the extensive written documentation in the Commission's hands and described above, consisting of copies of documents and other types of papers originating predominantly from the time the clinical WAS gene therapy study was conducted at MHH, which had been forwarded to the Commission mostly by Professor Klein.

Both experts are scientists of high national and international renown. One of them is a professor of medical law and an eminent expert in health and medical professional law as well as in medical ethics. The other is a professor/physician/director of a major university hospital specialized in hematooncology. Both experts work at different universities and were given no clue as to each other's identity.

The Commission's conclusion

After thorough assessment of all outstanding issues by the members of the Commission and the external experts, no evidence could be found suggesting that Professor Dr. Christoph Klein is guilty of scientific, medical, legal or ethical misconduct.

The assessment of press-law or journalism/trade-relevant aspects of the article published in SZ-Magazin on 22 April, 2016, and entitled "Arzt ohne Grenzen" ("Doctor without Borders"), could not and should not have been part of the scope of the investigation. The confirmation of the Order to Cease and Desist issued on 20 May, 2016, in reference to several items contained in the report of SZ-Magazin, proclaimed by the Civil Division 26 of the Hamburg District Court in the court hearing on 19 July, 2019 (Case File # 324 O 268/16), was for that reason not included in the investigation by the Commission on Professional Self Regulation in Science.

The hospital's executive board, including the medical director and the dean of the Faculty of Medicine of LMU Munich, the members of the Commission, and all persons involved in the study deeply regret the fateful course of the study which led to the demise of three of the study participants.


Please direct your inquiries to:

Philipp Kressirer

Press Office, Communication and Media

University Hospital of LMU Munich (Medical Center)

Telephone: 089 4400 - 58071




University Hospital of Munich (LMU)

The Munich University Hospital (LMU) treats around 500,000 outpatients, inpatients and semi-residential patients each year at its Großhadern and City Centre Campuses. Just over 2,000 beds are available to its 29 specialist clinics, eleven institutes and seven departments, and its 49 interdisciplinary centres. Of a total of 9,700 employees, around 1,700 are doctors and 3,200 are nursing staff. Munich University Hospital has been a public-law institution since 2006.

Together with the Medical Faculty of Ludwig Maximilians University, Munich University Hospital is involved in four special research areas of the German Research Foundation (SFB 914, 1054, 1123, 1243), three Transregios (TRR 127, 128, 152, 205) belonging to Clinical Research Group 809, and two Graduate Colleges belonging to the German Research Foundation (GK 1091, 1202). This is in addition to the Center for Integrated Protein Sciences (CIPSM), Munich Center of Advanced Photonics (MAP), Nanosystems Initiative Munich (NIM) and Munich Cluster for Systems Neurology (SyNergy) – all institutes of excellence – and the Graduate School of Systemic Neurosciences (GSN-LMU), the Graduate School of Quantitative Biosciences Munich (QBM) and the Graduate School Life Science Munich (LSM).

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