Seminar Report: 2nd Academia Clinical Development Seminar
“Light and shadow of the development of new drugs for rare diseases –to overcome the Darwinian Sea-”

The 2nd Academia Clinical Development Seminar in 2018 FY was held on Friday June 18th at the multi-media hall, Osaka University Hospital. We invited Prof. Kou NAKATA from Niigata University Medical and Dental Hospital as a lecturer. He gave us a talk with a title of “Light and shadow of the development of new drugs for rare diseases –to overcome the Darwinian Sea-“. He introduced episodes of challenges in the development of a drug, called Silorimus for Lymphangioleiomyomatosis (LAM).

The LAM is a rare lung disease that tends to affect young women.  LAM cells  proliferate in lungs (sometimes in kidneys), and result in gradual progression of dyspnea. A study in 260 Japanese patients conducted by the Ministry of Health Labor and Welfare (MHLW) Research Group showed that  70% of patients experienced pneumothorax and 30% of patients received oxygen therapy at home. Even though there have been great efforts of physicians and patients association for years  the LAM was specified as an intractable disease in Japan.

However, due to the problem of profitability, no any large pharmaceutical companies had the courage to develop the drug. Under such circumstances, he collaborated with the patients association and conducted an investigator-initiated clinical trial for LAM with Sirolimus in order to overcome “the Darwinian Sea”. Consequently, the Sirolimus was successfully approved for pharmaceutical law by Novel Pharma Co., Ltd in December 2014.  Three years after its release on the market, now over 300 patients with LAM have benefited from this drug.

At the beginning of the lecture, he introduced episodes of his research in Japan, when he came back to Japan after the training program in the USA. At that time, there was an insufficient experimental equipment in his laboratory, and his laboratory resembled to a novel, “Daichi no ko” written by Toyoko Yamazaki. However, the situations have been changed and his research was advanced after the LAM foundation donated by patients association in USA was established in 1995.The success story of drug development for LAM began with the only one female patient and her mother at the beginning, later on, the patients association supported by the LAM foundation became one of the biggest organizations in the USA in just a couple of years. We were really impressed with the fact that they have donated the research grant to the LAM foundation for the drug development.


The MILES trial, which was watched with interest as investigator-initiated international trial, was completed in 2011. Although the MHLW tried to examine efficacy and safety of the drug  by several kinds of new systems for regulatory approval, the MHLW did not approve  the drug, because of existing post-marketing safety concerns. He and patient associations of LAM tried to negotiate repeatedly with Pfizer for drug development, but failed. However, the right of drug sales in Japan was fortunately licensed to the Novel Pharma Co. Ltd. Then another investigator-initiated clinical trial was further conducted at  9 hospitals in Japan under a condition that Novel Pharma provided the drug without payment. Consequently, the Sirolimus was successfully approved for sale to patients with LAM in 2014. During 11 years of drug development, a total of 700,000,000 JPY appeared to be invested from the government. A support of patient association as well as the LAM foundation from the outside of Japan continuously played  a crucial role in the drug development.

At the end of his talk, he gave us comments that we need to discuss more about how to use an annual budget for medicine of 4000 billion JPY in Japan. Furthermore, he told us a concern whether drug development is decided only by profit from pharmaceutical companies. These topics were not fully discussed, and more proactive debates are necessary.  We felt that drug development in academia  mainly funded by government does not focus on profitability,  but  on the rarity of  diseases. We were also impressed that he made great efforts for the drug development patiently and we hope to follow his great negotiations.

After the lecture, there were a lot of queries from the participants and it took more than 30 minutes to end Q&A session. With the attractive title of his talk, many researchers and  graduate students attended the lecture. We appreciated his lecture and all participants.

The 3rd Academia Clinical Development Seminar will be held on Friday August 23th, starting from 5:30pm to 7:00pm. The lecturer will be Mr. Shigeki SHIMAZAKI from the Novel Pharma Co., LTD under the title of “ Efforts and Concerns on Drug Development for Rare Diseases by Drug Venture Company” Don’t miss it!

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