Yissum - Research Development Company of the Hebrew University

Sphingolipid metabolism modifiers

Posted by Yissum - Research Development Company of the Hebrew UniversityResponsive · Innovative Products and Technologies · Israel

Summary of the technology

Novel Sphingolipid molecules that have activity in: Inducing apoptosis and death to cancer; Reducing biosynthesis of sphingomyelin and glycolipids in lipid-storage diseasesas As well as inhibit sphingolipid hydrolases and can be used for “chaperone” therapy of these diseases. These compounds may also serve as anti-parasitic drugs. The synthetic compounds are non-natural analogs of sphingolipids. Their activities are based on modifying the metabolism of this group of complex lipids. Applications: As anti cancer and anti-parasitic drugs, they elevate ceramide leading to apoptosis and death. For lipid-storage diseases they inhibit the biosynthesis of sphingolipids or of their lysosomal hydrolases, enabling therapies based on both effects.

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Project ID : 12-2006-158

Description of the technology

Compounds for treating cancer, malaria and lipid storage diseases


Oncology, antimalarial, Niemann-Pick, Gaucher

Development Stage

A large number of analogs have been synthesized and screened for anti cancer activity in various tissue culture cell lines. Three analogs were selected and showed activity when tested in mice models of Human Prostate, Melanoma and Pancreatic tumors.

Patent Status

U.S granted (No. 6,756,504), Europe granted (No. 1430019), Filed in Japan

Market Size

The total worldwide estimated market for prostate, melanoma and pancreatic cancers is more than $20B. Gaucher disease is more than $840 million


  • Novel new compounds
  • Anticancer activity observed with subcutaneous and in metastatic cancer mice models of prostate, pancreas and melanoma. Analogs were injected intradermally or intraperitoneally, or given per os to mice bearing human tumors.
  • Provides powerful stand alone or adjunctive therapy with irradiation or chemotherapy for cancer
  • Compounds for treating cancer, parasitic diseases such as malaria, lipid storage diseases such as Gaucher, Niemann-Pick, Tay-Sachs and more
  • Elevates cellular ceramide, inducing cytotoxicity and death by apoptosis

Our Innovation

Novel synthetic compounds - non-natural analogs of sphingolipids – that modify sphingolipid metabolism

Key Features

  • Induce apoptosis and death to cancer cells.
  • Reduce human tumors (eg, prostate, melanoma, pancreas) in nude mice.
  • Reduce biosynthesis of sphingomyelin and glycolipids in lipid-storage diseases. Also inhibit sphingolipid hydrolases and can be used for “chaperone” therapy of these diseases.
  • Kill malaria parasites in nanomolar concentrations.

Development Milestones

  • Further in vivo mouse models
  • Pharmacokinetic pharmacodynamic experiments
  • In vivo human testing

The Opportunity

  • Provides more efficacious cancer treatment
  • Addresses needs for expanding cancer market
  • Legislative and economic incentives have created a positive climate for developing drugs and treatments for rare diseases; many orphan drugs are sold at high prices that can reach several hundred thousand US dollars per year; new orphan drugs now provide help for more than 11 million people in the USA alone
  • The global market for antimalarial drugs of the order of $100-120 million per year

Project manager

Ariela Markel
VP, Business Development, Healthcare

Project researchers

Arie Dagan
HUJI, Faculty of Medicine

Shimon Gatt
HUJI, Faculty of Medicine
Cellular biochemistry and human genetics

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About Yissum - Research Development Company of the Hebrew University

Technology Transfer Office from Israel

Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Founded in 1964 to protect and commercialize the Hebrew University’s intellectual property. Ranked among the top technology transfer companies, Yissum has registered over 8,900 patents covering 2,500 inventions; has licensed out 800 technologies and has spun-off 90 companies. Products that are based on Hebrew University technologies and were commercialized by Yissum generate today over $2 Billion in annual sales.

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