# US 9,750,712 B2 · Composition and method for treating neuronal ceroid lipofuscinosis

> Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt

- Number: US 9,750,712 B2
- Document ID: US9750712B2
- Status: Granted
- Kind: B2
- Application number: 14/649,069
- Filed: 2013-12-06
- Published: 2015-11-05
- Granted: 2017-09-05
- Earliest claimed: 2012-12-07
- Assignees: Rush University Medical Center
- Applicants: Rush University Medical Center
- Inventors: Kalipada Pahan
- Web page: https://patentgrep.com/patent/US9750712B2

## CPC

- A61K 31/216: of acids having aromatic rings, e.g. benactizyne, clofibrate
- A61K 31/192: having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid {(cannabinoids A61K31/658)}
- A61K 31/203: Retinoic acids {; Salts thereof}
- A61P 25/28: for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

Provided herein are methods for treatment of a neurodegenerative disease, such as neuronal ceroid lipofuscinosis including administering to a subject in need of such treatment a composition comprising a therapeutically effective amount of an agent that mediates upregulation of TPP 1.

## Claims

### Claim 1

A method for treatment of a neurodegenerative disease, comprising administering to a subject in need of such treatment a composition comprising a therapeutically effective amount of an agent that mediates upregulation of TPP1, the agent comprising a lipid-lowering drug, wherein the neurodegenerative disease is neuronal ceroid lipofuscinosis, wherein the lipid lowering drug is a fibrate.

### Claim 2 (depends on 1)

The method of claim 1, wherein the fibrate is gemfibrozil or fenofibrate.

### Claim 3 (depends on 1)

The method of claim 1, wherein the neuronal ceroid lipofuscinosis is late infantile neuronal ceroid lipofuscinosis or Batten disease.

### Claim 4 (depends on 1)

The method of claim 1, wherein TPP1 is upregulated by increasing TPP1 mRNA levels, increasing TPP1 protein levels, increasing TPP1 enzymatic activity, or activating a PPAR-RXR heterodimer.

### Claim 5 (depends on 1)

The method of claim 1, wherein the composition further comprises a therapeutically effective amount of all-trans retinoic acid.

### Claim 6 (depends on 5)

The method of claim 5, wherein administering all-trans retinoic acid and a fibrate provides a greater therapeutic effect in the subject than administration of all-trans retinoic acid or the fibrate alone.

### Claim 7

A method for treatment of a neurodegenerative disease, comprising administering to a subject in need of such treatment a composition comprising a therapeutically effective amount of an agent wherein the agent restores TPP1 activity, the agent comprising a lipid-lowering drug, wherein the neurodegenerative disease is neuronal ceroid lipofuscinosis, wherein the lipid lowering drug is a fibrate.

### Claim 8 (depends on 7)

The method of claim 7, wherein the fibrate is gemfibrozil or fenofibrate.

### Claim 9 (depends on 7)

The method of claim 7, wherein the therapeutically effective amount of the fibrate is lower when the fibrate is administered in combination with all-trans retinoic acid.

### Claim 10

A method for treatment of neurodegenerative disease, comprising administering to a subject in need of such treatment a composition comprising a therapeutically effective amount of an agent that mediates upregulation of a gene selected from the group of Cln1, Clin2, Cln3, and any combination thereof, the agent comprising a lipid-lowering drug, and wherein the neurodegenerative disease is neuronal ceroid lipofuscinosis, wherein the lipid lowering drug is a fibrate.

### Claim 11 (depends on 10)

The method of claim 10, wherein the fibrate is gemfibrozil or fenofibrate.