# US 5,716,616 A · Isolated stromal cells for treating diseases, disorders or conditions characterized by bone defects

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- Number: US 5,716,616 A
- Document ID: US5716616A
- Status: Granted
- Kind: A
- Application number: 08/412,066
- Filed: 1995-03-28
- Granted: 1998-02-10
- Earliest claimed: 1995-03-28
- Assignees: Thomas Jefferson University
- Inventors: Darwin J. Prockop; Ruth F. Pereira; Dennis B. Leeper; Michael D. O'Hara
- Web page: https://patentgrep.com/patent/US5716616A

## CPC

- A61K 35/28: Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
- A61K 48/00: Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- C07K 14/4702: {Regulators; Modulating activity}
- C07K 14/61: Growth hormone [GH], i.e. somatotropin
- C07K 14/745: Blood coagulation or fibrinolysis factors
- C07K 14/78: Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
- C12N 5/0663: {Bone marrow mesenchymal stem cells (BM-MSC)}
- A61K 38/00: Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00)
- A61K 2035/128: {capsules, e.g. microcapsules}
- C12N 2502/1311: Osteocytes, osteoblasts, odontoblasts
- C12N 2799/027: where the vector is derived from a retrovirus
- 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)
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

Methods of treating patients who are suffering from a disease, disorder or condition characterized by a bone cartilage or lung defect are disclosed. The methods comprising the step of intravenous administration of stromal cells isolated from normal syngeneic individuals or intravenous administration of stromal cells isolated from the patient subsequent to correction if the genetic defect in the isolated cells. Methods of introducing genes into a recipient individual are disclosed. The methods comprise the steps of obtaining a bone marrow sample from either the recipient individual or a matched syngeneic donor, isolating adherent cells from the sample, transfecting the adherent cells that were isolated from the recipient or a matched syngeneic donor with a gene and administering the transfected adherent cells to the recipient individual intravenously. Compositions that comprise isolated stromal cells that include exogenous genes operably linked to regulatory sequences are disclosed.

## Claims

### Claim 1

A method of treating a patient who is suffering from a disease, disorder or condition characterized by a bone defect comprising the steps of:
a) obtaining a bone marrow sample from a donor who is not suffering from a disease, disorder or condition characterized by a bone defect;
b) isolating adherent cells from said sample; and,
c) administering by intravenous infusion to said patient an amount of said isolated adherent cells effective to treat said disease, disorder or condition, wherein said patient undergoes bone marrow ablation prior to administration of said isolated adherent cells.

### Claim 2 (depends on 1)

The method of claim 1 wherein said adherent cells are administered by intravenous infusion to said patient together with non-adherent cells from a bone marrow sample from a donor who is not suffering from a disease, disorder or condition characterized by a bone defect.

### Claim 3 (depends on 1)

The method of claim 1 wherein said disease, disorder or condition is osteogenesis imperfecta.

### Claim 4 (depends on 1)

The method of claim 1 wherein said bone defect is due to a collagen gene mutation.

### Claim 5 (depends on 1)

The method of claim 1 wherein said donor is syngeneic with said patient.

### Claim 6 (depends on 1)

The method of claim 1 wherein said isolated adherent cells are cultured to expand the number of said cells and said expanded culture is administered to said individual.

### Claim 7 (depends on 6)

The method of claim 6 wherein said disease, disorder or condition is osteogenesis imperfecta.

### Claim 8 (depends on 6)

The method of claim 6 wherein said bone defect is due to a collagen gene mutation.

### Claim 9 (depends on 6)

The method of claim 6 wherein said donor is syngeneic with said patient.