INTRA-ERYTHROCYTE DEXAMETHASONE FOR TREATMENT OF ATAXIA-TELANGIECTASIA

1 . A method at treating Ataxia Telangiectasia in a subject less than about 10 years of age, comprising administering to the subject at least 5 mg of dexamethasone sodium phosphate (DSP) that has been loaded into erythrocytes.

2 . A method at treating Ataxia Telangiectasia in a subject less than about 10 years of age, comprising administering to the subject at least 5 mg of dexamethasone sodium phosphate (DSP) that has been loaded into erythrocytes.

3 . The method of claim 1 , wherein the subject is at least 6 years of age and less than 10 years of age.

4 . The method of claim 1 , wherein the subject is administered between about 5 mg and about 30 mg DSP.

5 . The method of claim 1 , wherein the subject is administered between about 5 mg and about 22 mg DSP.

6 . The method of claim 1 , wherein the subject is administered about 8 mg or about 22 mg DSP.

7 . The method of claim 1 , wherein the subject is administered about 8 mg or about 17 mg DSP.

8 . The method of claim 1 , wherein the subject is administered the DSP monthly, every 5 weeks, every 6 weeks, every 7 weeks or every two months.

9 . The method of claim 1 , wherein the DSP is administered by infusion.

10 . The method of claim 1 , wherein the treatment period is 3 months, 6 months, 9 months, or 12 months.

11 . The method of claim 1 , wherein the erythrocytes are autologous to the subject.

12 . The method of claim 1 , wherein the erythrocytes are loaded with DSP by:

(i) swelling erythrocytes, optionally isolated from whole blood, using a first hypotonic solution;

(ii) further swelling the erythrocytes in step (i) with a second hypotonic solution that is more hypotonic that the first hypotonic solution, wherein the further swelling is done without reaching lysis;

(iii) concentrating the erythrocytes obtained in step (ii);

(iv) placing the concentrated erythrocytes in contact with a lysing solution comprising, or alternatively consisting essentially of, or yet further consisting of DSP; and

(v) adding a sealing solution and thereby obtaining a population of erythrocytes loaded with DSP.

13 . The method of claim 1 , wherein the erythrocytes are loaded with DSP by:

(a) removing whole blood from the subject;

(b) using the whole blood, preparing a population of erythrocytes by

(i) isolating erythrocytes from the whole blood;

(ii) swelling the erythrocytes using a first hypotonic solution;

(iii) further swelling the erythrocytes in step (ii) with a second hypotonic solution that is more hypotonic that the first hypotonic solution, wherein the further swelling is done without reaching lysis;

(iv) concentrating the erythrocytes obtained in step (iii);

(v) placing the concentrated erythrocytes in contact with a lysing solution comprising DSP; and

(vi) adding a sealing solution for the purpose of obtaining a population of erythrocytes loaded with said one or more pharmaceutical products.

14 . The method of claim 1 , wherein the erythrocytes are loaded with DSP by:

(a) separating erythrocytes from the whole blood from 20-100 mL of whole blood;

(b) swelling the erythrocytes using a first hypotonic saline solution having an osmolality between 230 and 150 mOsm/kg;

(c) further swelling the erythrocytes using a second hypotonic solution having a lower osmolality than the first hypotonic saline solution, wherein the further swelling is done without reaching lysis, thereby providing swollen erythrocytes;

(d) contacting the swollen erythrocytes with a solution comprising between 2-10 mL of a solution comprising DSP at a concentration of 25 mg/mL; and

(e) incubating the swollen erythrocytes with a hypertonic sealing solution comprising phosphate-inosine-glucose-pyruvate-adenine (PIGPA).

15 . The method of claim 1 , wherein the erythrocytes are loaded with DSP by:

(a) obtaining between 20-100 mL of whole blood of the subject;

(b) separating erythrocytes from the whole blood;

(c) swelling the erythrocytes using a first hypotonic saline solution having an osmolality between 230 and 150 mOsm/kg;

(d) further swelling the erythrocytes using a second hypotonic solution having a lower osmolality than the first hypotonic saline solution, wherein the further swelling is done without reaching lysis, thereby providing swollen erythrocytes;

(e) contacting the swollen erythrocytes with a solution comprising between 2-10 mL of a solution comprising DSP at a concentration of 25 mg/mL; and

incubating the swollen erythrocytes with a hypertonic sealing solution comprising phosphate-inosine-glucose-pyruvate-adenine (PIGPA).

16 . The method of claim 14 , wherein the second hypotonic solution brings the intact erythrocytes to an osmolality between 200 and 170 mOsm/Kg.

17 . The method of claim 15 , wherein the second hypotonic solution brings the intact erythrocytes to an osmolality between 200 and 170 mOsm/Kg.

18 . The method of claim 14 , wherein about 50 mL of the whole blood is obtained from the subject.

19 . The method of claim 14 , wherein about 30 mL of the whole blood is obtained from the subject.

20 . The method of claim 19 , wherein the subject is between 9 and 15 kilograms in weight.